Learn in five days, what it takes to launch clinical trials

Holbrook Kohrt, MD, used his experience in the clinical research training course to launch three trials.

If Holbrook Kohrt seems impatient with how long it takes to get new medical therapies from bench top to bedside, it helps to know that it’s personal.

Because Kohrt, a medical fellow and a hemophiliac, knows that without pioneering translational research on synthetic blood clotting therapies in the ’80s, he probably wouldn’t be working on a PhD, running several clinical trials and mentoring the next generation of medical researchers. “In the year I was born, 90 percent of severe hemophiliacs ended up contracting HIV through unpurified blood products,” said Kohrt. “But because of innovative clinical trials in recombinant protein technology, I was able to grow up receiving sterile clotting factor VIII without risk of infection.”

Today Kohrt helps his colleagues jump-start their medical innovations through the Intensive Course in Clinical Research, run by Spectrum, Stanford’s Center for Clinical and Translational Education and Research. Senior residents, fellows and junior faculty selected for this elite training program take a five-day, four-night “vacation” from pagers and clinical duties to focus on turning a clinical trial concept into a formal research proposal that can be launched into regulatory and funding pipelines.

When Kohrt took this class three years ago, he had no idea how challenging it was to get a clinical trial off the ground. “ICCR taught me how to design cost-effective clinical trials that yield better patient outcomes. I learned how to use statistical analysis to enroll the fewest number of patients to prove my hypothesis. And to understand the principle of ethical equipoise to communicate the risks and benefits of each trial,” he said. “It was phenomenal to have a team of physicians, patient advocates, biostatisticians and trial designers, all focused on your proposal for a week.”

Using these lessons, Kohrt has launched three trials, including a phase-1 study, “Peptide immunization of matched related donors and recipients with acute myeloid leukemia,” with a $50,000 grant from the American Society of Clinical Oncology.

In this study Kohrt is aiming to reduce the relapse rate of AML patients by combining bone marrow transplantation with immunotherapy. His strategy is to vaccinate a healthy marrow donor, a few weeks before the transplantation process, with a string of nine amino acids that mimic the surface of an AML tumor. This tricks the donor’s immune system into creating antibodies against this type of tumor, with no apparent risk to the donor. When the donor’s marrow is transplanted, the AML patient benefits from an infusion of tumor-targeting immune cells.

In mouse studies using this approach, Kohrt has found that AML survival rates went from 0 to 80 percent. Using these statistical results, along with what he’s learned from the ICCR course, he obtained funding and begin a phase-1 clinical trial.

Kohrt believes that ICCR makes it feasible to open a clinic trial within a typical fellowship timeframe, and that the thoughtful structuring of ICCR proposals makes it easier to get follow-on trials approved.

On a personal level, it’s not surprising that Kohrt has made it his mission to accelerate translational medicine at Stanford, a leader in genetic research. “As a hemophiliac, people ask my wife and I if we’re thinking of having kids. After all, I will pass my X-chromosome mutation to my daughters, who will have a 50 percent chance of passing it to their sons. But I’m hoping that through advances in gene therapy, we’ll find new ways to treat my grandchildren. This is an important reason why I do what I do every day.” 

Kris Newby is a writer for Spectrum, Stanford’s Center for Clinical and Translational Education Research.