Those clever scientists at Roche's Genentech have found a way to slip an engineered antibody through the intricate blood-brain barrier that makes it so difficult to treat neurodegenerative diseases like Alzheimer's. In this case they relied on a receptor used by iron molecules which pass freely into the brain to allow the antibody to penetrate the complex defensive perimeter. And their breakthrough discovery opens the door to a new generation of therapies that target a host of conditions.
For Genentech's Ryan Watts, getting an antibody-based treatment in the brain like this resolves the crippling need to introduce extremely high doses of drugs in order to get enough of the therapy through the barrier and into the brain. "We needed a solution," Watts tells Bloomberg. And now they have it.
"This really opens a whole new frontier for antibody therapies," Genentech senior antibody scientist Mark Dennis tells Reuters. "Before, the brain was considered off limits."
Genentech's approach ties BACE-1, which inhibits an enzyme involved in the production of beta amyloid, to the receptor. Beta amyloid is a protein widely viewed as a prime suspect in the development of Alzheimer's. There's still quite a lively discussion in scientific circles, though, over exactly what role amyloid plays in the disease. Is it the cause or an effect? And what else happens to the body when you alter levels of amyloid in the brain?
As with any advance in science, solving one problem gives you the chance to face many more before a new drug can be approved. But as Sally Church notes on her blog Pharma Strategy, it also opens the way to other therapies for Parkinson's and gliobastoma, a deadly brain cancer. What if you used the same approach, she suggests, to smuggle Avastin into the brain?