“Families of SMA is excited to be awarding new goal-directed drug discovery funding for this gene therapy program. This work follows up on a 2010 grant from FSMA to test the age-dependence in primates of this gene therapy. The new funding will allow us to accomplish several key goals simultaneously”
“This is extremely important funding from FSMA to allow us to collect additional pre-clinical data for a CNS delivered AAV gene delivery into the cerebrospinal fluid, which will be important information to present to the FDA. It also jump starts research prior to obtaining government and commercial involvement which we are actively pursuing. We are quite hopeful for a positive funding decision on a recent NIH proposal for co-funding of this project with FSMA.” Brian Kaspar, PhD, Associate Professor, Principal Investigator The Research Institute at Nationwide Children’s Hospital, The Ohio State University.
The overall project goals are:
1) to optimize the dosing regimen for CNS-delivered SMA gene therapy;
2) to conduct the GLP toxicology, immune response, and bio-distribution experiments required by the FDA;
3) to prepare and hold a pre-IND meeting with the FDA;
4) to submit an IND to the FDA to begin human clinical trials; and
5) to produce clinical grade material for human studies.
The overall timeline for this work is expected to be three years.
This Program was chosen for funding by the FSMA Translational Advisory Committee (TAC), after reviewing multiple potential new drug programs. Every drug program carries risk of encountering hurdles at each of the stages described above. Therefore, a project specific Steering Committee has been put in place, which is comprised of experts in both gene therapy and in SMA biology, with representatives from academia and industry.
This committee will help manage the project, ensuring it progresses in an efficient and well-run manner. In addition, project funding will be awarded upon meeting predetermined milestones, decided on by the Steering Committee.
“I am incredibly excited by FSMA’s decision to support Dr. Kaspar and his team in this very important project. As a pharmaceutical scientist who works every day in drug discovery and development, I am encouraged by the quality of the science and the fact that it aims to address SMA treatment from a different vantage point from other programs in the SMA drug pipeline. This is only the first step, but it’s a critically important step toward assessing whether gene therapy is a viable approach in SMA. Time will tell but I, for one, am incredibly hopeful and look forward to working with FSMA to facilitate the efforts of Dr. Kaspar and his team. I should add that as a parent of an SMA child, I am always looking for a medical breakthrough that could the transform the lives of SMA patients.” Timothy P. Reilly, PhD, DABT Director, Drug Safety Evaluation, Bristol-Myers Squibb. TAC Member.
A major goal at FSMA has been to build the SMA drug pipeline, and we have been investing in drug research since 2000 towards this goal. Even with our community’s current progress in adding programs to the SMA drug pipeline and advancing programs to start clinical trials, FSMA believes it is critical to do more. Statistics show that only 10% of all drugs initiating human clinical trials ultimately receive FDA approval. The new funding announced here by FSMA for this preclinical drug program will help achieve this goal. FSMA has been involved in funding half of all the ongoing novel drug programs for SMA.
About Families of Spinal Muscular Atrophy
Families of SMA funds and directs the leading SMA research programs 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 $50 million in research and 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 85,000 members and supporters throughout the United States, and 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 healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. For more information: