Families of Spinal Muscular Atrophy Launches Next Phase of Drug Development Efforts for SMA

Nov. 11, 2010 - PRLog -- Families of SMA announces Request for Proposals (RFP) for new drug programs to develop therapies for Spinal Muscular Atrophy.
FSMA has made significant progress in advancing new therapies for SMA, starting with funding the first ever SMA drug program in 2000. The organization is now launching the next phase of therapeutic development work for SMA.  FSMA is aiming to fund two new preclinical drug development programs in 2011, with more to follow.  These multi-million dollar collaborations will focus on innovative methods of developing novel therapies for SMA, including both biologic and small molecule approaches. These programs will be multi-year in scope with a typical duration of three years.  The requested proposals will be reviewed by the FSMA Translational Advisory Committee next spring.

Families of SMA has been investing in and advancing pre-clinical drug research since 2000, with a total investment of $17 million in this area.  Over the last decade, our community has made great strides in this area, and currently there are a number of promising drug avenues for SMA emerging.  This progress is also clearly demonstrated by the successful transition of several potential therapies to industry and government funding after early stage investment and research leadership by Families of SMA.

Drug development is high risk with less than 10% of therapies that reach clinical trials ultimately receiving FDA approval. Our research model is based on advancing multiple opportunities at once, and then incentivizing and encouraging companies and the government to fund activities in later clinical stages. Having multiple “shots on goal” gives us the best potential for reaching our goal of a treatment and cure for SMA.

FSMA has made significant progress in the area of discovering and developing new therapies for SMA.  To date we have funded and advanced five such ventures:
1. Since 2000 with an investment of $13 million, the Quinazoline Compound to increase production of the back-up SMN2 gene, which was licensed to Repligen in 2009 for clinical development and is now in preparation to file for approval to start clinical trials. This new drug candidate recently received the first ever Orphan Disease Designation from the FDA for SMA.
2. Since 2003, the Oligonucleotide Program at UMASS, which identified an important therapeutic strategy using genetic material to modify the splicing of the back-up SMN2 gene.  This discovery was recently licensed to ISIS Pharmaceuticals, who now leads the clinical development program.
3. Since 2003 with an investment of $2 million, the MotorGraft Cellular Therapy Program at California Stem Cell Inc, University California, Irvine and Johns Hopkins, which completed the first ever pre-Investigational New Drug Meeting with the FDA for SMA.
4. Since 2004 with an investment of $2 million, the Tetracycline Program at Paratek Pharmaceuticals to correct SMN2 splicing, which is now being funded in part from a multi-million dollar award by the NINDS.
5. Starting in 2010, the Gene Therapy Program at Nationwide Children’s Hospital in Ohio, to replace the entire SMN1 gene.

As demonstrated by the projects described above, one of the long term goals at FSMA is to fund and de-risk early stage drug discovery programs for SMA.  At the very earliest stages of drug development programs have less than a 1% chance of FDA approval.  This inherent risk along with low potential for profit because of the small patient population has traditionally hindered industry from working on orphan diseases.

FSMA has actively reduced the barriers to early stage SMA drug discovery programs by providing:  1) early seed funding, 2) access to tools and reagents, 3) expert SMA advisors, and 4) established clinical trial protocols and networks.

The FSMA rational for providing these incentives to industry to work on SMA drug discovery is to build a large and diverse therapeutic pipeline.  Through this new RFP, FSMA will continue its long-term commitment to investment in this area. This next phase of SMA drug development will build on our success of advancing 5 programs over the last 10 years, and rapidly create a broader SMA therapeutic pipeline.

The selection of the most promising projects will be governed by the FSMA Translational Advisory Committee (TAC), which is made up of experts from multiple facets of drug development.  The use of TAC to select drug programs for funding fits perfectly with the overall 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 funded projects are run in a professional and efficient manner under the guidance of world-class experts.

About Families of Spinal Muscular Atrophy:
FSMA  is dedicated to creating a treatment and cure by: Funding and advancing a comprehensive research program; Supporting SMA families through networking, information and services; Improving care for all SMA patients; Educating health professionals and the public about SMA; Enlisting government support a; Embracing all touched by SMA in a caring community. www.curesma.org

Families of SMA funds and directs the leading SMA research programs. Our successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients.
-Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA.
-FSMA has funded 5 leading new drug development programs for therapies specially designed to treat SMA.
FSMA is a non-profit, 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters.

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Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:
- Funding and advancing a comprehensive research program
- Supporting SMA families through networking, information and services
- Improving care for all SMA patients