At the beginning of February. BioPontis Alliance announced a partnership with Massachusetts General Hospital. Together, the two set their sights on Huntington’s disease. Specifically, they hope to develop new treatment options. Keep reading to learn more about this partnership, or get the full story at the original source.
BioPontis Alliance exists to promote and support research of treatments for rare neurological diseases. Through the partnership with Massachusetts General Hospital (MGH), BioPontis Alliance advances research at MGH, MIT, and Northwestern University. Research focuses on sirtuin 2 deacetylase inhibitors as a potential treatment for diseases like Huntington’s.
At this moment in time, no therapies exist for Huntington’s disease, which is ultimately fatal. Huntington’s is a genetic degenerative disorder of the brain that may result in impaired cognitive ability, a lack of motor control, and emotional problems as the condition progresses.
The collaboration between BioPontis and MGH lays out a pathway. That path heads toward a treatment candidate. While basic, preliminary, and discovery research will be done, they’re making sure to never stop there.
“As clinicians and scientists, we are committed to not only unravel the basic mechanisms of diseases like Huntington’s disease, but to see this basic research fuel discovery of new therapeutics,” says and MHG’s former Chief of Neurology Anne Young, MD, PhD. “This is a unique opportunity for us, to collaborate with BioPontis on one of the major long term aims of our research, namely translating ground-breaking basic findings into treatment opportunities for patients.”
Advocacy groups such as the European Huntington Association (EHA) are also pleased to see this kind of cooperation.
EHA even actively endorses the collaboration between BioPontis and MGH. Astri Arnesen, president of EHA, describes it as “additional hope” to Huntington’s patients and families. Arnesen points to BioPontis’s plan to include patient perspectives early on during development as a huge benefit to the program. This, Arnesen hopes, will bring the project additional succes.
BioPontis also hopes to continue pursuing breakthrough treatments in the rare disease community. Rare diseases as a whole make up the largest underserved medical community world wide. Out of a group of around 7000, or 8000 rare disease, the genetic cause is known for nearly 4000. Treatment remains difficult to produce, however, even in cases where the cause is known. The knowledge base is often unorganized, and projects are starved for funding. As a result, it is estimated that only 6% of rare disease patients have approved therapies. That’s where BioPontis and MGH are trying to make a difference.