Michael J. Fox Foundation Research Update
I had the opportunity to sit in on a Michael J Fox Foundation research update this week, presented by Rachel Bulmer and Melanie Demakis, and wanted to share some updates with you that they shared with us. For those of you unfamiliar with the Michael J. Fox Foundation, they provide financial support to researchers working not only to advance the treatment of Parkinson’s Disease, but to find a cure.
While I’ve always tried to stay up to date on management of the patient with Parkinson’s to provide the best standard of care for my own patients, I also have personal interest in the topic as my mother is living with Parkinson’s disease. This webinar was a great opportunity to hear about current state of genetic research and also to learn about some recently approved pharmacological interventions.
Genetic Trials
The presenters discussed the importance of finding a concrete biomarker for Parkinson’s disease. A biomarker is a quantitative measurement tool that enables researchers to more definitively determine efficacy of a treatment in testing, and also better identify the current stage of a person’s disease. As there is no current validated biomarker for Parkinson’s disease, researchers have to rely primarily on clinical observation and functional outcome measures to determine whether a new medication or genetic treatment has made a significant and clinically relevant difference. They are able to perform brain scans that show some degree of dopamine loss, but this value is difficult to standardize and has not been used in a validated way as of yet.
They discussed three areas of genetic research that are potentially relevant to finding a better treatment, or possibly a cure, for Parkinson’s disease. Alpha-synuclein protein is one of these areas of interest. Located in the brain, mutation or overexpression of this protein is believed to play a role in the development and progression of Parkinson’s disease. This protein essentially folds into clumps, leaving Lewy Body deposits, which slow the transmission of dopamine within the brain, leading to the motor effects we see in patients with Parkinson’s disease. There are presently 13 Phase 1 to 2 trials investigating how to slow or prevent the expression of alpha-synuclein, in turn, slowing or stopping the progression of the disease.
The other two genetic avenues they presented were related to the genetic markers GBA and LRRK 2. With 6 GBA trials, and 3 LRRK 2 trials already in progress, two large companies, Biogen and Denali, just announced they will be combining efforts to support a Phase 2 LRRK 2 trial. This is significant, as with promised financing of $1,000,000,000, their support to the project speaks to their belief that this research may be particularly promising.
Pharmacological Treatments
The presenters also reported on three new medications that have recently hit the market. Interestingly, all three share a common goal - to reduce the experience of off-times, while each one approaches the problem in different ways. Kynmobi, a thin-film apomorphine strip can be taken sublingually as a rescue drug during off times. Ongentys, a COMT inhibitor, is also taken by mouth in pill form once daily, and though slightly older news, they also mentioned Inbrija, an orally-inhaled form of levodopa, which can be through an inhaler on as as-needed basis to reduce off-times as well.
Lastly, they touched upon the importance of volunteering for clinical trials, whether as a healthy control, or a person with Parkinson’s. You can access their trial database here and learn more about these opportunities.
I thought this was a great over-coffee opportunity to hear about the latest progress in Parkinson’s Disease research. You can see more webinars from Michael J. Fox Foundation here. If you would like to support this wonderful foundation, you can make a donation here.
