“This is major league news.”
That is how Stephen Salloway, MD, MS, director of neurology and the Memory and Aging Program at Butler Hospital, termed Wednesday the announcement that Biogen Inc., a biotechnology company based in Cambridge, Massachusetts, will pursue regulatory approval from the FDA for the investigational drug aducanumab used for treatment of the early stages of Alzheimer’s.
If approved, it would become the first drug to remove amyloid protein from the brain and slow the progression of Alzheimer’s.
The Alzheimer’s Association as well as its Rhode Island chapter hailed the development.
“No stone can be left unturned. We must advance all potential treatment avenues and also explore methods for combining these approaches. Alzheimer’s and other dementias are complex, and their effective treatment and prevention will likely also be a complex but achievable task. All currently pursued treatments that are considered safe should be continued to determine their efficacy,” the association says in a release.
Salloway is less restrained in his enthusiasm.
He worked with the 60-member Rhode Island cohort involved in the worldwide clinical trial of aducanumab and has seen first-hand the drug’s impact.
“This is a game changer,” Salloway said.
That wasn’t the feeling in March, when Biogen abruptly halted its studies of aducanumab after an analysis of the initial data showed it was unlikely to be successful in meeting the benchmarks necessary to be considered a viable therapy.
Salloway said many of those involved in the clinical trial, some for as many as five years, were distraught, as they believed the drug was beneficial. The drug is administered intravenously once a month for a period of 45 minutes to an hour.
Worldwide, about 3,200 patients were involved in the study that Biogen shut down in March.
“But as additional data came in, particularly from trial participants who had been administered a higher dose of the drug, it became apparent that aducanumab yielded clinically significant results for the successful treatment of early Alzheimer’s disease,” Salloway said in a release issued by Care New England. Butler is a Care New England hospital.
Salloway said patients in the study stopped in March have already inquired about the new re-dosing study. He said there would be not cost to them, but if and when the drug gains FDA approval he expects it “will not be a cheap medicine.” He didn’t specifically estimate what that cost might be.
Salloway said his study of those in the clinical trial found a side effect of “leakiness,” which he described as a film of proteins or plaque visible in MRIs of the brain. “Any medicine can have side effects,” he said. He said the “leakiness” is manageable.
“This is a very important moment for the Alzheimer’s field – for the first time in a long time, new treatment data being submitted to the FDA for review. This could be a game-changer. We are very hopeful,” Donna McGowan, executive director of the Alzheimer’s Association Rhode Island Chapter, said in a statement.
“The Alzheimer’s Association is an organization that puts the person with dementia and family members first. At the same time, we are driven by scientific evidence. We eagerly anticipate reviewing a full report of the findings from these studies – both locally and nationally – and participating in a robust discussion of data,” she added.
Salloway praised those involved in the clinical trial, calling them “very courageous people who want to make a difference.”
According to a release from Biogen, patients who received aducanumab experienced significant benefits on measures of cognition and function such as memory, orientation and language. Patients also experienced benefits in terms of 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.
If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer’s disease and would also be the first therapy to demonstrate that removing amyloid beta resulted in better clinical outcomes.
“With such a devastating disease that affects tens of millions worldwide, today’s announcement is truly heartening in the fight against Alzheimer’s. This is the result of groundbreaking research and is a testament to Biogen’s steadfast determination to follow the science and do the right thing for patients,” Michel Vounatsos, chief executive officer at Biogen said in a statement released Tuesday.
“We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid beta,” he said.