Cancer, once a terrifying black box, is slowly being opened up. Thanks to advances in technology and genomics, we know significantly more today about what drives cancer.
Precision medicine, the concept of giving the right treatment to the right patient at the right time, is flourishing in cancer treatment. But there is a blind spot: children are not benefiting enough from the progress we’re making.
We need a full-scale renaissance in pediatric drug development and care. And we need it now.
Researchers and clinicians have been chipping away at the devastating threats from cancer for the last 70 years. In the late 1940s, legendary cancer researcher Sydney Farber and colleagues reported successfully treating acute leukemia in children with a new drug called aminopterin. By the 1960s, an effective combination of chemotherapies had been assembled, renewing optimism in the fight against acute lymphoblastic leukemia (ALL) — the most common cancer in children — and many other cancers. This same strategy, high-dose combination chemotherapy, is still used to treat ALL.
In 1964, just 3% of children diagnosed with ALL survived for five years. Today, about 90% live that long — and longer. But survival rates don’t tell the whole story. For the 10% to 20% of children with ALL whose disease relapses or is resistant to treatment, options are limited. Approximately 95% of childhood cancer survivors develop one or more chronic health conditions by the age of 45 as a result of their treatment. And far too many children don’t survive cancer. Acute myeloid leukemia (AML) is particularly deadly.
Innovation is lagging behind for our youngest, most vulnerable patients. Only four cancer treatments have been approved for first use in children in the last 40 years, compared to hundreds approved for treating adults during this same time frame.
For too long, children have been treated with a one-size-fits-all approach. But as we learn that the biology of cancer is different in children than it is in adults — driven by different mechanisms and with different mutations — there is a pressing need for therapies designed just for children. It is time to change the standard of care for pediatric cancer patients. We need to rely less on chemotherapies that damage healthy cells, which can leave survivors with lifelong health challenges, and advance safer, more effective treatments based on precision medicine that target cancer precisely without harming the rest of the body.
As a physician, I’ve had to deliver the awful news to families that their child has cancer. As a parent, I can’t imagine hearing anything worse. As a researcher, I’ve banged on the doors of government, pharmaceutical companies, and industry leaders, but they remained shut because there wasn’t enough incentive to develop pediatric cancer drugs. And as chief medical officer of The Leukemia & Lymphoma Society (LLS), I hear every day from patients and families who have been devastated physically, emotionally, and financially by the far-reaching impact of a cancer diagnosis.
A few bright spots have already appeared. The FDA’s approval in 2017 of tisagenlecleucel (Kymriah), an immune-boosting, chimeric antigen receptor T-cell (CAR-T) therapy for pediatric ALL, was a historic victory. Work continues to advance CAR-T therapies so they’re safer and more effective for all cancer patients and less costly to produce. Looking ahead for both pediatric and adult patients, it will be increasingly important to predict who might reap the greatest therapeutic benefit from this game-changing approach.
I believe that precision medicine is the path forward for progress in treating pediatric cancer. But it will take a full-scale, global effort to deploy it.
We need more research dedicated to pediatric cancer to accelerate the accumulation of knowledge of the molecular underpinnings of this group of diseases, and we need new and efficient models of clinical trials that will put the unique needs of children with cancer at the forefront of trial design.
LLS has undertaken an unprecedented collaboration to launch the first global precision medicine clinical trial for pediatric acute leukemia, generally referred to as LLS PedAL. Through this multi-arm, multi-agent, and multi-pharma study, LLS is leading the effort to break down data and clinical silos in both the academic and pharmaceutical settings, allowing for widespread understanding of clinical trial outcomes and, ultimately, more precise treatment for children who deserve therapies designed for their needs.
This is just one part of The LLS Children’s Initiative, a new multi-year endeavor to take on children’s cancer through every facet of our mission, including research, education, patient support, policy and advocacy.
At the same time, clinicians and researchers must do more to help patients and families across the arc of their cancer experiences, from providing help to patients and families coping with cancer diagnoses to breaking down barriers to care and living with the aftermath of cancer.
Thanks to the bold and relentless efforts of previous generations, the outlook for children with cancer has brightened. But we must not stop there. Children deserve to benefit from precision medicine and the new, better-targeted therapies and scientific advances it is generating for adults with cancer.
Children should not only survive cancer, but thrive in their lives after it.
Gwen Nichols, M.D., is the chief medical officer of The Leukemia & Lymphoma Society and the former oncology site head of the Roche Translational Clinical Research Center and director of the Hematologic Malignancies Program at Columbia University.