SOURCE: Families of Spinal Muscular Atrophy

July 20, 2011 16:08 ET

Families of Spinal Muscular Atrophy Awards $3 Million in New SMA Research Funding

ELK GROVE VILLAGE, IL--(Marketwire - Jul 20, 2011) - Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program. The new funding awards will be allocated into three distinct research areas: 1)Basic Research to understand the disease and provide seed ideas for drug making, 2)Drug Discovery to develop new SMA therapies, and 3)Clinical Research to provide the means to test new drugs effectively.

$700,000 in Funding for New Basic Research Grants.
Basic research helps solve critical unanswered questions in SMA biology. This information then reveals new and more effective ways of making SMA drugs, helping build a robust SMA drug pipeline. The current round of funding focuses on understanding why motor neurons are particularly sensitive to lowered levels of SMN protein, identifying new drug targets for SMA, and assessing new assays and biomarkers for SMA.

$1.5 Million in Funding for Two New Drug Discovery Programs.
FSMA has been investing in and advancing novel pre-clinical drug research since 2000. The goal at FSMA for drug discovery funding is to build a deep and diverse therapeutic pipeline to maximize the chances for success in finding a treatment for SMA.

This new funding is critical as FSMA now begins to fund several programs concurrently which have different approaches to developing a therapy for SMA. This will both increase the chances of success and also accelerate the timeline to a treatment and a cure.

$500,000 in Funding for Clinical Research Projects.
The SMA community needs to be ready to conduct clinical trials on new SMA therapeutics by having validated trial endpoints, established means for patient recruitment, and experienced clinical trial sites. The funding goals in this area are to help develop the needed infrastructure to conduct pivotal FDA clinical trials on novel SMA drugs.

Specific project award details for each of these three important research areas will be announced over the next two months.

FSMA will also invest $250,000 for the 16th Annual SMA Research Group Meeting to be held in June 2012. This meeting is the largest scientific conference dedicated to SMA research in the world. It provides a forum for research experts from around the world to share unpublished data, to engage in open communication, and to foster research collaborations, all of which increase the pace of SMA research and ultimately accelerate the discovery of a treatment and cure for SMA.

"Families of SMA is excited to announce $3 Million in new research investments," says Jill Jarecki, PhD, FSMA Research Director. "FSMA is committed to funding a broad research program that includes targeted basic research projects, new drug discovery programs, and clinical research initiatives. We believe this coordinated approach to research is critical to accelerating the FDA approval of effective SMA treatments."

The FSMA research funding philosophy is based on 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. Please see for short biographies of FSMA scientific advisors.

In addition to funding critical research, FSMA is increasingly dedicating resources to areas that focus on improving patient care and supporting families. The organization will fund $950,000 for programs in this area over the next year.

The funds FSMA dedicates to advancing research, supporting families, and improving patient care are raised from family driven fundraising efforts across the United States.

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 and; Embracing all touched by SMA in a caring community.

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 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.

Contact Information