Cecil B. Day Laboratory for Neuromuscular Research
The Angel Fund for ALS Research supports the ALS research at the Cecil B. Day Laboratory for Neuromuscular Research at UMass Medical School in Worcester, MA. The lab is under the direction of Dr. Robert H. Brown, Jr.
Research update – September 2017
There has never been a more exciting and promising time in ALS research.
One main area of focus for Dr. Brown and his team at UMass Medical School is the genetic basis of ALS and its implications for treatment.
Over the last year, the UMass team has identified several new ALS genes. An international consortium, spearheaded by Dr. John Landers at UMass (who works closely with Dr. Brown) has identified five new ALS genes, bringing the total to more than 50. Perhaps most importantly, each gene is a clue to the biology of ALS and to possible ALS treatments. One way to dissect these genes further is to develop fruit fly models using the ALS genes. This year alone, Dr. Fen-Biao Gao also at UMass has published several seminal studies about ALS genetics based on the fruit fly studies.
A major lesson is that ALS genes appear to cause disease by having a toxic influence on motor neurons. This suggests that reducing the activity of these genes may be beneficial. Fortunately, key discoveries in the technology to silence genes were made by investigators at UMass.
Working with those experts, and with the Gene Therapy Center at UMass, Dr. Brown has been able to “turn off” some of the defective ALS genes. This worked extremely well in mouse models of ALS and has proven to be safe in larger animals. Accordingly, the UMass team believes that we are closer than ever testing these approaches in humans.
At present, the UMass team is focusing its efforts on two major ALS genes, known as SOD1 and C9orf72 (the two most common ALS genes). However, a key point is that these experiments set the stage for a much broader program to shut down many other ALS genes. Moreover, this therapeutic strategy may also prove applicable in non-familial ALS.
Today, we as a community hold great hope that these studies, moving from genetics to targeted therapies, will slow the progression of ALS and provide substantial benefit to ALS patients and their families. The entire team at UMass Medical is deeply grateful to the Angel Fund for its continuing support of this research program.
Robert Brown, Jr., D.Phil., M.D.
We will find the cure!!