June 9, 2014
ANCHOR LEAD: A BETTER WAY TO ASSESS ALS DRUGS MAY HAVE ARRIVED, ELIZABETH TRACEY REPORTS
ALS, amyelotrophic lateral sclerosis or Lou Gerhig’s disease, kills everyone it affects, in spite of thousands of dollars and many, many man hours confronting the problem. Now a cell system developed by Jeffrey Rothstein, an ALS expert at Johns Hopkins and colleagues, may change that.
ROTHSTEIN: Many drugs fail. About 80% of them fail. One of the reasons we fail is because we go to the patient, we give them the drug but we actually never really know if it works. Does it get to its target, does it change the biology of the brain? We don’t have what is known as pharmacodynamics markers. Oh, but wait a minute. We have real brain cells in the dish, human cells, that carry a mutation, and actually using those cells we’ve defined what that marker is. And we’re now testing that in patients, so by the time we go to patients we’ll not just have the drug, we’ll have a way of reading out if the drug works. :32
Rothstein says the system is already in use to assess drug candidates. At Johns Hopkins, I’m Elizabeth Tracey.