The University of Rochester Medical Center (URMC) has opened the doors on a new facility that will enable researchers to create, study, and ultimately use stem cells and their offspring in early-phase experimental human therapies. The Upstate Stem Cell cGMP Facility – which will be used by academic and private-sector scientists from across the state – was created with $3.5 million in support from the Empire State Stem Cell Board.
“One of the critical barriers to moving cell-based therapies into clinical trials is the requirement that these cells be manufactured in a facility that meets strict federal requirements,” said Steve Dewhurst, Ph.D., chair of the URMC Department of Microbiology and Immunology and principal investigator for the state grant. “Without this resource, much of this science remains stuck in the lab.”
The Upstate Stem Cell cGMP facility is located in the URMC’s DelMonte Neuromedicine Research Institute. cGMP stands for “current good manufacturing practice,” a term that means that the facility, its operation, and the people that work in it meet federal manufacturing guidelines necessary to ensure that biological materials produced there are suitable for human clinical trials. These guidelines require that the lab is a clean room facility with strict air quality standards and is designed, maintained, and monitored in a manner that prevents contamination. In addition, personnel must follow strictly defined manufacturing protocols and the cellular products must be subjected to rigorous testing that ensures their quality, safety and reproducibility.
In order to meet these requirements the Upstate Stem Cell cGMP Facility incorporates several design features including redundant air handling systems, walls covered with a fiberglass gel coat that aids in cleaning, and a building monitoring system that enables staff to remotely monitor air quality and room and equipment parameters 24 hours a day. The facility is designed to utilize unidirectional flow, meaning that researchers and materials enter the lab through an air lock and depart through a different exit to ensure that contamination from the outside is not carried into the facility or among different labs.
“Our scientists have made tremendous progress over the last several years unlocking the potential of stem cells to treat a long list of diseases,” said Bradford C. Berk, M.D., Ph.D., CEO of URMC and a member of the Empire State Stem Cell Board. “I anticipate that this new facility will accelerate research across that state and make Rochester a center for the development of new cell-based therapies.”
There are more than 40 labs at URMC that are working with stem cells. These labs employ more than 260 scientists and technicians and collectively have more the $80 million in total research funding.
“This facility represents the key bridge to early stage trials in humans,” said Mark Noble, Ph.D., director of the URMC Stem Cell and Regenerative Medicine Institute. “We are now poised for early stage clinical studies in a wide range of conditions, including efforts to repair damage to the central nervous system, re-grow bone and cartilage, and even selectively target and destroy the stem cells that are the source of some forms of cancer.”
The 3,600 square foot facility consists of three separate labs that can each support different cell production projects. Several pending projects will immediately begin using the facility, including:
- Noble, Chris Pröschel, Ph.D., and Margot Mayer-Pröschel, Ph.D. are investigating the use of glial progenitor cells, which are responsible for generating important support cells in the central nervous system, to repair spinal cord damage;
- Steven Goldman, M.D., Ph.D. and Maiken Nedergaard, M.D., D.M.Sc., Martha Windrem, Ph.D. and Su Wang, M.D., Ph.D. with the URMC Center for Translational Neuromedicine are working with researchers in Syracuse and Buffalo to use glial progenitor cells to treat patients with multiple sclerosis;
- URMC orthopedic research Edward Schwarz, Ph.D. director of the URMC Musculoskeletal Research Center, is developing a new method to grow mesenchymal stem cells, the cells responsible for healing bones and keeping them healthy. The intention is to use these cells to improve the performance of bone transplants. Schwarz is also working on a separate project that will produce antibodies for use against bone infections.
- Sally Temple, Ph.D. at the New York Neural Stem Cell Institute in Albany is developing a new approach to use retinal stem cells to restore vision in persons with age-related macular degeneration, the leading cause of blindness in Americans aged 60 and older.
“This facility is a true boon to stem cell researchers across New York State,” said State Health Commissioner Nirav R. Shah, M.D., M.P.H. “This state-of-the-art facility will help take the basic research findings of the state's outstanding scientists and help move them into the clinic, all the while ensuring that any cell products are produced in accordance with the strictest standards for use in human patients. Not only is the facility supported by the NYSTEM program, but it is a key component of several of the Consortia to Accelerate Therapeutic Applications of Stem Cells recently awarded by the Empire State Stem Cell Board. Together, the facility and the consortia will help bring therapies for devastating diseases to the people of New York State.”
The Upstate Stem Cell cGMP Facility is staffed by executive director Mike Fiske and two technicians. Fiske has developed and overseen operations at similar facilities in the private sector for Wyeth and Genencor International. For more information visit: http://www.urmc.rochester.edu/upstate-stem-cell-facility/.