Rhode Island researchers and study participants played large role in drug’s development
The U.S. Food and Drug Administration (FDA) has approved aducanumab for the treatment of Alzheimer’s disease (AD). It is the first drug approved by the FDA for the treatment of mild cognitive impairment and the first available to help slow the progression of Alzheimer’s, which afflicts some 5 million Americans and 50 million people around the world. The drug was developed by Massachusetts-based pharmaceutical company Biogen and Tokyo, Japan-based Eisai Co., Ltd.
The FDA approved the drug under its accelerated approval program, which is used for emerging treatments aimed at serious diseases for which there are currently few or no treatments. As part of its approval, the FDA said that Biogen must conduct a Phase 4, post-approval clinical trial to verify the drug’s benefit. That Phase 4 trial is expected to last several years, during which time the drug will be available to patients. If the Phase 4 trial data does not show the drug to be effective, the FDA can (but is not required to) revoke its approval and remove it from the market.
The drug, provided via a monthly infusion, will be made available to patients first at sites across the nation where the clinical trials took place. In Rhode Island this includes the Memory and Aging Program at Butler Hospital and the Alzheimer’s Disease and Memory Disorders Center at Rhode Island Hospital. Butler Hospital officials expect eligibility criteria and other information for those interested in seeking treatment with aducanumab to be available on their website at butler.org/memory in the coming days.
Rhode Island contributed one of the largest number of participants enrolled in the clinical studies that led to the approval, through study sites at the Memory and Aging Program at Butler Hospital and the Alzheimer’s Disease and Memory Disorders Center at Rhode Island Hospital, both of which are affiliates of the Warren Alpert Medical School of Brown University.
Stephen Salloway, MD, MS, director of Neurology and the Memory and Aging Program at Butler Hospital, the Martin M. Zucker professor of Psychiatry and Human Behavior and professor of Neurology at the Warren Alpert Medical School of Brown University, and Associate Director of Brown’s new Center for Alzheimer’s Research, served as co-chair of the global investigator steering committee for the aducanumab Phase 3 studies.
"This is truly a milestone moment in Alzheimer’s research. Approval of aducanumab is a major breakthrough that opens a new era of treatment for Alzheimer’s disease," Dr. Salloway said. “So many people have worked so hard and for so long to make this day happen. But as momentous as this achievement is, it is only the beginning. The approval of aducanumab provides a treatment foothold to build on as we continue to work toward developing new and even more powerful treatments,” Dr. Salloway said.
The clinical trials that led to aducanumab’s FDA approval included two Phase 3 trials, EMERGE and ENGAGE, in patients with early-stage Alzheimer’s disease (enrolled patients had mild cognitive impairment (MCI) due to Alzheimer’s disease and mild Alzheimer’s disease dementia with Mini-Mental State Examination (MMSE) scores of 24-30). In EMERGE, patients who received aducanumab experienced significant slowing of decline on measures of cognition and function such as memory, orientation and language. Patients also experienced slowing of decline on activities of daily living including conducting personal finances, performing household chores, such as cleaning, shopping and doing laundry, and independently traveling out of the home.
The aducanumab clinical program also included the Phase 1b PRIME study and its long-term extension (LTE) in patients with early Alzheimer’s disease. The results of this study indicated that aducanumab reduced the amyloid beta plaque in the brain that is associated with the development of AD. The drug was shown to reduce the presence of plaques in the brain as well as a reduction in the progression of symptoms a in a dose- and time-dependent fashion.
“Approval of aducanumab is the first step. The task now is to determine how best to administer the drug to the large number of people who can benefit from it, and we’ll be working hard to do that as quickly as possible here at Butler Hospital for patients in our region,” Dr. Salloway said. "This drug represents the opportunity for millions of people to reclaim precious time to spend with the people they love, and on the pursuits and passions that define who they are. I'm so grateful to the participants of this and all other clinical trials. Their participation is allowing critical Alzheimer's research to move forward. This isn't just a victory for them, it's a victory because of them.”
“I’m also so grateful for the incredible partnerships we have in the AD research community, especially the many strong Alzheimer’s research organizations and partnerships we have right here in Rhode Island, including the new Center for Alzheimer’s Research established at Brown University and the Rhode Island Alzheimer’s Association” Dr. Salloway continued. “I feel optimistic that together, additional significant advances will be made in fighting this disease in the near future."
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