Rewiring Pediatric Oncology: Why Better Connections Are Needed to Turn Discoveries Into Medicines

The path from a promising pediatric cancer discovery to an approved medicine is not broken because of a lack of scientific talent, clinical expertise, or resources. According to David Adler, MD, PhD, MBA, a senior oncology drug development leader, the field's core problem is one of connectivity: the exceptional components of pediatric oncology are "not always connected at the points where they could have the greatest influence on whether a promising program becomes a medicine."

Adler, who spent a decade leading oncology programs from preclinical research into the clinic within Bayer AG查看公司主页's Global Oncology Clinical Development organization, argues that the development process must be "rewired" so that development expertise, evidence, and decision-making arrive early enough to shape a program's trajectory.

Missed Connections Zap Energy From Research

Adler uses an analogy to frame the challenge: an electrician walks into a house equipped with modern appliances that are not working because the wiring does not allow everything to function together efficiently. "I think there is something similar happening in pediatric oncology," he writes.

The field, he notes, has "exceptional scientific expertise, experienced clinicians, specialized cancer centers, research networks, pharmaceutical capabilities, regulatory mechanisms, and philanthropic support." Yet a promising discovery can spend years in academic research before development expertise becomes involved. By that point, important decisions may already have been made about the evidence, models, biomarkers, clinical strategy, or other requirements for moving the program forward.

Drawing on his oncology development experience, Adler identifies a question that should be asked far earlier in the process: "What do we need to know to make the next development decision?" This question, he emphasizes, is distinct from simply asking whether a program is scientifically promising. A promising pediatric oncology program may still carry important uncertainties around its biology, models, pharmacology, biomarkers, safety, clinical population, manufacturing, or regulatory strategy. "The earlier those uncertainties are identified, the greater the opportunity to address them while there is still time to influence the direction of the program."

Four Things That Must Change Now

Adler outlines four concrete changes he believes are necessary to rewire the discovery-to-medicine pathway.

First, development-readiness needs to enter the process earlier. Academic institutions and pediatric oncology research networks should have structured opportunities to assess programs during discovery and preclinical development, not only when a program is ready for licensing. The purpose, he clarifies, "should not be to predict commercial success; it should be to identify what the science has established, what remains uncertain, and which uncertainties matter most for the next development decision."

Second, academic and industry expertise should be connected before there is a transaction. Pharmaceutical and biotechnology development teams often become involved only when an academic program is approaching a partnering or licensing discussion, by which time years of research may have shaped the program. Earlier interaction could bring translational pharmacology, biomarkers, toxicology, clinical development, manufacturing, and regulatory perspectives into the discussion while there is still time to act on them. Adler is careful to note that "this does not mean that academic research should become an extension of industry. It means that development expertise should be available early enough to be useful."

Third, funding should address the uncertainties that are actually blocking development. Some pediatric oncology programs sit between academic research funding and commercial investment. Rather than asking only whether more research should be funded, funders could ask which unanswered question is preventing the next development decision — whether that is model validation, pharmacology, biomarker development, safety, manufacturing feasibility, or another translational question. "The goal should not simply be to generate more data. It should be to generate evidence that can materially change what happens next."

Fourth, the connections need to remain in place once a program enters clinical development. Clinical development is not simply the final stage of the process; it generates new information that can change the therapeutic hypothesis. Safety findings can alter a development plan, biomarker results can change patient selection, and clinical observations can challenge assumptions made in preclinical research. "Those lessons should flow back into translational research, development strategy, regulatory thinking, and future pediatric oncology programs."

The Right Connections Matter

Adler stresses that pediatric oncology does not need to become adult oncology, nor does it need to adopt an industry operating model. "The biology, patient populations, clinical settings, and development challenges are different." But the field can benefit from thinking more deliberately about how its existing capabilities connect.

After a decade leading oncology programs through clinical development, Adler has concluded that "the challenge is not simply to generate more promising science or persuade companies to develop more pediatric cancer medicines. It is to create a development pathway in which promising pediatric oncology research encounters the right expertise, evidence, resources, and decision-making early enough to make a difference."

"The components are already there," he writes. "The task is to rewire the connections between them."

Adler currently serves as Chief Scientific & Medical Officer of the PATHORA Institute of Pathology & Tissue Medicine (搜索)搜索该词条 and holds academic appointments at the Hebrew University of Jerusalem, Ben-Gurion University of the Negev, and the University of Bonn.