"Holy Grail" Transplantation Technique Offers Hope to Patients with Rare Kidney Disease

In a study conducted by researchers at Stanford University, three pediatric patients with a rare genetic disease that often results in kidney failure underwent dual immune/solid organ kidney transplants. Three years later, the children remain healthy without the use of immunosuppression drugs or treatments. 

The researchers' findings were published Wednesday in the New England Journal of Medicine.

For patients who undergo a kidney transplant, their lives are often riddled with follow-up appointments and anti-rejection medications, and even with extensive care, many find themselves in need of another transplant in 10 to 20 years. Kidney transplantation requests take years for fulfillment and require an extensive medical review before a patient receives the new organ. Post-operation, powerful medications are needed for the rest of their lives to keep the immune system from rejecting the new organ.

The medications required for immune maintenance can heighten patient blood pressure, cause cancer or even lead to an infection severe enough to warrant hospitalization. All of these potential issues affect almost 90,0000 U.S. patients in queue for a new kidney.

Dr. Amit Tevar, surgical director of the Kidney and Pancreas Transplant Program at the University of Pittsburgh Medical Center, told NBC News about the current expectations tied to organ transplantation.

“There is no organ transplant, whether it’s kidney, liver, pancreas, lung or heart, that doesn’t have the same long-term complications from the body’s very effective immune system”, he said. “The Holy Grail of transplantation is immune tolerance.” 

To achieve immune tolerance, a group of participants with Schimke immune-osseous dysplasia (SIOD) was included in a study using a dual immune/solid organ transplant. Patients with this extremely rare diagnosis have an immune system that is particularly ill-equipped to fight infections, leading to kidney failure down the line. Only a few dozen patients with SIOD have been reported globally.

Tevar referred to the research conducted at Stanford as "groundbreaking." This technique is not new, but the inclusion of patients with this disease had not previously been done.

The dual immune/solid organ transplant was carried out in three pediatric patients, all of whom received stem cells and a kidney from a parent. The patients’ immune systems recognized the new kidney without rejecting it. Notably, three years after the double procedure was carried out, all three patients are enjoying life with a normally functioning kidney and immune system. 

Dr. Alice Bertaina, study author and an associate professor of pediatrics at Stanford, said that “these kidneys are going to last forever”.

The study began as two parents of children with SIOD began a non-profit called Kruzn for a Kure Foundation, with the goal of funding research for the rare disorder. Their efforts led to a collaboration with Stanford and a dedicated lab. The children later became participants in the experimental study.

Now, the parents are enjoying knowing they played a part in a procedure that might have saved not only their children but could potentially go on to be the standard of care for all SIOD patients. With additional research, the findings published today could influence the way organ transplant rejection is managed, even beyond SIOD patients.