June 17, 2008
12:00 a.m. Eastern Time
George Mason Scientists Say Determining Which Proteins Are Active During Diseases Could Allow Expensive Treatments To Be Better Targeted
In Second Of Two Health Affairs Interviews Focusing On Proteomics And Nanotechnology, FDA Official Says That The Agency Is Evaluating Nanotechnology Products Without The Need For New Regulatory Guidelines
Bethesda, MD -- In an interview published today on the Health Affairs Web site, two George Mason scientists describe technology they have developed to determine which proteins are active in cancerous tumors and other disease processes. This technology could aid physicians in targeting powerful and expensive drugs only to those patients who will actually benefit, say Lance Liotta and Emanual Petricoin, codirectors of the Center for Applied Proteomics and Molecular Medicine at George Mason University in Fairfax, Virginia. http://content.healthaffairs.org/cgi/content/abstract/hlthaff.27.4.w310
The interview with Liotta and Petricoin is one of two being published today in which Health Affairs contributing editor Barbara J. Culliton explores the emerging fields of proteomics, the study and manipulation of proteins, and nanotechnology, the study and manipulation of particles less than one-billionth of a meter in size. In the second interview, Culliton discusses the Food and Drug Administration’s views on nanotechnology with Norris Alderson, associate commissioner for science at the FDA’s Office of Science and Health Coordination in Rockville, Maryland. http://content.healthaffairs.org/cgi/content/abstract/hlthaff.27.4.w315
Both interviews are part of a series of interviews concerning innovation supported by a grant from the Pew Charitable Trusts.
Liotta states that the work he and Petricoin are doing sits “at the intersection of nanotechnology and clinical proteomics.” Among other advances, the two scientists have developed a “reverse phase protein microarray,” a technology that uses nanoparticles to determine which proteins are active in a given biological sample. Liotta and Petricoin, who have founded a company to commercialize this technology, note that much attention has been focused on using genetic analysis to target medical interventions. However, genetic analysis can only determine which proteins may be present in cells, and, to be effective, treatments must target proteins that are active -- not just present -- during disease.
“Genomics has led to the notion of ‘personalized medicine,’ but I believe that the road to personalized medicine will be paved with proteins,” Petricoin says. He notes that in cases where only 20 percent of patients respond to particular cancer drug 80 percent “are getting unnecessarily treated. They have unnecessary toxicity, they suffer, and they don’t respond at all, whereas the other 20 percent respond. Why not predict those responders ahead of time, up front? . . . How do you do that? You see if the actual drug target that the drug is going to inhibit is in use in that cancer cell -- in that patient.”
Alderson: The FDA’s Current Regulatory Framework Is Adequate
For Evaluating Nanotechnology Products
Because nanoparticles can behave differently from larger particles both chemically and physiologically, some have advocated that the FDA develop a new regulatory framework for evaluating the safety and effectiveness of nanotechnology products. However, the FDA’s Alderson tells Culliton that an agency task force determined that the FDA’s existing regulatory framework is adequate for evaluating nanotechnology products. Alderson cochaired the task force, which was appointed by FDA commissioner Andrew von Eschenbach in 2006.
Alderson says that the FDA did recognize a need for more information on nanotechnology products in areas such as biosafety. However, instead of modifying its regulations, the FDA is collaborating with other agencies, such as the National Cancer Institute, to develop such information. “It’s a novel approach. We’re bringing our expertise in terms of our data needs to show that a product is safe and effective. The NCI is providing the dollars and scientists to evaluate some of these basic nano materials to determine [their] biological, chemical, and physical properties,” Alderson says.
Alderson tells Culliton that the properties of nanoparticles allow cancer drugs to be delivered directly to a tumor and only to the tumor. Culliton asks: “Given the specificity and potential range of nanomedicine, do you anticipate appreciable effects on the cost of medical care using these products?” Alderson responds that “we’re all hopeful that nano materials will reduce costs,” but he warns: “I think it’s just too early to tell. Until we see some of these products make it to the marketplace, it’s all conjecture.”
After the embargo lifts, Culliton’s interview with Liotta and Petricoin will be available online at
Culliton’s interview with Alderson will be available online at
ABOUT HEALTH AFFAIRS:
Health Affairs, published by Project HOPE, is the leading journal of health policy. The peer-reviewed journal appears bimonthly in print with additional online-only papers published weekly as Health Affairs Web Exclusives at www.healthaffairs.org.
©2008 Project HOPEThe People-to-People Health Foundation, Inc.