Currently, there are no treatments that can slow or stop the progression of neuron death in multiple system atrophy (MSA). Survival for patients is usually around 8-10 years from symptom onset.
In patients with MSA, neurodegeneration is caused by the abnormal and progressive accumulation of the protein alpha-synuclein in neurons and glia. Alpha-synuclein, and other proteins, are usually removed from the brain by a system called autophagy. It is useful to think of alpha-synuclein as garbage, and the autophagy system as the garbage collecting truck of the body. However, in MSA and related disorders, the garbage truck is less effective than usual, which results in increased garbage accumulation in cells, meaning, excessive alpha-synuclein accumulation. This, in turns, causes degeneration of the neuronal cells.
The NYU Dysautonomia Center has a new study to test the promise of a drug that enhances autophagy, i.e., reactivates the “garbage truck” of the body. The drug, sirolimus (also called rapamycin), has been known for decades and is currently approved by the FDA to prevent organ transplant rejection, and to treat a rare lung disease. Studying a drug that is already FDA-approved for other purposes has several advantages, including increased safety, given that the side effects of this drug are well described and can, therefore, be anticipated and prevented. Moreover, there is research data showing that sirolimus administration to cells or mice reduces neurodegeneration. The trial will test if this is also true in humans.
The study, sponsored by the NIH, involves giving sirolimus or placebo for a year to patients with MSA who are still able to walk (with or without assistance). The clever design of the trial means that three out of four participants with MSA will get sirolimus, and 1 out of 4 will get placebo. By using neurological rating scales, brain MRI and retinal scans, investigators hope to determine if sirolimus can slow the progression of MSA.
While there are a number of active studies to improve symptoms of patients with MSA, this is currently the only study open to recruitment in the U.S. testing a medication that could potentially slow the progression of MSA.
Learn more about this study below and how to contact the Center to find out if you are eligible to enroll.
A futility trial of sirolimus in multiple system atrophy. NIH Clinical Trials Website: https://www.clinicaltrials.gov/ct2/show/NCT03589976
This study is only performed at NYU
Contact person: Jose Martinez, Clinical Trials Manager. Email: Jose.Martinez4@nyulangone.org.
Location:NYU Dysautonomia Center; New York University School of Medicine, 530 First Ave. Suite 9Q, New York, NY 10016.
Telephone: +1 212 263 7225.