Families of Spinal Muscular Atrophy Awards $150,000 to Dr. Lee Rubin at Harvard to Advance a New SMA Drug Discovery Program

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Dr. Rubin's project at Harvard University is the first of three drug discovery awards that will be given by Families of SMA in the next few months.

Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies. Dr. Rubin's project at Harvard University is the first of three drug discovery awards that will be given by Families of SMA in the next few months.

Description of FSMA Funded Project: A patient derived motor neuron screen to identify novel SMA Therapeutics.

Objective: To identify novel SMA therapeutics by using patient-derived motor neurons for drug discovery and preclinical testing.

Research Strategy: The researchers have made induced pluripotent stem cells from Type I and Type II SMA patient skin biopsies, and produced large numbers of motor neurons that can be used to model SMA. They will use these motor neurons in a screen for drugs that increase SMN levels. The researchers will test the candidate drugs in a 35 cell line panel that includes motor neurons from different patients and different SMA types.
Significance: By using human motor neurons to screen for new SMA drugs, the researchers hope to identify compounds that can increase functional SMN levels in the cell type most affected by the disease. They will prioritize compounds that are already approved for use so they can move through the development pipeline more quickly. The researchers will test the candidate compounds across a panel of SMA patient-derived cells so that they can identify drugs that are effective across many genetic backgrounds and disease severities. The researchers hope that this innovative approach will lead to the discovery of drugs that are mechanistically unique so that they may be used in addition to the therapies that are currently being developed. These cell lines can also help assess existing SMA drug candidates.

Earlier this year, FSMA’s Translational Advisory Committee (TAC) met to evaluate research funding for new drug discovery projects for SMA. Families of SMA is planning to award three new drug discovery projects over the next few months.

This current award to the Rubin lab at The Department of Stem Cell and Regenerative Biology at Harvard University is for one year for $150,000 with the goal of conducting cell-based screening in human motor neurons for new SMN inducing drug candidates. Subsequent funding to advance any newly identified molecules towards an Investigational New Drug application with the FDA will be evaluated by the TAC through the next planned funding cycle in Preclinical SMA Drug Development in 2014.

The SMA Drug Pipeline:
There are now have 13 SMA drug programs in development, including 3 in clinical trials. This pipeline has expanded from just 4 programs 5 years ago. Families of SMA has funded half of all the ongoing drug programs for SMA. The FSMA research approach funds programs at early stages, and then partners with companies to take them through clinical trials. Supporting multiple programs gives different approaches for a SMA therapy, which increases the chances of success and accelerates the timeline to a treatment and cure.

Funding for New Drug Discovery Programs.
Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Research in 2012. FSMA has been investing in and advancing drug research since 2000. The goal of drug discovery funding at FSMA is to build a large and diverse therapeutic pipeline to maximize the chances for success in finding a treatment for SMA. FSMA has been involved in funding half of all the ongoing SMA drug programs to date. New awards in the current round will focus on providing critical funding for early-exploratory projects assessing novel therapeutic approaches for SMA. The FSMA research funding philosophy is based on expert and independent 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.

Families of SMA is the world’s leader focused on funding SMA research 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. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization’s work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients.

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