Surgeons successfully transplant genetically edited pig kidney into living recipient
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Key takeaways:
- The pig kidney was modified with 10 gene edits for transplantation into a human patient.
- United Therapeutics is planning FDA investigational new drug application and clinical study.
A woman from Alabama is the latest to receive a gene-edited pig kidney in a recent xenotransplantation at NYU Langone Health, according to a university press briefing.
“We know much now about what the pig kidney can do and cannot do in terms of all the functions the human kidney is responsible for,” Robert A. Montgomery, MD, DPhil, FACS, H. Leon Pachter, MD, Professor of Surgery; chair of the NYU Grossman School of Medicine department of surgery; and director of the NYU Langone Transplant Institute, said during the briefing. “We have tested the simplest gene edits and most complex and found they both work. We tested different combinations of drugs to suppress the immune system. We do not have all the answers. This field is still in its infancy, but our learning curve has been steep and fast.”
Understanding the pig-to-human transplant
Towana Looney, aged 53 years, received FDA approval for the pig-to-human transplant under the expanded access program, which allows compassionate use of investigational products outside of clinical trials in urgent cases. Looney donated a kidney to her mother in 1999 before experiencing complications during pregnancy that led to kidney failure and dialysis in 2016. She was authorized for human organ transplantation in 2017 and spent 8 years on the waitlist.
The pig kidney with 10 gene edits was transplanted into the patient’s lower abdomen on Nov. 25 in New York City in a 7-hour surgery co-led by Montgomery and Jayme Locke, MD, MPH, a transplant surgeon at the University of Alabama at Birmingham, who was a catalyst for Looney’s procedure. Looney was discharged on Dec. 6, after 11 days of post-surgical observation, with wearable health monitors for physical activity, heart rate and blood pressure. She had daily outpatient evaluations and may sometimes require inpatient medicine administration for acute antibody sensitivity treatment, according to the briefing.
UKidney was developed by United Therapeutics Corporation-subsidiary Revivicor Inc. and removes immunogenic antigens while adding six human transgenes to lower rejection risk.
“This kidney is remarkable,” Locke said during the briefing. “Right after reperfusion, it began making urine. It functioned essentially exactly like a kidney from a living donor. Her creatinine fell by more than 50% within 24 hours and normalized very quickly.”
Looney currently has normal kidney function, Locke added, “but to be in her room post-transplant and to see her husband look at her and to describe her as having a life in her that he hasn’t seen, [to see] an immediate change … that is the miracle of transplantation.”
What may make the procedure successful
There is a key element that may make this transplant more successful than previous attempts, Montgomery told Healio during a question-and-answer session: “The last patient that we transplanted had both heart failure and kidney failure and was really probably days or weeks away from dying. The intention there was just to try to extend to her life,” he said. “The main difference is that Towana is in much better shape physically. Her disease has not extended to the point where she was at high risk for dying very soon, but she had no clear pathway to a transplant. And rather than waiting for her to develop all of the different comorbidities and problems that people who are on chronic dialysis for long periods of time develop, we caught her at a time when she was starting to lose access for dialysis to her blood vessels.”
Looney has since ended dialysis and reported feeling well during the press briefing.
“I am full of energy,” she said. “Got an appetite I've never had in 8 years. And, and of course, you know, I can go to the bathroom. Gotta get used to that.”
Looney said her outlook is good: “It's like I can feel the blood pumping through my veins. You can put your hand on my fistula and feel it buzzing. It's so strong.”
The procedure marks the seventh human xenotransplant surgery performed by the NYU Langone Transplant Institute and is the most recent since surgeons transplanted a genetically edited pig kidney into a 62-year-old man with end-stage kidney disease.
United Therapeutics is planning to submit an FDA investigational new drug application and begin clinical study, Leigh Peterson, PhD, executive vice president of product development and xenotransplantation at the company, told Healio.
Reference:
Gene-edited pig kidney gives living donor new lease on life. https://nyulangone.org/news/gene-edited-pig-kidney-gives-living-donor-new-lease-life. Published Dec. 17, 2024. Accessed Dec. 17, 2024.
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Press Briefing; Healio Interviews
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