Statement of Sarah E. Cavanaugh, Ph.D., Medical Research Specialist
The NIH announced on Feb. 4 a new partnership with major pharmaceutical companies and nonprofit organizations to better understand the underlying biological factors behind Alzheimer’s disease, type 2 diabetes, rheumatoid arthritis, and lupus.
NIH director Francis Collins, M.D., Ph.D., has said that drug development has a failure rate of 99 percent and costs $1 billion for each success. This is because pharmaceutical development relies heavily on the use of animals in preclinical phases. The hundreds of millions of dollars invested annually (more than $800 million since 2008) in animal studies of Alzheimer’s disease, for example, have resulted in little progress in our understanding of or ability to treat this devastating disease. But the Accelerating Medicines Partnership (AMP) proposes human-based methods of finding biomarkers of disease. This is a positive step, because these studies will produce results that are directly relevant to diseases as they occur in humans.
Of at least 38 drugs developed to treat Alzheimer’s disease that have reached the clinical trial stage in recent years—after producing promising results in animal studies—only five have been approved for use in humans. All five drugs only temporarily manage symptoms in a subset of patients without actually slowing progression of the disease. At least 23 have failed in clinical trials, either because they were ineffective, had severe side effects not observed in animal studies, or because they actually increased the rate of cognitive decline in humans. As of January 2014, two more drugs designed to treat Alzheimer’s disease, solanezumab and bapineuzumab, were proven ineffective in clinical trials.
The AMP research goals for Alzheimer’s disease will involve clinical trials and large-scale analyses of human patient brain tissue samples to identify targets that play key roles in disease progression. Similarly, the AMP studies on diabetes will involve analyses of human DNA and will pool data from clinical studies involving more than 100,000 people.
We hope this investment is indicative of a budgetary shift toward more human-based, human-relevant research and away from ineffective animal research. We also hope the NIH will expand its future investments beyond drug development to include prevention-focused research, which holds far greater promise for combating the epidemics of chronic disease in the United States.
Founded in 1985, the Physicians Committee for Responsible Medicine is a nonprofit health organization that promotes preventive medicine, conducts clinical research, and encourages higher standards for ethics and effectiveness in research.