A pediatric oncologist looks for progress in Alzheimer’s disease


A baby acutely ill with leukemia seems like the polar opposite of a woman with Alzheimer’s disease (AD), with her slow, insidious deterioration. Yet each disease can be progressive and fatal. I’ve cared for both, in different ways.  Can a childhood cancer doctor like me have insights about that other end of medicine, older adults with dementia?

As a husband and caregiver, confronting my wife’s Alzheimer’s disease, I am appalled by the lack of effective therapy for her. As a clinical investigator myself, I’m appalled by what I see as a lack of direction in clinical dementia research, a lack of structure, and a lack of ambitious leadership.

Every Minute Counts, the PBS TV documentary that aired recently, showed the heart-rending personal devastation of Alzheimer’s Disease (AD) and dementia, and the enormous cost of care for those affected.  It ended with a plea for more funding and research. But after decades of research and billions already spent, why aren’t we further?

Alzheimer’s is now the 6th leading cause of death in the U.S.; it was only 13th two decades ago. Many AD experts acknowledge the lack of progress. Recently, two researchers even wrote that seeking an AD cure is a notion “many believe unrealistic,” and that advocating for curative therapy “verges on the promotion of false hope.”

Since my wife was diagnosed, just one new drug, really a combination of two older drugs, has been approved for Alzheimer’s.  As a cancer doctor, I’ve watched more than 70 cancer treatments get approved during those same five years.

Even the Alzheimer’s Association plainly states “there is no cure for Alzheimer’s.”  Not so plainly stated is that we don’t know what causes it, how it happens, or how to prevent it.

Of course, we don’t really know what causes childhood leukemia either, how it happens, or how to prevent it, even with our current sophisticated molecular descriptions and theories.  But the survival rate for the most common childhood leukemia has gone from less than 10% in the 1960’s to over 90% now, with incremental progress every five years.  Most kids are cured with combination chemotherapy that was developed decades ago, before molecular testing.

Most AD funding goes for molecular or non-therapeutic research, at the expense of clinical work. Sickle cell anemia was called the first “molecular disease” over sixty years ago; we still don’t have a cure, even though just this year we might have a gene therapy for it. In AD, the molecular genetics seems more complicated. AD patients have waited years for ANY therapy, much less one from “precision medicine.” Is this the right strategy?

It’s not all bad news. Recently, three commercial drug trials announced results, and they showed glimmers of hope.  Two were reported as failures by the media, but company press releases (TauRx,Lilly) reported modest positive effects. The third drug, from Biogen, seemed better at slowing the decline in some patients, but evidently did not stop the disease.

AIDS therapy, like cancer, is an area of medicine that seemed hopeless at first.  The leadership of Dr. William Paul, an “AIDS Czar,” is credited with accelerating clinical progress in that condition.  Still incurable, nevertheless AIDS patients’ lives are now extended from months to years.

Nothing has really changed for Alzheimer patients over the past five years. Brilliant scientists are working, but in the usual atmosphere of creative academic chaos. There are a few AD clinical trial groups, much like cancer trials groups, but the comparison of their activity is stark. In the state of Washington, there are over 600 cancer studies recruiting patients; in AD there are about a dozen such studies.

Much foundational AD work still needs to be done at the bedside, in overall strategy, trial coordination, informed consents, vigorous subject recruitment, and consensus development, so appointing an accountable, identifiable, directive clinical research leader seems like an important way to accelerate progress (although “czar” or “emperor” may not be the right titles these days).

Ron Louie is a pediatric oncologist.  This article originally appeared in the Baltimore Sun.

Image credit: Shutterstock.com


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