April 14, 2008

Genentech, Inc. and Biogen Idec, Inc. announced that a Phase II/III study of Rituxan® (rituximab) for primary-progressive multiple sclerosis (PPMS) did not meet its primary endpoint as measured by the time to confirmed disease progression during the 96-week treatment period. Genentech and Biogen Idec will continue to analyze the study results and will submit the data for presentation at an upcoming medical meeting.

While this is very disappointing news, I'd like to point out that both companies deserve credit for taking on a study for PPMS.

Placebo controlled trials are used to test new drugs by comparing the results of those taking the active drug with those taking the inactive placebo. This allows us to know that the beneficial effects, if any, seen in the trial were due to the drug and not to some other aspect of the care surrounding the administration of the drug.

In a disease like MS, where there are several partially effective drugs, it becomes a concern to put someone on a placebo when they could be taking an approved medication. A group of clinicians, ethicists, statisticians, regulators, and representatives from the pharmaceutical industry convened to discuss this topic and published their thoughts.

They concluded that for patients with relapsing MS for which established effective therapies exist, placebo-controlled trials should only be offered with rigorous informed consent if the subjects refuse to use these treatments, have not responded to them, or if these treatments are not available to them for other reasons (e.g., economics).

Scientists in the Laboratoire de Neurobiologie des Processus Adaptatifs (CNRS/Université Pierre et Marie Curie) have shown that it is possible to repair an injured brain by creating a small number of new, specifically-targeted innervations, rather than a larger number of non-specific connections. Behavioral tests have demonstrated that such reinnervation can thus restore damaged cerebral functions.