More than 6 million Americans are living with Alzheimer's disease. The progressive neurological illness is the sixth leading cause of death in the United States and, with few treatment options available, symptom management is the only way sufferers cope with the illness.
On June 7, 2021, the FDA approved a new drug for the treatment of Alzheimer's disease called Aduhelm. It's the first FDA-approved treatment since 2003. The new drug targets and reduces amyloid protein accumulation in the brain; when amyloid proteins build up, they form plaque that collects between neurons and disrupts cell function.
Aduhelm, which is administered by infusion, was given the green light under the FDA's Accelerated Approval Program after undergoing about four years of clinical trials. More than 3,000 early-stage Alzheimer's patients participated in the trials and the drug was shown to positively alter the brain structures thought to contribute to the disease, removing amyloid buildup.
One of the medical facilities hoping to receive doses of Aduhelm is The Roskamp Institute in Sarasota. The institute, which researches and treats Alzheimer's and other brain disorders, has been keeping tabs on Aduhelm's FDA's approval and is anticipating its distribution nationwide.
Roskamp Institute executive director Dr. Michael Mullan, who discovered the amyloid protein's important connection to Alzheimer's more than 30 years ago, offers insight on how this new treatment works.
"Aduhelm was not approved on efficacy or on the basis that it showed positive benefit, but on the fact that it removed amyloid buildup in the brain," says Mullan. Amyloid buildup, along with neurofibrillary "tangles" within the neurons of the brain, are thought to accelerate Alzheimer's disease and impair memory and cognition. "A lot of pressure was put on the FDA by the Alzheimer's Association and advocacy groups to approve this drug. We haven't had a new treatment for the disease in 20 years."
Mullan adds that not all amyloids are bad. Unfortunately, drug treatments for Alzheimer's, including Aduhelm, cannot make this distinction. They target all amyloids.
"Our brains produce amyloids before we are even born, but for some reason, as we age, the protein starts to accumulate and puts us all at risk for Alzheimer's disease, but especially those who are genetically predisposed," says Mullan.
The approval of this drug has been unique, as previous drugs thought to operate in similar ways were not FDA-approved. However, the FDA stated that the potential benefits of Aduhelm to patients' quality of life over its uncertainties and side effects were enough to push it through approval.
Patients who will receive the drug will have it administered by infusion once every four weeks, presumably for the remainder of their life. Aduhelm is currently approved for use in the United States; the immediate next step is for Medicare, medical insurance companies and Biogen to agree on reimbursement of the proposed $56,000 yearly cost. This process can take about three to six months, and it's likely the drug will be available to patients before the reimbursement situation is settled.
"We hope to be able to give it to patients in the next few months," says Mullan.
The types of patients who qualify for Aduhelm will vary. Those who have co-morbidities of diabetes, hypertension and cardiac disease may not be eligible candidates. Clinical trials were also conducted on early-stage Alzheimer's cases, so the FDA has not published a statement regarding what stage of the disease it will best treat. Mullan adds that those with hypersensitivity to infusion will require careful monitoring during sessions.
"Other risk factors to consider are side effects," says Mullan. "They could include brain swelling and small brain bleeds." Those considering treatment must also have an official Alzheimer's diagnosis by a medical professional to ensure cognitive decline is not due to another illness.
Despite tribulations in getting the drug approved, Mullan is hopeful that it will improve patient's lives, and will be the first step of many toward reducing Alzheimer's disease.
"When I look at the pathology of treating this disease, it's like a three-legged stool," says Mullan. "One leg is the amyloid protein, which lives outside of brain cells; the other is the neurofibrillary 'tangles' living in the neurons, which are associated with cognitive decline; and the third is inflammation." Through a combination of therapies targeting each aspect, Mullan believes the disease can be more effectively treated.
As for Aduhelm, Mullan knows it won't be the cure, he's hopeful it will be a good option for those suffering with Alzheimer's.
For more information on The Roskamp Institute, click here or call (941) 752-2949. The Roskamp Institute is located at 2040 Whitfield Ave., Sarasota.