First-Ever Stem Cell Trial to Repair Kidney Damage

Murray, Utah — Intermountain Medical Center and Salt Lake City-based biotech company AlloCure are working together to conduct the world's first clinical trial using adult stem cells to prevent or repair kidney damage that can occur after open-heart surgery.

Currently, only a handful of adult stem cell studies are taking place across the globe and none of them have ever used stem cell therapy in this fashion and for this indication, according to AlloCure chief executive officer John Wirthlin.

Intensive medical procedures like open-heart surgery can place tremendous stress on the body's systems, and the kidney is often one of the first organs to suffer damage as a result. This stress can lead to acute kidney injury, particularly in patients who are over 65, have diabetes, hypertension, chronic obstructive pulmonary disease, congestive heart failure or underlying kidney problems.

During the clinical trial, Intermountain Medical Center cardiovascular surgeons – who do more open-heart surgeries than anyone else in the state – will administer a single dose of adult stem cells into the aortas of 15 patients. The cells are derived from bone marrow obtained from adult living donors and processed according to a technique developed by AlloCure co-founders Christof Westenfelder, MD, chief medical officer, and Axel Zander, MD, Hamburg, Germany, chief scientific officer.

When a kidney is damaged, it will send chemical signals into the bloodstream that attract AlloCure's stem cells to the injured organ. Once there, the cells initiate a complex repair process that preserves kidney function and promotes the survival and growth of kidney cells, leading the organ to repair itself. After two or three days, when the repair process has been fully initiated, the administered stem cells leave the kidney and are processed through the body. This aspect makes the therapy particularly safe.

The results from this trial could have a major impact on medical practice. More than a half-million Americans each year suffer from acute kidney injury. Many will die or require lifelong dialysis or a kidney transplant. Dialysis takes a toll on a patient's quality of life, and long-term survival is limited unless the patient receives a successful kidney transplant. The health care costs for these patients are extremely high.

"The extensive preclinical data we have generated in our laboratories in Salt Lake City and Hamburg have clearly demonstrated that AlloCure's cell therapy can reverse the course of acute kidney injury," said Dr. Westenfelder. "We are hopeful that we can help these patients with this novel form of treatment to retain adequate kidney function after open heart surgery, and thus avoid the need for dialysis and associated complications."

Intermountain Medical Center cardiovascular surgeon John Doty, MD, the principal investigator for the trial, said the 15 participants will be chosen from among a relatively healthy group of patients, because this part of the trial primarily will seek to show that the stem cells are safe.

The research team believes that the cells will be well-tolerated by the patients, since similar cells have been safely used in children and adults to treat other major diseases. AlloCure's cells are not recognized by the immune system, so patients will not need any anti-rejection drugs, said Wirthlin.

"This is cutting-edge medical technology being developed here in Murray, Utah. It's a part of history," said Dr. Doty. "It's very exciting to be involved."

This is the first phase of the clinical trial process, which is anticipated to end this spring. If all goes well, AlloCure will then initiate second and third phase clinical trials, which will include much larger groups of patients, before petitioning the FDA for market approval. The entire process is expected to take about six years.