A woman who has received a breakthrough gene-editing treatment on the NHS is hoping 2026 is the year she will be cured of a major blood disorder.<\/p>\n
Kavita Mehta, 36, from London, has beta thalassaemia, which means her body fails to produce enough haemoglobin \u2013 the protein in red blood cells that carries oxygen around the body.<\/p>\n
Beta thalassaemia is a genetic disease passed down through families and means patients need a blood transfusion every three to five weeks. People with the condition have a shorter life expectancy.<\/p>\n
Ms Mehta has spent her entire life needing transfusions and medication to keep her alive.<\/p>\n
But now she has become one of the first patients in the south of England to receive the gene-editing treatment Casgevy on the NHS.<\/p>\n
She underwent her treatment in November and is now at home recovering \u2013 with all the signs pointing to the fact she may be experiencing a cure.<\/p>\n
Ms Mehta, who works as a director of rooms in the hospitality industry, was diagnosed with beta thalassaemia when she was one month old.<\/p>\n
\u201cWe were living in Kenya at the time,\u201d she explained. \u201cMy grandmother noticed I looked unusually pale, my mum took me to hospital, and that\u2019s when they found out.<\/p>\n
\u201cMy parents are both carriers and so is my brother. I started transfusions straight away. I can\u2019t remember how many bags I needed, but from the beginning it was every six weeks without fail. That carried on for years.<\/p>\n
\u201cWhen I was five, I began iron chelation (a treatment to remove extra iron from the blood caused by blood transfusions). That was the hard part.<\/p>\n
\u201cThe needles back then were awful \u2013 huge butterfly needles \u2013 and my poor mum had to give me the injections every night.<\/p>\n
\u201cIron chelation used to be a slow 10-hour overnight infusion, five nights a week.<\/p>\n
\u201cIt dictated everything \u2013 my weekends, school mornings, even nights out when I got older.\u201d<\/p>\n
Ms Mehta has also experienced a lifetime of tiredness and pain in the week leading up to her blood transfusions, which could now be a thing of the past thanks to Casgevy.<\/p>\n
\u201cAs you get closer to needing a transfusion, you feel it,\u201d she said.<\/p>\n
\u201cI get out of breath, my chest pounds, my head aches, and even my lower back hurts.<\/p>\n
\u201cIt\u2019s eased as I\u2019ve got older, but as a child and in my 20s it used to hit me hard. I avoid the gym the week before a transfusion because I simply can\u2019t cope. I slow down until I\u2019m back in hospital topping up again.\u201d<\/p>\n
With Casgevy, blood stem cells are removed from the patient\u2019s body and edited in the lab before being returned to the patient via an infusion.<\/p>\n
The technique relies on a tool called Crispr, an award-winning method for targeting DNA and performing an edit.<\/p>\n
Shortly after people are born, foetal haemoglobin starts to be replaced by adult haemoglobin.<\/p>\n
But for people with beta thalassemia, their bodies are not able to make enough working adult haemoglobin.<\/p>\n
Casgevy overcomes this by switching on foetal haemoglobin production, which enables the body to overcome beta thalassaemia and offers the chance of a cure.<\/p>\n
Before undergoing treatment, patients are given an intensive course of chemotherapy.<\/p>\n
They then need to stay in hospital for around six weeks to allow the genetically-edited stem cells to embed and flourish.<\/p>\n
After receiving Casgevy in November at University College London Hospital (UCLH), Ms Mehta is now recovering at home.<\/p>\n
\u201cI feel fine now, I\u2019m back to chores and I\u2019m walking around,\u201d she told the Press Association.<\/p>\n
\u201cIt\u2019s looking really good. Even when I was in the hospital, things were looking really good.<\/p>\n
\u201cMy foetal haemoglobin is kind of holding steady and increasing \u2013 which obviously would never have happened before.<\/p>\n
\u201cSo in the doctors\u2019 eyes, it it is working. It\u2019s incredible and it\u2019s crazy.<\/p>\n
\u201cHonestly, I just keep thinking these scientists who discovered this whole procedure are just geniuses.<\/p>\n
\u201cTo change a whole group of people\u2019s lives is incredible.<\/p>\n
\u201cI\u2019m under the NHS, and I was one of the first people in the South so I know that I\u2019m very lucky, but I hope more people will have access to Casgevy.\u201d<\/p>\n
Ms Mehta said she is looking forward to being able to travel more and live a normal life.<\/p>\n
\u201cI\u2019d love to travel for longer than four weeks, that would be amazing,\u201d she said.<\/p>\n
\u201cI\u2019ve heard some people say that your energy levels just increase tenfold, and you never feel that kind of slump after three two to three weeks (like before), which is very exciting for me.<\/p>\n
\u201cYou have to be transfusion independent for one year to be cured, but that is my hope.\u201d<\/p>\n
Ms Mehta and her husband, who does not wish to be named, have also undergone fertility treatment to freeze embryos in the hope of starting a family in the future. The chemotherapy treatment she needed before Casgevy affects fertility.<\/p>\n
The couple have been told to wait two years before conceiving, but Ms Mehta said:\u00a0\u201cWith my bloods being so good, they\u2019re saying in a year, if my bloods are still the same, that they see no reason not to start. So that\u2019s quite exciting.\u201d<\/p>\n
She added: \u201cA life of regular blood transfusions is all I know.<\/p>\n
\u201cI cannot believe that doctors have already seen signs that I am now able to produce my own red blood cells.<\/p>\n
\u201cI can\u2019t tell you what this means to me, and to my family who have had to watch me go through this and care for me.<\/p>\n
\u201cAnd more than anything, I am thrilled that this drug is available on the NHS and may help patients who are much younger than me live a life independent of transfusions.\u201d<\/p>\n
In international clinical trials, Casgevy has shown impressive results, removing the need for blood transfusions for at least a year in 93% of patients.<\/p>\n
UCLH haematology consultant Dr Ben Carpenter, who has been treating Ms Mehta, told PA: \u201cWe are pleased with the way things are going.<\/p>\n
\u201cIt has been more than two weeks since Kavita\u2019s last red cell transfusion, and her levels are continuing to rise independently.<\/p>\n
\u201cWe are now seeing normal red blood cells being produced by her own bone marrow for the first time since she was born.\u201d<\/p>\n
He described the success rate for Casgevy as \u201cfantastic\u201d, adding: \u201cIt really is a game-changer for the transfusion dependent thalassemia community.\u201d<\/p>\n
He said the therapy is a \u201ca beautiful solution for these conditions\u201d, adding: \u201cIt does look like it is a curative treatment\u201d.<\/p>\n","protected":false},"excerpt":{"rendered":"A woman who has received a breakthrough gene-editing treatment on the NHS is hoping 2026 is the year…\n","protected":false},"author":2,"featured_media":261133,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[78],"tags":[134490,29073,4144,18,18822,1278,135,19,17,1565,58561,134491,134492],"class_list":{"0":"post-261132","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-health","8":"tag-beta-thalassaemia","9":"tag-blood-transfusion","10":"tag-chemotherapy","11":"tag-eire","12":"tag-fertility-treatment","13":"tag-gene-editing","14":"tag-health","15":"tag-ie","16":"tag-ireland","17":"tag-nhs","18":"tag-pa-exclusive","19":"tag-uclh","20":"tag-university-college-london-hospital"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ie\/115818249845862302","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts\/261132","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/comments?post=261132"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts\/261132\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/media\/261133"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/media?parent=261132"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/categories?post=261132"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/tags?post=261132"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}