Northwestern Medicine receives $12 million to continue organ transplant research with stem cells

Northwestern Memorial HospitalNov 8 2013

Northwestern Medicine promised $12 million to continue organ transplant research with stem cells to improve outcomes

An innovative Northwestern Medicine- research program investigating if stem cells may be the key to allowing organ transplant patients to stop taking immunosuppressive drugs has received $12 million in research funding. The grant will allow researchers to finish Phase II of the clinical trials and begin Phase III. Northwestern began the study's clinical trial in early 2009 as part of a partnership with the University of Louisville, which engineers the specialized stem cells used in each transplant procedure.

"During our clinical trials, we have been able to take the novel stem cell technology that the University of Louisville pioneered from the bench to the bedside," said Joseph Leventhal, MD PhD, director of kidney and pancreas transplantation at Northwestern Memorial Hospital, and professor of Surgery at Northwestern University Feinberg School of Medicine. "The results of our clinical trial were a catalyst for Novartis' long-term investment in this new cellular based therapy for solid organ transplantation."

The clinical trial uses specially engineered stem cells from kidney donors to make the organ recipient's immune system recognize the new kidney as its own. In mainstream transplant procedures, a recipient faces the danger of their immune system treating a newly transplanted organ as a foreign body that should be removed, causing it to attack and try to kill the new organ. To prevent a recipient's immune system from attacking their new organ, doctors must currently prescribe toxic anti-rejection medicines to weaken the immune system and keep it at bay.

In March of 2012, Leventhal and his colleagues published the study's initial clinical results from eight participants in Science Translational Medicine. The article outlined that of the study's eight participants, five were able to successfully stop taking all anti-rejection medicines in just one year after they received a new kidney, and two required only low-doses of anti-rejection medicine to be maintained instead of a normal regimen. The final patient suffered an unrelated illness that required another kidney transplant to be performed.

"Northwestern's Comprehensive Transplant Center has demonstrated for the first time in the history of organ transplantation that we can safely achieve durable transplantation tolerance in mismatched and unrelated donor/recipient combinations," said Leventhal. "I am very excited to be able to continue this groundbreaking translational research."

The first subjects to participate in the research study underwent surgery on February 26, 2009. To date, more than 20 patients have been enrolled and have received new kidneys as part of the clinical trial, the majority of which have been successfully taken off of all anti-rejection medicines. In order to qualify, the donor and recipient pairs must be blood-type compatible and have a negative cross-match, which means that testing has been done to confirm the recipient does not have antibodies in the blood that would cause rejection of the kidney.

The grant is a result of a license and research collaboration agreement between Regenerex LLC and Novartis to provide access to the novel technology developed by the University of Louisville's Institute for Cellular Therapeutics. Leventhal does not receive financial incentives or payment from Novartis or Regenerex LLC.