Elk Grove Village, IL, December 16, 2012 --(PR.com
)-- Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program. This new research funding will support a project at the Ann and Robert H. Lurie Children's Hospital of Chicago at Northwestern University to help discover new SMA drug targets using animal and cellular models of the disease.
The goal of this project is to study a novel biological pathway that may be impaired in SMA. Understanding the involvement of this pathway in SMA pathology could lead to a greater understanding of the mechanisms of the disease. It could also have potential therapeutic implications later by identifying new drug targets.
The researchers will use SMA mouse and zebrafish models, as well as human SMA and mouse motor neurons, to further investigate a novel mechanism that leads to motor neuron degeneration in SMA. This mechanism involves cellular regulation of two proteins called HDAC5 and Cdk5, when SMN is lowered.
Earlier this year, FSMA's Advisory Boards met to evaluate new research funding for 37 basic research grant applications and 7 drug discovery projects for SMA. The organization is planning to award $1.4 Million in new research funding over the next few months. This new round of research funding will be allocated into three areas: 1) Basic Research to understand the disease and provide ideas for drug making, 2) Drug Discovery to develop new SMA therapies, and 3) Clinical Research to help test new drugs effectively and to improve care for patients.
This grant to Dr. Ma of Northwestern University will be exploring the molecular pathways controlling degeneration in SMA motor neurons and looking for ways to identify and validate new drug targets in these pathways. This will help answer the key basic research question of are there SMA drug targets, in addition to SMN itself?
The basic research that Families of SMA has funded, through 145 research grants to 75 institutions around the world, has delivered major discoveries:
-The cause of SMA is now known. Which means treatments can be developed that correct the underlying cause of the disease rather than just reduce symptoms.
-A back-up gene for SMA has been identified. Which means a straightforward drug target is already in the body: a built-in switch for new therapies to work on.
Using this knowledge, there are now 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.