Despite Setbacks, PhRMA Awards Encourage Research into and Fight against Alzheimer's

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Delaying the onset of Alzheimer's disease by just five years could reduce the cost for care of Alzheimer's patients by $447 billion a year by 2050, according to a new PhRMA report.

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.

By the year 2050, one American will develop AD every 33 seconds, and the US healthcare system will spend an estimated $1 trillion per year on caring for AD patients over age 65. These statistics were highlighted as part of two new PhRMA reports connected to the awards program and its focus on Alzheimer’s: “Researching Alzheimer’s Disease: Setbacks and Stepping Stones” and “Medicines in Development: Alzheimer’s Disease.”

Delaying the onset of AD by just five years could “reduce the number of people with the disease by nearly half and reduce the cost for care of Alzheimer’s patients by $447 billion a year by 2050,” according to the PhRMA medicines in development report. This is just one of the reasons the association chose to focus on AD in its launch year for the Research and Hope Awards, which replace PhRMA’s Discoverer’s Award.

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 the PhRMA report on researching AD. “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 the PhRMA medicines in development report.

Castellani also used the example of the NASA Mars Rover, which recently and successfully landed despite 20 out of 40 previous failed attempts by NASA to orbit or land on the planet (a 1 in 2 ratio), to explain why the industry shouldn’t give up on AD research.

As part of its awards program and AD focus, PhRMA is highlighting the value of partnerships in disease research. The 2012 recipients, for example, feature not only industry scientists but also academic researchers, patient advocates, and volunteers working to educate the public about the disease. “The enormity of the challenges facing Alzheimer’s disease researchers means that no single company, institute, or organization can tackle the disease alone, particularly when it comes to diagnosing and evaluating the progress of the disease. There are many collaborations in the world of Alzheimer’s research…,” states the PhRMA research report.

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. Pharmaceutical Technology is a media partner to the 2012 awards, which were given at the Washington, DC, Newseum, an appropriate venue, explained Castellani, because of its focus on providing the public with information.

The full Pharmaceutical Technology interview with Castellani, including his thoughts on why Alzheimer’s Disease was selected for the launch award program over other global priority and neglected diseases and how regulatory approval systems can help AD research, can be download as a podcast at