When Phil Gutis was diagnosed with early-stage Alzheimer’s disease at 54, he immediately enrolled in a clinical trial for an experimental drug but had little hope of being helped. Over time, though, he started feeling better, his brain less cloudy.
“There was just a fogginess I remember having a couple of years ago that I don’t really feel I have now,” said Gutis, who has received monthly infusions of a medication called aducanumab for five years, except for a short interruption.
Now, he is hoping others with the disease will have a chance to try the drug. But he is worried that the Food and Drug Administration, which is weighing whether to approve the drug, will reject it, derailing the medication and jeopardizing his ability to get the treatment.
“Would my world become fuzzy again?” said Gutis, who lives in New Hope, Pa., with his husband and is a former reporter. “I don’t want to go backward.”
By June 7, the FDA was expected to make one of its most important decisions in years: whether to approve the drug for mild cognitive impairment or early-stage dementia caused by Alzheimer’s.
It would be the first treatment ever sold to slow the deterioration in brain function caused by the disease, not just to ease symptoms. And it would be the first new Alzheimer’s treatment since 2003.
The medication is a monoclonal antibody, a protein made in the laboratory that can bind to substances — in this case, clumps of amyloid beta, a sticky plaque compound that many scientists believe damages communication between brain cells and eventually kills them. The treatment is designed to trigger an immune response that removes the plaques.
Approval is far from assured. While everyone agrees there is an enormous need for new treatments — about 6.2 million Americans have Alzheimer’s, a number expected to more than double by 2050 — the drug’s messy, complicated history is sparking a battle among researchers, doctors, patients and advocates about whether the medication works and what regulators should do.
Supporters, including some doctors who treat Alzheimer’s and advocacy groups such as the Alzheimer’s Association and UsAgainstAlzheimer’s, acknowledge the drug’s clinical trial data is far from perfect. But they say it would help some patients, and argue that approval would launch an era of increased research and investment into therapies for a progressive, terminal illness that causes incalculable misery and costs society billions of dollars a year.
“This will give us a new biological foothold to build on,” said Stephen Salloway, director of neurology and the Memory and Aging Program at Butler Hospital in Providence, R.I., and a professor at Brown University. “To get the best in class, you have to have the first in class.”
But critics argue the drug’s trial data falls far short of proving the drug works. They note that one of the late-stage trials was positive and the other was negative — hardly convincing evidence. In an acrimonious meeting last fall, an FDA advisory committee recommended against approval and harshly rebuked FDA staff for what it called an overly positive view.
“The worst thing for people with Alzheimer’s would be to put out a product that doesn’t work,” Aaron S. Kesselheim, a professor of medicine at Harvard Medical School and a member of the panel, said in a recent interview. “It will be sold at an extremely high price and waste resources that could go to other things.”
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