Gene Therapy Current Affairs - 2020

ICMR: Task Force on Gene Therapy

The Indian Council of Medical Research (ICMR) will set up a task force on gene therapy in order to encourage research in the field. The purpose of setting up the task force is to explore gene editing based therapeutic approaches that will treat illness.

Highlights

The task force will work on genetic diseases affecting brain, eye disorders, muscles, heart, retina, cornea and blood disorders. The blood disorders include Thalassemia, Haemophilia and Sickle cell disease. The task force will also work on diseases such as diabetes, cancer and lung diseases. The work of the force will be completely abided to the guidelines on gene therapy released by ICMR.

Need

ICMR believes that many inherited disorders are not treated by existing drugs and therapies. While the western world has stridden far ahead in the field in the last 30 years, India is lagging in the field. Around 70 million Indians suffer from rare diseases whose cure can be achieved through gene therapy.

Even with advancements, the countries leading in the field are still in the stage of clinical trials and have not yet proved the success of the therapies. To mention a few, ICMR states that drugs like Luxuturna used to treat Retinitis Pigmentosa (illness that affects retinal cells of human eye) and Yescarta used to in cell therapy to treat cancer are still in clinical trial phase.

Therefore, it is important to build a strong task force that will work exclusively on gene therapy.

 

US approves first gene therapy for cancer

The US Food and Drug Administration (FDA) approved first gene therapy, a treatment that uses patient’s own immune cells to fight acute lymphoblastic leukaemia.

The approval will usher new approach to treatment of cancer and other serious and life-threatening diseases. Studies have shown that 83% of patients responded to this treatment, achieving remission within three months.

Key Facts

The gene therapy treatment was originally developed by researchers at University of Pennsylvania and licensed to pharma giant Novartis. It was previously identified as CAR-T cell therapy (CTL019) or tisagenlecleucel and now is called Kymriah.

The treatment is completely different compared to present popular immunotherapy drugs called “checkpoint inhibitors” used to harness immune system that treat variety of cancers by helping the body’s natural T cells better spot tumors.

CAR-T treatment

The CAR-T treatment is not a pill or form of chemotherapy. It uses gene therapy techniques not to fix disease-causing genes (cells) but to turbocharge T cells, immune system cells that cancer can evade. These cells removed from patient’s blood along with white blood cells are encoded with viral vector, reprogrammed in lab. They are reprogrammed to harbor chimeric antigen receptor (CAR) that specifically targets cancer cells. The revived and reprogrammed cells after returned to the patient continue multiplying to fight disease for months or years.