Stephan Grupp, MD, Ph.D., Chief and Medical Director of the Cell Therapy and Transplantation Section of the Institute for Cell and Gene Therapy at the Philadelphia Children’s Hospital (CHOP) and the first pioneer in cell immunotherapy in childhood. A collaborative team of researchers, including Cancer, recently released preliminary data showing that CRISPR-based gene editing therapies are safe and effective for hereditary blood disorders.
A one-time gene-editing therapy, known as CTX001, discovered and developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was given to 22 patients with 15 transfusion-dependent beta-thalassemias and 7 with sickle cell disease.Boosting fetal production hemoglobin To correct defective genes in hemoglobin associated with both diseases. All patients showed a sustained increase in fetal hemoglobin and total hemoglobin and had limited and manageable side effects associated with the transplant procedure.
Fifteen patients in beta-thalassemia have not received blood transfusions since they received the injection. Seven patients with severe sickle cell disease were not at risk of vascular occlusion. This is an acute episode of severe pain with organ damage. “What we see early in these is how variable this is for the patients with sickle cell disease we have seen,” says Grupp. “We hear that it is life-changing.” Until now, treatment options Beta thalassemia It was lifelong blood transfusion Or stem cell transplant.Treatment options Sickle cell The disease is hydroxyurea for pain management, blood transfusions, and increased fetal hemoglobin, and the only treatment option is stem cell transplantation from a donor.
“As we continue, the big question is whether it will last,” Grupp explains about experimental treatment. “Evidence so far shows that it is durable in the time frame we saw, and we need to keep track of the patient.”
Patients enrolled in these trials have their own hematopoietic stem cells and progenitor cells collected from peripheral blood. Patient cells are edited using CRISPR / Cas9 technology. The edited cell is now CTX001 and is injected into the patient as part of a stem cell transplant without the need for a donor or associated risk. Patients are monitored to track the impact of CTX001 on multiple measurements of the disease and to ensure safety.
“I think there is a general move towards gene editing, not gene insertion, in this area of cell and gene therapy,” Grupp said. “In this world where we are modifying hematopoietic stem cells, the movement I see is towards this more targeted approach.”
The first findings are New England Journal of Medicine..
Haydar Frangoul et al, CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia, New England Journal of Medicine (2020). DOI: 10.1056 / NEJMoa2031054
Philadelphia Children’s Hospital
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