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Solving the Alzheimer's Disease Therapy Drought

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How a litany of treatment options could be just a single breakthrough drug away.

Gregory Day, MD, MSc

Gregory Day, MD, MSc

In just a short decade of Gregory Day’s young career, the US multiple sclerosis (MS) therapy market has exploded from just a handful options to 17 US Food and Drug Administration (FDA)-approved agents for multiple mechanisms.

Day, MD, MSc, assistant professor, Washington University School of Medicine, Knight Alzheimer Disease Research Center, St. Louis, has reflected often on this fact — how a complex condition went from having only a few treatments to a cabinet’s worth of drugs in only 10 years. It especially lingers on him because it’s a trajectory he hopes for Alzheimer’s disease (AD) therapies.

In an interview with MD Magazine at the 70th annual meeting of the American Academy of Neurology (AAN) in Los Angeles, CA, this week, Day called AD a challenging disease, which somehow may be an understatement. At the end of 2017, the National Institutes of Health (NIH) reported an estimate that approximately 6 million adults in the US have either AD or mild cognitive impairment — a telling early symptom of the disease. Despite this, it has been 15 years since a drug was approved for AD.

The reasons why AD research fails to reach marketable progress is both varied and self-perpetuating: its aggressive pathology is complimented by limited diagnoses, reducing the pool of early-diagnosed patients for clinical trials. In diagnosing these issues, Day mapped out a way for researchers to bring about a clear resolution.

He explained that AD is a condition that presents insidiously in patients. Unlike the relapsing or remitting forms of MS, Alzheimer’s is relentlessly progressive. Indeed, a majority of Americans who have symptomatic AD either don’t receive a diagnosis in the clinic, or receive a diagnosis of dementia that doesn’t conclude on what has driven the symptom. When the diagnosis eventually comes, physicians can only tell patients about its progression and their eventual death.

“And without people knowing what the diagnosis is, I don’t think we’re going to have the opportunity to assign them appropriately to clinical research,” Day said. “We’re not going to have opportunities to learn about their care or their progression, or evaluate therapies in that population.”

This calls for emphasis on early diagnoses — identifying patients prior to any symptoms presenting themselves. Physicians need to begin realizing the current form of AD they recognize — the symptomatic phase — is actually the end-phase of a process possibly 1-2 decades in the making, Day said.

He pointed to trials such as the Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) Study that have focused on therapies for cognitively healthy people who have biomarker evidence of the disease present in their brain. Similar to statins for cardiovascular disease, these therapies could possibly serve as much benefit prior to disease progression as they would post-diagnosis.

While agencies such as the NIH have made financial contributions to AD research in recent years, Day does not fault drug companies for showing reticence in investing in the field.

“You don’t have to look too far to see the graveyard of past studies that have not panned out, and the billions and billions of dollars that have been spent pursuing therapies that have not been effective,” Day said.

What’s needed to change their minds is the same thing that changed the field of MS just a decade ago: a single effective drug. Day projected that a model of continued, sustained research investments, coupled with a push to advance promising investigatory therapies to later-stage clinical trials, could provide real results.

Lastly, Day called for more advocacy: patients with AD speaking up for what they want, and helping drive the push for more government-based funding to research.

Unfortunately, in a country of 6 million-plus patients and an aging population of legislators, AD is not in much need of an awareness campaign.

“This disease is on everyone’s mind, and I don’t care if their rationale is self-serving, that they want to prevent it in themselves or their colleagues,” Day said. “I think it’s going to benefit the constituents tremendously and benefit my patients, and that’s what got me excited for the current state of funding for this disease.”

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