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Tuesday, 29 April 2008

New promising treatment of tumors saves a girl

A lovely story of new and smart treatment to cancer that saved a little girl combining known medicines in unexpected way. Hopefully this will give a new boost to cancer treatments and pharmacists companies will back it. The article is from NY Times.
Melanie was 9 months old when her parents faced an agonizing decision. She had already had two operations for a malignant brain tumor, and doctors could not be sure they had removed all the cancer. She needed more treatment, but standard chemotherapy offered little hope in exchange for its harsh side effects.

Doctors at the Dana-Farber Cancer Institute in Boston offered another option, an experimental treatment. To qualify, a child had to have a progressive cancer, and it had to be terminal. The McDaniels took a gamble and a leap of faith, and signed Melanie up.

Recently, Mr. McDaniel sent me an e-mail message. “Melanie is now 7 years old, attending first grade, and doing very well,” he wrote. “The doctors told us last year that they do not see any residual tumor in her brain. Their original diagnosis was that her tumor had no known cure.”

Dr. Folkman founded a branch of research based on the theory that tumors need a blood supply in order to grow and can stimulate the formation of new blood vessels — angiogenesis — to feed themselves. If angiogenesis could be stopped, he reasoned, it might be possible to starve tumors. His work ultimately led to useful treatments but took years to gain acceptance in a field that was focused on attacking cancer cells directly.

Melanie McDaniel became one of 20 children with advanced cancer who were enrolled in a study that used drugs strictly to fight angiogenesis. The drugs included two standard anticancer medicines, but in small doses meant to stop blood vessels from forming, not the much bigger amounts needed to poison tumor cells.

The children also took two other drugs that had been found to block angiogenesis. One was Celebrex, usually given for pain and inflammation. The other was thalidomide, notorious for causing stunted limbs and other birth defects when pregnant women took it in the 1960s — damage, it was later learned, that the drug inflicted by halting the growth of blood vessels in the fetus.

Melanie and the other children were given small doses of medicine by mouth every day, instead of big doses intravenously every few weeks. The idea was that continuous treatment might keep blood vessel growth in check, whereas the usual schedule of therapy every few weeks could give new vessels a chance to sprout between doses. Doctors also hoped that the small doses would minimize side effects. The approach is called metronomic, low-dose or antiangiogenic chemotherapy.

“Our goal was to see whether we could keep the kids alive for an additional six months,” said Dr. Mark W. Kieran, Dana-Farber’s director of pediatric medical neuro-oncology.

The study was meant to test the feasibility of using the drugs for 26 weeks. But by the 26th week, seven children were doing so well that their parents refused to give up the drugs.

The McDaniels kept Melanie on the drugs for a year and a half. Then, she was monitored closely with M.R.I. scans.

Finally, last year, her doctors said there were no traces of the tumor left.

The next step is a larger study. One is already under way, involving 160 children at 12 medical centers, with eight categories of cancer.

“As much as we’re excited about how good she’s doing, there’s that much fear of it coming back,” said her mother, Amy McDaniel. “It’s always in your mind.

“We need the science to keep going. We need to be armed and ready if it does return.” source

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