A Necessary MatchA cure comes closer when clinical and basic scientists share “It’s important that our basic scientists see how clinical trials are designed, how they’re arranged, what the caveats are,” Packard Director Jeff Rothstein said, opening the annual symposium’s clinical trials sessions. “Here’s an example: In a meeting, one of our investigators asked why we don’t do clinical trials with people who carry a known familial ALS gene but who as-yet have no sign of the disease. Any clinician who runs trials, though, knows what a daunting task it is to try to study a drug in a patient population where you can’t tell when the disease is going to start. There’ve been attempts to do that in other diseases, like Huntington’s,” Rothstein explains. “But you don’t know how to power a trial properly to show that a drug is really preventing or slowing onset. “Our basic scientists, however, see that a way around this problem is to find a good early biomarker for ALS, one that shows the disease’s presence before symptoms start. That’s why we’re encouraging biomarker work. “A synergy between basic and clinical science benefits research,” Rothstein says, “and brings therapy closer.” | | |
