SMA Drug Pipeline Continues to Grow

This is an exciting time for the entire SMA community, as we celebrate another year of increasing momentum toward our goal of a treatment and cure for SMA.

It’s also an exciting time for us at Cure SMA, as we’re looking forward to announcing over $1 million in new research grants in the next three months—part of our $1.8 million commitment to research over this fiscal year. In total, we’ve invested more than $57 million in SMA research.

We’re marking these milestones with the release of an updated SMA Drug Pipeline. The pipeline is a chart that shows each of the current SMA drug programs, and it is one of the primary ways we evaluate the success of our research program.

The pipeline tracks:

  • The stages of each individual drug program, so we can see their progress.
  • The breadth of programs, so we can ensure we are attacking SMA from all sides and pursuing diverse therapeutic approaches.
  • The pharmaceutical and academic partners involved in SMA drug discovery, to be sure that we are building a broad community of support.

Important Results

This latest version includes a number of important results:

  • We have 17 active programs, more than ever before.
  • We have 12 pharmaceutical partners working with us, more than ever before.
  • We will have six programs in clinical trials by the end of 2014, more than ever before.
  • We have two programs in Phase 3 clinical trials, more than ever before.
  • One of those programs—Isis Pharmaceuticals—is the first-ever Phase 3 trial for a drug developed specifically to treat the underlying cause of SMA.
  • Cure SMA has provided funding for over half of the drug programs currently in the pipeline.

Future Goals

Researchers often test thousands of compounds to produce one drug for clinical trials. And of drugs that do make it clinical trials, only 10% will be successful. Drug development is a huge undertaking, which makes it all the more important for us to carefully track our progress.

In addition to sustaining the progress we’ve already seen, we are working to build our cumulative pipeline from 24 programs to over 30 total programs. The cumulative pipeline is the total number of active programs (currently 17) plus the number of failures (7).

A lot of hard work has already been done, and we have great hope for the near future. But more work remains to achieve our first approved treatment for SMA, and then to extend that treatment to every age and type of SMA.

With the support of our community, we know that together, we can cure SMA.

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