A new drug in pipeline to improve haemoglobin level in Thalassemia patients

New Delhi: With more than 5 crore Indians are suffering from Thalassemia gene and approximately 10,000 – 12,000 new Thalassemia major born every year, there is still ignorance about this serious blood disorder.

Now a new drug in the pipeline ‘Luspatercept’ will improve haemoglobin level in Thalassemia patients, claimed Dr J.S. Arora, General Secretary of National Thalassemia Welfare Society (NTWS). He added.,“This drug will drastically reduce the requirement of blood and iron chelating agent’s ultimately improving quality of the patient’s life”.

Dr Arora explained in detail about the research on patients who received Luspatercept subcutaneously every 3 weeks for up to five doses over 12 weeks while speaking at the 9th edition of National Thalassemia Conference held at Lady Hardinge Medical College (LHMC), New Delhi. “In the transfusion-dependent, 20 patients (83%) achieved at least a 33% decrease in transfusion burden over any 12-week period compared with baseline, and 16 (67%) achieved at least a 50% decrease. In the non-transfusion-dependent cohort, 21 patients (78%) achieved at least a 1 g/dL increase in mean haemoglobin over any 12-week period, and 15 patients (56%) achieved at least a 1.5 g/dL increase.”

Dr Sandeep Soni, Haemato-oncologist, who is doing research on gene therapy in Thalassemia at Lucile Packard Children’s Hospital, Palo Alto, CA stated, “Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. A gene that is inserted directly into a cell usually does not function. A carrier called a vector is genetically engineered to deliver the genes. The viruses are modified so that they can`t cause disease.”

Dr Soni further added that the virus used is a lentivirus that is capable of introducing beta-globin genes into the cells. “The virus is the mixed with bone marrow cells that have been removed from the patients of beta-Thalassemia. This virus injects the DNA into the cell. The gene joins the rest of the DNA and becomes part of the cell`s chromosomes. When this happens to bone marrow stem cells, the healthy beta-globin gene is passed to all future generations. These stem cells result in formation of normal haemoglobin. First successful gene therapy was done in France in an 18 year old patient in June 2007 and he is transfusion free since June 2008. After that many patients have been cured with gene therapy in USA & Europe,” he explained.

Mrs Vinita Srivastava, Senior National Consultant & Coordinator, Blood Cell, National Health Mission, Ministry Of Health and Family Welfare, Government of India apprised the steps taken by National Health Mission for the management and prevention of thalassemia and sickle cell disease.

The 2 day event was organized by NTWS in association with Department Paediatrics LHMC, on the occasion of its 28^th Anniversary in an endeavor to improve the quality of life of existing patients and prevent the birth of Thalassemia major children in future. It also saw the huge gathering of 200 eminent doctors, scientists along with renowned personalities and 800 patients/parents.

“Prevention is Cure” which is an ideal idiom for Thalassemia, was the emphasis of the conference which highlighted important issues such as Thalassemia management, Blood Transfusion, Latest developments in gene therapy, National Policy of Thalassemia, Benefits of Disability for Thalassemia, Quality of Thalassemia patients, etc.

A High level Thalassemia International Federation (TIF) delegation including its president, executive director and medical advisor arrived from Cyprus and thus organized Patient Capacity Building Workshop on Sunday 25^th November 2018. The Patient Capacity Building Workshop was dedicated to motivate and strengthen the voice of Indian Thalassemics to live a quality life with self-esteem and dignity.