Translating Discoveries into Treatment, Case by Case

Could an extract from the prickly pear cactus be the beginning of a new drug for type 2 diabetes? What steps would you take to find out, to take that insight from the laboratory bench and give that therapy to people for the first time?

For two weeks in July, 54 investigators from across the Harvard community were immersed in thinking through these issues in Intensive Training in Translational Medicine (ITTM), a new course offered by Harvard Catalyst | The Harvard Clinical and Translational Science Center.

Through lectures and case-based exercises, the course provided a comprehensive overview of the skills and principles of T1 clinical and translational research, the process of taking an idea or discovery and translating it into a first-in-human or proof-of-concept trial.

“The ITTM course grew out of a challenge from the NIH to change the way the academic biomedical community trains clinical and translational investigators,” said course director Tony Hollenberg, an HMS associate professor of medicine at Beth Israel Deaconess Medical Center. “To the best of our knowledge, this is the first educational offering in the nation focused solely on T1 translational medicine.”

The didactic portion of the course, taught by faculty drawn from several Harvard Catalyst–affiliated institutions and the private and government sectors, covered topics inherent to the T1 process, ranging from pharmacology to biomarker development, study design to regulatory processes, and academic–industrial relationships to the ethics of human subjects research. “We invited people from federal agencies, venture capital firms, and biotechnology and pharmaceutical companies who, to a person, said ‘If you have a project and you need some advice, call me or e-mail me, I’d be happy to give you some insight about my area of expertise,’” said Mason Freeman, one of the course co-directors and an HMS professor of medicine at Massachusetts General Hospital.

In the course’s case portion, groups of students assigned to one of three tracks—drug, biologic, or device—were charged with turning background case information (written for the ITTM course by a team of experts from academia and industry) into full-fledged development programs covering the range of T1-related preclinical and early clinical research activities and issues.

“In weaving the lectures and cases together, we encouraged the students to apply what they learned in real-time and find that balance between design, feasibility, costs, and benefits” said Eva Guinan, another course co-director and an HMS associate professor of pediatrics at Dana-Farber Cancer Institute. “You could see as they made their case presentations how they had absorbed what they’d heard.

“One of the practical benefits of the cases is that they really prompted us to go into much more depth on each of the course’s topics than could realistically be covered in the lectures,” said Cameron Keating, a research fellow in plastic surgery at Massachusetts General Hospital, whose drug track group developed the case for the hypothetical prickly pear–derived diabetes drug. “We used our case to demonstrate that you could use clinical and epidemiological observations—such as studies of a Pima Indian family that did not develop type 2 diabetes at nearly the same rate as others in that population—in a reductionist way to drive the discovery of an effective, first-in-class small molecule drug that could fit well into the market and which could be synthesized and taken forward into clinical trials.”

The ITTM course complements Harvard Catalyst’s Introduction to Clinical Investigation course, a weeklong survey of the skills and concepts at the heart of clinical and translational research. The two courses form the backbone of Harvard Catalyst’s efforts to create a comprehensive educational and training pathway for clinical and translational investigators at Harvard.

“I’ve been planning my research fellowship for about two years, and when I read about the course, it seemed like the perfect introduction to what I want to do,” said Keating. “In the long term, I plan to have an academic focus leading a translational research team. The course helped me put everything about my fellowship into context and has given me a much more complete perspective of both the research environment and the practical challenges of translational research.”

“The course received rave reviews from the participants,” noted Frances Jensen, the course’s third co-director and an HMS professor of neurology at Children’s Hospital Boston. “And since this was the first time we’ve offered it, we are taking what we’ve learned as educators from this first cohort of participants to offer an even better experience to next year’s.”

For more information, investigators may contact Erica Lawlor at ittmcourse@catalyst.harvard.edu.