Batten disease parents aim at screening

Batten disease parents aim at screening

San Francisco Business Times | May 20, 2011 | By Ron Leuty

Trying to find patients for clinical trials of rare disease treatments can be tough. But following StemCells Inc.’s abrupt shelving of an early-stage trial in Batten disease, patient advocates and parents of kids afflicted with deadly malady are charged up to ensure it isn’t so difficult anymore.

It’s a backstory that runs from Palo Alto to Jacksonville, Fla. — catching Vacaville, San Diego, Columbus, Ohio, Austin, Texas, Kansas City, Mo., Houston and Santa Fe, N.M., in between — and it says a lot about the power of individuals to make a difference.

The story didn’t begin with Palo Alto-based StemCells’ (NASDAQ: STEM) difficulty finding the right Batten disease patients for its trial, as I report in this week’s print edition. For years, patient advocates have been trying to get better screening of Batten disease carriers, so it reduces the risk of more children being born with the brain-wasting disease. Parents now can spend months, if not years, trying to get a correct diagnosis, allowing the disease to kill more neurons and making it less likely that companies will be able to generate the data they need to get therapies approved.

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