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More Stem Cells

Hadassah-University Hospital in Jerusalem’s Ein Kerem when Prof. Shmuel Slavin, who heads the bone marrow transplantation department, used a bone marrow stem cell transplant and gene therapy to cure two toddler sisters born without immune systems, a genetic disease known as severe combined immunodeficiency, or SCID. The disease is caused by the lack of an essential enzyme, adenosine draminase, or ADA, that creates a functioning immune system. Slavin is an Israeli and Hadassah is a prominent Israeli University Hospital!

After working with a team of Israeli and Italian researchers from the San Raffaele Institute in Milan, Slavin took one of the children’s stem cells and introduced the adenosine draminase replacement gene into her stem cells. The experiment was mostly successful, but she remained immune deficient because the number of cells treated were few and ineffective compared with the overwhelming number of unhealthy cells still in her body. Slavin then took bone marrow from her healthy brother, who was a successful stem cell match for his sister. He isolated the stem cells and transplanted them in the toddler, successfully treating and curing her of the condition.

In the gene-therapy procedure, Slavin’s team developed a protocol that would allow the genetically coerced cells to prevail in the patient’s field of stem cells. He injected the virus-enriched, genetically altered stem cells into the toddler’s body, adding medication to suppress her sick cells and to allow the new, healthy cells to develop and multiply for several days before encountering competition from the sick cells.

Two years later, Hadassah is now taking a multipronged approach to embryonic, fetal, and adult stem cell research, focusing on developing stem cells for the blood system and for the treatment of cancer, and working toward preparing nerve cells required for treating Parkinson’s disease.

“It’s a formidable undertaking,” said Rafi Hostein, CEO of Hadasit, Hadassah’s research and development company. “If we inject right neurons into the brain, we can fix the problem of Parkinson’s.” (Believe that when we see it!)

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