The 2026 Childhood Cancer Report
From diagnosis to research: measuring momentum in the search for cures
Drug Development and Emerging Technologies
Researchers are continuing to discover new cancer proteins, which are molecules in the cells that drive the growth of cancer cells. These proteins represent potential targets for drugs. There are thousands of these proteins,221 with the most infamous being TP53, a mutation in the p53 suppressor gene. This mutation is responsible for the development of many human cancers and is the main driver of Li-Fraumeni syndrome.222 But the challenge remains: How can these cancer proteins be targeted so that more children are cured?
One way of increasing treatment options for patients is by expanding technologies that can “drug the undruggable,” which consists of finding new ways to change the behavior of molecular targets that have a known link to driving cancers but are difficult to target with existing technologies. New technologies, like molecular degraders, can attach to these targets and break them down directly. This approach opens the door to targeting things that researchers couldn’t before, including certain “master control” proteins in cells called transcription factors.223 Early research shows promise, and most ongoing studies are looking at blood cancers, like lymphoma, and breast cancer in adults. In the future, these treatments may also be adapted for children with cancer.
Future directions will be potentially expanding existing molecular degraders into pediatric cancer indications and developing molecular degraders that can specifically target pediatric cancer driving factors.
Additional New Classes of Drugs
Antibody-drug conjugates (ADCs)
These are an additional class of drugs that can expand treatment options for pediatric cancer patients. There are three approved ADCs for the treatment of pediatric leukemia, and further efforts are ongoing to test ADCs outside of leukemia as potential treatment options. There has been progress made in developing ADCs to treat neuroblastoma and sarcomas, with the goal of launching clinical trials soon.224,225
CAR T-cell therapy
Although only one CAR T-cell therapy has been approved for use in pediatric cancer patients (see Section 3), significant progress has been made in advancing CAR T-cell therapies in both blood cancers and solid tumors. There have been many clinical trials focused on CAR T-cell therapy in pediatric and young adult cancer patients, with most focusing on leukemia; however, significant progress has been made in solid tumors as well, with clinical trials ongoing for brain tumors, sarcomas, and neuroblastoma.226 Future CAR T-cell therapy trials can greatly expand treatment options in pediatric cancer patients going forward.
Other immunotherapy
In addition to CAR T-cell therapies, ongoing clinical trials are exploring additional immune cell populations that can potentially be used to target cancer cells. Two such examples are Natural Killer (NK) cells and macrophages. CAR-NK and CAR-Macrophage studies are ongoing to determine if either immune cell population may be beneficial in slowing down the growth of pediatric cancers. While early, these studies are showing promise, both preclinically and in early clinical trials.227
Precision medicine
Technologies such as next-generation sequencing give clinicians the ability to identify mutations driving tumor formation and the possibility of creating highly individualized treatment plans, especially if there is a current approval therapy for the mutation. This type of approach is called precision medicine and is rapidly evolving as more sequencing technologies and anticancer therapies are made available to patients.228 Precision medicine approaches are especially valuable to patients that have very rare tumors without a standard treatment approach and to patients with relapsed or refractory cancers.229