We cannot accept the fact that researchers cite a 99 percent failure rate in drug development for treating Alzheimer’s dementia over 10 years.
CELEBRITIES Seth Rogen and Maria Shriver are raising awareness of Alzheimer’s dementia but clinical medicine is still far from producing a cure. New therapy reports are tantalizing, but to date they have little impact, while nutritional supplements, infant formulas and even dog-food makers are allowed to claim they help the brain, memory and cognition. To reconcile this, and make concrete progress, the time seems ripe to appoint an Alzheimer’s dementia therapy czar.
Washington state has 100,000 people living with Alzheimer’s dementia. There are only seven open Alzheimer’s dementia therapy studies listed here on the website clinicaltrials.gov. By contrast, there are more than 500 adult cancer trials for 300,000 patients, and more than 40 trials for our 12,000 HIV patients. Unlike national cancer and AIDS research infrastructures, a comparable Alzheimer’s therapeutic research structure seems lacking.
Alzheimer’s dementia researchers recently cited a 99 percent failure rate in drug development over 10 years. The scientific literature is full of interesting ideas, part of the creative chaos of academic inquiry, but it is also limited by academic and commercial incentives.
The early days of cancer and AIDS therapy were also frustrating, when things never worked well enough. On the face of things, isn’t Alzheimer’s a progressive disease that is eventually fatal, in that way cruelly similar to cancer and HIV infection?
HIV infection has gone from almost universal death to an average life expectancy measured in decades. Another once-dire disease, childhood leukemia, has gone from a survival rate of 4 percent to greater than 90 percent.
The first AIDS czar was appointed more than 20 years ago. AIDS therapy seemed to languish with single drugs until there was a concerted effort to change the clinical work, probe multiple and unconventional agents, and quicken the pace of trials. Coordinating the work of grass-root activists, academia, drug companies and the FDA is credited with the eventual development of highly active therapy.
Early investigators in childhood cancer proceeded without precise hypotheses of molecular or genetic mechanisms. Cancer therapy developed through tedious randomized trials with “shotgun” medications, learning from failures.
A case report did suggest a person with myeloma and Alzheimer’s dementia had improved brain function during his cancer treatment. Another cancer agent, nilotinib, is showing promise in Parkinson’s dementia. Are there other serendipitous observations that need follow-up?
Current Alzheimer’s dementia research strategy, reflected in the National Alzheimer Project Act 2015 Update, seems to concentrate the science and funding in descriptive research with genetics, imaging, pathobiology and services, rather than a concrete overall therapeutic plan.
There is an implied strategy for eventual therapy, rational but prolonged, a multi-step process of finding the right molecular target, then developing or designing precisely the right drug. Yet the stated national goal is effective therapy by 2025, only 10 years from now.
How could a therapy czar help the effort? He or she could create a strategy to quickly but definitively test “alternative remedies,” nutritional supplements and dosage combinations, create research templates, define outcome standards, further simplify consent issues, accelerate recruitment in nursing homes, prioritize and activate trials of existing drugs that seem promising but have no sponsors (“repurposing drugs”).
Brilliant minds in the Alzheimer’s dementia research community have been attacking the problem for years. We desperately want the miracle breakthrough, but we need to hedge our bets for 2025. Can we create a coordinated and comprehensive research structure to accelerate clinical improvements in treating Alzheimer’s dementia and be more aggressive, even radical in trials? It seems time to empower an Alzheimer’s dementia therapy czar.