19 February 2019

US scientists have developed a new treatment to prepare the body for stem cell transplant, by removing or suppressing unhealthy cells from the bone marrow.

Prof Judith Shizuru of Stanford University, California, USA, and colleagues say their technique could reduce the need for chemotherapy or radiation before transplantation.

The team studied an antibody which targets a protein called CD117 on the surface of haematopoietic stem cells (HSCs). This antibody – known as SR‑1 – was shown in tests on mice and human cells to “efficiently and safely eliminate” diseased HSCs by binding to the protein and blocking its ability to self-renew.

In mouse models of myelodysplastic syndrome (MDS), SR‑1 was able to deplete both low-risk and high-risk MDS HSCs donated by patients. Human umbilical cord blood cells subsequently transplanted into the mice showed long-term engraftment, with continued suppression of low- and high-risk MDS HSCs.

Details were published last week in the journal Blood.

Prof Shizuru says: “There are many blood and immune disorders that could be cured by a transplant of healthy stem cells. But the pre-treatments necessary to get the healthy cells to transplant effectively are so toxic that we can't offer this option to many patients.

“A treatment that specifically targets only blood-forming stem cells would allow us to potentially cure people with diseases as varied as sickle cell disease, thalassaemia, autoimmune disorders and other blood disorders.”

Dr Wendy Pang, who co-authored the study with Dr Agnieszka Czechowicz, adds that the approach could benefit people with myelodysplastic syndromes for whom chemotherapy or radiation is not feasible. “Many of these people are elderly and unable to qualify for a transplant,” Dr Pang says.

“We are very hopeful that this body of research is going to have a positive impact on patients by allowing better depletion of diseased cells and engraftment of healthy cells,” Prof Shizuru concludes.

Source: Pang, W.W., Czechowicz, A., Logan, A.C., Bhardwaj, R., Poyser, J., Park, C.Y., Weissman, I.L., Shizuru, J.A. (2019) “Anti-CD117 antibody depletes normal and myelodysplastic syndrome human hematopoietic stem cells in xenografted mice”, Blood, available at doi: 10.1182/blood-2018-06-858159


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