The University of Kansas Medical Center announced that the U.S. Food and Drug Administration (FDA) has cleared it to begin a first-in-human clinical trial of MSCTC-0010 in patients with de novo high-risk acute or steroid-refractory acute graft-versus-host disease (HR/SR aGvHD).
KU Medical Center is developing MSCTC-0010, a novel cellular therapy containing umbilical cord-derived Wharton's Jelly mesenchymal stem cells for the treatment of HR/SR aGvHD. The biologic drug product is manufactured at KU Medical Center's Midwest Stem Cell Therapy Center. The clinical investigation will be conducted at The University of Kansas Cancer Center.
"As our first IND submission and the first clinical trial to evaluate a product candidate produced by our Midwest Stem Cell Therapy Center, this represents a significant milestone for KU Medical Center," said Richard Barohn, MD, KU Medical Center vice chancellor for research.
"The survival rate for patients with steroid-refractory acute Graft-versus-Host Disease is extremely poor and many of the patients who succumb to it are children. This innovative treatment is capable of stopping a donor's T-cell attack against the patient after transplantation," said Joseph McGuirk, DO, principal investigator and director of KU Cancer Center's division of Hematologic Malignancies and Cellular Therapeutics.
"We leveraged the strengths and expertise of many throughout this process including researchers and clinicians at KU Cancer Center, KUMC's Midwest Stem Cell Therapy Center and regulatory experts at Cardinal Health Regulatory Sciences, to advance MSCTC-0010 from the laboratory to patients," said Scott Weir, PharmD, PhD, KU Cancer Center's associate director for Translational Research.
"This is another example of how KU Cancer Center is not only providing the standard of care, but shaping the future standard of care," Roy Jensen, MD, KU Cancer Center director, said. "We are committed to advancing more new therapies to our patients, including those with GvHD who currently have very limited options."
Multiple studies have demonstrated the potential of mesenchymal stem cells for the treatment of GvHD using a variety of tissue sources (e.g., bone marrow, adipose tissue and umbilical cord). KU Medical Center is investigating MSCTC-0010 for the treatment of HR/SR aGvHD based on the unique properties of mesenchymal stem cells obtained from the solid portion of the umbilical cord, also called Wharton's Jelly. After birth, the umbilical cord is typically treated as medical waste. Rather than disposing of the umbilical cord, scientists safely harvest the Wharton's Jelly stem cells and grow them during expansion in vitro. They are then transplanted into HR/SR aGvHD patients. KU Cancer Center expects to begin dosing patients in the first-in-human, Phase 1, safety and dose tolerance trial in mid-2018.
Graft-versus-Host Disease (GvHD) is an immunologically driven complication that occurs following allogeneic hematopoietic stem cell transplantation. In GvHD, the donated bone marrow stem cell graft contains T-cells that may recognize the recipient's body as foreign and subsequently attack the patient's healthy tissues and organs. Acute GvHD is a potentially life-threatening condition. The overall incidence of acute GvHD falls between 30 percent and 60 percent, with a significant mortality rate in those who fail to respond promptly to therapy. The primary treatment method for GvHD involves immune suppression by corticosteroids. Those patients who do not respond to the steroid therapy are labeled "steroid-refractory." Steroid-refractory GvHD patients have a less than 10 percent chance of survival.