The new Good Manufacturing Practice (GMP) like ISO 8 and ISO 7 compliant facility is part of the existing Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M). The center was established in 2016 and made possible by a $15.75 million gift from philanthropist Bernie Marcus, with a $7.25 million investment from Georgia Tech and another $1 million from the Georgia Research Alliance. 

MC3M is already helping researchers from Georgia Tech and partner organizations develop ways to provide therapeutic living cells of consistent quality in quantities large enough to meet the growing demands for the cutting-edge treatments. The center and this new facility also provide the infrastructural foundation for the Georgia Tech-led National Science Foundation Engineering Research Center for Cell Manufacturing Technologies (CMaT), which was announced in September 2017.

The Marcus Foundation’s gift along with the NSF’s expected funding over ten years in CMaT, together with potential private-sector contributions and the state of Georgia’s investment in infrastructure related bio manufacturing, could result in a combined statewide investment of more than $70 million in cell manufacturing. Beyond developing technologies to help make these life-saving cell therapies broadly available and affordable, the initiative will also help train the specialized workforce needed to manufacture these therapies at large scale. 

“This initiative has the potential to change the way we think about medical treatments, to change the way we think about medicine, and the way we approach cures for different diseases,” said Georgia Tech President G.P. “Bud” Peterson, who opened the dedication event. “Here, we will develop the tools and technologies to produce these cells at lower cost, more rapidly and for more people.”

MC3M is already supporting 23 research projects aimed at all components of the challenge, from understanding cell quality and developing scalable processes, to chip-based disease models for safety and efficacy testing and new models for supply-chain optimization and logistics. The center collaborates with several other institutions, supporting the work of 29 faculty members, and helping train 27 students and fellows for the emerging cell manufacturing industry.

The new facility dedicated on June 6 is a unique “sandbox” for collaboration among engineers, clinicians, and industry to develop and validate new scalable manufacturing processes for cell therapies under GMP conditions necessary to eventually obtain regulatory approvals. It will serve as the translational arm of the Marcus Center and CMaT to help researchers throughout the state of Georgia translate emerging cell therapies to clinical practice. This facility – designed to enable real time quality monitoring and control of cell products during manufacturing – is a one-of-a-kind space that will be instrumental in bringing affordable cell therapies to patients faster. 

The new cell-based therapies being approved for use in humans can have dramatic impact. But the therapies are costly, as much as a $500,000 per patient. The MC3M will help develop new technologies and processes to make these treatments consistent in quality and available to the average person.

“The center is about providing access for patients and enabling patients to benefit from these incredible therapies that could change their lives,” said Krishnendu Roy, who directs both MC3M and CMaT. “We need to scale these therapies up to treat hundreds of thousands of patients. This is the vision of Mr. Marcus – to make this available to everyone regardless of their socio-economic status.”

Marcus, who recalled working as a pharmacist before co-founding home improvement retailer The Home Depot, noted that common drugs such as aspirin are chemically consistent around the world, regardless of where they are sold. The consistency of living cell therapies can’t be similarly counted on because their properties may depend on the specific skills and facilities of the research center producing them. 

“Patients receiving these cells need to know that they are getting the right things,” Marcus said. “This is a very practical question for which we have no answer now.” Beyond consistency, the cells also need to be affordable, he said.  

The new cell manufacturing facility will connect cell-based therapies being developed in research facilities with the appropriate tools and technologies that ensure consistency in manufacturing and product quality while enabling scalability. “There is a gap right now between what we do in the research lab and what we need to do to get these therapies to a hundred thousand or even millions of patients,” Roy noted. 

Beyond developing quality control and analytical techniques to ensure consistency, the center will also develop novel feedback-controlled automation systems to lower the cost, Roy said. 

Peterson noted the potential economic impact of building a cell manufacturing industry in Georgia. “Working with our partner universities, the Technical College System of Georgia and the private sector, we will be able to attract new industries, create new jobs and help build the economy of the state of Georgia.”

The initiative began, he noted, with the development of a national cell manufacturing roadmap, an effort supported by the National Institute of Standards and Technology (NIST). The Marcus gift built on that foundation, and in turn, made it possible for Georgia Tech to lead a team including the University of Wisconsin, University of Georgia, University of Puerto Rico-Mayaguez, and other partners, to win the NSF Engineering Research Center award last fall.

Other collaborators in Georgia include Emory University and Children’s Healthcare of Atlanta.

The NSF ERC could provide up to $40 million over ten years, and attract private and local investment that could boost that amount much higher.

“We have incredible momentum,” Roy said. “We are bonded together by a single goal: getting these therapies to many patients at a lower cost to really help them.”

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Georgia Institute of Technology
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Media Relations Contact: John Toon (404-894-6986) (jtoon@gatech.edu).

Writer: John Toon

Tests had confirmed the developing child suffered from Krabbe disease, a genetic disorder that causes toxins to build up in the nervous system, progressively damaging the brain.

Just days after he was delivered, a medical team at Duke University began Owen on nine days of chemotherapy. His body was then infused with stem cell-rich donor umbilical cord blood. A second dose came four weeks later, through a spinal tap, delivering millions of cells directly to his central nervous system.

The rush to save the newborn came about two months after his 10-month-old sister, Mabry Kate, died from the same disease. Christin and Kyle Webb had spent months in and out of hospitals searching for answers as to why their daughter no longer smiled and was having muscle spasms and trouble eating.

By the time she was diagnosed with Krabbe at 6 months of age, the disease had progressed too far for treatment.

“We felt helpless,” Christin Webb said. “As parents we were supposed to be able to help her, and we couldn’t.”

But their search wasn’t in vain. It led them to Duke and to the discovery that for Owen, it was not too late. 

For years, medical researchers across the country have been working on a host of groundbreaking therapies using human cells to treat a range of diseases, from neurological leukodystrophies such as Krabbe disease, all the way to certain types of cancer.

For all of its promise, however, cell therapy still faces hurdles before it can be used to treat more than a handful of patients at a time. Challenges range from the need to standardize the way cells are manufactured to figuring out how to produce cells faster, in greater quantities, and at lower cost. Georgia Tech researchers have embarked on a multiyear effort aimed at helping doctors and scientists address these challenges and expand cell therapies to more people and more conditions.

“The fundamental challenge is that we’re dealing with a living entity,” said Krishnendu Roy, Robert A. Milton Chair and professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. “Classically we’ve always dealt with manufacturing an inanimate object, like materials or a car or an airplane. Even in biomanufacturing we have mostly dealt with a single molecule or protein, not a complete living product like a cell that can change with every manipulation you make.”

In January 2016, Georgia Tech announced a research center devoted to developing processes and techniques to manufacture living cells — the Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M). Made possible by a $15.7 million grant from the Atlanta-based Marcus Foundation, the $23 million center will include a facility to produce cells under strict federal quality control protocols — referred to as a good manufacturing practices facility — and will provide the framework for partnerships with industry as well as research and clinical institutions across the country.

The cell manufacturing effort is just one of myriad research initiatives ongoing at Georgia Tech geared toward advancing manufacturing technologies to solve real-world problems in a broad range of areas. 

Read the compete article from Research Horizons magazine.

Over the past decade, new and emerging cell-based medical technologies have been developed to manage and possibly cure many conditions and diseases. In 2012 alone, these technologies treated more than 160,000 patients. Before these treatments can be more widely available, however, the cell therapeutics community will have to develop the capability for advanced, large-scale manufacturing of high-quality and consistent living cells.

To advance that goal, the Georgia Research Alliance (GRA) and the Georgia Institute of Technology (Georgia Tech) have launched the National Cell Manufacturing Consortium (NCMC), an industry-academic-government partnership that recently released the National Roadmap for Advanced Cell Manufacturing. Establishment of the consortium and development of this 10-year national roadmap was sponsored by the National Institute of Standards and Technology (NIST).

The roadmap was announced June 13 at the White House Organ Summit.

“The cell manufacturing roadmap effort is mission critical to establish the United States as the world leader in cell therapy manufacturing,” said Greg Russotti, Ph.D., vice-president of technical operations for Celgene Cellular Therapeutics. “Cell therapies offer exciting next-generation opportunities that may help patients live longer and better lives, reduce the burden on health care and benefit society. Producing sufficient quantities of high quality cell therapies so that patients have access will not be possible without significant advances in the field of cell therapy manufacturing. Industrial, academic, and government stakeholders collaborated to construct this roadmap, which delineates our path to U.S. leadership in the emerging field of cell therapy production.”

Development of the roadmap required strong support and involvement from more than 60 representatives from industry, government and nonprofit organizations.

“MilliporeSigma (formerly EMD Millipore) supports consortia, like the National Cell Manufacturing Consortium, that bring together industry, innovators, clinicians and academics to advance the field of cell therapy,” said Martha S. Rook, Ph.D., head of novel therapies for the company. “The consortium’s cell manufacturing roadmap is a valuable resource to help identify and address challenges in cell manufacturing.”

While research has demonstrated the value of cell therapies – using adult stem cells and immune system cells – improvements are needed to make these cells broadly available to the medical community.

“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.”

Established in 2014 through a NIST Advanced Manufacturing Technology (AMTech) grant, the NCMC is an industry-driven consortium including cell manufacturing experts from industry, academic research, clinical good manufacturing practice (GMP) centers, government agencies and private foundations.

Georgia is positioning itself to be at the forefront of this new and growing market with its research institutions playing a vital role in the consortium. Researchers from Emory University, Georgia Tech, and the University of Georgia are contributing to the ongoing work of the NCMC. The Atlanta-based Marcus Foundation recently made a major gift to Georgia Tech to establish the Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M). The new center, the first of its kind in the United States, 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 NIST grant kick-started our efforts to develop a national roadmap 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 over $1 billion. Completion of this roadmap positions Georgia at the forefront of one of the most exciting new initiatives of this century.”

For more information on the National Cell Manufacturing Consortium and to view the roadmap, visit http://cellmanufacturingusa.org.

About Georgia Research Alliance
The Georgia Research Alliance (GRA) works to expand research and commercialization capacity in Georgia’s universities to recruit world-class talent, seed new companies and transform lives. For over twenty-five years, GRA has worked to strengthen the university research enterprise in Georgia by working in partnership with the University System of Georgia and the Georgia Department of Economic Development to create the companies and jobs of Georgia’s future. Visit www.gra.org for more information.

About Georgia Institute of Technology
The Georgia Institute of Technology is widely regarded as one of the world’s top technological research universities. Ranked 7th among public universities by U.S. News & World Report, Georgia Tech has more than 25,000 undergraduate and graduate students, and conduced $726 million in research during 2014. Visit www.gatech.edu for more information.

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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 healthcare 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.”

Research News
Georgia Institute of Technology
177 North Avenue
Atlanta, Georgia 30332-0181 USA

Media Relations Contact: John Toon (404-894-6986) (jtoon@gatech.edu).
Writer: John Toon