Children's Hospital Colorado

Getting New Pediatric Drugs to Market Faster

Cancer and Blood Disorders | August 18, 2016

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Under normal circumstances, the body's immune system can tell when mutated or abnormal cells are present and dangerous and is able to kill them off before  potentially fatal infections — or cancer — starts.

"The body's own immune system," says Children's Hospital Colorado's Lia Gore, MD, "is really most effective at fighting disease." In certain cases though, the immune system doesn't recognize the mutated cells of cancer as harmful, so it doesn't fight them. That's where a new leukemia drug called Blinatumomab comes in.

Helping the immune system

Initially developed by a small German pharmaceutical company, Blinatumomab acts as a molecular agent that binds healthy T cells to the surface of the most common type of childhood leukemia cells. Essentially, the drug helps the immune system kill leukemia by thrusting the malignant cell right in the T cell's face — a process Dr. Gore illustrates by thumbing her nose: "It's like, neener-neener-neener, come get me," she says. Or at least that's the theory. 

"Every drug was experimental once," Dr. Gore observes. "If you're the parent of a child with aggressive relapsed leukemia, you don't care how many people it's been tested on. You want somebody to take a chance."

Whatever the reason behind its efficacy, Blinatumomab has achieved compelling results: deep remissions with very few of the typical side effects of chemotherapy. On September 1, 2016, Dr. Gore and her team received exciting news: Blinatumomab (or BLINCYTO®) was approved by the FDA.

"BLINCYTO represents the first new treatment option for childhood acute lymphoblastic leukemia (ALL) approved in more than 10 years," said Dr. Gore. "The opportunity to enhance the landscape of ALL treatment for children with this drug is very exciting."

These kids can't wait

Dr. Gore led a globe-spanning team of 20 hospitals, which worked to get the drug to children as quickly as possible.

"These kids can't wait," says Dr. Gore. "Ten years ago, it took on average seven years for a new cancer drug to go from testing in adults to use in children," owing to a combination of rigorous bureaucracy and prohibitive development costs, says Dr. Gore.

Through tireless advocacy, wide-ranging collaboration and aggressive clinical trials, the Experimental Therapeutics team has been instrumental in lowering Children's Colorado's local average substantially, in some cases, to a year or less, in order to get new treatments as quickly as possible to the kids who need them most.

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