New partnership needed for child cancer - report

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The National Cancer Institute should work more closely with drug companies to help them find new and better drugs to treat childhood cancer, a panel of experts said on Monday.

Because cancer is so rare in children, and is different from cancer in adults, special help and incentives are needed to develop treatments that will work specifically for children, the Institute of Medicine panel said.

Doctors and drug companies have worked well together to make pediatric cancer highly curable. The Centers for Disease Control and Prevention says 79 percent of children under the age of 15 diagnosed with cancer live for five years or more, compared to 56 percent in the mid-1970s.

"Childhood cancer is rare. In the United States, an estimated 11,900 children and adolescents under age 20 were diagnosed with cancer in the year 2001 and about 2,200 died,” the report said.

“Between birth and 20 years of age, about 1 in 333 Americans develops cancer,” it added.

“A few types of cancer predominate—leukemias, and tumors of the brain and nervous system, the lymphatic system, kidneys, bones, and muscles. For the most part, these cancers are distinct biologically and clinically from the cancers common in adults, even if the names are similar.”

Though some drugs developed to treat adults work for children, some do not. This means some companies do not want to spend the money to develop drugs that have such a limited market.

“What appears to be missing in order to realize the potential for new childhood cancer drugs is an organized focus on childhood cancers to coordinate the pieces and drive a process toward shortening the developmental time line and multiplying the numbers of possible new agents,” the report said.

“Neither industry nor government can be expected to play this role alone. Not industry, because the market is too small and risky, even with orphan product incentives.”

Dr. Malcolm Smith, a pediatric cancer expert at the National Cancer Institute, said his agency was working on some of the ideas in the report.

“But there needs to be a more coordinated effort and it needs to be a larger effort,” Smith said.

More of the preclinical work—done in lab dishes, rats and mice to find promising agents to test in people—should be directed at pediatric cancer specifically, Smith added.

This will cost more, but a lot of the work can be done at universities with National Institutes of Health and private funding, Smith and the report both said.

The panel of experts on health policy, pediatric cancer and other areas also recommended that the National Cancer Institute should be an agent of “last-resort” to test cancer drugs that work only in children and that companies are reluctant to develop themselves.

And agencies such as the Food and Drug Administration should work with companies to speed the process of testing cancer drugs in children, it said.

The panel was chaired by Joseph Newhouse, a professor of Health Policy and Management at Harvard University, and included Dr. Peter Adamson, a pediatric oncologist at The Children’s Hospital of Philadelphia and RAND corporation researcher Richard Rettig.

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