A $50,000 Research Grant Awarded by The Multiple System Atrophy Coalition® for a “Stem Cell-Based Therapeutics Platform”

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Massachusetts General Hospital

The MSA Coalition announced in January 2014 the funding of five MSA research grants for a total of $219,000 including the project “Stem cell-based Therapeutics Platform for MSA.”

March 14, 2014

The Multiple System Atrophy Coalition has awarded a $50,000 grant to Dr. Vikram Khurana of Massachusetts General Hospital to develop a drug discovery platform for MSA using human patient-derived stem cell based models of the disease. The research project, entitled “Stem Cell-Based Therapeutics Platform for MSA” is set to begin in the second quarter of this year.

“I propose to develop stem cell-based models for MSA to look for signs of alpha-synuclein toxicity, the protein that aggregates in MSA and Parkinson’s disease,” stated Dr. Khurana. “If found, we will assess whether we can reverse such phenotypes by genes and small molecules we uncovered in recent work on Parkinson’s disease. I hope this work leads to a novel functional genomics and small molecule discovery platform for MSA.”

“As I transition to leading my own independent laboratory, I am committed to bringing to bear my training to develop a multidisciplinary clinical and research program focused specifically on MSA,” continued Dr. Khurana. “The data secured through this seed grant from the MSA Coalition will be critical to attract future federal NIH funding for this endeavor.”

Multiple system atrophy is a rare and fatal neurodegenerative disorder affecting approximately 50,000 Americans. At present there are no known disease modifying treatments or cures available.

“Because MSA is so rare there is not proactive interest from commercial entities to invest in discovering disease modifying treatments for MSA,” stated Pam Bower, a Board member for The MSA Coalition. “Additionally, governments are spending less today than they did ten years ago on rare disease research. As a result, researchers interested in multiple system atrophy struggle to find funding for even their most critical projects. We are pleased to be able to fund Dr. Khurana’s novel drug discovery platform. Hopefully our research grant will lead to the discovery of much needed life-saving medications for those currently affected by MSA.”

About The MSA Coalition® Research Grant Program
The Multiple System Atrophy Coalition established a dedicated MSA research fund in 2011. Then, the organization established an MSA Research Grant Program along with a scientific advisory board (SAB) in 2013. The goal of the MSA Research Grant Program is to identify and fund MSA research projects that have the potential to discover a cause, disease modifying treatments, and hopefully a cure. The world-class SAB ensures that The MSA Coalition is funding the most promising research. At present, 75% of each donation received by The MSA Coalition goes directly into the research fund. The other 25% used to fund advocacy, awareness, educational initiatives and minimal operating expenses.

Visit  Multiple-System-Atrophy.org for more information.

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