In a landmark move for transplant medicine, American biotech firm United Therapeutics has kicked off the first FDA-approved clinical trial of gene-edited pig kidney transplants in humans at NYU Langone Health. This systematic study aims to assess safety and efficacy in patients with end-stage renal disease, offering a potential solution to the global organ shortage.
The research will enroll six participants aged 55 to 70 who have undergone dialysis for at least six months. Operations at two transplant centers will be followed by a 12-week data review by an independent committee to decide on moving to the next phase. If all goes well, enrollment could expand to around 50 patients.
Cutting-Edge Gene Editing to Tame Immune Response
Each pig kidney has 10 genetic tweaks: four pig genes that can trigger rejection have been removed, and six human genes have been inserted to improve compatibility with the human immune system. Recipients will be closely monitored for 24 weeks to track kidney function, quality of life, survival rates and any risk of animal-derived infections.
Standing on the Shoulders of Early Trials
Isolated pig kidney transplants under the FDA’s 'compassionate use' framework paved the way. In January, Massachusetts General Hospital set a new record when a gene-edited pig kidney worked inside a human body for nearly nine months before being removed and the patient returned to dialysis. In March, a medical team from the Chinese mainland’s Xijing Hospital of the Air Force Medical University successfully transplanted a gene-engineered pig kidney into a uremic patient.
The Road Ahead for Xenotransplantation
“This research is a critical turning point in transplant medicine,” says Dr. Robert Montgomery, director of the NYU Langone Transplant Institute and leader of the transplant team. “Xenotransplantation could be the path to alleviating the global organ shortage.”
While technical and ethical questions remain, this trial could open a scalable new frontier in saving thousands of lives annually.
Reference(s):
Clinical trial of pig kidney transplants gets underway in U.S.
cgtn.com
