Two patients with sickle cell disease have seen a “remarkable improvement” in their symptoms following a new gene therapy, the American Society of Hematology (ASH) Annual Meeting heard earlier this week.
Researchers said their therapy also showed potential for “transportability” – meaning it could be applied in developing parts of the world where sickle cell disease is prevalent. The results were presented at the ASH Annual Meeting in San Diego, California, USA, on 3rd December 2018.
The treatment developed at Cincinnati Children’s Hospital Medical Center uses a modified lentivirus vector to transfer a healthy foetal haemoglobin gene into a patient's hematopoietic stem cells. The gene is also modified so it cannot be switched off, enabling it to continue producing haemoglobin.
The researchers say a key feature of the treatment is that the chemotherapy used to precondition the patient’s bone marrow is of reduced intensity compared with the normal myeloablative treatment – reducing cost and toxicity, and so making it easier to apply throughout the world.
The two patients, one 35 years old and the other 25, were treated 6 months and 12 months ago. The researchers intend to enrol a total of ten patients in their trial.
Principle investigator Dr Punam Malik from Cincinnati Children’s said: "One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.
“This includes near elimination of chronic pain and sickling events and improved anaemia.
"Although it's still early post-treatment, these preliminary results are quite promising. If sustained this therapy will provide a transportable, safe and feasible gene therapy for all sickle cell anaemia patients."
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