Girl receives kidney and stem cell transplant to avoid need for drugs

An eight-year-old girl is the first patient in the UK to have a kidney transplant and not require long-term immunosuppressive medication.

Researchers and clinicians from Great Ormond Street Hospital for Children (GOSH) in London 'reprogrammed' Aditi Shankar's immune system before performing a kidney transplant. This reprogramming meant that drugs to stop the body from rejecting the donated organ were not needed, and was achieved by firstly transplanting bone-marrow stem cells from her mother, who also later donated the kidney.

'Like any new treatment, it is not without risk and in this case as stem-cell transplantation means the patient must also undergo chemotherapy and radiotherapy.' Professor Jeremy Hughes, Kidney Research UK chair of trustees, said 'However, for a patient to receive a transplant and not require a lifetime of immunosuppressant medication is a significant breakthrough and whilst at this time the process is limited in scope, it does open the door for further future development that could have the potential to overcome one of the major challenges in transplantation care.'

While providing a vital function after transplant surgery, immunosuppressants work by dampening down the body's immune system, meaning anyone taking them is at higher risk of an infection, among other complications. They usually need to be taken for life but as the bone marrow stem cells and kidney came from the same donor, Aditi was able to stop taking immunosuppressant drugs a month after surgery.

Prior to the transplant Aditi, who has an extremely rare inherited condition, Schimke's immuno-osseous dysplasia (SIOD), was spending a significant proportion of her time receiving dialysis – a procedure that removes waste products and excess fluid from the blood when the kidneys have stopped working properly.

SIOD weakened Aditi's immune system to the point where her kidneys were failing and she was on dialysis for 24 hours a day. Six months following the bone-marrow stem cell transplant, Aditi was able to receive the kidney transplant.

The immune system 'reprogramming' approach has been used in other children with SIOD, but this is the first time it has been undertaken in the UK.

'She is the first patient in the UK who has had a kidney transplant to not require immunosuppressive medication after the surgery,' Professor Stephen Marks, clinical lead for renal transplantation at GOSH said. 'A month after the transplant, we were able to take her off all of her immunosuppression, which means she doesn't get the side-effects of the drugs.

Although a significant breakthrough in transplantation care, the procedure is unlikely to be widely used, as the double transplant carries increased risks to the patient.

Professor Marks added: 'There are subgroups of patients who have particular kidney diseases involving the immune system… you could postulate that for some of these conditions that increased risk may be worth taking because that patient may not be able to survive long term on dialysis.'