Preeminent Physician-Scientists at Children’s Hospital Colorado Set the Standard for New Therapies
Birthdays have always been a big deal for Ellory. But this year took the cake.
When Ellory turned 11 in November, it was the first time in three years that she celebrated her birthday outside the hospital. More significantly, she commemorated another year of life – no small feat for a young leukemia survivor who endured 850 days of chemotherapy as part of her treatment at Children’s Hospital Colorado.
While Ellory’s leukemia is now in remission, thousands of other children’s cancer battles are ongoing. But with the help of new therapies pioneered at Children’s Colorado, there is more hope for these patients than ever before.
Children’s Colorado is home to a cancer-fighting army of caregivers who are leading cutting-edge research to cure childhood leukemia – and to rapidly develop new cancer drugs and treatments for children who urgently need them.
“These kids can’t wait,” says Dr. Lia Gore, Chief of the Center for Cancer and Blood Disorders at Children’s Colorado, who has been a pediatric oncologist for more than 15 years.
The need for pediatric research
An estimated 15,000 children will be diagnosed with cancer in 2016, and 1 in 5 who are diagnosed will die from the disease within five years. In the United States, cancer is currently the leading cause of death by disease in children under the age of 15.
Despite these staggering statistics, government funding for pediatric cancer research is woefully low. The federally-funded National Cancer Institute spends just 4 percent of its budget on pediatric cancer research – the other 96 percent is dedicated to adult cancer studies.
As a result, new pediatric cancer treatments have stagnated, even as other medical advancements have seen rapid progress. Over the past 26 years, the FDA has approved only four pediatric cancer drugs, compared to more than 100 new adult cancer drugs. Moreover, an estimated half of all chemotherapy
First new drug in a decade
With the help of philanthropic partners, physician-scientists at Children’s Colorado are on the cutting edge of pediatric cancer research that is leading to promising new treatments.
Dr. Gore led a globe-spanning team of 20 hospitals that developed a new leukemia drug, which was recently approved by the Food and Drug Administration (FDA). Called blinatumomab, the drug targets acute lymphoblastic leukemia – the most common form of childhood cancer. The results have been impressive: widespread remissions and very few of the typical side effects of chemotherapy.
Pharmaceutical company Amgen Inc. is now distributing the drug under the name BLINCYTO®. The drug is the first new treatment option for childhood leukemia to be approved by the FDA since 2004. And with decreases in federal funding, it never would have happened without the support of donors, who provided critical funding to support Dr. Gore’s research and collaboration have the funding to support all of the studies we need to do. That’s why community support is vital. By partnering with donors who care as deeply as we do about eradicating childhood cancers, we’ll continue to pioneer new cancer breakthroughs that impact the lives of children here and around the world.” efforts. Dr. Gore is chairholder of the Ergen Family Chair in Pediatric Cancer and the Robert J. and Kathleen A. Clark Endowed Chair for Pediatric Cancer Therapeutics.
“The children who enrolled in this drug trial had undergone many prior regimens of therapy,” says Dr. Gore. “The fact that a single drug could put a patient with such a resistant disease into remission is quite remarkable and very promising.”
Blinatumomab uses the body’s immune system to fight cancer by helping healthy T cells bind to malignant leukemia cells. It’s a treatment approach known as immunotherapy, and it’s far less toxic than traditional chemo, which wipes out the body’s healthy immune system along with the cancer cells, making patients highly susceptible to illness.
“The body’s own immune system is most effective in fighting disease,” explains Dr. Gore. "We just have to give it the proper chance."
No time to lose
Children’s Colorado researchers are working every day to find less toxic, less invasive pediatric cancer treatments. And that could mean a future where cancer is effectively treated without chemotherapy.
The approval process for new pediatric cancer drugs is cumbersome to say the least. Between rigorous bureaucracy and prohibitive development costs, it can take a decade or more to make a drug available to all patients – not just those enrolled in clinical trials. For Dr. Gore, that’s simply too long.
“Ten years ago, it took an average of seven years for a new cancer drug to get from testing in adults to use in children,” she says. “That’s just not acceptable, which is why we are working hard to accelerate the research and approval process.”
Today, through advocacy, collaboration and aggressive clinical trials, Children’s Colorado has been instrumental in decreasing the time it takes to get new treatments to kids who need them – in some cases, shortening the process to one year or less.
Steve Winesett, President and CEO of Children’s Hospital Colorado Foundation, says that philanthropy is essential to getting new treatments from the laboratory to the bedside.
“Children’s Colorado is at the forefront of some of the most promising pediatric cancer research of our time,” explains Winesett. “But we simply don’t have the funding to support all of the studies we need to do. That’s why community support is vital. By partnering with donors who care as deeply as we do about eradicating childhood cancers, we’ll continue to pioneer new cancer breakthroughs that impact the lives of children here and around the world.”