Historically, it has been difficult to attract pharmaceutical companies to research projects on orphan diseases like Spinal Musular Atrophy because the small patient populations have small profit potential. Consequently, Families of SMA tries to reduce the risks for industrial partners and build incentives for them to work on SMA by providing funding, research tools, and scientific expertise. This strategy effectively lowers the barriers to embarking in SMA drug discovery. Our industry partners are then able to gather the needed preliminary data that ultimately leads to their own and the government’s financial investment in the research.

This also allows the community to build a therapeutics pipeline for SMA, which greatly increases our chances of successfully developing a treatment for SMA. In 2006 FSMA started funding a drug discovery program with the leading biotech company, Paratek Pharmaceuticals. FSMA has invested $2 Million in this program so far. The Paratek project aims to increase SMN protein levels by correcting SMN2 splicing. “For almost 3 years, FSMA has funded research at Paratek Pharmaceuticals to discover new drugs for the treatment of SMA. Further, they have put together an extensive network of scientific experts in the field, which has allowed us to form valuable collaborations and enabled our research to make significant progress. Our research has generated some very exciting results, and without the funding and guidance provided by FSMA it would not have been possible”, says Dr. Paul Higgins , Director of Inflammation Drug Discovery at Paratek Pharmaceuticals.

The NIH followed the initial Families of SMA seed funding for the Paratek drug program by giving an approvable score to a $4 million grant application aimed at bringing this program to the IND stage. This validates the FSMA funding model that includes early funding to biotech and pharmaceutical partners who pursue SMA research.

Professor Stuart Levy , Chief Scientific Officer at Paratek Pharmaceuticals, recently commented on the status of the SMA drug discovery and development project started in 2006 with the support of FSMA: “Paratek has discovered and is developing a non-antibacterial derivative of the tetracycline family which corrects the splicing defect in SMA, leading to increased amounts of the intact SMN protein. Through medicinal chemistry and biology efforts by Paratek scientists, other active non-antibacterial tetracycline derivatives are being designed and synthesized, helping to optimize this lead compound. More recently we have discovered a structurally different non-antibacterial tetracycline derivative which also shows partial correction of the splicing defect. These two compounds and their derivatives are being taken forward in animal studies in preparation for more advanced stages of preclinical drug development.”

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