Casgevy gene-editing therapy – For sickle-cell anaemia, a new cure

Source: The post Casgevy gene-editing therapy has been created on the article “For sickle-cell anaemia, a new cure — for millions, a new hope” published in “Indian express” on 25th November 2023.

UPSC Syllabus Topic: GS paper 3- Science and technology– new technology

News: The article discusses Casgevy, a new gene-editing treatment for sickle cell anaemia using CRISPR technology. It recently got UK approval and could soon be approved in the US. It’s highly effective but expensive. India, with many sickle cell patients, is researching similar treatments. India aims to eliminate the disease by 2047 through a national program.

What is gene editing?

Genome editing, also known as gene editing, comprises a set of technologies that empower scientists to modify an organism’s DNA. These technologies enable the addition, removal, or alteration of genetic material at specific locations within the genome.

Various approaches to genome editing have been developed, including the use of restriction enzymes, zinc finger nucleases, transcription activator-like effector nucleases (TALENs), CRISPR-Cas9, and Programmable Addition via Site-specific Targeting Elements (PASTE), among others.

For more information on gene editing read here

For information on CRISPR read here

What is Casgevy?

Casgevy is a revolutionary gene-editing therapy designed to treat sickle cell anaemia. It utilizes the advanced CRISPR-Cas 9 tool for editing genes.

It works by editing the patient’s genes to revive fetal haemoglobin, which is normally switched off after birth. This compensates for the defective haemoglobin in sickle cells, reducing the disease’s severe symptoms and complications.

In clinical trials, Casgevy proved highly effective, eliminating painful inflammatory attacks in over 90% of the participants.

Unlike current treatments like chemotherapy and hydroxyurea, Casgevy offers a more effective solution. However, its high cost, estimated at about $2 million per patient, poses a significant challenge for widespread accessibility.

What is sickle cell disease (SCD)?

In SCD, the problem lies with haemoglobin, the protein in red blood cells that carries oxygen. Normally, haemoglobin allows red blood cells to be disk-shaped and flexible, easily moving through blood vessels.
However, in SCD, haemoglobin is abnormal, causing cells to turn crescent-shaped when they lose oxygen. These sickle-shaped cells die faster than normal cells and often get stuck in blood vessels. This blockage prevents the proper flow of oxygen-rich blood, leading to painful crises and serious, sometimes life-threatening conditions.

For information on sickle-cell disease (SCD) read here

What are the implications of Casgevy for India?

High Prevalence of Sickle Cell Disease: In India, sickle cell disease is particularly prevalent, with 30,000 to 40,000 children born with the condition annually, mostly in tribal areas.

National Health Initiative Support: The National Sickle Cell Anaemia Elimination Programme, aiming to eliminate the disease by 2047, could be supported by the introduction of advanced treatments like Casgevy.

Cost Barrier: High cost of Casgevy, estimated at about $2 million per patient could hinder its accessibility and widespread use in the Indian healthcare system.

What are the government initiatives to eliminate SCA?

Outreach Programmes: The Indian Council of Medical Research (ICMR) and the National Rural Health Mission (NRHM) are implementing outreach programmes for better disease management and control.

Portal for Data Collation: The Ministry of Tribal Affairs launched a portal for individuals with SCA to register, aiding in the collation of disease-related information.

2047 Elimination Mission: Announced in the FY 2023-24 budget, this mission aims for universal screening of 0-40 years age group in tribal areas, backed by awareness and counselling, funded under the National Health Mission.

National Health Mission Guidelines on Hemoglobinopathies: These guidelines include establishing community-level services for pre-marital and pre-conception screening, supported by genetic counselling.

Integrated Centers in Tribal Districts: 22 tribal districts have established integrated centers for SCA treatment and diagnosis.

Question for practice

Discuss the implications of Casgevy’s effectiveness on India’s efforts to eliminate sickle cell disease through its national program by 2047.