NCPA - National Center for Policy Analysis


June 8, 2005

Smart drugs like Avastin, Tarceva and Gleevec are transforming the treatment of many cancers for adults, offering the hope of turning once-deadly conditions into manageable, chronic diseases. Now doctors are starting to test whether children will benefit from these drugs as well.

  • There are an estimated 15 to 20 clinical trials in the works around the country where children with cancer are being given so-called smart drugs.
  • Unlike standard chemotherapy agents, these drugs target only cancerous cells and leave normal cells alone.
  • Most of the drugs are approved for use in adults to treat cancers that children rarely or never get, such as lung, breast and prostate cancers or certain forms of leukemia.
  • But the belief is that the mechanism causing certain cancers to grow is similar, and that these drugs could be a less-toxic, more-effective, treatment for children.

Little is known about the long-term effects, and some oncologists worry that the way the drugs attack tumors and stop the formation of new blood vessels could inhibit children's growth. But the rising interest is driven by a growing concern that progress in treatment has stalled.

  • Survival rates for children increased to 78.6 percent between 1995-2001, but childhood mortality rates have flattened out.
  • Most of the drugs used to treat children have been around for decades and are effective, but 20 to 25 percent of children don't respond to them.

New approaches are needed, but drug companies are reluctant to subject sick children to trials until more is known about their safety. Additionally, the market for children is much less lucrative for drug companies and they are concerned that side effects in children might disrupt adult usage.

Source: Amy Dockser Marcus, "Testing 'Smart Drugs' On Children With Cancer," Wall Street Journal, May 31, 2005.

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