Biopharmaceutical company Genervon has announced what it considers encouraging results from a "compassionate use" trial of its experimental drug GM604 in a single patient with advanced amyotrophic lateral sclerosis (ALS).
Stem cells -- cells at an early stage of development that can mature and fulfill specific roles depending on their environment and signals they receive – have intrigued scientists as a possible treatment for amyotrophic lateral sclerosis (ALS) for several years. Now, two biotechnology companies – BrainStorm Cell Therapeutics and Neuralstem – are reporting encouraging results from ALS clinical trials.
Stanley Appel, a professor of neurology at Methodist Neurological Institute in Houston, was awarded an MDA research grant totaling $253,800 over three years to study immune mechanisms in amyotrophic lateral sclerosis (ALS). Experiments previously conducted by Appel and colleagues in mice with an ALS-like disorder have shown that the immune system acts to protect nerve cells during the early stages of the disease but that it damages nerve cells in the disorder’s later stages.
Muralidhar Hegde, a professor at the Houston (Texas) Methodist Research Institute, was awarded an MDA research grant totaling $253,800 over three years to study whether deficient repair of DNA strand breaks by a protein called TDP43 is involved in amyotrophic lateral sclerosis (ALS) causation. Hegde will conduct experiments in mouse and human cells and says the result could lead to a “major paradigm shift in our understanding of ALS pathology” and “open up new avenues for therapeutic interventions.”
Amanda Haidet-Phillips, a postdoctoral student at Johns Hopkins University School of Medicine in Baltimore, was awarded an MDA research development grant totaling $150,717 over three years to investigate how cells known as upper motor neurons, located in the top part of the brain, degenerate in amyotrophic lateral sclerosis (ALS).
Aaron Gitler, an associate professor of genetics at Stanford University in Stanford, Calif., was awarded an MDA research grant totaling $253,800 over three years to investigate how mutations in the gene for a protein called profilin 1 cause amyotrophic lateral sclerosis (ALS).
Maurizio Grimaldi, leader of the Neuropharmacology/Neuroscience Laboratory at Southern Research Institute in Birmingham, Ala., was awarded an MDA research grant totaling $253,800 over three years to study the development of molecules that have neuroprotective effects and have the potential to be used as therapies for the treatment of amyotrophic lateral sclerosis (ALS).
CHIGAGO, August 15, 2014 – Having a bucket of ice water dumped on your head may not seem dignified, but it is certainly helping MDA and other ALS-fighting organizations raise awareness and funds as the viral social media craze continues to sweep across America.
Today, Steven M. Derks, President and CEO of the Muscular Dystrophy Association, joined the ranks of tens of thousands of people willing to get chilled in the ALS Ice Bucket Challenge.