Sickle Cell Disease is a debilitating disease and impairs the quality of life. Bone marrow transplant is the only possible cure but carries its own risk. For those with a successful transplant with few complications, the results can be a great improvement in the quality of life, but for those who suffer the severe complications; it can lead to serious problems and may be life-threatening. Thus, the decision to undergo a bone marrow transplant for sickle cell disease is a complex one, involving medical, family, personal, and ethical considerations.

Gene therapy is a new approach to the treatment of many blood cell diseases that may provide an alternative to transplantation from another person. In gene therapy, the bone marrow is collected from the patient’s own body. Next, the stem cells are isolated in the laboratory and a normal copy of the relevant gene (one that produces normal hemoglobin) is either added to the cells or the defective gene of the patient is directly repaired. The gene-corrected stem cells are then transplanted back to the patient after they receive chemotherapy. In theory, gene therapy should be able to have the same benefits as a bone marrow transplant from another person, but not have risks for graft versus host disease, since it is an autologous (stem cells harvested from self) transplant from the patient. Research to develop methods for effective gene therapy has been ongoing for more than 20 years. It has been applied successfully to treat more than 50 infants with severe combined immune deficiency (SCID –also known as “bubble baby disease”), with recovery of their immune system after transplant of their own gene-corrected bone marrow. However, gene therapy can have a unique risk in that the methods used to add or fix the gene in the stem cells can trigger overgrowth of the cell, and in some cases caused a leukemia-like disease in treated patients. Newer methods to add or correct genes have been developed and are being studied in current trials for SCID and other diseases. Similar approaches for gene therapy of sickle cell disease are being developed and clinical trials will begin in the near future to determine if they provide a safe and effective way to treat sickle cell disease.

The research for stem cell gene therapy for sickle cell disease is a program of UCLA conducted by Dr. Kohn. His multi-disciplinary disease team includes experts in stem cell gene therapy, clinical bone marrow transplantation, and the care of patients with sickle cell disease.

With your continual contributions and support for research, Hina Patel Foundation remains hopeful that efforts of the UCLA team will find a safe cure for sickle cell disease.


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