Cancer, leukemia, and other malignancies are very rare in children. They came into the focus of medicine and research only when the general living conditions began improving, and research turned to new fields that had previously gone unnoticed in the flood of deadly childhood diseases. Flawed assumptions had to be overcome. Such as that cancer and leukemia only exist in adults, or that you can’t do anything anyway. First breakthroughs came with the treatment of childhood leukemia with chemotherapy.
New combinations (“cocktails”) of chemotherapy agents were tested and continuously improved survival rates. Today, we associate therapeutic breakthrough with new drugs. Childhood cancer was a historical exception. Effective drugs existed already, used and approved in adults. Researchers did not expect that minors with cancer would fare better, but they did. Minors have greater reserves, given the chance to prove this. Towards 2000, the fine-tuning of chemotherapy cocktails plus additional support such as the acceleration of red blood cell production by state-of-the-art drugs reached a plateau. Childhood cancer started to leave its habitat of a historical exception. People knew that new technology would be required. How?
Three movements. (1) New, targeted anti-cancer agents showed serious improvements in adults. (2) Childhood cancer became associated with “pediatric drug development”, based on the children-are-not-small-adults mantra that everything is different in “children”. (3) Breakthroughs occurred. Not because of, but despite “pediatric drug development”.
Childhood cancer researchers demanded easier access to new anticancer compounds for adults. US and European Union (EU) pediatric laws demand “pediatric” studies for all new drugs, including anticancer drugs, in minors. The catch: the 18th birthday is an administrative point in time. Bodily, nothing changes.
Chemotherapy is rather primitive. It poisons systematically the whole body in the hope to kill enough malignant cells and the immune system will clean up the rest.
The next breakthrough came in chemotherapy-resistant acute lymphoblastic leukemia (ALL), the most common childhood malignancy, by engaging the patient’s immune system. Blood is drawn, white blood cells are trained to kill ALL cells, the blood is re-infused. It’s also called CAR-T cell therapy. Of course, it’s much more complicated. For example, the immune system often overreacts (as we also know from COVID-19 infections). A cytokine storm causes high, life-threatening fever. The fever can be treated with high-tech drugs and biologics. Emily Whitehead was the first child who survived. Today she is a young, healthy woman. Youtube her name and learn more.
The key assumption behind the pointless “pediatric” cancer studies is the desire that what worked half a century ago with chemotherapy should also work with modern targeted drugs: Giving them to minors will help. But this assumption is wrong. Chemotherapeutic agents attack almost all cells. Targeted therapies are different. The only ones that profit from these
pointless “pediatric” cancer studies are researchers and scientists in academic medicine, regulatory authorities, and life science industry.
The FDA has relented. Today, it recommends inclusion of adolescents into adult anticancer studies, which is reasonable. But the US followed the EU by authorizing the FDA to demand studies with new anticancer drugs in children up to 11 years. The EU did so since 2007, without any breakthrough.
Childhood cancer research has taken two avenues. One saved Emily’s life by serious research. The other one runs pointless studies. Companies must pay them, or they don’t get adult approval. This second avenue is stronger in the EU. But today studies recruit worldwide, including the US. Would Emily have been recruited into such a pointless EU-demanded study, she would probably be dead.