UofL Bucks for Brains researcher delivers for the Commonwealth
The University of Louisville today announced that researcher Dr. Suzanne Ildstad, representing Regenerex LLC, has entered into a license and research collaboration agreement with Novartis to provide access to stem cell technology that has the potential to help transplant patients avoid taking anti-rejection medicine for life and could serve as a platform for treatment of other diseases.
The University of Louisville and Regenerex LLC announced the research collaboration agreement which will significantly enhance the university's Institute for Cellular Therapeutics' ability to carry out cutting edge research related to the Facilitating Cell, a novel cell discovered by Ildstad, a professor of surgery and director of the institute at UofL as well as CEO of Regenerex. Underpinning this collaboration is an exclusive global licensing and research collaboration agreement between Regenerex and Novartis.
Ildstad published results in a March 2012 Science Translational Medicine demonstrating the efficacy of this process, known as Facilitating Cell Therapy, or FCRx which is currently undergoing Phase II trials. Five of eight kidney transplant patients were able to stop taking about a dozen pills a day to suppress their immune systems. It was the first study of its kind where the donor and recipient did not have to be biologically related and did not have to be immunologically matched.
In a standard kidney transplant, the donor agrees to donate a kidney. In the approach being studied, the individual is asked to donate part of their immune system as well. The process begins about one month before the kidney transplant, when bone marrow stem cells are collected from the blood of the kidney donor using a process called apheresis. The donor cells are then processed, where they are enriched for developing "facilitating cells" believed to help transplants succeed. During the same time period, the recipient undergoes pre-transplant "conditioning," which includes radiation and chemotherapy to suppress the bone marrow so the donor's stem cells have more space to grow in the recipient's body.
One day after the kidney is transplanted into the recipient, the donor stem cells engraft in the marrow of the recipient and give rise to other specialized blood cells, like immune cells. The goal is to create an environment where two bone marrow systems co-exist and function in one person. Following transplantation, the recipient takes anti-rejection drugs which are decreased over time with the goal to stop a year after the transplant.
In 1998, Ildstad was one of the first recruits to the University of Louisville under the Commonwealth's Bucks for Brains initiative, advanced by former Gov. Paul Patton. As the Jewish Hospital Distinguished Chair in Transplantation Research, Ildstad brought a team of 25 families from Philadelphia to join the University of Louisville. In the following years the team has continued to examine the facilitating cell (FCRx) platform technology for the treatment of kidney transplant recipients as well as considering its potential for the treatment of red blood cell disorders, inherited metabolic storage disorders of childhood, and autoimmune disorders.
"Being a transplant recipient is not easy. In order to prevent rejection, current transplant recipients must take multiple pills a day for the rest of their lives. These immunosuppressive medications come with serious side effects with prolonged use including high blood pressure, diabetes, infection, heart disease and cancer, as well as direct damaging effects to the organ transplant," Ildstad said. "This new approach would potentially offer a better quality of life and fewer health risks for transplant recipients."
"In 1997, the University of Louisville was given a mandate to become a premier metropolitan research university that transforms the lives of the people of Kentucky and beyond," said Dr. James Ramsey, president of UofL. "Dr. Ildstad was among the first faculty members hired utilizing seed funds from the state to help us attract highly talented researchers through the Bucks for Brains program. Regenerex demonstrates the potential for that vision to be realized bringing new jobs to the city, adding to the revenue from the Tax Increment Financing district and providing funding to UofL in support of our academic mission."
The collaboration provides for investments in research, as well as milestones and royalty payments from Regenerex to the University of Louisville in connection with commercialization of the FCRx technology. The therapeutic potential for the technology is wide ranging. The collaboration also involves a sponsored research agreement to support a multi-year collaboration between Regenerex, UofL and the Novartis Institutes for BioMedical Research to pioneer new applications of the technology.
"The 'holy grail' of transplantation is immune tolerance, that is making the body recognize a transplanted organ as 'self' and not reject it as foreign tissue, but without the need for immunosuppressive drugs with their numerous serious side effects," said Dr. David L. Dunn, executive vice president for health affairs at UofL. "Dr. Ildstad and her team may well have solved this puzzle."
Ramsey noted that in addition to the supreme efforts of the research team, it would not have been possible for the work to move forward without the support of the state, the National Institutes of Health, the Department of Defense, Jewish Hospital Foundation, Kentucky Science and Technology Corporation and the National Stem Cell Foundation.
"It is immensely rewarding for our donors to know they helped move potentially life-changing therapies closer to being available for people in need worldwide," said Paula Grisanti, chair of the National Stem Cell Foundation.
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