A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the National Institute of Neurological Disorders and Stroke (NINDS) to accelerate the development of therapeutic candidates for this motor neuron disease.
A number of SMA voluntary organizations and patient advocate communities, including MDA, played a key role in the establishment of the SMA Project and have provided it with ongoing support.
NINDS' December 2012 progress report states “a late-stage lead compound has been developed that may be of interest to a pharmaceutical/biotechnology company to further develop and take into clinical trials.”
In addition, the report notes that “lessons from the SMA Project have guided the design of other drug development programs at the NIH, including the NIH Blueprint Neurotherapeutics Grand Challenge.
“Furthermore, NINDS’ choice of SMA for this pioneering effort has helped catalyze increased interest in SMA therapy development. Development of a variety of candidate drugs and biologics targeted to SMA is now under way at several pharmaceutical and biotechnology companies, as well as in multiple academic laboratories. Clinical trial resources, diagnostic screening tests and biomarkers are being developed to facilitate clinical trials of candidate therapeutics for the disease.”
The complete report, The NINDS Spinal Muscular Atrophy (SMA) Project Completes Discovery Phase of Therapeutics Development Program, is available on the NINDS website.
Annie Kennedy, MDA senior vice president for advocacy, credited NINDS for initiating the $20 million SMA Project in 2003 “before ‘translational research’ was a household term.” (Translational research seeks ways to turn promising laboratory findings into actual treatments.)
“Of all the diseases that NIH could have selected for this project — thousands of disorders to choose from — they chose SMA,” Kennedy noted. She said this selection was due to a variety of factors, including promising SMA laboratory research; existing infrastructure for conducting research (thanks to investments by SMA advocacy groups); advocacy efforts by SMA organizations and the clinical and research communities; and the good chance that the lessons learned in developing treatments for SMA also would benefit research into other diseases.
Now that this phase of the SMA Project is complete, the NINDS report notes that the agency “actively encourages feedback and input from the SMA community regarding the path forward.”