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SUPPORT GROUPS

Here is a list of groups supporting families of children with cancer.

Candlelighters Childhood Cancer Foundation:

http://www.candlelighters.org/

The Liddy Shriver Sarcoma Initiative:

http://www.sarcomahelp.org/

Drug development:

Children's Cause for Cancer Advocacy

http://www.childrenscause.org/index.shtml

Financial issues:

National Children's Cancer Society

http://www.nationalchildrenscancersociety.com/

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Who's fighting cancer in kids?

By Liz Szabo, USA TODAY

Stephen Sallan, chief of staff at Dana-Farber Cancer Institute in Boston, says adults attract more cancer research than children because they are a far larger and more lucrative market. Patients younger than 20 make up 12,400 of the nearly 1.4 million Americans stricken with cancer each year. Drug companies are generally unwilling to invest hundreds of millions of dollars into such a small market, he says.

Why adults instead of kids

Drug companies say there are sound reasons to focus on adults. Susan Desmond-Hellman, president of product development at Genentech, says she's compelled by the opportunity to help large numbers of patients. Not only does cancer strike far more adults, but their disease is less curable: 64% of adult cancer patients can be cured, compared with 80% of kids.

Organizing clinical trials for children also is difficult, Desmond-Hellman says. Because doctors are reluctant to try experimental drugs in kids who might be cured by standard ones, only a fraction of pediatric patients are eligible for early clinical trials. Enrolling enough kids to test a new drug can take years.

There are financial risks, as well. Companies typically invest more than 10 years and $800 million to bring new medicines to market, according to the Tufts Center for the Study of Drug Development.

Companies fear that if problems surface during pediatric trials, an otherwise promising drug might never be approved at all, even in adults. "It's a big risk for a small reward," Sallan says.

Because new, patented drugs are the most profitable, relatively few companies are interested in the older, generic drugs on which young patients depend, says Mary Relling, pharmaceutical department chair at St. Jude Children's Research Hospital in Memphis.

Production problems at one plant can disrupt the entire supply of a drug. Recently, doctors have struggled with shortages of at least five key oncology drugs. In one case, some children have had to go without a leukemia drug.

Doctors such as Sallan say they don't want to give up on children with cancer, who have perhaps more to lose from the disease than other patients. Young survivors pay a high price, their bodies scorched for up to three years with therapies so toxic that many are left permanently disabled.

New "targeted" therapies, which mostly spare patients from the ravages of conventional chemotherapy, might allow young survivors to lead normal, healthy lives, Sallan says. These breakthrough drugs are being tested almost exclusively in adults, however. Only one, Gleevec, has been tested in children and approved for their use.

Targeted drugs developed for adults might not help children, says Mitchell Cairo, chief of pediatric hematology and bone-marrow transplantation at the Morgan Stanley Children's Hospital at Columbia University.

Chemotherapy works for all ages because the drugs act broadly, poisoning growing cells — healthy and malignant — throughout the body. Today's "smart drugs" target only cells with particular genes, Cairo says. But the genes targeted in common adult cancers may not be the same ones that drive pediatric tumors. And targeted therapies often work for only a fraction of patients with particular types of cancer, which could shrink the market even further.

Maris says what children really need are drugs designed just for them.

The Food and Drug Administration has created a number of incentives to encourage companies to make pediatric drugs.

Through its orphan drug program, the FDA awarded Genzyme seven years to sell Clolar without competition from generics. Because Genzyme tested the drug in children, the FDA extended that exclusivity by six months. The FDA also gave Clolar "accelerated approval" in December, based on a study of 49 patients.

Yet such incentives haven't done enough to promote new pediatric drugs, according to the IOM report. Businesses have no incentive to test drugs in children early, because they receive the same benefits if they conduct trials before or after approval, the report concludes.

"There's a reason these drugs haven't gone forward," says David Parkinson, who collaborated on the report and heads the oncology development at Amgen. "They cost more to develop than they will potentially earn."

Promising strategies

Some doctors say they're starting to see progress. Researchers are testing more than 20 targeted therapies in children, says Malcolm Smith, associate branch chief of pediatrics for the National Cancer Institute's cancer therapy evaluation program. Several medications such as Velcade, Rituxan and Iressa already have been on the market for adults for several years. Many doctors would like to see drugs tested in children earlier.

That's why the NCI recently created a program to screen 10 to 15 promising drugs in the lab against common pediatric tumors.

Advances in molecular biology also may help. At Dana-Farber, scientists enroll patients in studies based on the biology of their tumors, not on their age, Sallan says. Doctors are planning to test a drug in adults and children with a type of leukemia.

St. Jude has launched a $10 million effort to begin testing drugs earlier. The hospital's director, William Evans, hopes industry eventually will collaborate with St. Jude, which recently opened a drug-making facility, to develop new therapies.

More coordination is needed

While such steps are encouraging, they are still far too rare, Parkinson says.

He suggests a more systematic approach: Government, university scientists, industry leaders and patient advocates could form a "public-private partnership" to jump-start drug development. Through this kind of partnership, biotech firms might share their "libraries" of experimental compounds, Parkinson says.

The NCI could help coordinate trials, and companies could keep the right to sell successful products. The partnership, acting like a non-profit corporation, could shepherd drugs through the approval process, then commercialize them.

"We will need someone to drive this process, to say, 'Our mission is to develop new cancer drugs for children,' " says Peter Adamson, an editor of the IOM report and chairman of the developmental therapeutics program of the Children's Oncology Group, a national research consortium. In the case of abandoned drugs, the IOM report calls on the NCI to step in as "developer of last resort."

Families say they are willing to do their part. Nearly half of child cancer patients participate in NCI-sponsored clinical trials, compared with only about 4% of adults, Smith says.

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