Families of SMA Invests in Clinical Trial Initiatives with Project Cure SMA

Families of SMA believes that it is critical to invest in clinical trial infrastructure to reach our ultimate goal of finding a safe and effective therapy for SMA.  To this end, our investment in this area allows us to accomplish several critical steps on the path to our final goal:

• Establish validated protocols for clinical assessment in every SMA type
• Test re-purposed drugs, already approved for other dieases, in SMA
• Implement clinical trial sites across the US to facilitate pivotal novel drug trials, conducted for FDA approval
  
• To collaboroate with the broader SMA community to achieve clinical trial readiness
  

In 2001 Families of SMA single-handedly established the clinical trial network Project Cure SMA in order to achieve these goals and has solely funded the network since its inception. The network first worked on developing the necessary clinical infrastructure and drug testing protocols required to assess candidate drugs for SMA and later to actually conduct clinical trials. To date Project Cure SMA has conducted five clinical trials with an investment of over $6 million by Families of SMA.

Please click here for the Project Cure SMA website.

Currently, the Project Cure SMA clinical network consists of seven North American clinical testing sites (as well as two affiiate sites in Argentina and Germany) and the entire supporting infrastructure needed to complete a clinical trial. Over 30 professionals are involved. This includes a full-time clinical trials manager (who runs the daily operations), a central pharmacy, a group for statistical analysis of data, an informatics core for data collection, and an outcome measures development group

With a fully operational clinical network in place, Project Cure SMA is poised to expand and do new projects, which can now be initiated faster, more cheaply, and more effectively. Ideally, we now want to expand our clinical work in three main ways:

• Initiate trials in a broader population of SMA patients.
• Add clinical testing sites in North America.
• Prepare to test novel drugs, not yet approved by the FDA and specifically designed for SMA.

In fact now that the Project Cure SMA team has completed their first randomized Placebo-Controlled Phase II trial, called CARNI-VAL, to test the combined efficacy of Carnitine and Valproic acid in children with type II and III SMA, they have widened the scope of their projects.  For example, the SMA population included in the two trials they are currently conducting has broadened to the following:

• The VALIANT Trial to test the safety and efficacy of valproic acid in ambulatory adults with SMA

• The CARNI-VAL Type I Trial to test the safety of valproic acid and carnitine in infants with Type I SMA.

Please click here to learn details about these two ongoing clinical trials.

More details about Project Cure SMA projects can be found in a recent issue of Compass.

We anticipate that the clinical testing of novel therapeutics (non-FDA approved drugs) will commence for SMA in the next 2 years. Having a fully functional clinical network with validated testing protocols and a sufficient number of sites to conduct a Pivotal SMA drug trial will help attract and encourage biotech and pharmaceutical companies to invest in SMA drug development.

Families of SMA and the Project Cure Clinical Network are collaborating with the broader SMA community on clinical research issues.  Both FSMA and Project Cure Representatives are part of the International Coordinating Committee for SMA Clinical Trials (ICC).  As part of the Patient Advisory Group (PAG) for the ICC, FSMA actively helped organize and fund the SMA Drug Summit in Bethesda Maryland in September 2007. More information about the Drug Summit can be found here.