UMMS Researchers Make Progress in ALS Research

UMMS Researchers Make Progress in ALS Research

It has already been a year since the ALS Ice Bucket Challenge took the internet by storm, causing countless American households, celebrities and public figures to show support for ongoing efforts to find a cure for amyotrophic lateral sclerosis. In the wake of the viral campaign, a team of scientists at UMass Medical School have made significant progress in the laboratory.

“There has been enormous progress and there are grounds, very legitimately, for hope and encouragement,” said Robert H. Brown Jr., DPhil, MD, the Leo P. and Theresa M. LaChance Chair in Medical Research, and chair and professor of neurology.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that causes a gradual breakdown of the body’s motor neurons in the central nervous system. While the exact cause of this debilitating and fatal disease is not fully understood, scientists estimate about 10% of all ALS cases are hereditary. Research is still a long way from finding a definite cure, because even though investigators at UMMS and other institutions have already determined a number of genes known to trigger the disease’s inherited form, nearly half of these cases still have an unknown genetic origin. What Dr. Brown and his team of researchers are working on now is a way to inactivate these troublesome genes.

“In terms of therapy development, there has been real progress. Combining forces with the incredible staff in the gene therapy unit [at UMMS], it’s been possible to show that one can put gene silencing elements into the nervous system using viral delivery, and in so doing, turn off some of the toxic genes that promote the disease,” said Brown. “So far, using animal models, this approach has led to increased survival. The question is, can we take this most rapidly and far enough along to get it into humans and the answer is, ‘Yes, I think we can,’” said Brown.

Watch the video below to learn more about the progress genetic research has made so far in the field of ALS:


Last year a monumental push to increase research funding for Amyotrophic Lateral Sclerosis (ALS), known as the ALS Ice Bucket Challenge, raised over $220 million globally for ALS research and patient care.  The video based social media challenge is set to return this year.

Co-founder Pat Quinn stated in the announcement for this years’ upcoming challenge, “Last year gave the ALS community hope for the first time in a long time. But we still need the public’s help to keep the momentum going. We plan to do the ALS Ice Bucket Challenge again this August and every August until we find a cure.”

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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