ROAD TO THE CURE UPDATE DECEMBER 2013Category: Road to the Cure
Alpha-synuclein is the sticky protein that clumps up in the brain of Parkinson’s patients. While its role in disease progression is not fully understood, increasingly scientists believe that deciphering how alpha-syn accumulates and spreads in the brain is a key to deciphering the disease. But there is one major problem and that is alpha-synuclein cannot be seen until the brain is autopsied after the patient dies.
“The development of an alpha-synuclein imaging agent could be transformative for Parkinson’s disease research and drug development. The ability to image alpha-synuclein in the brain would enable tracking of the degree and location of pathology over time and monitoring of therapies aimed at reducing alpha-synuclein levels” [Jamie Eberling, et al (Michael J Fox Foundation), Journal of Parkinson’s Disease Research, 2013].
Since its inception in 2008, the SDBC scientists have believed that the ability to image alpha-synuclein deposition in the brain would be a game-changing achievement for early diagnosis, monitoring therapeutic efficacy of drugs and disease progression in patients at risk or afflicted with Parkinson’s disease. However, this is a monumental task. First, the imaging agent must cross the blood-brain barrier (BBB), which restricts the entry of most biologics into the central nervous system. Second, it must not only bind to an extracellular alpha-synuclein but also enter cell wall of neurons and bind to the intracellular alpha-synuclein. Third, it must be highly specific, which means it must only bind to alpha-synuclein.
The SDBC has developed a highly specific imaging agent (aka: ligand) for detecting alpha-synuclein in the brain of Parkinson’s patients. This imaging agent was tested in Parkinson-like transgenic mice last year in collaboration with UCSD neuroscientists. When injected in the tail vein of these transgenic mice, this imaging agent traveled to the brain where it specifically bound to the aggregated alpha-synuclein and Lewy body like structures, which are composed of alpha-synuclein.
The SDBC scientists are now gearing up to putting a radioactive tracer on its imaging agent, inject into transgenic mice and then scan the brain to detect and quantify the amount of alpha-synuclein in the brain of these mice. These studies require the use of a PET scanner, which the Company has been trying to outsource but the prices quoted have been forbiddingly too high to work with outsourcing facilities. The Company has come to realize that it better bite the bullet, buy its own PET scanner to conduct experiments at its facility. Therefore, efforts are underway to arrange funds to order a PET scanner as soon as possible.
Blood Test for PD: As stated previously, the SDBC is contemplating developing a diagnostic test for PD from blood. Its scientists are planning to determine if alpha-synuclein can be detected in the blood of PD-like transgenic mice. For this purpose, blood will be collected from PD-like transgenic mice at different ages and levels of serum alpha-synuclein will be compared in these mice. If indeed alpha-syn is effluxed by the PD brain into blood, these measurements will not only help to diagnose the condition but also help to monitor effect of the treatment.
Parkinson’s Resource Organization is appreciative to have been introduced to this life-changing science. This science needs funding to bring it to fruition.
Being a part of this historic, humanitarian effort requires an investment. If you have Parkinson’s it may be an investment into your life, if you don’t have Parkinson’s it may be an investment in humanity. Contact Jo Rosen of the Parkinson’s resource Organization who will introduce you to the right parties for making your investment possible. The only obstacle to getting to the human side of this science is funding. The sooner the funds are raised the sooner human clinical trials can begin. The possibility of being in human clinical trials in 12 months is possible if full funding happened tomorrow. Reiterating, the sooner it’s completely funded the sooner it will be in human clinical trials. Call and be surprised at how little full funding is.