Young Brothers Lives Saved By Unique Stem Cell Transplant Procedure

Jun 23, 2019

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.

 

To your health,

The Healing Miracle Team

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12 Comments

  1. Celvin

    Who can afford stem cell therapy?

    Reply
    • THM Team

      While affordability is subjective to the individual, a network of stem cell specialists is available to answer the question, “Are Stem Cells Right For You?” at http://www.StemCellHotline.net/today. They will have more information on the costs associated with the various treatment options. As with many innovations, as it becomes mainstream practice, costs will likely reduce. Costs will vary with each treatment center and the specific treatment required.

      Reply
  2. Dalit Tayar

    Can I help my grandson who has Rasmussen syndrom with stem cell treatments?

    Reply
    • THM Team

      While we are unable to make specific recommendations about personal health, a network of stem cell specialists is available to provide more personalized answers – http://www.StemCellHotline.net/today. They can connect you with a stem cell specialist in your area to answer the question, “Are Stem Cells Right For You?” and to help you find the solution that works best for you.

      Reply
  3. Tilly Dunn

    Yes, I enjoyed this. 23 years ago, my son received my left kidney. He has been on the antirejection drugs, Neoral and Cyclosporin, ever since. I love the idea that soon transplant recipients don’t need cancer inducing drugs anymore.

    xoxox

    TillyDunn/Ottawa

    Reply
    • THM Team

      It is definitely an exciting time!

      Reply
  4. NASRULLA

    Excellent clever step in this field of Transplantation

    Reply
    • THM Team

      It really is amazing!

      Reply
  5. Wilf

    I find the progress made with stell stem
    A very encouraging trend in medicine and
    Look forward to the day when my peripheral
    Neuropathy pains in both legs can be treated
    With regenerative stell stem procedures.

    Reply
    • THM Team

      That will be truly wonderful. 🙂

      Reply
  6. audrey sanfilippo

    the problem for me is it costs too much. I have diabetes and kidney failure from synthroid so looking to treat that I guess id like to be interviewed as to the dangers of drugs and what they cn do . armor thyroid wouldn’t have done this.

    Reply
    • THM Team

      While affordability is subjective to the individual, a network of stem cell specialists is available to answer the question, “Are Stem Cells Right For You?” at http://www.StemCellHotline.net/today. They will have more information on the costs associated with the various treatment options. As with many innovations, as it becomes mainstream practice, costs will likely reduce. Costs will vary with each treatment center and the specific treatment required.

      Reply

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