A $15.7 million grant from the Atlanta-based Marcus Foundation has helped launch a new Georgia Institute of Technology research center that will develop processes and techniques for ensuring the consistent, low-cost, large-scale manufacture of high-quality living cells used in cell-based therapies. The therapies will be used for a variety of disorders such as cancer, lung fibrosis, autism, neuro-degenerative diseases, autoimmune disorders and spinal-cord injury – as well as in regenerative medicine.
The work of the new Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M) will help provide standardized production and quality testing for these living cells, which have great therapeutic potential. Standardized manufacturing techniques already exist for drug-based pharmaceuticals; the new center will help provide similar methods and standards for manufacturing therapeutic cells.
Expected to be the first of its kind in the United States, the center will include a validation facility for good manufacturing practices in cell production. In addition to The Marcus Foundation, funding will come from the Georgia Research Alliance and Georgia Tech sources for a total investment of $23 million. The center will also seek support from federal agencies, clinical research organizations and other sources.
"The aspirin you buy today from one pharmacy is essentially the same as the aspirin you buy from another pharmacy, but cell-based therapies may have different efficacy depending on the source and manufacturing processes," said Krishnendu Roy, Robert A. Milton Chair and professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. "There are established ways to quickly assess the efficacy and safety of small-molecule drugs that are acceptable around the world. We want to develop and establish similar processes for therapeutic cell manufacturing."
Ultimately, the growing need for these cell therapeutics could require large-scale production facilities similar to those used in today's pharmaceutical production. But living stem cells and immune system cells are readily affected by the varying conditions under which they are grown, stored and packaged, meaning the same type of cell produced at different facilities could behave very differently. Unless those cells can be produced with consistency, in large scale and at low cost with high quality, use of the new cell therapies could be limited and their promise would not extend to large numbers of patients.
"The critical goal is to either minimize differences caused by varying manufacturing conditions, or to have a very defined characterization process so we exactly know how much the cells have changed and what specific characteristics are predictive of their efficacy in patients," explained Roy, who will lead the new center. "That consistency will allow us to produce affordable products that can make this new technology available to the large community of people who need it."
The new center will collaborate with research and clinical institutions around the country, especially those at which The Marcus Foundation funds research on cell-based therapies, including Duke University, the University of Miami, City of Hope, Emory University, as well as the University of Georgia and other national and international universities.
"Access to this network will provide us a huge advantage by bringing together experts to work on a common problem," Roy said.
"Stem cell treatments and cell-based immunotherapies are, and will be, the treatment of the future," said Bernie Marcus, who co-founded The Home Depot. "Manufacturing and characterization of stem cells and immune cells is a major first step, and that is why The Marcus Foundation chose Georgia Tech and its teams – they have the experience and the personnel to achieve key goals in this process."
The new center will be a collaboration among research groups at Georgia Tech, as well as numerous outside institutions, noted Georgia Tech President G.P. "Bud" Peterson.
"Reproducible production of high-quality therapeutic cells and understanding what markers predict cell effectiveness could give clinicians worldwide new tools in the battle against some of the most difficult human health challenges we face today," Peterson said. "Transitioning these cells into broad clinical use will require the kind of multidisciplinary collaboration that Georgia Tech is known for. Beyond Georgia Tech, this effort will involve The Marcus Foundation, top clinical institutions, the private sector and the Georgia Research Alliance."
The center will involve multiple research organizations at Georgia Tech, including the Institute for Electronics and Nanotechnology, the Georgia Tech Manufacturing Institute and the Parker H. Petit Institute for Bioengineering and Bioscience. Also involved will be faculty researchers from the College of Sciences, College of Computing, and various schools in the College of Engineering, which includes the Coulter Department of Biomedical Engineering operated by Georgia Tech and Emory University. The center will also work closely with the Center for Immunoengineering at Georgia Tech, the Georgia Immunoengineering Consortium, and the Regenerative Engineering and Medicine (REM) Center, a partnership between Georgia Tech, Emory University and the University of Georgia.
"There is no question that stem cell and immune cell manufacturing have the potential to significantly impact our lives, especially as we age," said Ravi Bellamkonda, chair of the Coulter Department of Biomedical Engineering. "We are fortunate to have a visionary foundation in The Marcus Foundation, and the foresight of the Georgia Research Alliance providing leadership in this endeavor."
Work of the center will help make new cell-based therapies more widely available to patients.
"The timing of this investment in cell manufacturing by The Marcus Foundation is absolutely critical," said Robert E. Guldberg, executive director of Georgia Tech's Petit Institute for Bioengineering and Bioscience. "Cell therapies are being evaluated in nearly 9,000 clinical trials worldwide, but their potential to impact human health care will be severely limited until we can scale up their production reproducibly and at low cost. There are currently FDA-approved, clinically effective cell therapy products sitting on the shelf and unavailable to patients because the cost of manufacturing them is simply too high."
The cell manufacturing effort grew, in part, out of a major planning grant awarded by the National Institute of Standards and Technology (NIST) to the Georgia Research Alliance in 2014. That effort focused on developing a road map for cell manufacturing in the state of Georgia – an initiative expected to provide significant economic development benefits. Georgia Tech has been leading this road mapping effort that involves more than 30 industry partners and 16 academic institutions as well as key federal agencies.
"The NIST grant kick-started our efforts to develop a national road map for cell manufacturing," said Michael Cassidy, president and CEO of the Georgia Research Alliance. "The cell manufacturing industry is an emerging and growing industry with annual revenues of about $1 billion. This initiative has the potential to turn scientific research into new businesses and jobs for Georgia."
Initial funding is for five years, and ultimately the center will be expected to support itself with corporate, government and nonprofit funding, Roy said.
"This is a unique public-private philanthropic partnership to address a grand challenge," he added. "We hope to make significant contributions to improving cell-based treatments and lowering their cost. This could provide huge benefit not only to the health of our fellow citizens, both adults and children, but as a manufacturing initiative, could be transformative to the economic development and workforce in Georgia."