A new initiative at the University of Rochester Medical Center (URMC) will provide academic and industry researchers the expertise and scientific collaboration necessary to conduct early stage clinical studies. The Center for Human Experimental Therapeutics (CHET) represents the first university-based program focused on accelerating the development of novel medical therapies.
“This Center consists of both infrastructure and a group of people with decades of combined experience in conducting experimental human therapeutics and running some of the more complex clinical studies in the world,” said URMC neurologist Karl Kieburtz, M.D., director of CHET. “It is a unique approach that represents a marriage of scientific expertise and logistical competence, with a focus on the early stage of drug development.”
The Center is designed to address a critical juncture in the process of developing new treatments, one that is probably the most significant and persistent barrier to progress in medical research: the initial translation of novel interventions from preclinical evaluation – research performed in the lab – into the first human clinical trials. This phase is often referred to as T1, meaning it represents the first stage of translating biomedical discoveries into new human treatments.
This phase of research is one that has become a high hurdle for scientists to overcome. The process of conducting initial human investigations has not traditionally been a focus of academic institutions. It is also a function that is being outsourced by larger pharmaceutical and medical device companies that are increasingly dedicating their resources to later stage clinical studies as opposed to newer approaches that may have a higher risk of failure.
Consequently, many of the truly novel approaches to treat disease being developed in a university setting or by smaller biotechnology companies often fail to advance to the clinical stage. One of the functions of CHET will be to assemble under one roof the resources needed by academic and industry scientists that are necessary to answer fundamental questions about their early stage research. These include whether or not the intervention is safe, tolerable, feasible, and targets the intended mechanism.
“These questions are the same if you are talking about kidney, heart, lung, or neurologic diseases,” said Kieburtz. “There are a broader set of questions that apply to the application of any novel interventions to human disease. Does the compound go where it is supposed to go, does it do what is supposed to do, and can we measure the relationship between the dose and the intended effect?”
CHET brings together a unique set of resources at the Medical Center designed to assist in translational research. The Clinical Trials Coordination Center (CTCC) and the Clinical Materials Services Unit (CMSU) will now operate under the direction of the new Center.
The CTCC was created in 1986 and over the years has grown to support a full array of services essential to the conduct of clinical trials for industry, foundations, and government sponsors. These services include: study protocol development; data collection, management and analysis; administrative support; and oversight of study sites. The CTCC has managed over 70 multi-center, international clinical trials and is used by several consortia of investigators in neurological disorders. Collectively, these groups are comprised of more than 350 academic investigators from the United States, Canada, Europe and Australia.
CMSU offers a comprehensive clinical trial supply services including package and label design and production, assembly of patient kits, national and international distribution, secure storage with robust environmental controls, and the tracking and destruction of returned products.
CHET will also coordinate closely with the activity of the URMC Clinical and Translational Science Institute.
“CHET serves as a critical link between our basic science and patient-oriented research enterprises,” said Thomas Pearson, M.D., M.P.H., Ph.D., director of the Clinical and Translational Science Institute. “It is an essential component in our efforts to re-engineer medical research here in Rochester and beyond by accelerating the process of translating scientific discoveries into new ways to understand, prevent, and treat diseases.”
CHET will initially consist of existing URMC faculty and staff who are currently part of the CTCC and CMSU. The Center will also recruit 6 new faculty and staff including biostatisticians and clinical pharmacologists. The Center and its staff will reside in the new Clinical and Translational Sciences Building which is scheduled to open in March of 2011.
More information on the Center can be found online at www.urmc.rochester.edu/chet.