Wow! How? Cancer Treatment Innovation

This post is the latest in my series of “Wow! How? Health” stories, also shared on LinkedIn if you want to follow the conversation on that platform. 

When Michael Katz was diagnosed with multiple myeloma, he was fortunate to connect with someone who had recently completed treatment, the “just-in-time someone-like-me” that could guide him. He vowed to do the same for others.

He joined two patient-led organizations, the International Myeloma Foundation and the Association of Cancer Online Resources (ACOR), listening, learning, and making connections among patients who were both seeking and sharing advice based on their lived experience. Patients in both groups were grateful for the existing treatments, but they wanted more. Nobody with real power was focused on the problems they wanted to solve.

Over the course of a decade, Katz rose to become the chair of the Patient Representative Committee for the Eastern Cooperative Oncology Group (ECOG), which conducts multicenter clinical trials for cancer research. He sat at the table with clinicians and scientists as an equal, advocating for the investigation of questions that patients wanted answered.

In 2002, Vincent Rajkumar, MD, chair of ECOG’s myeloma committee and an expert on blood cancer at the Mayo Clinic, was excited about the prospect of testing new drugs for multiple myeloma. Katz pushed back. That is not what patients want, Katz said, adding that “all these new drugs don’t help if patients cannot take them.” Too many people were unable to survive the established first-line treatment, dexamethasone, which was being given at a high dose by default because nobody had ever tested its effectiveness at lower doses.

The rest of the committee was skeptical. Why test the dosing of a drug that worked to kill cancer cells and was widely accepted by clinicians? “To us the idea seemed destined to fail,” Rajkumar recalls. “It seemed so boring. We had waited 40 years for new drugs and Mike wants us to test dex dosing! However, we respected Mike. We knew he was aware of what patients were going through. We saw 100–200 myeloma patients a year. He interacted with thousands. He was also leading meetings of support group leaders who were leading meetings with lots of other myeloma patients.” The clinicians convinced their institutional sponsors to try Katz’s idea.

Patients were so eager to participate in the clinical trial for lower-dose dexamethasone that all the spots were filled in record time—faster than any other national, cooperative multiple myeloma trial. And, as it turns out, patients were right to push for the change. At the one-year mark, 96% of participants on the low-dose regimen were alive, compared with 87% of those on the high-dose, standard-of-care treatment. Serious side effects, like blood clots, were also reduced among those on low-dose dexamethasone, which has become the new standard. And, just as Katz had predicted, using a lower dose of dex has allowed oncologists to build new drug combinations.

When the randomized trial of high-dose versus low-dose dexamethasone was published in The Lancet Oncology, it became one of the most cited myeloma papers ever.

In the lexicon of my book, Rebel Health, Katz was a Seeker–Networker and Rajkumar is a Champion. They bridged the gap between what patients need and what scientists and clinicians are able to deliver. They showed that it is a competitive advantage for clinicians, researchers, and funders to work with patients.

Image: Test tubes by TNS Sofres on Flickr.