St. Jude invests in drug research

Leaders of St. Jude Children's Research Hospital say they're seeking new drugs to treat childhood cancer and other rare diseases because they don't believe the pharmaceutical industry will.

The overall cure rate for major childhood cancers is around 70 percent today, and leaders at St. Jude, a leading research center for childhood cancer and other rare diseases, say better drugs could save more children's lives.

But companies are unlikely to invest the hundreds of millions of dollars it takes to develop the new drugs, said Dr. James Downing, the hospital's scientific director.

"Probably the biggest obstacle is these are rare diseases, and there is little financial incentive for a large pharmaceutical company to pursue work in these areas," he said.

The Pharmaceutical Research and Manufacturers of America, a group representing drug manufacturers, didn't provide information about member research in children's cancer drugs Thursday. But it said in a statement that drug manufacturers have made strides in fighting rare diseases and that the Food and Drug Administration has approved more than 160 new medicines for such illnesses over the past decade.

However, the authors of a 2005 National Academy of Sciences report, titled "Making Better Drugs for Children with Cancer," say the number of children with cancer is so small that the extra incentives the federal government gives to the makers of drugs for rare diseases aren't enough to encourage companies to invest in research.

Roughly one in 6,400 children was diagnosed with cancer in 2001, according to the report.

The result of the lack of company research is that most children are treated with drugs that were designed for adults, the report says.

"But in fact, the biological and clinical characteristics of nearly all childhood cancers differ substantially from adult cancers," the report states.

Those differences mean there is a chance to discover new drugs and raise survival rates, the report's authors write.

The leaders of St. Jude don't express resentment against pharmaceutical companies. Indeed, Downing said the hospital wants to work with pharmaceutical firms to bring the drugs to clinical trials and eventually to market once the basic research is complete.

The company would pay St. Jude for its intellectual property to prevent money from hospital donors from flowing to a private company, he said. And St. Jude would look for deal terms that would ensure that the new drug was reasonably priced and available to patients, he said.

The hospital recently began a new chemical biology and therapeutics department, and leaders have said they plan to invest $60 million in it over the next five years.

St. Jude has also built a sophisticated automated facility that screens thousands of compounds for their effectiveness against cancer.

Despite the scale of the investment, St. Jude is setting modest goals.

"We hope over the next few years to be able to move one of those compounds forward into a clinical trial," Downing said.

 

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