medicine
The first drug for a one-in-a-million disease just arrived
A mistake in a mouse lab in the 1990s. Thirty years later, a cure.
The FDA has approved the first drug designed to directly treat Alexander disease, a neurological disorder so rare it affects fewer than one in a million people worldwide. The treatment, called Zanvastro, is the first that targets the underlying disease rather than easing its symptoms. Until now, doctors could only manage the seizures, the loss of movement, and the difficulty swallowing as the condition slowly advanced. The science began three decades ago at the University of Wisconsin-Madison, in the lab of Albee Messing. He and Michael Brenner engineered mice to overproduce a protein called GFAP and, without intending to, created animals with the exact brain lesions seen in human patients. That accident revealed the genetic cause. In an international trial of 54 patients, those given Zanvastro walked faster after a year, and some regained motor function. "This is a wonderful and long-awaited day for the Alexander disease community," Messing said. The drug reaches the US in the coming weeks.
