Extensive work in the lab and some encouraging ALD studies provided the basis for this trial. This trial was subsequently terminated before completion. Enzyme replacement therapy (ERT)
(current as of February 2021)
Takeda acquired the MLD ERT from Shire in early 2018 and continues to develop and studying their intrathecal SHP 611 (formerly HGT-1110) ERT [Enzyme Replacement Therapy]. Clinical Trial
A third global trial studying the late infantile form of MLD for 42 patients aged 6 – 72 months launched in April 2019 and was fully recruited in January 2021. This is the first time ERT study sites are open in the US. Clinical trial information & inclusion criteria can be found on the MLD Foundations ERT page and at the Clinical Trials.gov site. Substrate reduction therapy
Biomarin South (formerly Zacharon before being acquired by Biomarin in January 2013) from San Diego had initiated a drug discovery program for MLD. This program is based on using assays which measure sulfatide accumulation in cultured fibroblasts as a means to discover and develop small molecule drugs for MLD. (This approach differs from other approaches which have measured enzyme activity to discover effective drugs.) As of July 2011, Zacharon has begun adapting the assays it developed for other lysosomal storage diseases so that they can be employed to discover and develop drugs for MLD. (current March 2013)
The Cooper Health System (New Jersey) sponsored a clinical trial underway to determine the safety and efficacy of a Vitamin K antagonist (Warfarin) in treating Metachromatic Leukodystrophy (MLD) in 2009.
Oxandrolone, sold under the brand names Oxandrin and Anavar, among others, is an androgen and anabolic steroid (AAS) medication which is used to help promote weight gain in various situations, to help offset protein catabolism caused by long-term corticosteroid therapy, to support recovery from severe burns, to treat bone pain associated with osteoporosis, to aid in the development of girls with Turner syndrome, and for other indications. It is taken by mouth.Side effects of oxandrolone include symptoms of masculinization such as acne, increased hair growth, voice changes, and increased sexual desire. The drug is a synthetic androgen and anabolic steroid, hence is an agonist of the androgen receptor (AR), the biological target of androgens such as testosterone and dihydrotestosterone. It has strong anabolic effects and weak androgenic effects, which give it a mild side effect profile and make it especially suitable for use in women.Oxandrolone was first described in 1962 and was introduced for medical use in 1964. It is used mostly in the United States. In addition to its medical use, oxandrolone is used to improve physique and performance. The drug is a controlled substance in many countries, so nonmedical use is generally illicit. Medical uses
Oxandrolone has been researched and prescribed as a treatment for a wide variety of conditions.