Earlier this week, the FDA approved aducanumab – a new drug developed to slow progression of Alzheimer’s disease in patients who show early signs of the condition. Aducanumab, which will be sold under the brand name “Aduhelm” is the first therapy for Alzheimer’s disease to have gone through the FDA’s accelerated approval pathway. However, since aducanumab’s effectiveness in treating Alzheimer’s disease showed mixed results in early clinical trials some memory care experts are divided on the benefits of the drug.
How Does Aducanumab Work?
Aducanumab or Aduhelm is an antibody treatment that targets amyloid beta proteins. The build up of these proteins in the brain is implicated as a main causal factor of Alzheimer’s disease. The drug is intended for use in patients with mild cognitive impairment to slow down the progression of Alzheimer’s disease – not to treat advanced dementia or to restore cognitive capabilities.
Aducanumab is administered via intravenous infusion once every four weeks. The FDA says patients must receive an MRI before initiating treatment, as well as MRIs before the seventh and twelfth infusions to monitor for brain abnormalities.
What Do The Experts Think?
Some patient advocate organizations, including the Alzheimer’s Association, are supportive of the FDA’s decision to approve the drug. Even though aducanumab isn’t a silver bullet for Alzheimer’s disease, the potential for benefit is worthwhile to patients and their loved ones.
“Our team is extremely familiar with this medication since we’ve been involved with this development since the very beginning,” said Dr. Marc Agronin, Chief Medical Officer for MIND Institute at Miami Jewish Health. “This is an exciting new treatment for Alzheimer’s disease and is bringing hope to so many individuals and their families.”
Why Is There Controversy?
In 2019, the late-stage testing program for aducanumab consisted of two phase 3 clinical trials. One trial met the primary endpoint, showing reduction in the decline of cognitive health. The second trial did not meet the primary endpoint.
According to Biogen, the pharmaceutical company developing aducanumab, patients from the successful trial treated with high-dose aducanumab showed 22% less clinical decline in their cognitive health at about 18 months, indicating that the progression of their early-stage Alzheimer’s disease slowed.
In 2020, the Peripheral and Central Nervous System Drugs Advisory Committee assembled by the FDA to review the clinical trial data did not recommend approval of aducanumab, stating that the data did not show “primary evidence of effectiveness” in treating the neurological disease.
Taking that information into consideration, the FDA decided to approve aducanumab through its accelerated approval program, which “allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate or intermediate clinical endpoint.” A surrogate endpoint is a marker (i.e. a laboratory measurement, radiographic image, physical sign, or other measure) that is thought to predict clinical benefit – but is not itself a measure of clinical benefit. In the case of aducanumab, the FDA cited data showing that aducanumab is effective at breaking down amyloid in the brain – indicating that it’s “reasonably likely” that aducanumab will eventually show a clear clinical benefit in slowing cognitive decline.
“In all studies in which it was evaluated,” said Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, “Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent fashion. It is expected that the reduction in amyloid plaque will result in a reduction in clinical decline.”
As part of the FDA’s accelerated approval program, Biogen will be required to fund a post-approval clinical trial of the aducanumab to verify its efficacy.
As one of Florida’s official memory disorder centers with direct experience in the development of aducanumab, MIND Institute at Miami Jewish Health can answer all of your questions about this medication and guide you to making the best decision about treatment for you or a loved one. There are ongoing clinical trials, including with aducanumab and similar medications, that are open, without cost, to eligible individuals with mild to moderate cognitive impairment. Our entire practice is devoted to empathic diagnosis, brain fitness, innovative research, and one-on-one care and support for individuals and their families. Every family and every individual is unique. Their treatment should be too. For questions about aducanumab or other treatment plans for memory care, contact us at 305.514.8710.