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FDA approval must not lead to complacency

The FDA approved aducanumab a new Alzheimer's disease

The FDA approved aducanumab a new Alzheimer's disease treatment, on an accelerated pathway, Credit: Getty Images/Sean Gladwell

The approval process from the FDA for a new Alzheimer’s disease treatment has been highly controversial. One of the two pivotal studies of this medication failed to demonstrate clinical efficacy, and in November an FDA advisory committee voted — 10 members against and one uncertain — that there was insufficient evidence of clinical benefit on delaying progression of the disease.

The FDA approved aducanumab, or Aduhelm, on an accelerated pathway, which means they relied on results from an imaging test that showed a reduction of amyloid plaque in the brain, which they felt was likely to predict clinical benefit. Under the accelerated approval provisions, which allow patients earlier access to the treatment, the FDA is requiring a new randomized, controlled clinical trial to verify the drug’s clinical benefit. If the trial fails to verify clinical benefit, the FDA may withdraw approval of the drug.

Controversy aside, there is an enormous unmet need to develop better treatments for a debilitating disease that affects more than 6 million people in the United States. With the approval of aducanumab, it is not yet time to take a victory lap. We need to keep our foot on the gas pedal as we race for a true cure. We need more studies, more clinical trials, more treatments, and more options for the families and patients suffering from this disease.

Amyloid is by no means the only potential therapeutic target for Alzheimer’s disease. Other approaches need to be tested, including treatments that focus on the abnormal accumulation of tau protein tangles in the brains of patients with Alzheimer’s, inflammation in the brain, abnormal energy metabolism, and loss of connectivity between nerve cells. Aside from potential disease-modifying drugs, there is still a need to study treatments that may help to alleviate the symptoms of the illness, such as behavioral problems in patients with advanced Alzheimer’s.

Along with my colleagues at the Feinstein Institutes for Medical Research, Northwell Health’s science arm, we have been looking into repurposing drugs already approved for other conditions to see if they may prove beneficial for patients with Alzheimer’s. We also participate in large, multicenter studies to test drugs with novel mechanisms of action.

We need to support and encourage medical research. As the response to COVID-19 has shown, with proper government and private support, adequate funding and collaborative efforts across the entire global science community, medical research can progress quickly and safely. We need to do the same for Alzheimer’s disease. I hope that the approval of aducanumab will not discourage patients and family members from enrolling in new studies. There is a still lot of work to be done.

Dr. Marc L. Gordon is a professor at the Feinstein Institutes for Medical Research.

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