New ALS Drug

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Anchor lead: A drug combination slows progression of ALS, Elizabeth Tracey reports

A two drug combination tested in people with ALS, also called Lou Gehrig’s disease, has slowed their rate of decline compared with those who did not receive the drug, a study in the New England Journal of Medicine reports. Jeffrey Rothstein, an ALS expert at Johns Hopkins, comments.

Rothstein: The trial was successful. It’s based on the use of a reportable scale that rates how people are doing. The FDA often likes not just a biochemical marker they often want how do people function, and this is a functional rating scale. Previously in ALS especially in the 90s trials were based on survival with ALS being a fatal disease, but those are long trials, and in the end they don’t tell the patient have you done better or not? Have you slowed your disease down? So this trial used the ALSFRS and surprisingly, and excitingly found that it could slow down the disease. That’s very good.  :32

Rothstein says he’s heartened by the findings and is still on the hunt for even more efficacious drugs and possibly a cure. At Johns Hopkins, I’m Elizabeth Tracey.