We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment.
But according to a recent article in GlobeNewswire, we will be seeing many novel therapies being tested over the next decade. The Globe’s article lists over twenty companies with novel MSA therapies currently in or about to enter clinical trials.
MSA is a neurodegenerative disease with multiple symptoms affecting autonomic (involuntary) actions. This includes the nervous system, digestion, blood pressure, motor control, and other involuntary actions.
Estimates of the number of MSA patients range anywhere from fifteen to fifty thousand people. The disease generally appears in individuals over the age of fifty and advances rapidly over the next five to ten years. The patient experiences loss of motor function and eventually becomes bedridden. There is a risk of pneumonia in the late stages of MSA with possible fatal respiratory or cardiac complications.
Symptoms generally include tremors, stiffness, incoordination, speech impairment, and bladder control issues. Fainting can occur at times due to changes in blood pressure when rising from a chair or bed.
MSA symptoms are very similar to those of Parkinson’s disease and therefore difficult to diagnose.
The Cause of MSA is Unknown
Researchers found an accumulation of the alpha-synuclein protein in cells that support the brain’s nerve cells. This is one of the characteristics of MSA.
The alpha-synuclein accumulates in cells that make the coating on nerve cells. Although alpha-synuclein also accumulates in Parkinson’s disease it differs from MSA in that it appears in nerve cells.
Currently, no treatment exists for MSA. Drugs that treat Parkinson’s patients, such as levodopa administered in Sinemet tablets, are prescribed for MSA patients. Levodopa is used to control MSA symptoms in the early stages of the disease.
MSA Drugs in Development
Over twenty-five therapies are currently at various stages of development to treat MSA. One of the most advanced drugs is verdiperstat by Biohaven Pharmaceutical. Verdiperstat is designed to reduce neuroinflammation that causes brain cell death in neurodegenerative diseases such as MSA and ALS.
In July 2020 Biohaven announced that it had completed enrollment of three hundred MSA patients worldwide. M-STAR is an interventional Phase III clinical trial (NCT03952806) designed to evaluate the efficacy and safety of the Verdiperstat.
The FDA granted Verdiperstat Fast Track and Orphan Drug designations for the treatment of MSA. The EMA granted the Orphan Drug designation as well.
Companies awarded an Orphan Drug designation by the FDA are given incentives to develop a drug for a rare disease that may not otherwise be profitable. Developing a drug for a rare disease generally does not allow a return on investment. In other words, the drug is not developed for profit but to meet a public health need.
Judging by the number of clinical trials and involvement of over twenty-five key companies, it may be safe to say that we are turning the corner on this, so far, incurable disease.