Not enough Alzheimer’s patients?!

by Jeongyeon Shim, Program Analyst, FasterCures

As part of the FasterCures’ Philanthropy Advisory Service program, we invited four eminent scientists in Alzheimer’s disease research to learn more about their perspectives on key opportunities and challenges that the field is facing. We discussed many of the exciting development occurring in the field. In addition to progress in basic research, many potential treatments are showing promise in animal studies and various stages of clinical trials. Many of the efforts targeting what scientists believe to be the causes of the disease are moving along the pipeline, and novel mechanisms of interventions are also discovered.

The meeting also addressed challenges in AD research. Among others, low enrollment in clinical trials was highlighted as a key challenge in transforming exciting scientific discoveries into treatments that benefit the patients. Alzheimer’s is not exactly an orphan disease—it impacts 4.5 million patients in the US today, with a drastic increase of disease burden expected. How is it possible that there are not enough clinical trial participants?

Many clinical trials in Alzheimer’s target the patients in early stage of the disease, since that is when the debilitating effects of dementia, caused by the death of neurons, can be averted. However, a diagnosis is less straightforward at early stage, given that clear biomarkers of the disease are yet to be established. Even in cases where diagnosis is made early, additional challenges to clinical trial participation exist. For example, under the current system of care where primary care physicians are in charge of most Alzheimer’s patients, it is difficult to have patients enroll in clinical trials, which are conducted in more specialized healthcare centers. Trial participation may also increase the burden to the caregiver due to the need for additional visits, especially when the trial site is at a distant location.

While scientists and trial administrators focus on finding solutions to this challenge by developing biomarkers for early diagnosis, reaching out to primary care physicians, educating participants on the importance of trials and developing technological solution to reduce the need for physical visits, the most important lever would be that patients and caregivers learn more about trial options, and when possible, enroll in trials. Participating in trials requires substantial effort from the patients and caregivers as well as courage to commit to a therapeutic regimen that is yet to be proved. No other research tool can substitute for clinical trials in developing novel therapeutics. Trial participants make an invaluable contribution to medicine by making themselves available—we cannot overemphasize the fact that none of the medical breakthroughs we have today would have been realized without their contribution.

To highlight one trial, the Alzheimer’s Disease Cooperative Study, a clinical trial network funded by the NIH, is currently recruiting participants for a trial aiming to curb the toxic effects of beta-amyloid .* This is not the only trial targeting Alzheimer’s disease—ClinicalTrials.gov lists 121 trials in the US and 216 when counted across the globe, actively recruiting participants, as of September 24, 2008. FasterCures has been highlighting clinical trial participation as a key element to accelerate medical research. I look forward to a day when an informed conversation about clinical trial participation becomes part of routine medical care.

*FasterCures does not endorse any specific clinical trial. Decisions on clinical trial participation should be made based on consultation with healthcare professionals.