Clinical trials underway on gene editing as potential therapy for sickle cell disease

New Delhi: As traditional treatments such as blood transfusion, hydroxyurea, and other medicines have primarily focused on managing symptoms and preventing complications, experts on World Sickle Cell Day on Wednesday said that recent advancements in sickle cell anaemia and innovations in medical technology have led to the development of several promising new therapies.

According to Dr Meet Kumar, Clinical Director, Haematology & Bone Marrow Transplant, Marengo Asia Hospitals, Gurugram, clinical trials carried out gene editing as a potential treatment method for sickle cell disease.

As per preliminary findings, normal haemoglobin production may be enabled, which could significantly reduce sickle cell disease symptoms and effects.

As per experts, in sickle cell disease, the blood can get too thick and clot, causing various brain problems like silent strokes, regular strokes (both where blood flow is blocked and where there is bleeding), and other issues. This happens because the thick blood can block the blood vessels in the brain.

"Silent cerebral infarcts occur in about 40 per cent of children by 18 years. Headaches occur in about 36 per cent of children with sickle cell anaemia. Ischemic stroke occurs in about 10 per cent of children, and hemorrhagic stroke occurs in children at a rate of about 3-10 per cent," Dr Praveen Gupta, Principal Director & Chief of Neurology, Fortis Hospital, told IANS.

Sickle cell disease may affect the brain in various ways, including cognitive dysfunctions like working memory, verbal learning, visual motor function, inadequacy in general intellectual functioning, problems with language and attention, as well as headaches, seizures, and others, the experts noted.

Dr Gaurav Kharya, Founder and Director, Cellogen Therapeutics, said that gene therapy represents one of the most promising advancements in the treatment of sickle cell disease. This approach involves modifying the patient's own hematopoietic stem cells to correct the genetic defect responsible for the disease. He further mentioned that the modified stem cells are "then reintroduced into the patient's body".

"Early clinical trials have shown promising results, with some patients achieving significant improvements in symptoms and quality of life," Kharya added.