Daily Current Affairs : 25-November-2023
In a historic development, the UK drug regulator has granted approval for Casgevy, a gene therapy designed to cure sickle cell disease and thalassaemia. This revolutionary breakthrough employs the Nobel Prize-winning Crispr-Cas9 gene editing technology and marks a significant milestone in the field of medical science.
Key Details:
- Casgevy is the world’s first licensed therapy utilizing Crispr-Cas9 for gene editing, a technology acknowledged with the Nobel Prize in 2020.
- The therapy targets and edits the defective gene responsible for sickle cell disease and thalassaemia, potentially offering a lifelong cure.
- Traditional treatments for these blood disorders often require a bone marrow transplant, a procedure contingent on finding a closely matched donor.
- Casgevy, however, is a one-time treatment involving the extraction of blood stem cells through apheresis, a process used to filter various blood components.
How Does the Therapy Work?:
- Sickle cell disease and thalassaemia result from errors in the gene for haemoglobin, the protein responsible for oxygen transport in red blood cells.
- Casgevy utilizes the patient’s own blood stem cells, precisely edited with Crispr-Cas9 technology.
- The therapy targets the BCL11A gene, crucial for the transition from foetal to adult haemoglobin.
- By enhancing the production of foetal haemoglobin, which lacks the abnormalities associated with adult haemoglobin, Casgevy alleviates symptoms in both conditions.
Sickle Cell Disease:
- Genetic errors cause red blood cells to assume a crescent shape, impeding their movement through vessels and leading to blocked blood flow.
- Symptoms include severe pain episodes, life-threatening infections, anaemia, and stroke.
- Manifestation occurs in individuals inheriting damaged genes from both parents, while carriers of a single gene lead normal lives.
Thalassaemia:
- Similar to thalassaemia, individuals inheriting gene pairs from both parents experience severe anaemia, leading to fatigue, shortness of breath, and irregular heartbeats.
- Blood transfusions are a lifelong necessity, resulting in excess iron accumulation, requiring chelation.
- India harbors the largest number of children with thalassaemia major globally, approximately 1-1.5 lakh.
Challenges of the Treatment:
- Cost: The therapy’s price, yet to be announced, is expected to be substantial, potentially reaching up to $2 million per patient.
- Absence of Local Manufacturing: Lack of local manufacturing facilities necessitates the transportation of harvested blood stem cells across countries.
Important Points:
- Casgevy Gene Therapy Approval:
- Groundbreaking approval by the UK drug regulator for Casgevy, a gene therapy targeting sickle cell disease and thalassaemia.
- Utilizes Crispr-Cas9 gene editing technology, awarded the Nobel Prize in 2020.
- Key Details:
- First licensed Crispr-Cas9 therapy globally, offering potential lifelong cure.
- Casgevy edits the faulty gene causing blood disorders, eliminating the need for closely matched bone marrow donors.
- One-time treatment involves apheresis to collect blood stem cells from the bone marrow.
- Mechanism of Action:
- Targets errors in the gene for haemoglobin, the protein in red blood cells carrying oxygen.
- Uses patient’s blood stem cells edited with Crispr-Cas9 to enhance production of foetal haemoglobin.
- Foetal haemoglobin lacks abnormalities found in adult haemoglobin, alleviating symptoms.
- Sickle Cell Disease:
- Genetic error leads to crescent-shaped red blood cells, causing blocked blood flow.
- Symptoms include severe pain, infections, anaemia, and stroke.
- Manifests in individuals inheriting damaged genes from both parents.
- Thalassaemia:
- Inherited gene pairs from both parents result in severe anaemia.
- Symptoms include fatigue, shortness of breath, irregular heartbeats.
- Lifelong blood transfusions and iron chelation required.
- India has the largest population of children with thalassaemia major globally.
- Challenges of the Treatment:
- Cost: Estimated to be high, potentially reaching $2 million per patient.
- Absence of Local Manufacturing: Harvested blood stem cells must be transported across countries due to the lack of local manufacturing facilities.
Why In News
The UK drug regulator recently approved a groundbreaking gene therapy for the cure of sickle cell disease and thalassaemia, marking a significant milestone in the advancement of medical science and offering renewed hope to patients worldwide.
MCQs about Casgevy Gene Therapy
-
What is Casgevy?
A. Traditional blood transfusion method
B. Nobel Prize-winning gene editing technology
C. Bone marrow transplant procedure
D. One-time treatment for diabetes
-
Which gene does Casgevy target for editing in sickle cell disease and thalassaemia?
A. BCL11A
B. FOXP2
C. BRCA1
D. HER2
-
What is the primary symptom of sickle cell disease?
A. Fatigue
B. Severe pain episodes
C. Irregular heartbeats
D. Shortness of breath
-
What is a significant challenge associated with Casgevy gene therapy?
A. Low treatment efficacy
B. Lack of global approval
C. Estimated high cost
D. Excessive side effects
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