Families of Spinal Muscular Atrophy Awards $600,000 for Basic Research to Identify New Approaches for SMA Therapies

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These funds were awarded as part of Families of SMA’s basic research grant program. Basic research looks into the causes of SMA, revealing new and better ways of making drugs to target the disease.

Families of SMA has announced seven grants, totaling $600,000 in new funding, for research into the causes of Spinal Muscular Atrophy (SMA). SMA affects an estimated 1 in every 10,000 children, and is the number one genetic killer of children under age two.

These funds were awarded as part of Families of SMA’s basic research grant program. Basic research looks into the causes of SMA, revealing new and better ways of making drugs to target the disease. Families of SMA also funds a drug discovery grant program, which applies the findings of basic research to drug development. Grants in that area will be announced later this year.

We are leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. There are currently five SMA drugs being tested in clinical trials, advances that would not be possible without the important discoveries made in basic research.

Many important questions in SMA basic research remain unanswered today. The current round of new research awards from Families of SMA will help answer some of these, including:

-What function does SMN protein perform in motor neurons?
Dr. Han from the University of Colorado will investigate what controls the correct distribution of SMN protein into motor neurons, providing a greater understanding of SMN function.

-What tissues are affected by reduced SMN protein?
The grants to Dr. Ko at the University of Southern California, to Dr. Ebert at the Medical College of Wisconsin, and to Dr. Burnett will help determine the exact cells that influence SMA disease pathology.

-Are there SMA drug targets, in addition to SMN itself?
The funding to Dr. Murray of the University of Edinburgh will explore the molecular pathways controlling degeneration in SMA motor neurons to identify possible new drug targets in these pathways. Dr. Kothary at the from the Ottawa Hospital Research Institute will investigate non-SMN mediated mechanisms that provide therapeutic benefit in mouse models of SMA.

-What are the best measures in animal models of SMA to predict human drug responsiveness?
Drs. Lutz and Bogdanik from Jackson Laboratory will lead a multi-center team investigating electrophysiological endpoints, that are currently used in patients, in drug testing in mice.

Families of SMA invests in four areas of research: Basic Research, Drug Discovery, Clinical Research, and Care Research. FSMA has invested over $55 Million in SMA research since our inception in 1984, with $35 Million in funding in the past decade alone.

About SMA
Spinal Muscular Atrophy is a genetic disorder caused by a mutation in the survival motor neuron gene 1 (SMN1). This mutation affects the motor neurons, which are nerve cells in the spinal cord that control muscle movement. Depending on the severity of the disease, individuals with SMA may experience difficulty crawling, sitting, walking, controlling their head and neck—or even breathing, swallowing and eating. SMA is the number one genetic killer of children under two. An estimated 1 in every 10,000 children are affected, and nearly 8 million Americans are genetic carriers.

About Families of SMA
Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. Families of SMA has invested over $55 million in research, including $35 million in the last decade alone, and has been involved in funding almost two-thirds of all the ongoing novel drug programs for SMA. Families of SMA invests in four areas of research: Basic Research, Drug Discovery, Clinical Research, and Care Research.

Families of SMA is also a leader in offering ongoing support for families affected by SMA. In the last year alone, over 3,000 families received support from the organization. Families of SMA also hosts the Annual SMA Conference, the largest gathering of SMA families, researchers, and healthcare providers.

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Kenneth Hobby