Advancements in the Treatment of Neurodegenerative Disease

In this video, Ghazaleh Sadri-Vakili, PhD, director of the NeuroEpigenetics Laboratory and the Mass General Institute of Neurodegenerative Disease, discusses her genetic research, specifically concerning neurodegenerative diseases like ALS and X-linked dystonia parkinsonism. 

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Transcript

My laboratory focuses on Neurodegenerative Disease. In particular, we focus on amyotrophic lateral sclerosis or ALS. We also study Huntington's disease.

We are actively testing out the neuro-protective effects of a drug called cromolyn sodium, which is an FDA approved drug for the treatment of ALS. We're testing the effects of this compound in the SOD1 mouse model of ALS. If we have positive results from our mouse study, which will be ending shortly, then we have a plan in place with the clinicians here at Mass General and the Neurology Department who are ready to go and get this drug into clinic.

Another unique project or opportunity that I'm able to work at here at Mass General is a program project centered around the disease called X-linked dystonia parkinsonism. It's a disease that effects people from the Philippines. My colleagues have now honed down on the gene that's important or the disease-causing locus and now my lab is trying to figure out the mechanisms whereby this gene is dysregulated. We have done some more imaging work, again this is not being done in my lab, but being part of this project I feel like I can take ownership together with my other colleagues here. They're learning more about the brain and its connectivity and how it's all changing using MRI.

Given that most of the funding for my lab, especially for the ALS projects, comes from foundations and philanthropy, it would mean a great deal if any of the projects were fruitful or we have positive results. The patients and their families are extremely dedicated and extremely hopeful, waiting for us to do something good in the laboratory. And I really hope that I can give them something like that and that would mean a great deal to me, and I think the patients as well.

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