India conducts clinical trials for ‘haemophilia A’ gene therapy

• Union Minister Jitendra Singh said that India has conducted the first human clinical trial of gene therapy for ‘haemophilia A’ at Christian Medical College, Vellore.

• Jitendra Singh said the trials involved deploying a novel technology of using a lentiviral vector to express a FVIII transgene in the patient’s own haematopoietic stem cell that will then express FVIII from specific differentiated blood cells.

• The minister expressed hope that manufacturing of this vector will commence in India soon and proceed with further clinical trials.

Haemophilia A

• Haemophilia is usually an inherited bleeding disorder in which the blood doesn’t clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. 

• The disorder primarily affects males. 

• Haemophilia is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot.

• This change or mutation can prevent the clotting protein from working properly or to be missing altogether.

• People with haemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factor in the blood. 

• Hemophilia A is characterised by a deficiency of the blood clotting protein known as Factor VIII that results in abnormal bleeding. 

• Hemophilia B (Christmas Disease) is caused by a lack or decrease of clotting factor IX.

• The frequency and severity of bleeding episodes depends on how much FVIII protein a person produces. Severe hemophilia A is characterised by especially low levels of FVIII (less than 1 per cent in the blood) and represents about 60 per cent of all cases. 

• Severe hemophilia A may result in bleeding into vital body organs such as the kidneys and brain, which can be life-threatening if left untreated.

• Treatment for severe hemophilia A usually involves use of FVIII replacement therapy or an antibody-based medication to improve the ability of blood to clot and reduce the likelihood of bleeding.