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.” The project is being led by Vikram Khurana, M.D, Ph.D. (Massachusetts General Hospital) and could lead to genomics and small molecule discovery platform for multiple system atrophy (MSA).
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.”
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The data secured through this seed grant from the MSA Coalition will be critical to attract future federal NIH funding for this endeavor.
“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.
For more information or to donate to The Multiple System Atrophy Coalition, please visit: http://www.Multiple-System-Atrophy.org.
About Multiple System Atrophy
Multiple system atrophy (previously known as Shy-Drager Syndrome) is a rare and fatal disorder with less than 15,000 Americans diagnosed and 35,000 undiagnosed at any given time. According to the National Institutes of Health, “Multiple system atrophy is a progressive neurodegenerative disorder characterized by symptoms of autonomic nervous system failure such as fainting spells and bladder control problems (neurogenic orthostatic hypotension), combined with motor control symptoms such as tremor, rigidity, and loss of muscle coordination. MSA affects both men and women primarily in their 50s. MSA tends to advance rapidly over the course of nine to 10 years, with progressive loss of motor skills, eventual confinement to bed, and death. There is no remission from the disease. Currently no cure is available.”