Great Strides 2006
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Medical News Briefs

CFFT Looks for a Winning Combination

Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) recently awarded CombinatoRx, Inc. up to $13.8 million to discover novel drug combinations for the treatment of CF from existing drugs already approved for the symptoms of other diseases. This strategy opens up a new avenue for stocking the CF Foundation’s drug discovery and development pipeline.

To discover therapeutic drug combinations that hit multiple targets relevant to CF, CombinatoRx uses high-throughput screening, a technique that allows scientists to screen hundreds of compounds simultaneously. Scientists hope to find a combination of approved drugs that improve the function of CFTR in CF cells. If successful, the path from laboratory to clinic should be smoother and easier, as many safety questions for the individual compounds will have already been addressed in prior studies for other diseases.

New Enzyme Products in Clinical Trials

Clinical trials evaluating different forms of pancreatic enzymes are starting across the country. In response to a CF Foundation request, the FDA has mandated that pancreatic enzymes undergo more rigorous evaluation in clinical trials. The Foundation believes that this additional clinical testing and evaluation will ultimately result in the improvement of existing pancreatic enzymes and will therefore benefit patients.

A new form of enzyme product, ALTU-135, developed by Altus Pharmaceuticals, using enzyme produced from sources other than the internal organs of animals, is currently being prepared for Phase 3 testing. It is hoped that, following this research, patients will require fewer capsules and that a liquid formula will be available for younger children.