In the last 40 years, just six cancer drugs have been specifically designed and tested for children. And only 5% of all oncology drugs have been approved for first use in pediatric cancer.
The Leukemia and Lymphoma Society (LLS) wants to change that.
LLS is leading the way to new pediatric blood cancer treatments through the Pediatric Acute Leukemia (PedAL) Master Trial, an unprecedented international collaboration led by LLS, designed to transform treatment and care for pediatric acute leukemia. The research will focus on two of the most common childhood blood cancers: acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). The goal of LLS
PedAL is to expedite development of new targeted treatments for hard-to-treat childhood leukemias and replace one-size-fits-all chemotherapy with treatments tailored to each child’s unique tumor biology.
The Children’s Initiative, to be renamed the Dare to Dream Project this summer, will expand access to trial medications. PedAL trials will be available to children and families worldwide, bringing the dream of safer, more effective pediatric leukemia treatments closer to home for more people.
MEDICATION MADE FOR CHILDREN
Children diagnosed with blood cancer not only have to endure a heartbreaking diagnosis, but they are also often treated with repurposed drugs (tested only on adults) which can cause lifelong side effects.
“Chemotherapy, in general, is made to attack the whole body and everything in it that’s fast-growing,” explains Dawn Berryman, the LLS Ohio River Valley Region Campaign
Development Director. The movement now is toward developing more targeted treatments that attack the cancer without damaging the entire body.
Dawn has witnessed the long-lasting negative effects of chemotherapy firsthand. At just three years old, her daughter was diagnosed with ALL. While the dosage of chemotherapy she received was effective in bringing her cancer into remission, the treatment resulted in numerous chronic health conditions.
Children undergo significant brain development from the ages of three to five, Dawn explains. Chemotherapy’s toxicity keeps cancer cells out of the brain and other parts of the body, but it can negatively impact children physically, cognitively and emotionally — at any age.
This is one of many reasons why clinical trials to find targeted treatments for pediatric cancer are so important. But the smaller population of pediatric cancer patients — compared to adults — has limited the testing and approval of treatments specifically for children.
THE FUTURE OF CANCER CARE
Another obstacle in the development of targeted pediatric leukemia treatments? Many parents continue to opt for the better known, albeit toxic, chemotherapy treatments rather than try medications that are being developed and tested.
Amy and Greg Kappen, however, view trial medications as the future of cancer treatment.
When their daughter, Sophia, was diagnosed with ALL, they were told that it was “the best cancer to get,” referring to the standard risk level and a high cure rate of greater than 92%.
Sophia achieved remission after one month of treatment.
But when Sophia’s cancer returned a few short months later, it proved resistant to chemotherapy. “We didn’t know if it was the cancer or the drugs wreaking more havoc in her little body,” Amy says in reference to her daughter’s severe pain levels.
Amy recalls how she and Greg decided to pursue what they called a “six-week Hail Mary” — a clinical trial drug to make a targeted attack on Sophia’s cancer. “Normal procedure had failed. We were willing to do whatever it took to stop this beast of a cancer.”
Sophia was quickly transferred to Children’s Hospital of Philadelphia for treatment, but her cancer had mutated and spread. Additional trial treatments gave the family another four to six months with their daughter, but Sophia died on April 5, 2017.
Greg says that even though the experimental treatments didn’t save Sophia, he and Amy still speak up and advocate for trial medications like those LLS is funding through the Dare to Dream Project. “If available sooner, that treatment can save kids like Sophia,” says Amy. “What we do today could save someone in the future.”
The Dare to Dream Project is only one aspect of the LLS mission. The Leukemia and Lymphoma Society also offers education and support to pediatric patients and their families, such as financial assistance to ease the financial burden of cancer care and one-on-one consultations with pediatric oncology specialists. In addition, LLS funds groundbreaking research and drives advocacy efforts to help all kids with blood cancers get affordable, quality health care.
Dawn’s hope is to increase awareness of all that LLS offers.
“The worst thing we can hear when meeting someone new is that they wish they had known about LLS while they or a loved one were going through treatment,” Dawn says. “We can offer so much support and hope to help people who need it.”
To learn more about the Leukemia and Lymphoma Society, Ohio River Valley Region, and the various ways you can support its mission, visit lls.org/ohio or email dawn.berryman@lls.org.