FDA Commissioner open to curative potential of gene-editing
FDA Commissioner Robert M. Califf has said that advances in gene knowledge could lead to gene-editing for the cure of diseases. The relatively recent advances in data sharing and genomic study will allow for precision medical therapies, Califf said in his keynote address to the “Improving Clinical Research in the Age of Precision Medicine” symposium on 14-15 September. The ability of gene editing to remove or replace genes that cause diseases could be used in the near future for genetic-related illnesses, such as sickle cell disease, which has been treated successfully in mice using a genetically-engineered virus.

Four years ago, if someone had told FDA Commissioner Robert M. Califf that, soon, it might be possible to remove disease-causing genes from organisms and replace them with healthy ones, he would have called such genetic manipulation the stuff of science fiction.

“But it turns out that we now have technologies that can do this,” Califf said Wednesday while delivering the keynote address to open the symposium “Improving Clinical Research in the Age of Precision Medicine,” held at the University of Miami’s BankUnited Center September 14-15.

“We’ve gone from looking at the downstream products of what genes do and using genes to make a diagnosis to actually being able to go in and to either delete a gene, insert a new gene in place of a gene that was there, or add a gene to the genome—and doing it very specifically with little molecular scissors,” Califf said.

The two-day symposium, hosted by UM’s Office of Research Compliance and Quality Assurance in collaboration with the FDA Office of Regulatory Affairs Florida District Office and the FDA Office of Minority Health, focused on the scientific, regulatory, and ethical aspects of clinical trials as well as quality assurance practices.

Speaking to an audience of about 450 researchers, physicians, scientists, nurses and other medical professionals, Califf pointed to sickle cell disease, which affects about 300,000 babies a year around the world, as one illness that could perhaps be cured through gene therapy.

“We have therapies that make it [sickle cell disease] better, but we don’t have a cure for it,” said Califf. “It looks like now we might be able to apply this technology.”

Indeed, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center recently reported in the Journal of Clinical Investigation that a genetically engineered virus that triggers the production of an alternative form of hemoglobin reversed sickle cell disease symptoms in mice, clearing the way for possible human clinical trials in the near future.

Advancements in genomics are just some of the tools in an ever-growing arsenal to wipe out disease and boost the field of precision medicine. The direct involvement of patients themselves in the development of therapeutics is also playing a major role, Califf explained, pointing to electronic health records as an example of that involvement.

“In the last 10 years we’ve gone from almost no electronic health records to every American having an electronic health record,” said Califf, a former Duke University professor of medicine who was handpicked by President Barack Obama to lead the FDA and confirmed for the job by the Senate last February. “This gives us an incredible opportunity to use digital data in a way that was just not possible in the old system.”

But it was Califf’s comments on the field of genomics and its impact on the future of health care that was the highlight of his keynote address. “We’re about to see some very dramatic changes in therapeutics related to genomics,” he said.

The National Institutes of Health’s new Precision Medicine Initiative (PMI) Cohort Program could spawn many of those dramatic changes. The bold research effort, announced by Obama in his 2015 State of the Union address, promises to revolutionize how we improve health and treat disease, engaging a million Americans to volunteer to contribute their health data to improve health outcomes, fuel the development of new treatments for disease, and catalyze a new era of data-based and more precise preventive care and medical treatment.

Califf urged symposium attendees to sign up for the initiative. “If health care professionals volunteer, it will send a message to everyone else that this is a good thing to do.”