A biotech company has recently combined CRISPR with stem cell therapy to treat a human patient. CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene altering technology that uses bacteria to edit DNA. This is the first time this type of treatment has been performed on a human outside of China.
The research is being conducted by Vertex Pharmaceuticals, an American biopharmaceutical company based in Boston, Massachusetts. The treatment is being used to treat a patient who suffers from a rare blood disease called beta thalassemia. The therapy, called CTX001, is a CRISPR/Cas9 hematopoietic stem cell therapy. The treatment is still in an early stage to assess safety and effectiveness. The data so far is promising.
Beta thalassemia is a genetic blood disease that prevents people from making enough hemoglobin, the protein in the blood that carries oxygen to feed cells throughout the body. A shortage of hemoglobin leaves people tired, short of breath and anemic. People who suffer from this illness traditionally require blood transfusions to stay alive.
With the CRISPR/Cas9 treatment, researchers extract stem cells from the patient then alter them to produce fetal hemoglobin in red blood cells. When the cells are successfully modified, they are reintroduced into the patient’s bloodstream.
In addition to this project, the researchers are also working on using this same procedure to treat sickle cell blood disease. This side project already has a patient who will receive the treatment, and the project will begin in the middle of 2019.
Dr. David Altshuler, Executive Vice President of Vertex, explains that the goal of this therapy is to provide a cure in one treatment for Beta thalassemia and sickle cell diseases. The CEO of CRISPR Therapeutics, Dr. Samarth Kulkarni, has stated that the results with the CTX001 procedure in treating beta thalassemia show great progress for the technology. This research is an important milestone toward CRISPR/Cas9 therapies becoming a new, transformative advancement for treating serious, life-threatening diseases.
