Universal treatment for rare diseases? Cure for sickle cell, immune deficiencies created from umbilical cord blood

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[I]nfusing umbilical cord blood — a readily available source of stem cells — safely and effectively treated 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies. This is the largest trial of its kind to date.

The idea was to create a fairly universal treatment, rather than chasing individual therapies for all of these rare diseases, and to do so with minimal risk to the patients. The results were published in [the July 14] edition of Blood Advances.

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To make room in their bone marrow for donor stem cells to take root and prevent them from being rejected, study participants received a low dose of chemotherapy and immunosuppressant drugs in a careful sequence. Once the cells integrated into the patients’ bodies, these drugs were tapered off. To kick the immune system back into gear, the researchers reserved a small fraction of the cord blood and gave it to participants a few weeks after the initial infusion.

Related article:  Podcast: Has sickle cell disease met its match in CRISPR gene editing?

It’s important to note that this procedure doesn’t require the donor and recipient to have matching immune profiles.

“That’s huge for ethnic minorities,” [researcher Paul] Szabolcs said. “The probability of a perfect match is very low, but with a cord blood graft, we have a chance to overcome this discrepancy over the course of a couple months and then taper immunosuppressants away.”

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