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Madame Butterfly

Who's Fighting Cancer in Kids?

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Who's Fighting Cancer in Kids? Drug Firms Not Interested


By Liz Szabo, USA TODAY


(July 20) -- Fourteen-year-old Max Levine owes his life to experimental cancer therapies. Yet a drug that helped keep him alive might never reach the market.


University pharmacists mix the medication, called 131I-MIBG, for patients who have no other options, says his doctor, John Maris, an associate professor at The Children's Hospital of Philadelphia. Max's disease - neuroblastoma, a tumor of the nervous system - afflicts just 650 children a year and is too rare to attract drug developers, Maris says. The tumor, like all childhood cancers, is considered an "orphan" disease.


"If our trial is successful, and we prove this drug cures patients, there is no guarantee that anyone is going to make it," Maris says. "We could say, 'We've proved this drug works, but we don't have any.' " (Related story: Kids' cancer drugs run short)


Maris is one of many doctors who are frustrated by the lack of interest in developing drugs for young cancer patients. A government report in April found a "near absence" of research into pediatric cancer drugs. About half of the oncology drugs used to treat children are at least 20 years old, according to the report by the Institute of Medicine, a non-profit group that advises the government on health policy.


Most drugs given to children were developed for adults, then passed down to children. In the past 10 years, only one cancer drug, Clolar, has received initial approval for children.




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.


Max's mother, Sue Levine, says she's counting on scientists to come up with new drugs. Experimental therapies have given Max two years. Although 131I-MIBG stopped working after six months, it kept the Cherry Hill, N.J., boy alive long enough to become eligible for another experimental drug, which has controlled his cancer for a year and a half.


"The fact that my son is here playing with his GameBoy is a miracle," Levine says. "If my son can give these doctors some knowledge, he's willing to try it."

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I donate as much as I can,as often as I can to St.Jude's Childrens Hospital's Cancer Research Center.

Cancer doesn't distinguish children from adults.It's a monster,and we need to eliminate it.

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ugh... that's why I hate mitosis. So many things can go wrong... :roflmao:

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A friend of mine lost her daughter last May.


She was 10.


She'd been battling leukemia, and it wasn't the cancer that killed her, it was the lack of immunities.


Most horrible experience I have ever been a part of.


Sadly, her father lost his brother to the same cancer at the same age.


The way his body rocked with tears and anguish will make me cry any time it comes to memory for the rest of my life.

Edited by Madame Butterfly

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