A new treatment for sickle-cell dieses and Thalassemia has been authorized by Medicines and Healthcare Products Regulatory Agency (MHRA) for patients aged 12 and above after a rigorous assessment to certain it’s safety and effectiveness.
This method is the use of gene therapy which involves the use of innovative genes editing tools CRISPR, for which the inventors had been awarded Nobel prize in 2020. It has been named Casgevy and is the first medicine to be licensed and of this kind.
Both sickle cell diesese and Thalassemia are genetic conditions caused by errors in the genes for haemoglobin l, which is used by red blood cells to carry oxygen around the body. Sickle cell disease is common in people with an African or Carribean family background. Thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian and Middle Eastern origin.
People with sickle dieses has genetic error that might lead to attacks of very severe pain, serious and life-threatening infections including Anaemia.
Thalassemia can lead to severe anaemia, patient often need a blood transfusion every 3-5 weeks and injections or medicines thought out their lives.
Julian beach an interim Executive Director at healthcare quality and access at MTRA says that both sickle cell and thalassemia are fatal dieses and leads to death. And that It is very difficult to treat.
“Both sickle cell disease thalassemia are painfull, life-long conditions that in some cases can be fatal. To date, a bone marrow transplant which must come from a closely matched donor and carries a risk of rejection has been the only permanent treatment option,” he says
He adds that this new treatment is new and of it’s onw kind and would benefit people with sickle cell disease. He also appreciate the effort made by the patients who engaged them as part of test during the process.
“I am pleased to announce that we have authorized an innovative of it’s own kind gene editing treatment called Casgey, which in trials has been found to restore healthy hemoglobin production in majority of the patients with sickle-cell dieses and transfusion dependent thalassemia, relieving the symptoms of disease.”
“I would like to thank the patients with lived experiences who engaged with us as part of the assessment process and gave us valuable insight into their lives and the challenges of managing their conditions,” he concluded.
Trial results
According to MTRH clinical trial for sickle-cell, 45 patients received Casgevy but 29 were in the trial long enough to be eligible for the primary important analysis. Of this patients, 28 (97%) were free of severe pain crisis for at least 12 months after treatment.
On the hand, thalassemia trial had 54 patients receiving casgevy with 42 being in trial long enough to be eligible for the primary efficient analysis. Of these, 39(93%) did not need red blood cell transfusion got at least 12months after treatment. The remaining three had more than 70% reduction in the need for red blood cells.
Administration
Casgey is administered by taking stem cells out of a patients bone marrow and editing a gene in the cells in a laboratory. Patients must undergo conditioning treatment to prepare the bone marrow before the modified cells are infused back into the patient.
After that, patients may need to spend at least a month in a hospital facility while the treated cells take up residence in the bone marrow and start to make red blood cells with the stable form of hemoglobin.
The decision to authorize Casgey was endoresed by the government’s independent scienctific advisory committee, the commission on human medicines after a thorough review of the available evidence.
With this new method of treating sickle-cell disease, the entire world is about to move a greater height in health, science Information and technology.
