Current:Home > InvestUK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia -BrightFuture Investments
UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia
Fastexy Exchange View
Date:2025-04-09 19:04:15
LONDON (AP) — Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K.
In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.
The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. To date, bone marrow transplants, an extremely arduous procedure that come with very unpleasant side effects, have been the only long-lasting treatment.
“The future of life changing cures resides in CRISPR based (gene-editing) technology,” said Dr. Helen O’Neill of University College London.
“The use of the word ‘cure’ in relation to sickle cell disease or thalassemia has, up until now, been incompatible,” she said in a statement, calling the MHRA’s approval of gene therapy “a positive moment in history.”
Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.
In people with sickle cell — which is particularly common in people with African or Caribbean backgrounds — a genetic mutation causes the cells to become crescent-shaped, which can block blood flow and cause excruciating pain, organ damage, stroke and other problems.
In people with thalassemia, the genetic mutation can cause severe anemia and patients typically require blood transfusions every few weeks, and injections and medicines for their entire life. Thalassemia predominantly affects people of South Asian, Southeast Asian and Middle Eastern heritage.
The new medicine, Casgevy, works by targeting the problematic gene in a patient’s bone marrow stem cells so that the body can make properly functioning hemoglobin. Doctors take stem cells from the patient’s bone marrow and use genetic editing techniques in a laboratory to fix the gene. The cells are then infused back into the patient for a permanent treatment.
Britain’s regulator said its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated. In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year afterwards.
Gene therapy treatments typically cost thousands of dollars and experts have previously raised concerns that they could remain out of reach for the people most in need of treatment. Vertex Pharmaceuticals said they had not yet established a price for the treatment in Britain and were working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible.”
Medicines and treatments in Britain must be recommended by a government watchdog before they are made freely available to patients in the national health care system.
Casgevy is currently being reviewed by the U.S. Food and Drug Administration; the agency is expected to make a decision early next month, before considering another sickle cell gene therapy.
Millions of people around the world, including about 100,000 in the U.S., have sickle cell disease. It occurs more often among people from places where malaria is or was common, like Africa and India, and is also more common in certain ethnic groups, such as people of African, Middle Eastern and Indian descent. Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria.
__
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely responsible for all content.
veryGood! (1518)
Related
- 'Squid Game' without subtitles? Duolingo, Netflix encourage fans to learn Korean
- 2 Holland America crew members die during incident on cruise ship
- Why Joey King Doesn't Consider Kissing Booth a Stain on Her Resume After Jacob Elordi Comments
- South Carolina court official resigns as state probes allegations of tampering with Murdaugh jury
- Senate begins final push to expand Social Security benefits for millions of people
- Spurs rookie sensation sidelined for at least one game with sprained ankle
- Below Deck Trailer: See an Iconic Real Housewife Rock the Boat With Her Demands
- Trump could learn Monday how NY wants to collect $457M owed in his civil fraud case
- IRS recovers $4.7 billion in back taxes and braces for cuts with Trump and GOP in power
- Mindy Kaling Responds to Rumors She and B.J. Novak Had a Falling Out
Ranking
- Man can't find second winning lottery ticket, sues over $394 million jackpot, lawsuit says
- After tumultuous 5 years for Boeing, CEO will depart as part of broader company leadership shakeup
- Girl dies from gunshot wound after grabbing Los Angeles deputy’s gun, authorities say
- Environmentalists Sue to Block Expansion of New York State’s Largest Landfill
- What do we know about the mysterious drones reported flying over New Jersey?
- 'American Idol': Former 'Bachelor' Juan Pablo Galavis makes surprise cameo for daughter's audition
- Upsets, Sweet 16 chalk and the ACC lead March Madness takeaways from men's NCAA Tournament
- March Madness picks: Our Monday bracket predictions for 2024 NCAA women's tournament
Recommendation
Can Bill Belichick turn North Carolina into a winner? At 72, he's chasing one last high
Boys, ages 12, 7, accused of stabbing 59-year-old woman in Harris County, Texas: Police
Trump’s social media company to start trading on the Nasdaq on Tuesday
Full transcript of Face the Nation, March 24, 2024
Israel lets Palestinians go back to northern Gaza for first time in over a year as cease
Is the war on drugs back on? | The Excerpt podcast
The Sweet 16 NCAA teams playing in March Madness 2024
Baltimore Orioles owner Peter Angelos dies at 94