Roche spotlights PhI efficacy of Alzheimer's antibody

Roche has taken the first big clinical step in a long journey it hopes will lead to a mega-blockbuster market. The pharma giant says that a small, early-stage study of its experimental Alzheimer's drug gantenerumab worked as hoped for. And now researchers will set out to determine if they're on the right path.

Testing two doses of the drug over 6 months among 16 patients, investigators reported that the treatment triggered a significant drop in amyloid plaque while the same suspect biomarker for the disease continued to build in the placebo arm. Scientists were also able to confirm its effect on amyloid plaque in an ex vivo lab test using patient tissue samples.

"These results and especially the rapidity of the effects observed on amyloid removal are very encouraging and pave the way for the development of a novel treatment for Alzheimer's disease," said Luca Santarelli, head of Roche's global neuroscience disease division, in a statement.

Roche's medicine is an antibody which has proven its ability to penetrate the tricky blood-brain barrier, nature's defense against any intruders trying to get into the brain. It was identified by investigators from Roche in collaboration with Germany's MorphoSys.

So far, so good. But the researchers will now have to try and determine if the desired effect translates into a clinical benefit for the patients. While scientists have known for years that the plaque clogs the brain in advanced cases, there's no hard evidence to prove that getting rid of it will help people retain their memories.

Roche also has some high clinical hurdles ahead. The pharma giant wants to develop ganetnerumab for early-stage patients, hoping that an effective treatment will long delay a terrible illness that affects some 25 million people. That huge target has helped inspire a slate of experimental drug programs, but for now Alzheimer's provides countless mysteries.

- here's the Roche release
- read the report from Reuters