December 11, 2007

SMA Groups Hear Biotech Presentations, Plan Trials

Representatives of four spinal muscular atrophy (SMA) advocacy groups -- MDA, Fight SMA, Families of SMA and the SMA Foundation -- met in Bethesda, Md., Sept. 28 and 29 to hear presentations from the National Institute of Neurological Disorders and Stroke (part of the National Institutes of Health, or NIH) and from biotechnology companies and others interested in drug development for SMA.

Sharon Hesterlee, MDA’s vice president of translational research, represented the Association.

The Patient Advisory Group of the International Coordinating Committee for SMA Clinical Trials, under the auspices of NIH, organized the meeting.

Among the presenters were NIH, Tikvah Therapeutics, deCODE Genetics, Paratek Pharmaceuticals, Trophos, MethylGene and PTC Therapeutics.

Most of the drug development research is aimed at increasing production of SMN, the protein needed but deficient in SMA, either by changing the way cells process instructions from a gene known as SMN2 or by increasing the stability of the protein made from the SMN2 gene.

Several candidate compounds were discussed. Among them were analogs (chemical relatives) of indoprofen; sodium phenylbutyrate; quinazoline analogs; tetracycline analogs; TRO19622; and histone deacetylase (HDAC) inhibitors.

Also discussed were the establishment of standardized end points (measurements of strength and other aspects of the disease) and the development of a large, unified network of institutions for the conduct of SMA clinical trials.

The Patient Advocacy Group also released SMA standard of care guidelines in September. (See “SMA Disease Management Guidelines Released”)

This group and the International Coordinating Committee encourage all SMA-affected families to join the International SMA Patient Registry.