Young Brothers Lives Saved By Unique Stem Cell Transplant Procedure
The Dogan brothers. Ronnie and Levi, were born with a rare, life-threatening, genetic disease called IPEX syndrome, which causes their immune system to attack their healthy tissues.
This story details how a unique stem cell transplant procedure performed at Lucile Packard Children’s Hospital Stanford saved these boys lives, who are now 2 and 1 years old.
Stem cell transplants were first developed for blood cancer victims, but are now starting to be used more widely in the treatment of genetic diseases. If combined with solid-organ transplants, they could also remove the need for immune-suppressing therapy in organ recipients.
However, traditional stem cell transplants are very risky and are only done to patients who have very little chance of surviving. This is mainly because of a fatal complication known as graft vs. host ailment that could potentially occur when the donated organ responds to the protective markers of the patient, referred to as HLA antigens. They mount an infestation against the body of the patient. A team of Stanford researchers is currently studying how to make the transplants safer and gentler to ensure more people receive cell transplants successfully.
Such advances in the field of medicine are what helped Ronnie and Levi. Alice Bertaina, a medical doctor, and the professor who came up with a technique for reducing graft vs. host ailment risk, was broadening the cell donors potential pool before she moved to Stanford.
As mentioned, a person inherits their HLA antigens from both parents so the best scenario is to carry out a transplant using cells from a fit sibling with a ten-for-ten antigen match. Unfortunately, Ronnie and Levi did not have any other siblings.
The next best alternative was to look for a match from the registry. The fact is that ethnic minority populations have a lesser matching probability to a registered giver as compared to white people. Being black, Ronnie and Levi did not get a match.
Doctor Bertaina’s technique was pioneered in Italy and used a different process. Cells from a donor are processed by experts to remove Beta T cells that are responsible for activating ailments. This way, patients can have a safe cell transplant from a person with five of ten matching HLA antigens same as that of the parent.
Using this processing method allowed Ronnie and Levi to successfully receive a stem cell transplant from their father who is free from the autoimmune ailment. The procedure used was not accessible in other hospitals in the US but is now used on most transplant recipients at Packard Children’s.
Rosa Bacchetta, a medical doctor and Bertaina’s colleague, is going further to conduct more research in terms of establishing gene remedy techniques for the syndrome. Bacchetta says that the significant difference is that gene treatment will correct cells of the patients, hence eliminating compatibility issues between donors and patients.
Together with her team, Bacchetta is researching two gene-therapy approaches, with one being close to moving into clinical trials. They are optimistic that their research studies will offer valuable therapies not only for the sporadic syndrome but also for the other common autoimmune diseases.
The Healing Miracle Team
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