Families of SMA believes that it is critical to invest in clinical trial infrastructure.  This allows us to accomplish several goals.  The first is to prepare for the future by developing useful drug-testing protocols for each SMA type.  Clinical trial protocols need to be tailored to SMA type since functional abilities differ.   Having established protocols will allow novel drug candidates for SMA to be clinically assessed in all SMA patients as rapidly as possible after identification.  Our second and more immediate aim is to begin testing drug candidates, FDA-approved for other diseases, for therapeutic benefit and safety in SMA.

FSMA has established and has solely funded Project Cure SMA, a clinical trials network. The network has focused first on developing the necessary clinical infrastructure and drug testing protocols needed to assess candidate drugs for SMA and later to actually conduct clinical trials.

 Currently, the Project Cure SMA clinical network consists of six North American clinical testing sites (as well as one in Argentina) 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.

The Project Cure SMA team has just completed the Phase II CARNI-VAL clinical trial to test the combined efficacy of Carnitine and Valproic acid. With a fully operational clinical network in place, Project Cure SMA is poised to expand and begin several new projects, which can now be initiated faster, more cheaply, and more effectively. 

 These include the VALIANT Trial to test the safety and efficacy of valproic acid in ambulatory adults with SMA. and the CARNI-VAL Type I trial to test the safety of valproic acid and carnitine in infants with Type I SMA. 

We are now looking to expand our clinical work in three main ways:

1. Initiate trials in a broader population of SMA patients.
2. Add clinical testing sites in North America.
3. Prepare to test novel SMA drugs not yet approved by the FDA.

More details about recent 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 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.