NIH Convenes Meeting on SMA Treatment Development

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In a sign of the significant progress being made in spinal muscular atrophy (SMA) research, the National Institutes of Health (NIH) convened a meeting in October that examined the status of laboratory research, clinical trials and strategies for drug development in this disease, with an eye toward speeding the process.

The meeting, which took place in Rockville, Md., Oct 27-29, 2010, and which was webcast in real time to online participants, brought together representatives from disease-specific advocacy organizations like MDA, as well as the pharmaceutical and biotechnology industries, the academic research community, and government agencies, such as the U.S. Food and Drug Administration (FDA) and NIH.

Scientific, medical, regulatory and industry perspectives were included in the presentations and discussions.

Several MDA-associated physicians and researchers participated, as did MDA Medical Director and Executive Vice President for Research Valerie Cwik, and Annie Kennedy, MDA Vice President for Advocacy.

Conferees discussed:

• the possibilities for development of molecules to increase production of full-length SMN, the protein that's deficient in SMA;
• the status of and future for gene therapy to supply needed SMN genes; and
• the potential use of stem cells in treating SMA.

Among the many challenges are defining which SMA subgroups to target with which therapies; deciding whether and how to conduct newborn screening to identify and treat SMA as early as possible; and designing clinical trials that ask the right questions and measure meaningful outcomes.

The meeting organizers said a full summary of the proceedings can be expected.

Meaning for people with SMA

That NIH saw fit to convene a three-day meeting of stakeholders in SMA therapeutic development can be taken to mean that such development is a reality and has progressed far beyond the realm of "something, someday."

Rather, this was a "let's roll up our sleeves" meeting, in which several biotechnology and pharmaceutical companies expressed an interest in developing treatments for SMA.

MDA will persevere in its central role in therapeutic development for SMA by funding basic and clinical research and working with other stakeholders in this community.