Build a Therapeutic Pipeline for SMA:
While human therapies are not usually an immediate outcome of fundamental research programs, this type of research helps us build a therapeutic pipeline for SMA.  Basic research discoveries will fuel the SMA therapeutic pipeline and drive innovation in SMA drug discovery by revealing new ways to make SMA drugs.  Basic research expenditures are an investment in the future discovery of innovative and alternative drug development strategies.  Having a therapeutic pipeline of drug approaches with more than one route for developing an SMA drug is essential to successfully finding an effective SMA treatment.  Our current drug strategies may not benefit every SMA patient, and different drug options that work in distinct ways may be needed to help each and every person living with this disease. Finally, drug development in a very high-risk exercise, and over 80% of drugs that are tested in clinical trials fail to obtain approval by the FDA.

Advance the Quinazoline Program to Human Testing:
With a clinical candidate in hand, we have recently begun generating an Investigational New Drug application (IND) for the FDA.  This application includes an extensive series of preclinical safety studies, which take about 12 months to complete. If the FDA accepts the IND application, first-in-human testing can be initiated with safety studies, typically in healthy volunteers.

Expand the Number of Clinical Sites and Test Novel Drugs:
Currently, the Project Cure SMA clinical network consists of six clinical testing sites and the entire supporting infrastructure needed to complete a clinical trial, over 30 professionals are involved.  Now that an efficient drug-testing infrastructure has been established we are poised to expand and begin several new projects.

• Add clinical testing sites.
• Prepare to test novel SMA drugs not yet approved by the FDA. 

With a fully operational clinical network in place, new trials can be initiated faster, more cheaply, and more effectively.  We anticipate that the clinical testing of novel therapeutics 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.

Conduct Safety Testing with Stem Cells for SMA:
Doing the key safety study for stem cells in parallel with the efficacy study will save approximately one year in getting to clinical trials.  Ultimately, we hope to use these studies as a springboard to clinical trials in humans with paralysis from a variety of causes including spinal muscular atrophy, and then the more complex situations of traumatic spinal cord injury and ALS.
Our specific goals in this area are:

• Production of human motor neurons for human clinical trials. 
• Actual IND to FDA to begin testing stem cells in humans for SMA.