New Alzheimer’s drug therapy shows promise in treatment breakthrough

An antibody drug therapy that is targeted against Alzheimer’s disease recently showed promising results in its initial trial, which could potentially lead to a breakthrough in the treatment of the brain disorder that has devastates over 5 million Americans.

The antibody, aducanumab, is still in the early stages of testing. It targets a protein called amyloid beta that builds up in the brain and creates plaques associated with Alzheimer’s disease.

In a paper published in the journal Nature, a trial to evaluate the drug’s safety and tolerability showed positive results. Among 165 patients who were studied, those who received monthly infusions of the drug for a year showed a significant reduction in amyloid beta and slower cognitive decline than those receiving a placebo.

If aducanuman proves successful and effective in larger trials being currently tested, it could be the first ever drug to reverse the signs of the disease and the first new Alzheimer’s drug to be approved in over a decade.

“Overall this is the best news that we’ve had in my 25 years doing Alzheimer’s clinical research and it brings new hope for patients and families most affected by the disease,” according to Stephen Salloway, director of neurology in the Memory and Aging program and professor of neurology and psychiatry at Brown University, one of the study’s authors.

Participants of the study were divided into four groups, receiving either a placebo, a low dose of aducanumab, a medium dose or a high dose.

Another study author, Roger Nitsch, director at the Institute for Regenerative Medicine at the University of Zurich, said that the higher the dose and the longer the treatment were, the larger the degree of reduction was.

In PET brain scans taken of people receiving the highest dose, “after one year you can see no red on the image, meaning the amyloid has almost completely disappeared,” Nitsch said. Compared to past studies, he added, “the effect size of this drug is unprecedented.”

There was some adverse reactions seen among the patients carrying the APOE4 gene associated with Alzheimer’s who were given higher doses. These included slight brain swelling and bleeding seen in MRI brain scans.

But the authors said that for most recipients, the side effect was manageable by careful monitoring and adjusting of the drug.

Currently, there are five FDA-approved drugs that can alleviate some symptoms. But in the past 12 years, no new drugs have been approved for the disease, which at US$236 billion a year is the most expensive disease in the US.

In recent years, several promising therapies have failed to show positive results in later trials. But the technology that can identify amyloid plaques in the brains of living patients – which was used in the new study – has been a game-changer for researchers.

“This trial has reinvigorated the field and clearly created a lot more excitement about amyloid as a target,” James Hendrix, director of global science initiatives at the Alzheimer’s Association said about the aducanumab study.

Successful Phase 3 trials of the drug would also add strong support for the hypothesis that preventing or clearing amyloid buildup can help patients, he said.

And while experts believe the key to stopping Alzheimer’s will ultimately require a combination of approaches, “right now we need one good one, a new one.

“If aducanumab and others in the pipeline have the promise of maybe preventing and slowing down the progression, it could let (people) live out their golden years the way they want to.”

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Category: Features, Pharmaceuticals