Cancer drugs for children remain scarce in U.S.

May 23, 2013 8:16:53 PM PDT
The Texas Medical Center is known for offering the latest treatments. And Houstonians have come to expect the best medical care in the world. But there's one area where medicine is lagging far behind -- cancer drugs for children. In fact, there's only been one new cancer drug for children approved in the past two decades.

When you're a child with cancer...

"The options are very grim," Rice student James Ragan said.

Ragan knows first hand.

"I had a large tumor," Ragan said.

He was 13. He has scars from half a dozen cancer surgeries to remove bone cancer. But it still spread to his lungs. Ragan had the newest surgical techniques to save his leg. But when it came time for chemotherapy, his family was shocked.

"There weren't any good drugs out there for kids with osteosarcoma," Ragan said. "And it's not just that way for kids with osteosarcoma, it's like that for kids with so many different types of cancers."

Dr. Eugenie Kleinerman heads MD Anderson Children's Cancer Hospital.

"I've been frustrated for 28 years," she said. "There has been one new drug for pediatric cancer that has been approved in the past 20 years in this country."

Dr. Kleinerman says you hear about the success with children's leukemia -- 80 percent cure rate. But that's not true with other pediatric cancers. Because they're rare, drug companies don't want to invest millions of dollars developing drugs for children when it's not profitable.

"The amount of dollars that go to fund pediatric cancer research is minuscule, 4 percent," Dr. Kleinerman said.

"That's the saddest reason to lose the lives of kids," Ragan said.

Since we first looked at this issue almost two years ago, Congress has passed the Creating Hope Act. It offers pharmaceutical companies a financial carrot for pharmaceutical companies; if they develop a pediatric cancer drug, another of the company's more profitable drugs will be fast tracked for FDA approval.

So far, MD Anderson knows of no company that's bitten.

"There's always hope and we're not giving up," Dr. Kleinerman.

To speed new drugs, they're working to:

  • Use old drugs in new ways
  • Test adult cancer drugs on children's cancers
  • Include teenagers in adult studies
  • Increase research spending
  • Speed up the FDA

"For kids to die because they don't have enough drugs available or treatment options is just a poor reason," Ragan said.

So Ragan and his sister have started a foundation that is raising hundreds of thousands of dollars that they hope will change the future for children with cancer.

Meanwhile, a cancer drug developed in Houston is saving lives of children all over the world, but not in the United States. It's an experimental drug called Mepact, and it was developed by Dr. Kleinerman here at MD Anderson.

But the drug was approved in Europe and in Mexico, but couldn't get the FDA's stamp of approval. The FDA says it wants another study, which would take years and cost $100 million.

The decision has forced American families to take a trip to Europe or Mexico to get that chemotherapy.

Christi Myers will explain why on Eyewitness News at 10.

Find Christi on Facebook at ABC13-Christi Myers or on Twitter at @ChristiMyers13


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