By STEVEN ZECOLA This study tracks the decades-long journey to harness alpha-synuclein as a treatment for Parkinson’s disease. Steven Zecola, an activist who closely follows Parkinson’s research and recently discussed it on THCB, offers three crucial changes needed to overcome the underlying challenges.
**A Quick Start for Alpha-Synuclein R&D**
In the mid-1990s, Parkinson’s patient advocacy groups grew frustrated with the lack of therapeutic advancements since the approval of L-dopa for Parkinson’s disease. The Director of the National Institute of Neurological Disorders and Stroke (NINDS) organized a workshop in August 1995 that included scientists specializing in human genetics who could bring new insights to PD research. One of these scientists, Robert Nussbaum, emphasized the importance of identifying genes responsible for familial Parkinson’s to enhance understanding of the disease. By 1997, Spillantini et al. discovered that alpha-synuclein (A-syn) played a key role in abnormal protein clusters in Parkinson’s patients. Fast progress was made, with notable remarks from President Clinton highlighting the rapid advancement in Parkinson’s research.
**The NIH is Asked to Take a Leadership Role**
Following President Clinton’s call to action, the National Institutes of Health (NIH) and NINDS collaborated to develop a comprehensive five-year PD Research Agenda. This agenda aimed to cover all facets of PD research, including understanding the disease better, developing new treatments, and advancing research capabilities. Noting the significant research shift due to the discovery of alpha-synuclein’s effects, the NIH invested close to $1 billion from FY 2000 to FY 2004 to implement the PD Research Agenda.
**Private Interests Finally Move Forward with Alpha-Synuclein**
Recognizing the ongoing lack of progress in A-syn research, the Michael J. Fox Foundation initiated a $10-million competition to develop an essential imaging tool for Parkinson’s disease. MJFF hailed the progress made by various teams in developing different alpha-synuclein tracer methods. Private companies have also stepped in, with companies like Merck, Neuropore Therapies, UCB, Prothena Biosciences, Roche, Biogen, AFFiRiS, AC Immune, and Vaxxinity conducting trials and studies related to A-syn development and therapies.
In conclusion, while challenges remain in developing A-syn therapies, the progress made so far indicates a potential approval of a treatment utilizing A-syn within the next 5-8 years. Despite the lengthy research journey, advancements in understanding the underlying biology of Parkinson’s disease are paving the way for more effective treatments.
