From workbench to bedside: an inventor’s tale

Dean Kamen’s portfolio of medical inventions ranges from infusion pumps to home dialysis machines to the iBOT, a mobility device for disabled people that is soon to be marketed by Johnson & Johnson. While he may slog through years of trial and error before solving a problem, creating technology is the easy part, Kamen recently told the Associates of the Cushing/Whitney Medical Library. Getting technology to patients is the challenge.

It often means waiting decades for FDA approval, marshaling support from the scientific community and persuading insurance companies to cover new devices. While not proposing specific reforms, Kamen called for a faster, more streamlined process for taking medical devices to market. “Just because it took longer, is it any better?” asked Kamen, president and CEO of DEKA Corporation. “Did it make it any safer? And what is the lost opportunity cost of not using it in between?”

Technology, Kamen added, is often misunderstood. He amazed the Harkness audience with a video of the iBOT in action. The machine allows the user to climb stairs, navigate rough terrain, reach high shelves and look a standing person in the eye. Kamen’s fear about the iBOT? That it will be perceived as just a souped-up wheelchair.

“Technology is moving faster,” he said, “but our adjustment to it is not. It took people 15 years to stop calling the Model T a horseless carriage.”

—Colleen Shaddox

The human genome—more than a list

Among the estimated 30,000 human genes are more than 250 genes that are implicated in oncogenesis. That, according to Michael R. Stratton, Ph.D., the Distinguished Lecturer for 2003 at the Cancer Center grand rounds in May, is extraordinary: close to 1 percent of known genes are risk factors for cancer.

Stratton, head of the Cancer Genome Project at The Wellcome Trust Sanger Institute in England, is tracing the history of such mutations and looking for patterns of behavior to try to develop methods of analysis. His team has found, for example, that kinases are heavily represented in cancer genes, while phosphatases are not. “There has to be something biologically configured about phosphatases that does not allow them to become cancer genes. It is this sort of analysis—that is not hypothesis-based, that is purely based on statistical constructs from analysis of the genome sequence—that will give us new biological insights.”

That could change the way cancers are classified, or lead to new treatments, Stratton said, before cautioning, “We just don’t know whether it is going to end up that way. But I think it is a reasonable aspiration that through these global descriptions of the cancer genome we will begin to move closer to an understanding of the core processes through which cancer develops.”

—John Curtis

From Seattle to Botswana, partnering to fight AIDS

For nearly three years Merck & Co., the Bill & Melinda Gates Foundation and the government of Botswana have worked together to help prevent the spread of HIV and mitigate the impact of the AIDS epidemic. The Merck Company Foundation and the Gates Foundation are each contributing $50 million; Merck is also donating its two HIV medicines.

Why Botswana? “Botswana has the highest HIV prevalence in the world,” said Jeffrey L. Sturchio, Ph.D., vice president for external affairs, Europe, Middle East & Africa at Merck. But because it also is one of the wealthiest nations in Africa, basic health care infrastructure was already in place and Botswana’s leaders were committed to the fight against HIV/AIDS. The African Comprehensive HIV/AIDS Partnerships has helped build health resource centers at district hospitals and trained nurses and doctors in HIV/AIDS prevention and treatment, Sturchio told an audience at the Center for Interdisciplinary Research on AIDS in April.

“Implementing a comprehensive HIV/AIDS program like this is a complex, dynamic and time-intensive process,” Sturchio said. “It has to be done in the context of broader development policies. Mobilizing local capacity and local resources is absolutely critical. Working together it is possible to make a world of difference in the lives of people living with HIV/AIDS.”

—John Curtis

Using genomics to craft a safer pharmacopeia

Every year some prescriptions go awry, causing the deaths of about 100,000 patients from toxic responses to medications. “We can’t predict who will respond to a drug,” said Janet Woodcock, M.D., director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA). That could change with the application of genetic knowledge to predict individual responses to medications. It is “more than promise now,” Woodcock said. “It’s starting to happen.”

Woodcock, the keynote speaker at the annual Pharmacogenetics and Medicine Lectures sponsored by Genaissance Pharmaceuticals in April, oversees federal regulation of emerging knowledge about the role genetic variability plays in reactions to medications. Labels for a small number of drugs, she noted, already include information about genetic differences that could affect a patient’s response. The FDA has begun to study electronic formats for providing more of such information to physicians.

According to Woodcock, the FDA has requested that companies provide genetic information when available, but has no plans to require pharmaceutical companies to submit genetic response data about their medications. “We need to understand the science,” she concluded, “so that we can develop an appropriate policy” which will allow the public to benefit from this knowledge.

—Marc Wortman