Landmark Breakthrough: UK Approves Gene Therapy for Sickle Cell Disease and Thalassaemia

By


    The UK drug regulator recently approved a groundbreaking gene therapy for the cure of sickle cell disease and thalassaemia, marking a significant milestone in medical advancement. 

    This therapy, named Casgevy, is the world's first licensed treatment based on the revolutionary gene editing technology Crispr-Cas9, which earned its innovators a Nobel Prize in 2020.

The Crispr-Cas9 Innovation

  • The Crispr-Cas9 technology, often referred to as "genetic scissors," became available in 2012 and has since revolutionised the field of biotechnology. 
  • Casgevy utilises this technology to edit the faulty gene responsible for blood disorders, offering a potential lifelong cure.

Understanding the Therapy

  • Casgevy targets the gene BCL11A, crucial for the transition from foetal to adult haemoglobin. 
  • By editing the patient's own blood stem cells, the therapy stimulates the production of foetal haemoglobin, mitigating the symptoms of sickle cell disease and thalassaemia. 
  • This approach differs from the conventional treatment, which involves a bone marrow transplant requiring a closely matched donor.

Overview of Sickle Cell Disease and Thalassaemia

Sickle Cell Disease

  • Sickle cell disease causes red blood cells to assume a crescent shape, hindering their movement through vessels and leading to blocked blood flow. 
  • This results in severe pain, infections, anaemia, and the risk of a stroke. 
  • In India, an estimated 30,000-40,000 children are born with this disorder annually.

Thalassaemia

  • Thalassaemia, like sickle cell disease, involves errors in the haemoglobin gene. 
  • It leads to low haemoglobin levels, causing fatigue, shortness of breath, and irregular heartbeats. Individuals with thalassaemia often require lifelong blood transfusions, leading to iron accumulation in the body.


Trials and Results

  • Casgevy underwent trials for both sickle cell disease and thalassaemia. 
  • Results showed promising outcomes, with a significant reduction in pain crises for sickle cell disease patients and a substantial decrease in the need for transfusions in thalassaemia patients.

Treatment Process

  • Casgevy is administered as a one-time treatment. 
  • The patient's blood stem cells are collected, edited, and tested over a six-month period. 
  • The edited cells are then transplanted back into the patient's bone marrow. 
  • This process requires a month-long hospital stay, during which the edited cells integrate and begin producing red blood cells with normal haemoglobin.

Challenges and Considerations

  • While the therapy represents a significant advancement, challenges such as high treatment costs pose potential limitations. 
  • Estimates suggest a considerable expense, possibly around $2 million per patient. 
  • This raises concerns about accessibility, particularly in countries with limited resources. 
  • Efforts to develop local manufacturing facilities and ongoing research aim to address these challenges.

Future Prospects

  • Despite the current challenges, experts anticipate that prices may decrease over time as more research is conducted. 
  • Developing local manufacturing facilities could enhance accessibility and affordability for a broader population. 

Source : The New Indian Express




Location: Karnataka, India

Comments

Post a Comment

Popular Posts

KPSC KAS 2015 Preliminary Exam Paper- 1 (Questions & Solutions)

By
Image
Click Here for the the question paper:  Questions and Answers: 1.  Match the Name of the Shifting Cultivation (List I) with the Country (List II) : List I (Name of the Shifting Cultivation) List II (Country) A Huma/Ladang I Brazil B Milpa II Vietnam C Ray III Central America D Roca IV Indonesia          Select the code for the correct answer from the options given below:                   A       B     C     D           (1)    IV     III    II      I          (2)    IV     III     I      II          (3)    III     IV    II      I          (4)    III     IV     I      II Answer: Option (1) Explanation: Shifting Cultivation or Jhum Cultivation is a form of agricultural practice in which a plot of land  is cleared of vegetation and cultivated for few years and the
Read more

KPSC KAS 2017-18 Preliminary Exam Paper- 1 (Questions & Solutions)

By
Image
Click Here for the question paper Questions and Answers: 1. Match List-I with List-II: List I List I 1 Stamp duties and duties on medical I Duties levied by the union government but collected and appropriated by the state 2 Duties on succession to property other than agriculture land II Taxes levied and collected by the Union but assigned to the state 3 Taxes on income other than corporation tax and taxes on agricultural income III Taxes levied and collected by the Union and distributed between the Union and the states 4 Excise duties on items other than medical and toilet preparation IV Taxes levied and collected by the Union but which may be distributed between the Union and the States   Choose the correct answer from the options given below :         A      B       C     D (1) 
Read more

The Supreme Court's Landmark Decision: Striking Down Electoral Bonds

By
Image
This Post is based on the article  " The clear message in the Court’s ‘no’ to electoral bonds "  Which is Published in the The Hindu on 17th February 2024.  Introduction: The Supreme Court of India, in its ruling on February 15, 2024, has made a significant decision by striking down the electoral bonds scheme .  This move highlights the crucial need for transparency in democracy, particularly concerning campaign funding. Transparency Restored The electoral bonds scheme was criticised for its opacity, leaving voters unaware of who funded political parties and the amounts involved.  Transparency in campaign funding, a cornerstone of leading democracies, has been reinstated by the Court's decision. Limits on Corporate Funding The Court's judgment addresses legal issues, including the removal of limits on corporate funding, which can lead to undue influence on the government.  By reinstating these limits, the Court aims to prevent the potential misuse of funds for politi
Read more