One of the few bright spots in the Covid-19 pandemic has been the swift development of therapies and vaccines that — beyond all odds — made it to patients in mere months versus years. The government, specifically the Food and Drug Administration, has played an essential role, flexing to expedite activities and prioritizing speed without sacrificing safety. I applaud their efforts and hope they won’t stop there.
As a mother who watched her 9-year-old son, Zach, die from aggressive leukemia following four years of harrowing therapy and three bone marrow transplants, I urge the government and the biopharma industry to apply a similar spirit of innovation and resolve in tackling another critical challenge: childhood cancer.
Despite great progress, cancer remains the No. 1 disease-related cause of death in kids, even amid Covid-19. Survivors can face debilitating treatment-related side effects, secondary cancers, and early death.
Take Brennan Simkins as an example. Diagnosed with acute myeloid leukemia (AML) at age 7, Brennan, now age 18, endured four grueling bone marrow transplants. Though he survived, he now struggles with cognitive challenges, seizures, cataracts, stunted growth, fatigue, lung and thyroid issues, emerging diabetes, and more — all late effects of treatments that haven’t changed much in 30 years. Brennan now pivots his gritty and herculean efforts from surviving cancer to independent living, an exhausting existence but one he and his family are grateful to have.
Many other pediatric cancer survivors share similar challenges.
These children often rely on decades-old, highly toxic therapies designed primarily for, and tested in, adults. And despite pioneering — and even inspiring — advances like chemotherapy, bone marrow transplants, and cellular therapies, kids today are pushed to the back of the line to try new treatments, waiting an average of 6.5 years longer than adults to try them (and often get access only to those that have been proven effective in older patients). Among these promising therapies are targeted agents like small-molecule inhibitors, monoclonal antibodies, and cellular therapies (including CAR-T treatments) that could improve cure rates with reduced toxicities.
Despite their historical contributions and willingness to be trailblazers, kids fighting cancer are neither prioritized nor fully considered in drug development and testing. That needs to change.
Look what can be accomplished when the FDA, the National Institutes of Health, biopharma companies, and others prioritize and partner to solve a problem. Watching the lightning-speed approvals of Covid-19 vaccines and therapies I can’t help but ask, “Can’t we pick up the pace and do better for children with cancer?” Many parents like me see childhood cancer as our pandemic.
As a good start, the FDA needs to adopt the flexibility, creativity, and urgency it applied to Covid-19 to cancer drug development, especially precision therapies for childhood cancers. These treatments offer great hope and have already proven to be a total game changer in the most common form of childhood leukemia.
Maximizing and accelerating the benefits of precision medicine in childhood cancers (and other diseases with smaller patient populations) can’t be accomplished by clinging to traditional models for drug development, rigid clinical trial constructs, and outdated ways of doing business. I’m encouraged that the FDA has already begun embracing this notion. It has, for example, clarified how researchers can leverage master protocols — streamlined, multiarm umbrella trials — to accelerate drug development, especially for rare diseases. But there’s more to do.
Kids with cancer — especially those with rare, poor-prognosis subtypes — need the FDA to keep following the science and exploring better ways of considering the unique circumstances of children with cancer. Just as one-size-fits-all therapies aren’t sufficient for all cancer patients, drug development guidance and approaches written primarily with adults in mind fall short of the needs of children.
The FDA proposed new guidance this summer for researchers developing treatments for AML. This aggressive blood cancer, with a notoriously poor prognosis, is the most common kind of leukemia in adults. The FDA’s guidance offers an invaluable look at the “scorecard” the agency intends to use when deciding whether to approve a new treatment for AML. When drug makers know what to expect from the FDA, they can bring new treatments to patients more quickly.
But acute myeloid leukemia also occurs in children like Zach and Brennan, and continues to be a pediatric cancer with a very poor prognosis. Current AML drug development guidance briefly mentions children but doesn’t take into account kids’ unique disease biology, which differs significantly from adults, as well as their overall resilience, treatment path, and response to therapy. Unlike adults, kids can and do endure multiple rounds of “maximum-a-human-can-tolerate” chemotherapy and repeated transplants. They want to live and are often willing to sacrifice and take significant risks to do so.
They need FDA guidance that accounts for childhood biology, patient resilience, and the smaller numbers of kids eligible to enroll in clinical trials.
Pediatric oncologists are acutely aware of these dynamics, but it’s a struggle to advance new therapies within current frameworks. The FDA needs to work with these clinicians, parent advocates, and the broader cancer community to create pediatric-specific guidance that matches the realities of kids’ treatment protocols and paths, which differ significantly from adults.
As the FDA and others take stock of the lessons learned during the Covid-19 pandemic, we should consider elevating the needs of children and embracing approaches that are faster, more innovative, and more flexible for kids — our unsung cancer pioneers.
Zach and our family would have welcomed more options and accepted the accompanying risks. Many kids with cancer and their families would do the same. Because childhood cancer is our recurring nightmare — our pandemic — we pray for the day it’s treated like one.
Julie Guillot is a former IT/business operations executive who now works as a full-time volunteer/advocate with the Children’s Oncology Group, Leukemia & Lymphoma Society, and other organizations to accelerate less toxic, more curative treatments for pediatric cancer. She currently serves as the national volunteer chair of Leukemia & Lymphoma Society’s Children’s Initiative and PedAL Master Trial — a large-scale, strategic effort to speed precision medicine for kids fighting high-risk leukemias.