theindependent--2019-05-03--Super-gonorrhoea cure could come from new treatment for drug-resistant STI

A new combination of medication could treat a previously drug-resistant strain of gonorrhoea, according to scientists.  There has been increasing concern that resistance to treatment could become widespread, but now researchers have found that an antibiotic known as gentamicin can be effective when taken alongside another drug.  The latest treatment combines this antibiotic with azithromycin, which is usually prescribed in combination with a different drug called ceftriaxone.  According to the results of a study which was published today in The Lancet medical journal, the new combination was found to be almost as effective in treating genital gonorrhoea, which is good news for doctors who are concerned that the existing treatment “is beginning to fail”.  In January, Public Health England revealed that two women had been infected with super-gonorrhoea, which was resistant to the first-choice treatment. Other cases have also been reported.  "Our current antibiotic treatment for gonorrhoea is beginning to fail and experience with previous drugs strongly suggests that this could become a widespread problem," said Professor Jonathan Ross, chief investigator from the University of Birmingham where the research was conducted.  The trial took place at 14 sexual health clinics in England between October 2014 and November  2016, and 720 patients were given either the current antibiotic treatment of ceftriaxone or the new combination.  A total of 98 per cent of those given the traditional combination were cured of gonorrhoea after two weeks, compared to 91 per cent of those treated with gentamicin.  While the trial found that gentamicin combined with azithromycin works almost as well as for genital gonorrhoea, it did not clear throat or rectal gonorrhoea as effectively, and researchers said further work is needed to find alternative treatments, with antibiotic resistance a growing problem.  Professor Hywel Williams, director of the NIHR's health technology assessment programme, said: "It's very worrying that cases of treatment-resistant gonorrhoea are now appearing across the globe.  "This research provides important new evidence that suggests that gentamicin with azithromycin could become a second-line treatment for patients who are resistant to ceftriaxone with this infectious disease.  Gonorrhoea is a sexually transmitted infection caused by bacteria called Neisseria gonorrhoeae, also called gonococcus, which is usually found in penis discharge and vaginal fluid. It does not usually lead to complications, but if left untreated can cause serious health issues including pelvic inflammatory disease among women.


theindependent--2019-09-28--Skin cancer Drug treatment increases patientsapos long-term survival chances by 50 per cent stud

Skin cancer: Drug treatment increases patients' long-term survival chances by 50 per cent, study claims

A combination of two powerful drugs for skin cancer can increase a patient’s long-term survival chances by 50 per cent, according to a study.  Results from a new trial have shown that ipilimumab (Yervoy) and nivolumab (Opdivo), taken together, can stop or reverse the progression of advanced melanoma for five years or more in one in two patients.  Melanoma is a form of skin cancer, known for its mole-like appearance on the skin, that accounts for about 2,300 deaths in the UK every year.  The most recent figures showed about 16,000 people were diagnosed with the disease in 2016.  Both of the drugs, which were developed by pharmaceutical company Bristol-Myers Squibb, help the immune system find and destroy cancer cells as they spread.  Only one in 20 patients with advanced melanoma would survive for five years a decade ago, with many living for just six to nine months.  Professor James Larkin, a consultant medical oncologist at the Royal Marsden NHS Foundation Trust, said: "In the past, metastatic melanoma was regarded as untreatable. This is the first time we can say that the chances of being a long-term survivor of advanced melanoma are now over 50 per cent, which is a huge milestone."  Patients in the trial were divided into three groups and given either both nivolumab and ipilimumab or one of the drugs with a placebo.  The five-year overall survival rate for the combination group was 52 per cent, with 74 per cent of those patients going treatment-free after five years.  The overall survival for the nivolumab group was 44 per cent, while for the ipilimumab group, it was 26 per cent.  Mr Larkin, who is also a professor at the Institute of Cancer Research, added: "By giving these drugs together you are effectively taking two brakes off the immune system rather than one so that the immune system is able to recognise tumours it wasn't previously recognising and react to that and destroy them."  Researchers also found that patients who stopped treatment because of side-effects such as fatigue, skin rashes and diarrhoea saw outcomes that were just as good as those who were on the combination for longer.  However, Mr Larkin warned that there is currently no method to predict which patients are most likely to benefit from combination immunotherapy.  “The decision on which treatments to give is a matter for doctors to discuss with individual patients and their families,” he said. "The two drugs together definitely have a role in treating metastatic melanoma and will be the choice for some patients. For others, the decision may be to give the drugs in sequence.”  The findings from the trial, which was led by the Royal Marsden NHS Foundation Trust, will be presented at the European Society for Medical Oncology meeting in Barcelona, Spain, and published in The New England Journal of Medicine.  The treatment is recommended by the National Institute for Health and Care Excellence and is available in this combined way on the NHS, following assessments by doctors.


theirishtimes--2019-03-21--Israeli cancer treatment could be something special

As we all know, cancer is a great scourge. The Irish Cancer Society website tells us that one person dies from cancer every hour in Ireland and cancer accounts for almost one-third of all annual Irish deaths.  Internationally, cancer is the second leading cause of death, second only to cardiovascular disease. Intensive research efforts have been under way worldwide since the 1970s to discover cures for the various forms of cancer. Progress has been very slow, but now Israeli researchers have announced a cure for all kinds of cancer and predict that this cure will enter clinical trials within a year (The Jerusalem Post, January 28th). This claim has been greeted with widespread scepticism internationally.  The new research is the work of a company called Accelerated Evolution Biotechnologies Ltd (AEB), based in Ness Ziona, Israel. The Jerasulem Post article reported information provided by AEB’s chairman Dan Aridor and chief executive Ilan Morad. They call the cancer treatment a multi-target toxin (MuTaTo), describing it as a cancer disruption technology of the highest order – on the scale of a cancer antibiotic. Aridor promised: “Our cancer cure will be effective from day one, will last a duration of a few weeks and will have no or minimal side-effects.”  AEB has conducted successful experiments on mice, completed several in-vitro trials and say they are close to starting clinical trials to be completed within a few years when the treatment will be made widely available. AEB claims they have not published their results so far in a scientific journal because they are generating final patents on their intellectual property.  International experts have mostly reacted negatively to the Israeli claim, pointing out that the limited tests carried out so far have been on mice, that it is quite difficult to translate success in mice into a successful human treatment, that no results have been published in peer-reviewed scientific literature and that, even if everything goes well, it will take many more years than the Israeli scientists predict before this new cure is through clinical trials and available to the general public.  Morad explained the AEB approach. Firstly, AEB set out to identify why most anti-cancer drugs either don’t work, or eventually fail, and then devised a way to get around these deficiencies. Most anti-cancer drugs attack specific targets in or on the cancer cell, thereby affecting a physiological pathway that promotes the cancer. But mutations in the cancer cell often render the target irrelevant to the cancerous nature of the cell, rendering the attacking drug ineffective.  AEB devised their new MuTaTo approach using peptides to attack the cancer cells. Proteins are polymers of units called amino acids joined together through peptide bonds in a long chain. Peptides are relatively short chains of amino acids (2 to 20) and they can be very effective at targeting cancer cells. MuTaTo uses a combination of several peptides that simultaneously target the cancer cell, combined with a strong peptide toxin that specifically kills cancer cells.  AEB claims their MuTaTo approach, attacking each cancer cell with several cancer-targeting peptides combined with a strong peptide toxin to kill cancer cells, ensures the treatment will not be affected by mutations because it kills all the cancer cells before mutations can mount an escape from the drug treatment.  When attacked by drugs, cancer cells also activate detoxification mechanisms that can either expel the drugs from the cancer cells or chemically inactivate them. But detoxification takes time and AEB claims that the new MuTaTo approach will kill the cancer cells before they can successfully deploy the detoxification defence.  Most current cancer treatments have sickening side effects mainly because the conventional drugs interact with the wrong targets or with additional targets on the cancer cells and on non-cancer cells. The MuTaTo approach is highly and specifically targeted at the cancer cells only and this should dramatically decrease side effects. AEB also promises that, when the MuTaTo technique is fully developed, it will be possible to personalise individual treatments to match the exact cancer type expressed in the patient.  Despite the widespread international scepticism that greeted AEB’s announcement, I have a feeling that AEB is on to something special. Let us hope they are.  William Reville is an emeritus professor of biochemistry at UCC


theirishtimes--2019-11-07--Record numbers seek treatment for cocaine use

Record numbers of people in the State are seeking treatment for cocaine use, with an unprecedented 50 per cent surge in new cases last year.  Demand for treatment from users of all other drugs combined, including cannabis and heroin, increased by only eight per cent during the same period.  The sharp spike in the number of cocaine users presenting for treatment is being linked to rising consumption of the drug due to the economic recovery.  Chief executive of the Health Research Board (HRB), Dr Darrin Morrissey, said the rapidly rising number of cocaine users now seeking treatment “dominates” Ireland’s latest drug trends.  “It represents the majority of the increase in all treatment cases,” Dr Morrissey said of the cocaine treatment cases.  “HRB figures show a consistent rise in treatment for cocaine since 2013, with the biggest increases in 2017 and 2018, and highlight a changing pattern of drug use during the recent economic recovery.”  The worsening national cocaine problem has emerged in data for last year from the HRB’s national drug treatment-reporting system which was due for release on Thursday.  The report found 10,274 drug treatment cases last year, up 15 per cent on 2017. Heroin accounted for 4,349 of the cases, cannabis 2,358, and cocaine 2,254.  The report said between 2012 and last year the total number of all drug treatment cases had increased by 28 per cent with cocaine users accounting for the vast majority of that increase.  Some 3,962 of last year’s drug treatment cases involved people new to the drug treatment system, the report found, made up of 1,505 for cannabis, 1,232 for cocaine and 719 for opiates.  “Last year was the first time the number of first-time treatment cases for cocaine was higher than for heroin,” it said.  The majority, 65 per cent, of cases were treated at out-patient facilities, prisoners accounted for 10 per cent, in-patient facilities 14 per cent, and GPs two per cent.  Cocaine accounted for nine per cent of new drug treatment cases in 2012, rising to 31 per cent of new treatment cases last year. While the cocaine problem was worsening there were some signs that problem heroin and cannabis use was more stable or decreasing.  Opioids, or heroin, were the main problem drug in treatment cases between 2012 and 2018. It accounted for 42 per cent of all treatment cases in 2018, down from 52 per cent in 2012.  Similarly, cannabis accounted for almost 29 per cent of treatment cases in 2012, falling to 23 per cent last year.  The new figures come just weeks after Garda Commissioner Drew Harris said he believed the improving economy was impacting crime rates.  He said rising disposable incomes had resulted in increased consumption of drugs and alcohol. This, he said, was in turn leading to more public disorder and assaults.  The latest crime data also reveals an increase in drug crime; up nine per cent last year followed by a 20 per cent increase in the first six months of this year.


thesun--2019-02-11--Boy 9 receives life-saving cancer treatment after umbilical cord stem cell transplant

Boy, 9, receives life-saving cancer treatment after umbilical cord stem cell transplant

A BOY of nine is cancer free after a revolutionary transplant using umbilical cord stem cells.  Medics said the op was Jenson Wright’s only hope when leukaemia returned a second time.  The cells, which had been stored frozen in Texas, began to kill the disease within five days.  Two years on, and Jenson is now fully recovered.  The brave youngster was first diagnosed with lymphoma and leukaemia in November 2013 when he was just four-years-old.  Thrilled mum Carolyn, 46, of Penketh, Cheshire, said: "The early stages of the treatment were quite hard to comprehend, and quite difficult for us emotionally.  "Without the transplant we would be in a completely different situation - that saved him.  “We’ll never know who the donor was, but their donation has given life to someone else.”  Jenson, Carolyn, dad Steve and 18-year-old older brother Lewis had a family meal to celebrate receiving the news that the youngsters is now free from cancer.


thesun--2019-06-02--Search and destroy radioactive treatment could extend lives of patients with incurable prostate ca

‘Search and destroy’ radioactive treatment could extend lives of patients with incurable prostate cancer

A “SEARCH and destroy” radioactive treatment could extend the lives of patients with incurable prostate cancer.  Early results show men with no other options could get more than two years’ extra life.  Medics described the tumour-targeting approach as “the next big thing” for the disease.  Two men with advanced prostate cancer last week became the first to get it in Britain.  It costs £12,000 a session privately, but experts say it could soon be available on the NHS.  Given as a drip, it lets medics deliver a “radioactive payload” directly to tumours without damaging surrounding tissue.  Hans Schaupp, 77, from Hants, received the treatment in a four-hour visit to a clinic last week.  He said: “Rather than poisoning your whole body with chemotherapy it goes straight to the tumours. I feel perfect. No side effects.”  Average life expectancy for men with the advanced disease is nine months. But one in five in a trial with 50 patients got an extra 24 months.  Dr Arun Azad of Melbourne’s Peter MacCallum Cancer Centre is running a trial involving 200 participants. He said at the American Society of Clinical Oncology conference in Chicago: “If the results are positive it really will change the landscape.”  Around 47,000 men a year are diagnosed in the UK, and experts say 5,000 may benefit.


thesun--2019-09-29--New treatment could help thousands of women beat one of the deadliest forms of breast cancer

THOUSANDS more women could beat one of the deadliest forms of breast cancer thanks to a new treatment.  Scientists found that a particular drug combined with chemo “turbocharges” patients’ immune systems.  This means people with “triple negative” tumours are more likely to see them disappear before they have surgery — boosting their chances of the disease never coming back.  And the treatment could be available on the NHS in the next two to three years.  Researcher Peter Schmid, from Queen Mary University of London, said: “The potential of this is massive — this approach could save thousands of lives.”  Presenting the data in Barcelona to the European Society for Medical Oncology, the professor said 1,200 women took part in the study.  Two-thirds were given the immunotherapy drug pembrolizumab with chemotherapy while the rest had chemo only.  A NEW ultrasound technology could pick up far more cases of cancer and cut the need for biopsies. At present, ultrasounds can help identify potential problems with key organs but are not sensitive enough to detect cancer. Scientists in Edinburgh say they can produce images with five to ten times the resolution of traditional methods. The process works by injecting tiny bubbles into the bloodstream and scanning organs.  For the first group, the chance of their cancer disappearing — known as a pathological complete response — before surgery was 65 per cent. For those given just chemo it was 51 per cent — a difference of 27 per cent.  Breast cancer affects around 55,000 Brits a year. The triple negative form accounts for one in six cases and kills a quarter of patients within five years. It is more common in those under 40.  Prof Charles Swanton, of Cancer Research, called the new treatment “a promising option”.


thetelegraph--2019-06-19--NHS plans for faster treatment of stroke will save thousands of lives

NHS plans for faster treatment of stroke 'will save thousands of lives'

Small hospitals must stop treating stroke emergencies in order to save thousands of lives, England's top doctor will today say.  The national medical director will say NHS trusts across the country must centralise services, so that victims get the right help sooner.  Professor Stephen Powis will say hospitals should follow a controversial model pioneered in London and Manchester, which is now saving around 170 lives a year.  In both cities, local stroke wards were closed, with ambulances instead taking victims not to nearest hospital, but to larger centres with access to brain scans, clot-busting drugs and specialist procedures.  As a result, the numbers dying or suffering long-term disability have fallen significantly.  Now health chiefs want to introduce the same changes across towns and cities in England, in the hope of saving more than 800 lives a year.  Professor Stephen Powis, NHS national medical director, said rolling out expert stroke teams will ensure thousands more people “survive and thrive”.  Speaking at the NHS Confederation conference in Manchester, he will say: “Introducing quicker access to better treatment for stroke in London and here in Manchester has saved hundreds of lives and we now want to see them rolled out across the whole of the country.  “As clinicians and as leaders we have a responsibility to drive this forward and to make the case for change, because we know that the prize is so great: thousands more people surviving and thriving after stroke.”


vox--2019-12-30--1,000 people sent me their addiction treatment stories. Here’s what I learned.

Since September, I’ve asked Vox’s readers to tell me their stories about drug addiction treatment for our Rehab Racket project. In less than four months, we’ve gotten a lot of responses — now more than 1,000.  The responses range from tragic stories about overdoses and early deaths to more optimistic ones about recovery. They’ve come from all over the US, including every state and Washington, DC, and Canada, the United Kingdom, Germany, Switzerland, South Africa, and Australia. Most of the stories come from addiction patients, although parents, spouses, siblings, and other relatives and friends of patients have sent in their stories as well.  From all of these experiences, one theme emerges: Addiction treatment in the US is expensive, hard to navigate, and far too often fails people. In the middle of an opioid epidemic that’s helped lead to more than 700,000 drug overdose deaths since 1999, America’s main defense against addiction is deeply flawed.  The stories we’ve published so far speak to these problems. Kim and Tim Blake spent more than $110,000 on treatment for their oldest son, yet much of the treatment was unhelpful and he died anyway. Michel Cote’s daughters went through $200,000 worth of treatment over a decade before they finally found something that worked for them. Nan Warren faces financial ruin after she lost her oldest son to addiction and has spent years trying to avoid the same fate for her surviving daughter.  Health insurance alone is not enough to protect patients from high costs and low quality. Maureen O’Reilly lost her son to addiction after their health insurance plan wouldn’t cover addiction care near home and he was sent to shoddy treatment facilities — some of which were later shut down — in Florida.  Most recently, Ian McLoone told me about how methadone helped him overcome opioid addiction — an example of how treatment can work if it’s evidence-based and supported, but also about the barriers McLoone faced due to the stigma and strict regulations around medications like methadone.  This is just the beginning. The Rehab Racket will last at least a year, possibly longer, and we’re less than four months in. With the end of the year approaching, though, I wanted to take a moment to reflect on what I’ve learned to this point.  Here are four lessons from the project so far — and where I plan on going next.  Just about everyone I spoke to reported at least some problems with getting addiction care covered — because they didn’t have insurance or because their insurer refused to cover the care they needed.  This is a big problem nationwide: Based on the National Survey on Drug Use and Health, federal researchers estimated that roughly 314,000 people in the US in 2018 needed drug addiction treatment but couldn’t get it because they didn’t have health coverage and couldn’t afford the costs. About 100,000 couldn’t get it because, while they had health coverage, it didn’t cover treatment at all or it didn’t cover the full cost. (There’s some overlap between the groups because participants were able to select multiple responses.)  A recent report by Milliman, a consulting company, found commercial insurance coverage of addiction treatment may be getting worse, at least relative to more conventional health care. It found that inpatient drug addiction treatment facilities were 10.1 times more likely to be out-of-network compared to medical or surgical inpatient facilities in 2018, up from 4.7 times more likely in 2013. There were similar and rising disparities for outpatient facilities too — making drug addiction treatment overall more expensive and harder to access.  Maureen O’Reilly experienced some of these problems firsthand. Her son, Ed Fahy, tried to get into addiction treatment facilities near home in New Jersey. But Horizon Blue Cross and Blue Shield, the family’s insurance plan administrator, refused to cover the programs. Fahy ultimately found addiction treatments that the health plan would cover in Florida, but there he got caught in the “Florida shuffle,” a quagmire of low-quality, weakly regulated addiction treatment centers and sober homes. He overdosed and died at a sober home that was later shut down by law enforcement.  The story reflects the problems with insurance coverage: Insurers often don’t want to pay for addiction treatment that can be fairly expensive, and they also don’t know what’s good care that’s worth covering.  O’Reilly is now suing Horizon and its behavioral benefits manager, Beacon Health Options. Horizon said in a statement that “the claims being made in [O’Reilly’s] lawsuit are untrue, unfounded, and unfair.” Beacon declined to comment.  O’Reilly is not alone. While reporting her story, I spoke to other family members of addiction patients who sued insurers. Some of these families have landed big public victories: In Wit v. United Behavioral Health, a federal court found United had illegally denied mental health and addiction treatment coverage to tens of thousands of patients. The ruling will likely be appealed, but if it succeeds, it could set a precedent for the industry.  Insurers, at least, seem to be aware of a problem. Many of them, including Horizon, Beacon, and United, have teamed up with the advocacy organization Shatterproof to develop better standards for addiction care.  All of this helps explain why US drug addiction treatment can be so expensive: Insurance is the system that’s meant to shield Americans from huge health care expenses, but it’s often not doing that for addiction care.  2) We know disturbingly little about addiction treatment in America  While reporting on addiction treatment in the US and asking experts about the evidence for addiction care, there has been one answer I’ve gotten used to hearing a lot: “I don’t know.”  It was a common response when I asked if certain addiction treatments worked. Many facilities, for example, now use equine therapy, in which patients connect with horses. But we have next to no research on whether this works. The same is true for wilderness therapy that emphasizes outdoors activities, which also has little reliable scientific evidence behind it.  It’s not that these methods are proven to not work. We just don’t know if they work. This is a very strange thing to occur in what’s supposed to be health care: Normally, treatments follow evidence. Patients usually don’t want doctors trying a bunch of stuff on them and hoping something works out.  But the lack of evidence for effectiveness is largely standard in addiction treatment. The vast majority of treatment facilities don’t even track real outcomes for what they do. Some of these facilities claim to do so, but they usually only use follow-up surveys that are riddled with bias and errors; for example, a patient can claim he hasn’t used drugs, and the survey taker will make no effort to actually verify that.  This is not just a failure of the industry, but of the law and regulators as well. The Centers for Medicare and Medicaid Services “has over 4,000 quality measures,” Tami Mark, a health economist at the research foundation RTI International, previously told me. “There are none for addiction programs — zero.”  At the same time, we have little indication about what kind of financial impact addiction treatment is having on American families. There’s just not much research on it.  Mark, along with the advocacy group Shatterproof, is now undertaking an effort to fix that by creating a sort of “Yelp for rehab” that will try to guide patients to evidence-based care with the use of provider surveys, insurance claims, and user evaluations, starting next year in at least some states.  For now, though, patients are largely left in the dark about whether a treatment facility really is following the evidence.  3) We don’t make good use of what we do know about addiction treatment  There are, however, some things we know about addiction treatment. The problem is we don’t make good use of this information.  For example, the research shows that medications for opioid addiction — methadone, buprenorphine, and naltrexone — are very effective: Studies show they cut the mortality rate among opioid addiction patients by half or more and keep people in treatment better than non-medication approaches.  Yet in the US, the majority of addiction treatment facilities don’t offer any medications. According to federal data, only 42 percent of the nearly 15,000 facilities tracked by the Substance Abuse and Mental Health Services Administration (SAMHSA) provide any type of medication for opioid addiction. Less than 3 percent offer all three federally approved medications.  In fact, treatment centers may even discourage the use of medications due to the mistaken belief that the medications are simply replacing one drug with another. Ian McLoone said one of the rehabs that he went to, RS Eden in Minnesota, pushed him to get off methadone. It was only thanks to an intervention from his mom that McLoone agreed to stay on it.  That “was by far the best decision I could have made,” McLoone said. “Then, I was able to finish rehab, get a job shoveling asphalt that summer, working my ass off. I got into grad school. I got through grad school. And I was able to really have the life and the family and the career that I had dreamed of. Methadone absolutely facilitated that and helped to make that possible.”  The hurdles to evidence-based treatment reflect not just the industry’s decisions but public policy choices too. Needle exchanges have decades of evidence behind them and support from the American Medical Association, Centers for Disease Control and Prevention, and World Health Organization, but they remain illegal in much of the US. Prescription heroin, in which people with treatment-resistant addiction are provided heroin, is supported by major studies — including an extensive review of the research by the RAND Corporation — but it’s received next to zero attention from US policymakers.  The point here is straightforward: If we don’t even use what we know works, then we’re not going to get very far in addressing the opioid crisis or addiction in general.  When writing about anything addiction-related, there’s an explanation I can expect for just about any problem: stigma. Indeed, it’s the thing that ties the lessons on this list together.  This has stuck with me ever since I went to Vermont in 2017 to see how the state built up its addiction treatment system. When I asked officials about the hurdles to building up the new system of care, I expected to hear a lot about money (a common problem in policymaking). But officials told me, in fact, that money wasn’t that big of a deal. Instead, the main barrier was stigma around addiction, fueled by the notions that addiction is a moral issue — not a health issue — and people with addiction don’t deserve public resources. Once that stigma was overcome, the money came easily.  As I’ve written before, this is the core problem with how the US approaches drug addiction in general. It’s the lasting impact of the country long treating addiction as a moral failure — one that was relegated largely to the criminal justice system to handle — instead of a medical condition that warrants public health resources.  I’ve heard the same, time and time again, with the Rehab Racket project. Why don’t we know much about addiction treatment facilities and their outcomes? How can addiction treatment remain so expensive and so ineffective for many? Why don’t we use the evidence we do have for treatment? Why don’t insurers adequately cover addiction treatment? There is one common answer: Stigma leads to a skewed perception of addiction, or apathy about it, so the public, policymakers, and other actors don’t do enough to change the status quo.  “We’ve had these problems for a very long time,” Keith Humphreys, a drug policy expert at Stanford, told me. “I’ve heard politicians making speeches about, you know, outrage that people with heart disease are waiting in the [UK’s National Health Service] or the quality of cancer care or the dirty conditions at Walter Reed. I have never heard a politician say, ‘I am outraged at the quality of alcoholism care in our community. This is not acceptable.’ Never. I mean, it’s almost unthinkable.”  That’s how these problems keep going: Until enough people overcome their stigma toward addiction, they’re not going to give the problem the attention it requires to solve.  One bit of good news is that a lot of people are ready to combat this stigma. The fact Vox has received more than 1,000 responses to its survey, with most including their full names and personal information, demonstrates as much.  The Rehab Racket project will last well into 2020, and the responses my editors and I have gotten to our survey have given us several leads and ideas we want to continue exploring.  One story I’ll tackle soon is the dominance of the 12 steps. This treatment approach, built around the teachings of Alcoholics Anonymous, is helpful to some, and it has solid empirical approach when it comes to alcohol addiction. But there’s not solid evidence for it when it comes to other kinds of drugs, and some people don’t like the approach at all — yet it’s often what’s foisted on patients in American addiction treatment.  Emilie and Danika Cote, for instance, previously told me that they didn’t like the 12 steps’ emphasis on submitting to a “higher power.” As Emilie, who’s “not religious at all,” said, “I had a really hard time with the whole God thing.” Danika agreed.  But it took roughly a decade for them to finally find a treatment clinic that didn’t drive them to the 12-step approach and that they otherwise liked.  I’ve heard similar things from dozens, if not hundreds, of other patients. Why is that? And what can be done about it?  Another is the financial problems surrounding methadone treatment. Methadone is one of the most well-studied treatments for opioid addiction, with strong evidence behind it. But I’ve now heard from multiple patients that insurance doesn’t cover it, or methadone clinics will refuse insurance — leading to costs that can be as high as $600 a month. Given that some patients can take methadone indefinitely, the cost can add up very quickly. I want to figure out why that is, especially given that, as journalist Alison Knopf has written, methadone itself can be less than $1 a dose.  Finally, I’d like to zoom in on some of the more specific issues in addiction treatment. That will involve shining a light on bad actors in addiction treatment and holding them accountable, but also digging into the examples of addiction treatments that do work and what they involve.  One of the outrageous things about the failures I’ve uncovered so far is that we know that drug addiction treatment can work when it’s evidence-based, accessible, affordable, and flexible. This makes all of the problems in the field even more inexcusable. Hopefully, by putting a spotlight on the places that are getting things right, we can help set an example that the rest of the industry and policymakers can follow.  We want to hear from you  We’re shining a light on the problems with the addiction treatment system in America and how they’re affecting people, financially and otherwise, every day. Please help our reporting on the high cost of addiction rehab by sharing your story with us. You can also sign up for our email newsletter to get updates on the project.  If you or someone you know needs addiction treatment, you can seek help online at FindTreatment.gov or by phone at 1-800-662-4357. If you need more information, Vox put together a guide for how to find good addiction treatment.


yna--2019-11-01--Researchers develop method that can find treatment for multidrug-resistant TB

It said in the past two years over 3,700 compounds that could be used against TB were tested on artificially infected macrophages, with six candidate materials being shortlisted. Of these, one substance, called 10-DEBC, showed considerable effectiveness in combating TB while not being toxic to the white cell itself or the body as a whole.


birminghammail--2019-05-15--How alpaca therapy is being used to help a string of conditions - with amazing results

Victoria Barrett is used to the odd raised eyebrow when she introduces her co-therapists at the counselling service she runs.  She uses alpacas - south American animals often confused with llamas -  to help people of all ages come to terms with trauma, mental health issues, and to boost confidence and self-esteem.  And while it is far from conventional, the unusual treatment can be remarkably effective.  Victoria says: “Working alongside the animals offers a different way of exploring difficult and sensitive issues as clients may find it easier to express their feelings and recount painful experiences.  “It is well documented that holding and stroking animals can reduce blood pressure, lower pulse rates and alleviate feelings of tension and anxiety.  “The animals offer attention and unconditional acceptance. They are responsive and don’t mind who you are or what you look like.  So they are ideal for working with people who find themselves confused or threatened by human relations, many clients feel safer and less threatened around animals. A child I work with who is in foster care struggles with empathy and social cues. But being with the alpacas has changed everything.”  Victoria, 55, came up with the idea for alpaca therapy after working as a paramedic for more than 30 years.  Alongside her frontline role with the West Midlands Ambulance Service, she was also a trained counsellor, helping people battling depression and mental health problems for almost twenty years.  She relaxed by caring for alpacas and llamas at her farm in Worcester. So when she decided to leave the ambulance service, she realised she could combine her experience and skills with her hobby to help other people.  After some research it became clear she was in the perfect position to offer therapy using her alpacas and llamas. The alpaca is a species of South American camelid similar to, and often confused with, the llama. However, alpacas are often noticeably smaller than llamas. Victoria retrained and now offers a Camelid Assisted Therapy programme. It uses the animals as “co-therapists” alongside a human therapist in a structured treatment plan. Clients come from all walks of life including children in the care system.  They come to nurture and care for the animals, which in turn builds self-esteem, confidence and morale while reducing anxiety and depression.  Meeting the animals it is clear just what makes them so special. With their large baleful eyes, and soft fluffy fur, the alpacas are incredibly endearing. They are also intelligent. The llamas are much bigger but also captivating. It is obvious why it might be easier to open up in their company.  For youngsters with behavioural issues, just being around the animals and learning to feed or groom them induces calm. “I have a group of autistic children who come and the alpacas won’t respond if the child scares them so they quickly learnt to adjust their behavior.”  Victoria adds: “The alpacas can encourage our nurturing and empathic traits and, for survivors of abuse, offer an opportunity for ‘safe touch’.”  Just one person whose life has been transformed is Daisy, 33, who suffers from multiple personality disorder following years of neglect and abuse as a child.  Daisy remembers her first encounter with one of the animals. “I remember looking up and seeing this huge llama. I thought I would feel scared but I actually felt calm. He nuzzled me and it made me smile. I had not smiled in a very long time. Meeting Eddie gave me my smile back. We have such a special bond and I love him.  “Four years ago I was having a very traumatic time. I also had become a double amputee due to complications from diabetes and the neglect.  Victoria is an amazing woman and being around the llamas and alpacas is the only thing that has really helped my mental health. I can’t express how uplifting they are.”  For more Stories of Local Pride, click here


eveningstandard--2019-02-06--CBT What is Cognitive Behavioural Therapy and how can it help anxiety depression and pain

CBT: What is Cognitive Behavioural Therapy and how can it help anxiety, depression and pain?

With society's increasing awareness surrounding mental health, more and more of us are turning to therapy for problems such as depression and anxiety.  Over 900,000 people now receive psychological therapy each year through the The Improving Access to Psychological Therapies (IAPT), a government scheme rolled out in 2008 intended to improve accessibility to mental health services.  In 2013-14, Cognitive Behavioural Therapy (CBT) became the most popular form of therapy in the IAPT programme, accounting for 38 per cent of total appointments.  But why is CBT in such high demand?  According to Doctor Elena Touroni, Psychologist and Clinic Director of Chelsea Psychology Clinic, it’s down to two reasons:  So, is it worth exploring?  We interviewed Dr Touroni to find out.  CBT therapies are based on the theory that thoughts, feelings, what we do and how our body feels are all connected. If we change one of these things, we can alter the others.  Dr Touroni elaborates: “At the heart of psychological distress lie negative beliefs that we have about ourselves, the world and other people. These are often distorted and cause us suffering.  “CBT tries to help us understand how these beliefs have developed and challenge our moment by moment negative thoughts in order to live a fuller and more meaningful life.”  According to Dr Touroni, CBT is effective in the treatment of many conditions including:  "However, it’s an excellent therapy for people who like practical coping strategies and a problem solving approach. It’s very effective at reducing symptoms.  “CBT is a great place to start with if this is your first time receiving psychological therapy.”  Dr Touroni recommends CBT to “people with mild to moderate difficulties.”  “The more complex and severe someone’s difficulties and the more attempts someone has previously had at having therapy without finding it particularly effective, the less likely it is that CBT will help.”  "Mindfulness based cognitive therapy (MBCT) can be used to treat physical and chronic pain," says Dr Touroni.  “CBT can offer a range of tips on sleep hygiene routines that can assist getting a good night’s sleep and breaking unhelpful habits,” she adds.  Although some therapists might offer CBT to couples, Dr Touroni believes that “CBT isn’t the most effective model when working with relationship difficulties.”  “CBT can offer couples some practical strategies to manage their relationship more effectively, however schema therapy is a more sophisticated approach that is far more effective in shifting entrenched relational dynamics.”  According to Dr Touroni, you should be looking for a psychologist first and foremost.  “Psychologists have a minimum of seven years of training across multiple therapy models with CBT being a key one.  “CBT therapists can often become accredited within two years and only deliver CBT, therefore they cannot adapt the approach to fit the needs of an individual as flexibly.”  “A qualified psychologist will be registered with the Health Professions Council,” explains Dr Touroni.  “In an ideal world, you could look for a qualified psychologist who is also an accredited CBT therapist.  “So, look out for the letters BABCP as well. This is the accrediting body for cognitive behavioural therapists.”  “The NHS offers CBT in some GP practices in primary care and also through a big initiative called Improving Access to Psychological Therapies (IAPT) which has made CBT widely available for people with a range of emotional difficulties.”  “It is very hard to say as a general rule how many you might need as this varies depending on the nature and severity of patient’s mental health.  Dr Touroni suggests: “Practice monitoring your thoughts when faced with a situation that provokes strong feelings in you.”  Here are her top CBT tools:  Keeping a simple record of situations that trigger difficult emotions and breaking down what the thoughts and feelings are can be a very powerful technique that can help us develop self-observation.  Noticing all the ways that our mind might engage in cognitive errors and distortions can help put a triggering situation into a more helpful, realistic perspective.  Finding alternative ways of thinking about the situation that open up a range of possibilities can be very effective in reducing negative feelings.  Relaxing one muscle group at a time until your body is in a state of relaxation. Sometimes it is helpful to do this with the aid of a relaxation tape that can guide you through such an exercise.  Dr Elena Touroni is the Clinic Director of Chelsea Psychology Clinic, a private psychiatry and psychology clinic in London. She is also a co-founder of My Online Therapy in the UK, which aims to make high quality therapy available in the form of a virtual clinic.


eveningstandard--2019-11-14--The most expensive therapy retreats in the world

You’re met at the door to your private jet by a tall, impeccably dressed Swiss man who whisks you through security and into a blacked-out Bentley.  After a short drive, you arrive at a residence beside Lake Zurich, where your room comes complete with suede beds flanked by abstract expressionist paintings. There’s an Apple TV, Bose speakers and a well-stocked kitchen ready to be used by your personal chef. A maid, butler and secretary will also be on call throughout your stay. There will be no other guests while you’re here and, if you want, no one ever has to know you’ve been. Luxury getaway for the super-rich? Not quite. Welcome to Paracelsus Recovery, the £62,500-a-week Swiss therapy centre designed to cure the world’s wealthiest of everything from eating disorders to addictions and OCD in an ultra-exclusive and discreet setting.  Once guests have settled in, they are driven to a top-of-the-range Swiss clinic for a comprehensive three-hour physical exam, that involves being hooked up to a Bicom machine for biochemical testing, a vitamin C drip and a full psychiatric assessment. Forget group therapy; here, a team of psychologists, nurses and addiction specialists tend to just a single patient at a time for 10 hours a day (one of them is available 24 hours a day, and sleeps next door to the client). Total privacy is the clinic’s USP, though actors, supermodels and members of the world’s royal families are said to have been treated here.  On average, they stay for four weeks, at a cost of £230,000. ‘There’s not a typical client, but what they all have in common is that they are looking for the very, very best treatment available and require 100 per cent confidentiality,’ says managing director and co-founder Jan Gerber. ‘It’s a 360-degree approach with seven-star service, which is what our clients are used to. The luxury we have is not about spoiling someone. It’s just their reality.’  Paracelsus, which also offers acupuncturists, yoga, a personal trainer and a nutritionist, is far from the only therapy centre boasting radical treatment programmes for the radically rich. Indeed, more and more centres are being opened to cater for the upper echelons in search of a private place to detox or reset (there’s a reason Succession’s Kendall Roy heads to a discreet clinic in Iceland to ‘decompress’).  Starting at £72,000 a week, real-life Roys can check in to The Kusnacht Practice, also on Lake Zurich, which calls itself the ‘best luxury mental health treatment centre in the world’. It’s also one of the most exclusive — only 30 people gain entry each year. George Michael was said to be one of them, spending £2 million over six months in 2015 to treat addiction and depression. Other high-flyers said to have passed through its luxury villas (complete with views over the Alps) include royals and heads of state, who come for the team of 18 world-class psychiatrists who treat everything from burnout to depression and co-dependency issues. Their methods include transcranial magnetic stimulation, in which electromagnetic pulses are delivered to the brain to treat low moods. Full-spectrum blood tests, electrocardiograms, renal, coronary and circulatory examinations and GI tests, as well as detoxes, are also available.  There’s also the similarly exclusive Clinic Les Alpes, a £25,000-per-week centre housed in an old Swiss Alpine chateau, where treatment includes art and music therapy, adventure therapy, clinical psychology and sleep hygiene (a spa, high-end restaurant and ‘serenity space’ are also part of the package). Beyond Switzerland, the Sonoran Desert is also fast becoming a byword among billionaires who want to get help. The Meadows in Wickenburg, Arizona, is set on a 35-acre former dude ranch, and has become famous for its ‘Gentle Path’ sex addiction treatment programme (Tiger Woods, Kevin Spacey and Harvey Weinstein have all reportedly checked in), as well as treating anxiety and depression. Clients are given colour-coded name badges depending on the issue they’re here to address (sex addiction is red), but the therapeutic timetable is similar for all, including carrying around rocks to symbolise the burden you’ve been holding on to for decades, hugging horses during ‘equine therapy’ and taking a walk through a ‘serenity circle’.  The 45-day treatment programme costs £42,000, but the aesthetic at The Meadows is deliberately spartan, with shared bedrooms and bathrooms. Alcohol and narcotics are banned, as are caffeine and sugar. Tight or revealing clothing is also not permitted. ‘The first night I was there, I was stuck in this tiny little room with two complete strangers,’ Sean Brock, a highly successful American chef, has said. ‘I was listening to my room-mates detoxing, and thinking, woah, this is not the luxury resort I was expecting.’  A more pleasant experience may lie in alternative therapies, a route increasingly being favoured by one percenters. ‘Many of my clients have anxiety, insomnia or depression, or they just feel that something is missing and they want a fast track to healing,’ says Lucyne Pearson, who trained as a psychotherapist and worked at The Priory, before learning shamanism and energy medicine and setting up her own retreats in Europe and Central America. ‘They live busy lives and they want to get to the root cause of why they’re struggling.’ She leads shamanic ceremonies of around 50 people at the Rythmia Life Advancement Center in Costa Rica, which is the only medically licensed ayahuasca facility in the world.  ‘You’re opening yourself up to another level of consciousness, but you’re around doctors and nurses in a luxurious spa setting with mud treatments,’ adds Pearson. It costs around £5,000 a week, plus treatments. ‘I’ve seen people try pharmaceuticals and years of therapy and have little progress, and then they use plant medicine and miracles happen overnight.’ Pearson has just returned from a sold-out, four-day psychedelic-assisted therapy workshop in Amsterdam, where psilocybin truffles are given out ‘with the support of sober healthcare professionals’ as a tool for subconscious reprogramming.  Things, however, might not have to be so far-out — or away. On a quiet street in Mayfair you’ll find Addcounsel where, for £45,000-£150,000 a week, clients are treated by a personalised one-to-one treatment squad. Addcounsel deals with no more than three clients at a time, who will never see one another during treatment. They are each given large houses and apartments in Belgravia, Knightsbridge and Green Park and the fee covers 24-hour care from a crack team of specialists including a psychiatrist, a yoga teacher, a ‘neuro-feedback practitioner’ and a chef. These experts are at the top of their game, such as David Behrens, a mindfulness coach who spent 27 years living as a monk in India, and visits twice a week to teach the patients how to meditate.  To begin, you’ll have a top-to-toe health check — CT scan, blood tests, ultrasounds, liver check, along with functional medical tests which assess nutrition, deficiencies and gut health. All treatment is overseen by your private GP who liaises with the best psychiatrists, psychologists and counsellors in the world. Two nurses on a five-day rotating shift will be with you every waking moment, supervising your treatment. A recovery manager — companion, confidante, opera buddy, tennis partner, midnight Scrabble opponent, whatever you fancy — is also there to keep you on the straight and narrow.  ‘We’re a new concept in the UK,’ says Paul Flynn, co-founder and chief executive of Addcounsel. ‘Many of our clients come from the Middle East, but a lot live in London. For them the financial commitment isn’t the issue, it’s the personal commitment.’ Flynn says that 90 per cent of the people they work with have some kind of addiction — whether it’s Xanax, their jobs or the video game, Fortnite. Most enquiries come via word of mouth. Its youngest client is 16, but most are men over 35. ‘The people we treat tend to be incredibly demanding and specific,’ says Flynn. ‘So you have to be strong when you tell them there are no quick fixes.’  Indeed, the question will always remain: can money really buy you happiness? Hilda Burke, a Hammersmith-based psychotherapist is not so sure. ‘This kind of intense work wouldn’t be suitable for everyone,’ she says. ‘People who go on these retreats tend to be Type A — they want results and they want them fast. But then they go back into their high-powered existence where they have no time to reflect — where does it go? Has your life really changed? ‘Most of us need time to unfurl.’


freedombunker--2019-06-03--Why This New Gene Therapy Drug Costs 21 Million

Why This New Gene Therapy Drug Costs $2.1 Million

Last year, drug producer Brainstorm Cell Therapeutics received significant criticism for attempting to offer patients access to an experimental treatment procedure for amyotrophic lateral sclerosis (often called ALS or Lou Gherig’s disease) for $300,000. A drug recently approved by the Food and Drug Administration dwarfs this price.  A new gene therapy drug named Zoglensma became the most expensive drug in the world, costing patients over $2.1 million for one-time use. Zoglensma joins a small (and outlandishly expensive) group of treatments called gene therapy drugs. Gene therapy uses actual genes to treat or prevent diseases.  Before Zoglensma’s approval, its predecessor treatment, named Spinraza,Nearly one in eight-thousand people carry the SMA gene. SMA is considered the most common genetic cause of death in infants. cost patients $750,000 for the first year’s treatment and an additional $375,000 for each additional year. Other FDA approved gene therapy treatments cost between $375,000 and $875,000.  Zoglensma treats spinal muscular atrophy (SMA), a rare genetic disease that restricts the nervous system’s ability to coordinate voluntary muscle movement. Nearly one in eight-thousand people carry the SMA gene. Tragically, the disease typically affects infants. In severe cases, infants with SMA are unable to sit upright, suffer from severe joint pain, and ultimately die from respiratory failure. SMA is considered the most common genetic cause of death in infants.  Despite providing rare and promising results, many gene therapy drug prices place them beyond financial reach for many families in tragic situations. But why are these treatments so expensive?  Unlike other drugs regulated by the Food and Drug Administration, gene therapies are subject not only to the regulatory structure of the FDA, but also to the Office of Biotechnology Activities, and the Recombinant DNA Advisory Committee. Excessive regulatory oversight creates an elongated and expensive route to approval.Gene therapies provide those with rare, serious, and possibly terminal conditions with the ability to significantly improve their quality of life. By one estimate, an approved gene therapy drug costs nearly $5 billion (five times as high as the average cost of FDA approval). Covering these costs is difficult because many of the diseases gene therapy drugs treat are extremely rare.  Gene therapies are also often tailored to specific individuals, making completing clinical trials for FDA approval very challenging and costly. One estimate holds it costs drug providers nearly $1 million per clinical trial participant. For reference, the first phase of the FDA approval process typically requires twenty to eighty participants. The third and largest phase usually requires at least 3,000 participants.  Gene therapies provide those with rare, serious, and possibly terminal conditions with the ability to significantly improve their quality of life. When pioneering medical breakthroughs provide such an opportunity, it is imperative to provide treatment where possible. Existing regulations serve to hinder access to treatment by making it prohibitively expensive. Drugs costing over $2 million are the latest symptom of the overregulation disease.  This article is republished with permission from the Independent Institute.


naturalnews--2019-09-17--Music therapy improves psychological well-being quality of life of terminally ill patients

Music therapy improves psychological well-being, quality of life of terminally ill patients

(Natural News) Researchers from Ewha Woman’s University in South Korea investigated the positive effects of music therapy, especially in relieving pain and improving the quality of life of terminally ill patients. The results of their study were published in the journal Alternative and Complementary Therapies.  Based on these findings, the researchers concluded that a structured singing program designed to meet the individual needs of patients with serious conditions can help improve their psychological well-being while they receive hospice care.  Oh JE, Chong HJ, Kim AJ. CASE STUDIES OF A STRUCTURED SINGING EXPERIENCE FOR THE PSYCHOLOGICAL WELL-BEING OF HOSPICE PATIENTS. Alternative and Complementary Therapies. 01 April 2019;25(2):85–94. DOI: 10.1089/act.2019.29211.jeo


npr--2019-04-17--Gene Therapy Advances To Better Treat Bubble Boy Disease

Gene Therapy Advances To Better Treat 'Bubble Boy' Disease

David Vetter, pictured in September 1982 inside part of the bubble environment that was his protective home until he died in 1984. Today most kids born with severe combined immunodeficiency are successfully treated with bone marrow transplants, but researchers think gene therapy is the future. **AP** ****hide caption****  ****toggle caption****  AP  David Vetter, pictured in September 1982 inside part of the bubble environment that was his protective home until he died in 1984. Today most kids born with severe combined immunodeficiency are successfully treated with bone marrow transplants, but researchers think gene therapy is the future.  AP  Sometimes rare diseases can let scientists pioneer bold new ideas. That has been the case with a condition that strikes fewer than 100 babies a year in the United States. These infants are born without a functioning immune system.  The disease is called [severe combined immunodeficiency](https://www.niaid.nih.gov/diseases-conditions/severe- combined-immunodeficiency-scid), or SCID. "It was made famous in the mid '70s when the 'Bubble Boy' was described in a documentary, and I think it captured the imagination of a lot of people," says [Matthew Porteus](https://profiles.stanford.edu/matthew-porteus), a pediatrician at Stanford University.  David Vetter was the boy who spent most of his short life [inside a plastic bubble to protect him](https://primaryimmune.org/living-pi-explaining-pi- others/story-david) from infection. He died at age 12 in 1984.  All babies born in the United States are now screened for this condition, and the best treatment today — a bone marrow transplant — succeeds more than 90 percent of the time. The disease remains a source of great interest to researchers.  "This is one of those diseases in which there's probably more doctors and scientists studying the disease than patients who have the disease," Porteus says.  In the 1990s, European scientists actually cured SCID in some patients, using a technique called gene therapy. This process involves removing defective blood cells from a patient, inserting a new gene with the help of a virus and then putting the cells back into the body. Those cells then build up the patient's immune system.  At first, this treatment in the 1990s and early 2000s looked really promising.  "Of the 20 patients, they all had immune recovery," says [Donald Kohn](https://stemcell.ucla.edu/member/kohn), an immunologist at UCLA's Broad Center of Regenerative Medicine and Stem Cell Research. "But, over time, five of them went on to develop a leukemia."  He says 18 of those original patients are still alive today, but the leukemias put an understandable pall on the whole field of gene therapy.  Scientists went to work to figure out how to inject new genes into cells without triggering leukemia, a cancer of blood cells. And they think they've succeeded.  Over time, there has been gradual improvement in gene therapy. The latest advance, [reported in the __ New England Journal of Medicine](https://www.nejm.org/doi/full/10.1056/NEJMoa1815408) on Wednesday, details a study of eight infants who have a type of SCID called SCID-X1.  The gene to correct the problem was inserted into a modified version of HIV, the virus that causes AIDS. That engineered virus can't cause AIDS, and it has been further tweaked to reduce the risk that it could trigger leukemia.  Gene therapy has been used successfully over the past decade. Scientists at St. Jude Children's Research Hospital in Memphis, Tenn., modified the procedure for SCID by giving the infants a short course of chemotherapy before introducing the new gene. This helped the new cells take up permanent residence. The babies developed apparently healthy immune systems, according to the new study.  "I am thrilled to see these outstanding results," says Ewelina Mamcarz, a transplant physician and first author on the new paper.  "To be able to see these babies in my clinic now as toddlers is very rewarding," she says. "They live normal lives. They aren't any different from my daughters." Two more infants have been treated since the paper was prepared for publication, the team says.  Standard treatment for SCID is a bone marrow transplant. But that procedure often restores only part of a child's immune system. As a result, patients require monthly infusions of antibodies called immunoglobulins. [Jennifer Puck](https://profiles.ucsf.edu/jennifer.puck), a pediatrician at UC San Francisco and a collaborator in the latest study, says infants who got the gene therapy don't need that medication.  These children are "growing normally, they're getting colds like everyone else and they're getting over infections — so I would say that is a cure," Puck says.  Of course, she adds, they'll be watched carefully for signs of leukemia and to see if the effect of the therapy is wearing off.  In her view, the key to treatment is finding these children early — through newborn screening — before they start to get life-threatening infections. Screening for SCID is now done throughout the U.S., though its introduction was gradual and state by state.  Before screening was instituted, these children used to show up in the hospital with life-threatening infections, "and now we're seeing happy, bouncy little newborns who just look perfectly normal and they're never sick," Puck says. "Sometimes their families don't understand just how profoundly affected their immune system is."  St. Jude hopes to commercialize its treatment. It has an exclusive licensing agreement with Mustang Bio to develop a product. A similar treatment, called [Strimvelis](https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis), has already been approved by Europe's equivalent of the Food and Drug Administration. It targets a different mutation that causes SCID, but the technique is very much the same, including the brief dose of chemotherapy.  This latest advance is not only encouraging news for these rare patients. SCID is a test case for all those scientists working to develop better gene therapy techniques.  For example, instead of inserting a healthy gene into blood cells, [Porteus](http://med.stanford.edu/porteuslab.html), the Stanford pediatrician, has used a precision gene-editing technique called CRISPR [to correct the genetic error](https://www.nature.com/articles/s41467-019-09614-y) in blood cells drawn from SCID patients. It works in the laboratory, "and this really sets the stage then for a clinical trial, hopefully in the next 12 to 18 months," he says.  All this makes the leukemia setback from the 1990s feel like a fading memory. Kohn at UCLA says that for more than a decade, it seemed that the field of gene therapy was a dead end.  Clearly it has made a comeback and has been used to treat other rare diseases, including [adrenoleukodystrophy](https://www.nejm.org/doi/full/10.1056/NEJMoa1700554), a neurological condition better known as Lorenzo's Oil disease, after a [1992 movie](https://www.imdb.com/title/tt0104756/) that highlighted a boy with the condition and his parents' hunt for a cure.  Now, with continual advances in treating SCID, "it's just nice to see another success for gene therapy," Kohn says.  _You can contact NPR science correspondent Richard Harris at_[ _rharris@npr.org_](mailto:rharris@npr.org) _._


politico--2019-05-24--FDA approves 2M gene therapy for rare birth disorder

FDA approves $2M gene therapy for rare birth disorder

Novartis announced today that it will sell a newly approved gene therapy for $2.125 million for a single infusion, making it the most expensive single-dose drug ever. The Swiss drugmaker said it will implement a five-year payment plan for an annual cost of $425,000.  Novartis’ Zolgensma, which FDA approved earlier in the day, is designed to replace the defective or missing gene causing spinal muscular atrophy or SMA, a rare but often fatal disease. Many children born with it die by their second birthday, and it is the leading genetic cause of infant mortality in the country.  The announcement came shortly after President Donald Trump appeared on television boasting about lowering drug prices.  The nonprofit Institute for Clinical and Economic Review today estimated a cost-effective price for the new medicine would be $1.1 million to $1.9 million per treatment in one of its analytic models. Executives said in a call with media that the $2 million price falls within the upper bounds of ICER's benefit ranges.  "Were committed to ensuring children have access to this product," Novartis CEO Vas Narasimhan said.  Critics noted that the bulk of Zolgensma's development was done by AveXis, which Novartis bought last year.  “The question in drug pricing isn’t how much is a life worth; it’s what makes a fair return on an investment in R&D and an accessible price,” said David Mitchell, president of Patients for Affordable Drugs, an advocacy group.  SMA is believed to affect about one in 10,000 people, though not all forms of it are fatal to children.  Novartis said in a statement that the $2.125 million price tag is half the current 10-year cost of Spinraza, the only other approved SMA treatment, estimated at $4.1 million, and half the cost of medicines for other rare pediatric diseases.  Biogen’s Spinraza was cleared by the FDA in 2016 and sold at a $750,000 list price in the first year, then $375,000 annually for regular infusions. Other countries have balked at the price, in some cases refusing to cover it. England's National Health Service this month struck a deal with Biogen to cover the drug for a limited time at an undisclosed price to collect data.  Novartis executives said Friday that besides the five-year payment model, the company is exploring an outcomes-based contract with certain payers, doling out rebates to return some of the cost of the treatment if a patient dies or goes on a ventilator permanently.  But executives said certain laws that cover government health plans are constraining how creatively they can construct those deals. In particular, a law mandating that a drugmaker give Medicaid the lowest or “best” price it negotiates with payers limits the size of rebates in the outcomes-based plans.  “We’re continuing to work and advocate with the federal government to put in place relevant reforms or relevant interpretations of current guidances to enable us to use these novel models we’re talking bout here, either pay-over-time or outcomes-based payments,” Narasimhan said.


theguardianuk--2019-02-18--Gene therapy could treat rare brain disorder in unborn babies

Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains of foetuses in the womb.  The treatment, which has never been attempted before, would involve doctors injecting the feotus’s brain with a harmless virus that infects the neurons and delivers a suite of molecules that correct the genetic faults.  Tests suggest that the therapy will be most effective around the second trimester, when their brains are in the early stages of development.  “We believe that this could provide a treatment, if not a cure, depending on when it’s injected,” said Mark Zylka, a a neurobiologist at the University of North Carolina.  The therapy is aimed at a rare brain disorder known as Angelman syndrome, which affects one in 15,000 births. Children with the condition have small brains and often experience seizures and problems with walking and sleeping. They can live their whole lives without speaking a word.  Zylka said children with Angelman syndrome can have such severe sleeping difficulties that parents can feel they must lock them in their rooms at night to prevent them from getting up and having accidents around the house.  Healthy people tend to have two copies of every gene in the genetic code, one inherited from their mother and the other from their father. But both copies are not always switched on. For normal brain development, the mother’s copy of a gene called UBE3A is switched on, while the father’s copy is silenced.  In Angelman syndrome, the mother’s copy of UBE3A is either missing or mutated, meaning there is no UBE3A active at all. Zylka’s therapy uses a version of a powerful gene editing tool known as Crispr-Cas9 to switch the father’s copy back on.  Studies in mice show that when the gene therapy is given in the womb, it activates UBE3A in key parts of the brain. Speaking at an American Association for the Advancement of Science meeting in Washington DC, Zylka said the therapy switched the gene on in the cortex, which is important for cognition, the hippocampus, which is crucial for learning and memory, and the cerebellum, which controls movement.  Follow-up work showed that it worked in human brain cells grown in a dish, too. “It really does raise the possibility that this gene therapy might be usable in humans,” Zylka said.  No one has ever developed a gene therapy that is injected directly into the brains of foetuses. But Zylka believes that if future work goes well, his therapy could be an important first that paves the way for treating other, more common disorders, such as certain forms of autism. “For many of these neurodevelopmental disorders, where the brain has been developing inappropriately because of a gene mutation, the best time to intervene is probably going to be early, very early,” he said.  Angelman syndrome can be detected as early as 10 weeks after conception with a test that picks up DNA from the foetus in the mother’s blood. But there are no treatments available for women who are told their baby has the disorder. “People are getting this diagnosis, and there’s nothing to be done, they have to make pretty difficult decisions at that point,” Zylka said. “This may give them another option.”


theindependent--2019-01-23--Teenage boy with rare brain tumour to receive pioneering proton beam therapy at UK treatment centre

A 15-year-old with a rare brain tumour is to undergo pioneering proton beam therapy at the UK's first dedicated treatment centre.  Mason Kettley, from Angmering in West Sussex, will receive the highly targeted therapy which helps shrink tumours and cuts the risk of side-effects.  It will be carried out by the Christie NHS Foundation Trust in Manchester – home to the world's newest proton beam therapy centre.  Mason is one of the first patients to undergo proton beam therapy in the UK and the first to go public.  Until now, British patients needing the treatment had to travel to countries including the US.  He was diagnosed with an inoperable tumour in October after suffering headaches and failing to put on weight.  Doctors found that the tumour was growing in a critical part of his brain but could not operate due to the risk of causing blindness and damage to vital brain tissue.  Mason, who wants to work as a doctor specialising in tumours when he is older, said he did not initially have many symptoms.  He said: "I had some headaches and stomach pains and usual things, and got check-ups at the doctors.  "My mum said, 'he's not gaining weight or growing', and eventually, when we moved last year, we saw doctors who told us to go to Worthing Hospital for an MRI scan.  "We had the scan and the result showed it was a tumour."  Following a biopsy and an operation to insert a shunt, doctors referred Mason's case to a national panel of experts.  They decided that his tumour – known as a benign pilomyxoid astrocytoma – made him a suitable candidate for proton beam therapy.  Mason, who lives with mother Cally, stepfather Ryan and four siblings – Taylor, 20, Logan, 10, Scarlett, seven, and Elijah, four – said he feels apprehensive about the treatment.  He said: "I'm a bit nervous because the machine is intimidating because of its size.  "It's a bit nerve-wracking but this is a better choice than chemo because it's more effective. Because of my age, (doctors) thought radiation would be a better choice.  "Their goal is to stabilise the tumour. It may shrink, but they are aiming to stabilise it."  Mason will have treatment Monday to Friday for almost six weeks - 28 sessions in total.  A specially made radiotherapy mask has been created to keep his head still during the therapy.  He said: "The short-term effects are that you may vomit and get a headache now and then, but in the long term the side effects are rare."  Mason's family has been hugely supportive, though his younger siblings are unaware of his treatment.  "My little sister and little brother don't know because they are young," he said.  "My 10-year-old brother understands a bit and he's a bit upset about it."  Mason, who will sit GCSEs next year, will have six weeks off school.  Proton beam therapy is a highly targeted treatment which hits tumours much more precisely than conventional radiotherapy.  This makes it beneficial for patients with difficult-to-treat tumours in critical areas, such as in the brain or spinal cord, and for young people whose tissues are still developing.  Two new proton beam therapy centres have been built at The Christie and University College London Hospital (UCLH) with £250m of government money.  Gillian Whitfield, consultant clinical oncologist who is leading Mason's care at The Christie, said: "With proton beam therapy, compared to conventional radiotherapy, there is less dose to surrounding normal tissues and less risk of permanent long-term effects of treatment.  "This is particularly important for children and teenagers with curable tumours, who will survive decades after treatment and are at much greater risk of serious long-term effects of treatment than adults.  "Mason's tumour is a low grade (slow growing) tumour with a high chance of cure.  "For Mason, in comparison to conventional radiotherapy, proton beam therapy should carry a lower risk of some important long-term side effects of treatment, particularly effects on short-term memory and learning ability and the risk over the next eight decades of the radiation causing other tumours."  Professor Stephen Powis, medical director for the NHS in England, said: "This is a hugely exciting development for the NHS and we are delighted that we are able to provide this life-changing treatment for patients like Mason."  Proton beam therapy hit the headlines in 2014 when the parents of Ashya King, who was due to undergo chemotherapy and radiotherapy at Southampton General Hospital, fled with him to Spain.  They were arrested in Spain but were eventually able to take him for proton treatment in Prague.  His father Brett has said Ashya is now cancer-free.


theindependent--2019-04-18--Gene therapy cures babies of rare aposbubble boyapos immune disorder in world first

Gene therapy cures babies of rare 'bubble boy' immune disorder in world first

Ten newborns with the rare genetic disorder known as “bubble boy” disease were cured with gene therapy, researchers said on Wednesday.  The treatment appears to have completely rid the babies of their immune disorder with no side effects or complications – a result scientists have sought for decades through painstaking research and heartbreaking setbacks.  An earlier attempt to use gene therapy to treat severe combined immunodeficiency disease, or SCID, was halted in 2003 after researchers realised the therapy was giving the children cancer.  The treatment announced on Wednesday does not appear to carry such calamitous side effects, and experts hope it could help advance cures for other rare genetic disorders, such as sickle cell disease.  “It’s a game changer,” said Jennifer Hemall, a paediatric immunologist at Children’s Hospital of Philadelphia, who was not involved in the study.  “For immunologists following this disease, gene therapy has always been out there as the hope of the future. It’s exciting to see this wave of treatments actually becoming a reality.”  Infants with SCID are essentially born without a functioning immune system.  Without treatment, they rarely survive past their first birthday and can be killed by infections as innocuous as the common cold.  Such children were once kept isolated in sterile environments, giving rise to the term “bubble boy”. Their unusual predicament has caught national attention and has been featured in movies and TV shows.  The survival rate in recent years has soared with the advent of widespread screening tests and refinement of lifesaving procedures such as bone marrow transplants.  But such treatments have come with complications, often leaving children dependent on regular infusions of immunoglobulin or causing their new immune systems to attack their own bodies.  The new gene therapy – developed by St. Jude Children’s Research Hospital and UCSF Benioff Children’s Hospital in San Francisco and published in the New England Journal of Medicine – corrects the genetic defect in the DNA of babies soon after they’re born, prompting their bodies to generate missing parts of their immune system.  In interviews, the researchers said they first extracted blood stem cells from the infants’ bone marrow.  They used a modified virus as a vehicle to deliver the correct copy of a defective gene into those patients’ stem cells. Those corrected cells were then re-infused back into the patient, where they proliferated and created healthy immune cells.  To avoid accidentally turning on cancer-causing genes as previous gene therapies did, the researchers built “insulators” into the virus so neighbouring genes wouldn’t be affected when the virus made its insertion into the DNA.  In another innovation, the researchers gave their infant patients a tiny amount of chemotherapy to clear existing cells from the bone marrow before they re-infused the treated cells, giving the corrected cells a better chance to take hold.  The announcement was bittersweet for many at St. Jude Children’s Research Hospital.  Their team leader and senior author on the paper, Brian Sorrentino, spent the final months of his life racing against a fatal cancer to finish his work on the experimental treatment.  Dr Sorrentino was diagnosed at a young age with Hodgkin’s lymphoma, said his widow, Suzanne Sorrentino.  Because better treatments did not yet exist, his doctors used radiation that weakened his heart and produced the lung cancer that killed him last November.  “The loss of hair, the pain and agony of the treatment, it’s what made him determined to find new treatments for other diseases in kids,” his widow said in an interview.  Since the 1990s, Dr Sorrentino had been working alongside other immunologists on gene therapy for SCID, but his work took on new urgency in the final year and half of his life after his cancer diagnosis, Suzanne said.  When he could no longer get to the hospital, St. Jude colleagues started arranging conference calls and setting up shop at Dr Sorrentino’s home dining table.  By then, it was apparent that the experimental procedure was succeeding. Several of their infant patients had grown into toddlers with normal immune systems.  “He told me he thought he was saved as a kid from Hodgkin’s because there was something he thought he should be doing with his life,” said Suzanne, her voice cracking as she recalled one of the last conversations with her husband.  “With the trial working, and the children doing so well, he felt it was a sign that he achieved what he was supposed to. That it was time for him to go.”  In recent years, as screening newborns for SCID has become mandatory across the country, experts have found the disease to be more common than previously thought – occurring in as many as 1 in 50,000 infants.  There are several forms of SCID.  Gene therapy for a variant called ADA-SCIDS is available in Europe. But a cure for the most common form – X-linked SCID, which is only found in boys – had remained elusive.  The disease drew national attention in the 1970s with the case of David Vetter.  News outlets chronicled his life as he grew up cocooned in plastic.  His story spawned the 1976 John Travolta movie “The Boy in the Plastic Bubble.”  And even after David Vetter’s death, something about the disease’s vulnerable isolating effects kept it in the national consciousness, making a famous cameo in a “Seinfeld” episode and becoming the premise of a panned 2001 Jake Gyllenhaal movie, Bubble Boy.  Until now, the most effective therapy for SCID had been transplanting blood-forming stem cells from a tissue-matched sibling donor, but most patients lack such donors.  Half-matched and unrelated donors have also proved lifesaving but often resulted in complications.  The first successful gene therapy trial, in 2000 by French doctors, gave many researchers and patients hope that they could fix the molecular underpinnings of the disease instead of simply treating its symptoms.  But when leukemia was found in some of those patients, it temporarily halted gene therapy trials in America and much of the world and sent dispirited researchers searching for answers.  “The leukaemia stopped the field for a long time and rightfully so, because people had to figure out what went wrong,” said Jordan Orange, a paediatric immunologist at Columbia University, who was not involved in the St Jude study but helped develop a gene therapy for another immunodeficiency disease called Wiskott-Aldrich.  “But that led us to the point now where we’re finally beginning to see actual cures to incurable conditions.”


themanchestereveningnews--2019-01-23--Teenager among first in UK to have beam therapy for a brain tumour

A schoolboy with a rare brain tumour was today undergoing pioneering treatment as one of the first patients at the new Proton Beam Therapy Unit based at The Christie.  The treatment is a type of radiotherapy that targets certain cancers precisely, shrinking tumours, increasing success rates and reducing side-effects.  Mason Kettley's treatment in Manchester means the 15-year-old is one of the first patients to undergo proton beam therapy in the UK - and the first to go public.  He said he's looking forward to enjoying a McDonald's after his treatment ends.  The life-saving cancer therapy unit at The Christie is the first of its kind in the UK and has been under construction since the summer of 2014.  The specialist form of radiotherapy was formerly only available to patients abroad.  Mason, from West Sussex, was diagnosed with an inoperable tumour in October after suffering headaches and failing to put on weight.  Doctors found a tumour growing in a critical part of his brain but could not operate due to a risk of blindness and brain tissue damage.  Science-loving Mason, who wants to work as a doctor specialising in tumours when he is older, said he didn't initially have many symptoms.  He said: "I had some headaches and stomach pains and usual things, and got check-ups at the doctors. My mum said 'he's not gaining weight or growing' and eventually, when we moved last year, we saw doctors who told us to go to Worthing Hospital for an MRI scan.  "We had the scan and the result showed it was a tumour."  Following a biopsy and an operation to insert a shunt, doctors referred Mason's case to a national panel of experts.  They decided the tumour - known as a benign pilomyxoid astrocytoma - made him a suitable candidate for proton beam therapy.  Mason will have treatment Monday to Friday in Manchester for almost six weeks, 28 treatment sessions in total.  A specially-made radiotherapy mask has been created to keep his head perfectly still .  "It's a bit nerve-wracking but this is a better choice than chemo because it's more effective," he said.  "Their goal is to stabilise the tumour. It may shrink, but they are aiming to stabilise it."  Medical experts have hailed a 'hugely exciting development' in the NHS.  Consultant clinical oncologist Gillian Whitfield, who is leading Mason's care at The Christie, said: "With proton beam therapy, compared to conventional radiotherapy, there is less dose to surrounding normal tissues and less risk of permanent long-term effects of treatment.  "This is particularly important for children and teenagers with curable tumours, who will survive decades after treatment and are at much greater risk of serious long-term effects of treatment than adults.  "Mason's tumour is a low grade, slow-growing tumour, with a high chance of cure."  Professor Stephen Powis, medical director for the NHS in England, added: "This is a hugely exciting development for the NHS and we are delighted that we are able to provide this life-changing treatment for patients like Mason."  Up to 1,500 patients every year could benefit from proton beam therapy in the UK.  The Christie has two treatment rooms able to accept patients, while a third will be up and running in the next few months.


thetelegraph--2019-01-23--Schoolboy with rare brain cancer among first to have NHS proton beam therapy in UK

A 15-year-old boy has spoken of his excitement at being among the first to have NHS proton beam therapy in the UK.  Mason Kettley, who suffers from a rare brain tumour, began treatment on Tuesday - five years after the parents of Ashya King sparked an international manhunt when they took him abroad in search of treatment.  Until last month, NHS patients were sent as far away as the US for treatment, if specialist doctors said it was required.  Now the Christie Hospital in Manchester has begun offering the highly targeted treatment, with Mason the fourth case to undergo it, and the first to speak publicly about it.  It is the first centre in the UK offering such treatment to NHS patients.


dailybeast--2019-12-20--In 2020, Could Artificial Intelligence Help Cure Cancer?

Time is of the essence when it comes to treating cancer, the second leading cause of death in the U.S. according to the Centers for Disease Control and Prevention. Between diagnosis and the first day of treatment, days and even weeks may tick by as doctors convene to discuss treatment plans and order testing to gather as much information as possible. But as a new decade dawns, artificial intelligence may buy more time for those who need it most.  Both President Donald Trump and former Vice President Joe Biden have promised to prioritize curing cancer should they win the 2020 election. But because of its complex biology, cancer has been historically difficult to cure with a pill or injection. As new treatments like immunotherapy undergo further research, health systems are starting to harness data-sharing and artificial intelligence to better predict a patient’s prognosis, and determine the most effective treatment plan for their cancer based on other patients with similar medical histories.  The Knight Cancer Institute at Oregon Health & Science University, in partnership with Intel, created a “collaborative cancer cloud” with this vision. Once doctors have met a patient, within hours, they can diagnose them and begin treatment. Thanks to AI’s ability to analyze massive amounts of real patient data, doctors can better predict the best possible treatment for their patient, based on the responses of others with genetically similar cancers. OHSU says the project uses “machine learning techniques against a collective set of molecular and imaging data in order to support big data analytics in a federated, aligned environment,” and hopes to shorten the diagnosis-to-treatment time period to 24 hours in 2020. Similar programs include CancerLinq, ORIEN and the Cancer Moonshot Research Initiative.  “In the past, vast quantities of information were lost to file cabinets and unconnected servers. The big data era is allowing CancerLinQ and other initiatives to access this large and growing amount of de-identified data to help physicians provide high-quality care,” said Dr. Robert Miller, medical director of CancerLinq, which gathers anonymized patient data from electronic health records nationwide. “Instead of learning only from the 3% of patients who join clinical trials, CancerLinQ aims to enable practices to learn from everyone with cancer.”  “Using informatic techniques to mine data in this way is useful, and we’re in the very beginning stages of understanding it. The promise is there,” said Dr. John Vu, a board-certified medical oncologist and director of clinical informatics at Baptist MD Anderson Cancer Center in Jacksonville, Florida. “With advanced analytics, we might be able to get more specific information, like prognosis in a particular situation, or have a better idea of their genetic makeup and the turnaround time for successful treatment.”  Vu believes that same-day diagnosis and treatment would help patients beat cancer because every day counts with a notoriously fast-progressing disease. But for this to become a reality, the rest of medical technology would need to catch up. For example, pathology labs may not have same-day biopsy results. If other steps in the diagnosis process speed up, Vu said, he would feel equipped to make treatment decisions more quickly than is currently possible.  “If someone needs treatment right away, and I had results that day, that patient could be treated with targeted therapies that day. We don’t have the technology to turn it around in one day, but when we get there, yes, that would help that patient,” he said.  The NCI’s Cancer Moonshot initiative aims to improve cancer care and accelerate research, and one arm of that mission focuses on data sharing. Cancer Moonshot-funded projects require investigators to submit a plan of how their conclusions, and data throughout the trial, will be shared with the public. Their answer should end with the data being FAIR: findable, accessible, interoperable and reusable. While data sharing has always happened between colleagues and between experts who know of each other’s work, data sharing on an international level has only gained traction in recent years.  “One of our overarching goals is to enhance data sharing, making sure that data that’s coming from any of the projects funded through Moonshot funds are getting into the hands of people who can use that information well,” Dr. Jaime Guidry-Auvil, director of the Office of Data Sharing in the National Cancer Institute (NCI), told The Daily Beast. “It hasn’t always been appreciated how useful data sharing is. Once a hypothesis is published, it can be thought that there has been a sufficient scientific process. What’s coming about as more data is being generated, is we can take the data and use it in different ways: both to validate a hypothesis, and also to generate a hypothesis.”  Vu agrees that, while AI is not a replacement for a medicinal cure that may one day end cancer, advances in data sharing and analysis in health care could lead to what does. By finding associations within the data, AI can point researchers in new directions they may not have considered otherwise.  “You can see an association that, if you give this drug to this patient, in 1,000 patients, it seems to help, but it doesn’t show a cause-and-effect. It can drive hypothesis,” he explained. “Looking at these large data sets, you start thinking of clinical questions about the data that need to be answered, and generating clinical trials to answer them, and we may find answers that otherwise wouldn’t have been found. In that way, AI can shorten the life cycle of the clinical trial. It can generate hypotheses we may not think of otherwise.”  For example, Vu said, most lung cancer patients today receive genome sequencing testing, which looks for specific mutations within their cancer that doctors know respond well to certain treatments. Hypothetically, AI analysis of patient data may reveal a trend that a specific mutation predicts longer survival regardless of treatment, or show a new link between a less-understood mutation and a developing treatment.  AI can also help customize treatment plans for patients with complex cancers, which some cancer data-sharing platforms have begun offering.  “There are more than 100 types of cancers with different genetic variations,” said Miller. “Data sharing and AI have the potential to help us further personalize care for each individual patient. For example, if a physician is treating a patient with a rare cancer, he or she can examine the outcomes of patients across the country with the same cancer and similar characteristics to help choose the right therapy for the right patient at the right time.”  Vu cited the Eastern Cooperative Oncology Group (ECOG) performance scale, which physicians use to determine how fit a patient is to undergo cancer treatment. The system is subjective, and adding objective data based on AI’s analysis of objective data to the process could result in more appropriate plans for treatment.  “Analytics could help us parse that scale into further detail. Do they have a history of smoking? Diabetes? Obesity? Then when we have a patient in front of us, we could say, ‘Based on your history and this algorithm, you’ll tolerate treatment really well,’ or ‘No, we don’t recommend it for you.’ Currently ECOG is very vague. With analytics, we could get much more specific, but no one has done that yet.”  In 2020, the National Institutes of Health (NIH) will formalize their new data sharing policy, which will require data gathered from any NIH-funded project to be made available to the scientific community. This policy has not been updated since 2003, and the changes reflect a growing attitude that the more scientific data is available, the faster innovations in care can be discovered.  “Much of it centers on the idea that data sharing should be considered throughout the life cycle of research and not just at the end of research. If we promote better data sharing, then we can accelerate our field further,” said Guidry-Auvil.  Ultimately, AI is not the cure the world has been waiting for. But oncologists like Vu are beginning to use it as a way to improve treatment until the cure is discovered. And, if researchers take the opportunity, AI may be the tool that helps science discover a cure sooner.  “All of this AI stuff is really new. It’s been out about five years and hasn’t taken hold in many places. It has a lot of promise: I think the way we treat cancer in 10 years will be a lot different than we do today, and some of that will be due to advanced informatics,” said Vu. “You can use AI to help accelerate discovery, to find new processes and situations to suppress cancer’s growth. I think AI can help us get there faster than traditional means, like standard clinical trials. Definitely it’s moving us in the right direction.”  As Guidry-Auvil says, “having very quick access to a data set that is much larger, that you could not easily obtain on your own, will hopefully lead to an improvement in treatments. Maybe it’s not a direct cure, but without that information, it will be harder to get to one. Adding AI to that, if the AI can analyze the data well, would add to that progress.”