Families of Spinal Muscular Atrophy Announces Launch of New Translational Advisory Council (TAC) to Select and Govern New Drug Discovery Projects for SMA.
February 18, 2010.

The ultimate research goal at Families of Spinal Muscular Atrophy is to develop safe and effective therapies for SMA.  In the drug discovery area, our strategy has been to provide incentives for industry to invest in SMA drug discovery to build a robust and diverse therapeutic pipeline.  Families of SMA has been funding drug discovery translational research since 2000 and will be increasing our efforts in the coming years.

Traditionally, it has been difficult to attract major pharmaceutical companies to conduct research for orphan diseases like SMA, which have small patient populations with small potential for profit. FSMA has taken the strategy of providing seed funding to encourage biotech and pharmaceutical partners to engage in SMA drug research.  Our objective is to reduce the risk for industrial partners by simultaneously providing funding, research tools, scientific expertise, and established clinical networks.

With this philosophy, we have invested in three distinct programs to date, each of which recently reached successful milestones:
1) The Families of SMA Quinazoline Program has been licensed to Repligen Corporation for clinical development.
2) The Tetracycline Program at Paratek Pharmaceuticals received a multi-million dollar award from the NIH. 
3) The FSMA Motor-Neuron Replacement Program at California Stem Cell, Inc. and UCI completed a Pre-IND Meeting with FDA.
Each of these programs is based on a different scientific approach to developing a therapy for SMA and together represent an investment of over $16 Million from Families of SMA.  A detailed description of our strategy for drug discovery can be found by clicking here.

In order to build on these recent successes and rapidly create a robust SMA therapeutic pipeline, Families of SMA plans to start and fund several new translational research projects.   To achieve this goal effectively, Families of SMA is now launching a formal translational research program that will provide funding and direction to new and diverse SMA drug discovery projects. 

To govern this new translational research program at FSMA, we are launching a new Translational Advisory Council (TAC).  Our immediate goals for the TAC will be to:
1) Assess feasible strategies for SMA drug discovery and development,
2) Release a request for proposals focusing on those strategies,
3) Evaluate the submitted proposals, and then
4) Fund and then manage the best new translational projects.

Establishing the TAC fits very well into the general FSMA research funding model, which is based on the need for expert and independent prioritization and oversight of research projects.  This approach ensures that FSMA funds only the most promising research, and that the funded projects are run in a professional and efficient manner under the guidance of world-class experts. 

The members of the TAC will be announced in March, 2010.  We anticipate these members will be experts in a wide range of drug discovery areas, including in assay development, high throughput screening, neurobiology, medicinal chemistry, and toxicology, and will be from diverse backgrounds in academia, industry and government.

This new advisory group and funding program will fill a critical position in the step between early academic research that creates seed ideas for new therapies and later clinical and medical stages.  Translational research is the critical middle step that takes research ideas and makes them into practical solutions for patients.

The FSMA Basic Research Program is governed by our Scientific Advisory Board (SAB).

The FSMA Patient Care and Family Support Program is governed by our Medical Advisory Council (MAC).