Earlier this week, on Sept. 12, 2012, the Pharmaceutical Research and Manufacturers of America (PhRMA) honored nine individuals for their research into and fight against Alzheimer’s Disease (AD) as part of the association’s new Research and Hope Awards. The event was at the Washington, DC, Newseum and featured as keynote speakers Marc Kennedy Shriver, who wrote a book about his father’s experience with Alzheimer’s, and Meryl Comer, an Emmy award-winning journalist.
The evening was about inspiration and motivation to keep up the fight against AD, whether as caregivers, patient advocates, health professionals, or scientists.
It’s no secret that ongoing as well as recent setbacks have challenged research and discovery scientists working to find a treatment for AD. “Years of research has yielded five medicines that provide some symptomatic relief in some cases but has not yet resulted in an approved disease-modifying medicine,” states a new PhRMA report on researching AD, released in connection with the awards. “What sets AD apart from other diseases is that scientists still do not know what causes it. We may very well find a treatment first, that leads us back to the cause,” PhRMA President and CEO John J. Castellani told Pharmaceutical Technology in an interview before the award recipients were unveiled on Sept. 7, 2012.
There are two additional key challenges to developing AD medicines listed in the PhRMA research report: the limited utility of current models of the disease, which creates a barrier in preclinical testing of drug candidates; and the absence of validated noninvasive biomarkers of disease activity and progression, which delay diagnosis until patients become symptomatic. As a result, long and expensive clinical trials have become the mainstay of human studies.
This past summer, Johnson & Johnson, Pfizer, and Eli Lilly all released reports of failed clinical trials targeting the beta-amyloid protein, which is known to form brain plaques in AD patients. Some reports have alluded to lost confidence and momentum in the fight against AD as a result, not to mention potential decreases in R&D investment, but Castellani hopes that these ups and downs are just part of the biopharmaceutical discovery process. “…Researchers take the findings from the unsuccessful projects and use that new information to step forward and continue their quest,” states the PhRMA research report.
Castellani acknowledges that AD is particularly challenging and complex, with 101 unsuccessful drug trials targeting AD since 1999 and a success ratio of 1 in 34 compared with most new medicines, which have a 1 in 5 chance of moving forward. The fact that traditional animal models cannot be used in trials for AD and that human trial recruitment is difficult only add to the complexity of finding a successful AD therapeutic. Castellani has high hopes, however.
He points to the fact that there are nearly 100 new medicines in development right now for AD and dementias, boasting a full pipeline that has potential to reverse, treat, and hopefully prevent this catastrophic disease. These drugs include a medicine that inhibits the formation and accumulation of amyloid-beta and tau protein deposits; an intranasal medicine that is able to penetrate the blood-brain barrier for mild cognitive impairment, a precursor to AD; and a gene therapy, according to a second PhRMA report on medicines in development that was released this week.
PhRMA is encouraging increased public understanding and awareness of AD and its unique challenges as well. The association has issued a list of 108 ongoing clinical trials for AD patients. Many caregivers of AD patients simply don’t have the energy or time to enroll their loved ones in a trial and the patients themselves often do not have the cognitive ability to enroll themselves, Castellani told Pharmaceutical Technology, but PhRMA hopes this may change as more information about the disease and about the research being done to help treat it reaches the public.
For a list of the 2012 PhRMA Research and Hope Award recipients, including the Merck BACE team, see the PharmTech awards announcement story.