Removing barriers to battle disease

NIH plan promotes work between fields, involves more doctors in tests

October 01, 2003|By Jonathan Bor | Jonathan Bor,SUN STAFF

WASHINGTON - The National Institutes of Health unveiled a sweeping plan yesterday to involve more doctors in clinical trials, make technology widely available and reward scientists hoping to pursue bold but risky ideas.

Dr. Elias Zerhouni, the agency's director since last year, said the plan would remove barriers that keep scientists from turning some of the great advances of recent years - such as the charting of the human genome - into treatments and cures.

The plan, which he called the "NIH Roadmap," would unite scientists from disciplines as varied as engineering, physics and mathematics with medical specialists in the fight against specific diseases. In the process, the agency wants to break down what he called the "silo mentality" that keeps scientists from sharing their ideas, he said.

"We want to completely reshape the way we are doing clinical research," said Zerhouni, who until his NIH appointment was vice dean for research at the Johns Hopkins School of Medicine. "This is not business as usual."

The plan will cost $2.1 billion across the next five years, including $130 million in the fiscal year that begins today. It was developed with consultation from more than 300 leaders in academia, industry, government and the public.

One of the plan's key features is the expansion of clinical trials to include patients seeing doctors in community practices. Patient advocacy groups, such as those organized around cancer and neurodegenerative diseases, would also play a greater role in recruiting study participants.

Most trials are now confined to academic research centers - such as Hopkins and the University of Maryland in Baltimore - which Zerhouni said limits their scope.

Models for this approach exist, Zerhouni said. For example, most children with leukemia and lymphoma participate in clinical trials through their community physicians as well as teaching hospitals. Also, advances in the treatment of athlerosclerosis and high blood pressure occurred because tens of thousands of patients were recruited for a single trial, many through community doctors.

Yet only 1 percent of Parkinson's disease patients and 3 percent to 4 percent of adults with cancer take part in research, largely because trials are overly centralized, he said.

Patient involvement

Involving more patients in clinical trials has become crucial as researchers turn their attention to chronic illnesses such as osteoporosis, rheumatoid arthritis and even AIDS. Patients with these disorders are more likely to be seen by community physicians than by academic centers.

"With the shift to chronic diseases, one of the challenges and barriers is public participation," said Dr. Stephen Katz, director of the National Institute of Arthritis and Musculoskeletal Diseases.

Reaching out to community practices will not only increase the number of patients in a trial, but also ensure that participants are more representative of the general population, Katz said.

Doctors who are not ordinarily engaged in clinical research will have to be trained in research ethics and practices, especially those designed to protect the safety of volunteers, Zerhouni said.

The NIH also plans to establish "molecular libraries" housing tens of thousands of compounds that scientists can use in their research. Using robotic devices, scientists would be able to test each chemical in a blind search to find one that switches a gene on or off or keeps a protein from getting inside a cell.

For example, Katz said, scientists are using this approach to find a chemical that might switch on a gene that creates bone. Such a chemical could be used to treat osteoporosis, a disorder in which bones become porous and brittle, particularly in older women.

The plan also calls for national computing centers, which would enable scientists from across the country to tap into a supercomputing network to share and analyze data.

In addition, Zerhouni announced the creation of awards to help scientists pursue bold ideas that have a high risk of failure but would produce important results if they succeed. Five-year grants of $500,000 will be awarded to 10 scientists in the first year, Zerhouni said.

While leaders in medical research were sifting through the plan's complex details, many applauded its ambition and scope.

"I think first of all, it's really great to see [Zerhouni] take on this leadership role," said Dr. Michael J. Klag, vice dean for clinical investigation at the Johns Hopkins School of Medicine. Hopkins receives $500 million in NIH funding a year - more than any other research center in the United States.

"The plan for clinical research really hits the nails on the heads," said Klag, who attended one of the NIH planning meetings. "The issues that people think are important are all in here."

Praise for concept

Dr. Howard B. Dickler, associate dean for research and graduate studies at the University of Maryland School of Medicine, also liked what he saw.

"The fact is, that because of the explosion of biomedical information, we have more potential new therapies coming down the pipeline than ever before in history," he said.

"They only actually do people good and advance health and longevity if we test them. You have to go where the patients are, and they aren't necessarily in this day and age in the academic health centers."

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