Administrator, Centers for Medicare and Medicaid Services (CMS)
Merit Cudkowicz, MD, directs the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, in Boston.
There, her team is testing three new therapies for amyotrophic lateral sclerosis, a fatal disease that destroys the nerve cells controlling movement. The type of clinical study they’re using, known as a platform trial, allows them to test several drugs in different people at the same time.
In a few months they say they'll test a fourth therapy. Taking the approach can halve the time to find an effective treatment, Cudkowicz has said.
“This is an incredibly hopeful and exciting time with more targeted treatments, more knowledge of ALS biology, and more innovation on trial design and conduct,” Cudkowicz says. “The speed has finally accelerated.”
The lab continued operating its trials during the coronavirus pandemic by setting up virtual consent, visits over video, and assessments in people’s homes. The center has more than 10 other trials ongoing in different forms of ALS.
Awareness around ALS grew significantly six years ago thanks to the viral Ice Bucket Challenge, which raised $220 million. Cudkowicz, who also oversees the neurology department at Mass General, says it helped bring new people into the field and greatly increased funding.
“We need much more, though, to really make a huge impact,” she says.