Advances in understanding cancer on a molecular level and the identification of subgroups of cancer patients with rare diseases are expected to have an effect on drug development and supply.

“The vision of what cancer care will be like in the future is this very precise personalized care, where the patient’s tumor genome and germline genome will tell the doctor the specific agents that the patient will need. They will need two or three targeted agents from among several possibilities, and the oncologist will choose these based on how patients metabolize drugs, and what drugs are likely to cause them specific toxicities,” ASCO President Michael P. Link, MD, explained. “When you think this through, it becomes very clear that each therapy will be individualized. In fact, it will be so individualized, the question will become, is there sufficient incentive for industry to develop drugs that are only going to be used in a limited number of patients?” he noted.

Lessons from Pediatric Oncology

“What was learned in pediatric oncology is that we are treating patients with relatively rare tumors,” Dr. Link said, and therefore very few drug companies have been manufacturing drugs specifically for pediatric cancers. “There usually needs to be some sort of signal that the drugs are going to be useful in an adult cancer first,” he said.

“A good example is crizotinib [Xalkori],” he said. “The target of that drug was cloned from a child with non-Hodgkin lymphoma a relatively long time ago. We hadn’t seen much activity in developing drugs for that target, because it is a relatively rare tumor. It turned out that it is also present in a couple of other rare tumors. Once it became evident that the ALK tyrosine kinase receptor was a target in a subset of lung cancer,” he added, “there was an incentive to develop a drug for that.” (Crizotinib was approved by the FDA for the treatment of patients with locally advanced or metastatic non–small cell lung cancer that is ALK-positive as detected by an FDA-approved test.)

“As a sideline, crizotinib actually also works for that rare lymphoma, and it works with a couple of the other rare tumors where ALK is an important driver,” Dr. Link added. “But it is not clear that anybody would have developed the drug just for the rare cancers that could be targeted.” ■