In the Spring of 2011, HSCI initiated the Translational Research Workshop series in order to stimulate and facilitate the development of projects that take the understanding of the basic mechanisms of stem cell biology and translate them into effective treatments for diseases.
While the goal of basic stem cell research is to understand the fundamental molecular and biological mechanisms of stem cells and their role in development and disease, translational research explores ways in which these molecular features can be exploited as targets for treatment. Some of the hurdles researchers must face in taking research from its basic to translational phase is that each phase involves different scientific focus, different types of funding mechanisms, different specialized equipment and facilities, and different expertise in experimental design and execution.
The first Translational Research Workshop was aimed at providing an overview of the main differences between basic and translational research and the expertise and facilities available to HSCI investigators to help them navigate the transition. Faculty members also shared their personal experiences of working in a translational setting, and described both the obstacles that they faced and how those obstacles were overcome.
This past December, the second workshop focused on the many experimental and logistical considerations that a researcher must take into account when conducting early stage translational work. For example, Jon Rowley, PhD, from Lonza, a company that specializes in processing cells for therapeutic purposes, explained that the type of media in which scientists choose to grow their cells could have a significant impact on the cost of mass-producing the cells for a clinical setting — a key issue for market acceptance that a basic scientist typically never thinks about.
HSCI Principal Faculty member Michael Young, PhD, also shared his experiences partnering with biotech companies, and discussed how researchers could keep such collaborations operating smoothly to help move the research along as efficiently and effectively as possible.
At the most recent workshop, guest speakers addressed the regulatory considerations that must be addressed when conducting translational research.
HSCI Principal Faculty member David Williams, MD, discussed how one of his own discoveries was brought from bench to bedside over the course of 10 years, and shared his experience working to gain approval to conduct clinical trials.
Patrick Au, PhD, from the Division of Clinical Evaluation and Pharmacology/Toxicology at the US Food and Drug Administration, spoke about how the FDA can serve as a resource for researchers and how the process of gaining approval can proceed more smoothly if researchers make early and frequent contact. Matthew Wladkowski, MS, the Regulatory Affairs Coordinator at the Translational Research Program at Children’s Hospital Boston, reiterated this point, saying while some researchers hesitate to contact the FDA, it is actually in a scientist’s best interest to address potential stumbling blocks as early as possible.
Building on the success of these events, the Translational Research Workshop series will continue over the next year to examine the barriers and bridges in taking stem cell science from bench to bedside and share this valuable expertise with the HSCI community.