New Drugs Needed in Battle Against Childhood Cancers

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Pedidome.jpgOn Sept. 23, the House Childhood Cancer Caucus hosted its Second Annual Childhood Cancer Summit on Capitol Hill in Washington, D.C. The caucus is co-chaired by Congressmen Michael McCaul (R-TX) and Chris Van Hollen (D-MD).

In addition to hosting the summit, McCaul and fellow Congressman G.K. Butterfield (D-NC) introduced the "Creating Hope Act of 2011" legislation that would incentivize pharmaceutical companies to develop new drugs for rare pediatric diseases such as cancer.

Eugenie Kleinerman, M.D., head of the MD Anderson Children's Cancer Hospital, was there to speak before the caucus on issues concerning the development of new agents for pediatric cancers and improving patient access.

Excerpts from Kleinerman's remarks before Congress explain the challenges and potential solutions to improving the cure rates for childhood cancer.

Unacceptable treatment
In her words:

"Cancer continues to claim the lives of more children than any other disease. This statistic has not changed in more than 20 years.

Even more disturbing is that the actual cure rates for children with acute myeloid leukemia and solid tumors such as sarcomas, brain tumors and neuroblastoma have not improved.

This is an unacceptable statistic for a disease that claims the lives of so many of this country's youth. It robs us of future talent and tomorrow's leaders, tax payers and contributors.

Curing cancer requires new drugs. This is a fact.

Left Behind
In the past 20-plus years, there have been numerous new agents approved to treat adults with cancer, but only one new agent has been approved for children with cancer and that was chlofarabine in 2004.

Pediatric oncologists are using the same drugs that we used 40 years ago.

Ironically, the first clinical trials combining several different chemotherapy drugs were used to treat children with leukemia. The pioneers in institutions in Memphis, Boston and Bethesda were told that combining these cytotoxic agents together would be too toxic and would kill patients. But rather than kill, these combinations cured children who faced certain death.

These children became survivors of childhood cancer. This concept of using combination chemotherapy for cancer treatment is what we use today for many different adult cancers. It was first in children, then in adults. Yet today, children are being left behind with long delays or often no access to new drugs. What happened?

Somewhere along the line we adopted an attitude of protecting children from toxicity rather than being proactive in finding them a curative therapy. We have forgotten the lessons of the past. We have taken our eye off what I believe to be the goal of "cure" to one of "fear of inducing toxicity."

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Improving Access to New Cancer Therapies for Children

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