A drug that actually slows or reverses the brain damage inflicted by Parkinson’s disease, rather than just alleviates its often debilitating symptoms, remains elusive. But Prevail Therapeutics, a startup just launched in New York, has become the latest to try.
This morning, the Silverstein Foundation, a nonprofit organization formed by OrbiMed partner Jonathan Silverstein, announced the formation of a new startup in New York called Prevail Therapeutics. Headed by Columbia University neurology professor and Parkinson’s researcher Asa Abeliovich, the startup has launched to press forward with a gene therapy, and possibly other approaches, for a genetically-defined subset of people with the condition.
Prevail has a deal in place with RegenXBio (NASDAQ: [[ticker:RGNX]]) to use a tool used in gene therapy that shuttles genetic instructions into the body to fix a disease-related malfunction. Prevail can now use the delivery tool, a type of engineered virus, to develop a gene therapy for Parkinson’s or other neurodegenerative diseases. The goal is to introduce a working copy of a gene that is mutated in people with a rare genetic form of Parkinson’s. The hope is that the new gene would alter the progression of the disease.
The startup is the first industry investment for the Silverstein Foundation, which formed earlier this year to fuel Parkinson’s research. CNBC reported Wednesday morning that Silverstein, a longtime life sciences investor at OrbiMed, started the foundation after being diagnosed with an aggressive form of Parkinson’s. According to its website, the foundation has given grants to the Michael J. Fox Foundation, the Parkinson’s Institute and Clinical Center in San Francisco, and Columbia University.
Abeliovich, whose research at Columbia has been focused on the genetics underlying Parkinson’s, worked with OrbiMed when he co-founded Alector, an OrbiMed-backed neurological drug developer in San Francisco (he’s currently that startup’s chief innovation officer). The OrbiMed connection led Abeliovich to join the Silverstein Foundation’s scientific advisory board, and eventually to discussions about forming new companies to combat the disease. The first is Prevail.
“He’s put together a real ‘SEAL team’ to try to figure out how we can advance things” against Parkinson’s, Abeliovich says of Silverstein.
Both the Silverstein Foundation and OrbiMed are investors in Prevail. Abeliovich wouldn’t say how much has gone into the company so far. The Silverstein Foundation didn’t immediately respond to a request for comment Wednesday.
According to the National Parkinson Foundation, about 1 million Americans and 4 to 6 million people worldwide suffer from the disease, which causes motor symptoms like tremors, loss of movement, and stiff limbs, and cognitive problems like confusion and memory loss. Those numbers are expected to increase substantially in the next few decades as the population ages. As the disease progresses, patients can have trouble walking, speaking, or functioning without the help of a caregiver.
Levodopa has been the standard of care for Parkinson’s since the 1960s. While much remains unknown about the disease’s underlying biology, its hallmark motor symptoms arise from the death of neurons in the brain that produce the neurotransmitter dopamine. There are no drugs that actually slow or reverse the course of the disease, but levodopa has made a huge difference. It helps nerve cells make dopamine, which in turn helps patients manage symptoms and function normally for a longer period of time.
The problem is, over time, patients’ responses to levodopa start to decrease. To compensate, patients boost their levodopa doses, but that can cause dyskinesia, or uncontrollable, spastic movements.
The good news is
