May is Amyotrophic Lateral Sclerosis (ALS) awareness month. The Muscular Dystrophy Association (MDA) announced the awarding of eight new MDA grants totaling more than $2 million, four of which directly rely on patient samples and banked materials, funding research focused on ALS. This pledge reinforces MDA’s unwavering commitment to the progress of scientific and clinical ALS research and builds on the more than $165 million MDA has already invested in ALS.
The newly funded projects will promote therapy development on multiple fronts. The awarded grants include studies to help provide a better understanding of what causes ALS and pinpoint new drug targets, to develop improved therapeutic strategies, and to ensure that the right tools such as biomarkers are in place to pave the way for successful clinical trials. MDA is also committed to maintaining a robust pipeline of promising early-stage scientists who will make the important discoveries of tomorrow.
Banked patient samples feature in the following awards:
Tania Gendron, PhD | Mayo Clinic, Jacksonville, Fla. | Research grant, $285,000
As many as half of all ALS patients will develop impairments in cognition during their disease course. Dr. Gendron will identify protein biomarkers in blood and spinal fluid that may be useful to predict which ALS patients may develop cognitive impairments. These biomarkers could be helpful to improve patient care as well as inform how clinical trials should be designed.
Jonathan Glass, MD | Emory University, Atlanta, Ga. | Research infrastructure grant, $151,592
The NEALS Consortium is an international organization of 125 research sites that collaborate to support and conduct clinical research in ALS and other motor neuron diseases. This grant will help support the NEALS Consortium’s biorepository, which contains tissues and fluids that are widely used by the community — including MDA-funded researchers — to advance ALS research. The grant will also support the annual NEALS Consortium meeting, where NEALS clinicians share advances and receive training.
Csaba Konrad, PhD | Weill Cornell Medical College, New York, N.Y. | Development grant, $210,000
Developing drugs to treat ALS has proven difficult because there are many causes of the disease, resulting in a highly diverse patient population and a dearth of biomarkers. Biomarkers can be used for predicting disease progression and response to therapy, early detection, and patient stratification in clinical trials — making biomarker discovery for ALS a high priority. Dr. Konrad will use his large bank of skin cells derived from non-familial ALS patients to determine if the cells can be clustered into groups based on changes in size and shape. He hopes to use these models to discover improved biomarkers for ALS.
Marka Van Blitterswijk, MD, PhD | Mark Ebbert, PhD | Mayo Clinic, Jacksonville, Fla. | Research grant, $300,000
Discovery of the C9ORF72 gene repeat expansion has provided some knowledge of ALS disease mechanism, but it’s still unknown how or whether interruptions in the repeated genetic material affect disease variability, development, and progression. Dr. Van Blitterswijk will use a novel technology to analyze the C9ORF72 gene mutation in clinical samples from ALS patients to determine whether the size of the mutation can be used to predict factors such as survival time and cognitive changes. It could eventually pave the way for future therapies and diagnostics based on the variability found in the repeat expansion. This work is a collaboration with the lab of Mark Ebbert, PhD.
The four other research projects, awarded grants, can be viewed in the original press release.
“These new grants show MDA’s commitment to fund groundbreaking research that will one day lead to treatments and cures for ALS.” … “Every grant will have an impact on therapy development, from improving understanding of disease mechanisms to discovering new therapeutic targets and moving these into clinical trials.” – Amanda Haidet-Phillips, PhD, a scientific portfolio director at MDA