Selim Corbacioglu of Germany’s University of Regensburg, who treated the 20-year-old. Nine months after the hopefully one-and-done treatment, her total hemoglobin reached near-normal levels, boosted by a meaningful increase in fetal hemoglobin Fully 99.8% of her hemoglobin was the fetal type, a clear signal that CRISPR had done what it was supposed to.įreedom from transfusions promises to improve a patient’s life dramatically, said Dr. The beta thalassemia patient became transfusion-independent after years of needing about 16 transfusions per year. In both patients treated with CTX001, the infused cells successfully engrafted in the marrow, the companies reported. Technicians zap the cells with a pulse of electricity the “electroporation” opens a portal that the CRISPR complex passes through.Īfter the patient undergoes what’s called busulfan treatment to obliterate the mutation-carrying bone marrow cells remaining in the patient, the CRISPR’ed cells are infused into the bloodstream and, if all goes well, make a beeline for the marrow, there to set up production of healthy, hemoglobin-packed red blood cells. Physicians - one in Germany treated the beta thalassemia patient, another in Nashville, Tenn., treated the sickle cell patient - draw blood and isolate hematopoietic (blood-producing) stem cells. READ MORE: Stephen Hahn, Trump’s FDA nominee, pledges ‘bold action’ on vaping but sidesteps questions on flavor banĭoing so involves an arduous procedure. That’s what CTX001 aims to do: use CRISPR to disable a DNA brake on the production of fetal hemoglobin, thereby giving the body a healthy supply of the vital molecule. In beta thalassemia, a slew of different mutations each produces the same result: too little production of hemoglobin, which carries oxygen throughout the body that produces anemia and organ damage.īoth diseases, research has shown, might be cured if a form of hemoglobin that the body usually stops making soon after birth can be reactivated. In sickle cell, the mutation causes red blood cells to be misshapen, with the result that they struggle to push through blood vessels, leading to organ damage, strokes, bouts of intense pain, and, often, early death. Sickle cell and beta thalassemia are both caused by catastrophic mutations in the hemoglobin gene. The CRISPR/Vertex treatment, called CTX001, targets the two blood disorders in an indirect way. Weiss commented without seeing the data, which the companies shared with STAT on the condition that it not show them to any independent experts. “Now we have a confluence of scientific understanding and technology that can come together to make things happen.” “For decades, we knew about the sickle cell disease mutation but we didn’t know about other genes and we didn’t have the necessary tools for genetic correction” of blood-making stem cells, Weiss said. Jude Children’s Research Hospital, a leader in the genetics of sickle cell who is not involved in the clinical trial. The variety of approaches, after years of neglect for sickle cell in particular, represents a “perfect storm” of basic research on the diseases converging with new genetic technologies, said Dr. Several other companies and academic scientists are also pursuing curative treatments for beta thalassemia and sickle cell disease based on genome editing or gene therapy. Last June, a one-time gene therapy for beta thalassemia from Bluebird Bio secured approval in Europe, with a U.S. The companies, CRISPR Therapeutics ( CRSP) and Vertex ( VRTX) Pharmaceuticals, have competition. The two patients, enrolled in a pair of ongoing clinical trials, have been free from blood transfusions and disease symptoms for a relatively short time, but the encouraging data offer hope that genome editing might one day offer a safe, durable cure for both blood diseases. The first two patients to receive a CRISPR-based treatment for the inherited blood disorders sickle cell disease and beta thalassemia have benefited from the experimental therapy and experienced only temporary and treatable side effects, the companies developing the treatment announced on Tuesday.
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