A world-first gene therapy which turns white blood cells into a disease-busting \u201cliving drug\u201d has reversed previously untreatable blood cancers, according to recently released research funded by Blood Cancer UK.<\/p>\n
Great Ormond Street Hospital (GOSH) and King\u2019s College Hospital in London treated nine children and two adults with T-cell leukaemia using the technique, which scientists said triggered a \u201cdeep remission\u201d in the majority. Seven are still disease-free three years later.<\/p>\n
Developed by GOSH and University College London (UCL), the technique involves editing the genetic code of donor T-cells to target cancer cells without being rejected by the body. This base-editing is an advanced version of CRISPR technology.<\/p>\n
\u00a0<\/p>\n
\u201cA few years ago this would have been science fiction,\u201d said UCL\u2019s Prof Waseem Qasim. \u201cNow we can take white blood cells from a healthy donor and change a single letter of DNA code in those cells and give them back to patients to try to tackle this hard-to-treat leukaemia.\u201d<\/p>\n
In 2022, 13-year-old Alyssa Tapley from Leicestershire was the first in the world to receive the BE-CAR7 treatment. She had not responded to standard therapies, such as chemotherapy or a first bone marrow transplant, and her family had been discussing palliative care options before the research opportunity was proposed.<\/p>\n
Dr Rob Chiesa, study investigator and bone marrow transplant consultant at GOSH said: \u201cAlthough most children with T-cell leukaemia will respond well to standard treatments, around 20% may not. It\u2019s these patients who desperately need better options and this research provides hope for a better prognosis for everyone diagnosed with this rare but aggressive form of blood cancer.\u201d<\/p>\n
\u201cI\u2019ve now been able to do some things I thought earlier in my life would be impossible,\u201d said the now 16-year-old, who is cancer-free and has been discharged to long-term follow up. \u201cMy ultimate goal is to become a research scientist and be part of the next big discovery that can help people like me.\u201d <\/p>\n
Main image: National Cancer Institute<\/p>\n","protected":false},"excerpt":{"rendered":"A world-first gene therapy which turns white blood cells into a disease-busting \u201cliving drug\u201d has reversed previously untreatable…\n","protected":false},"author":2,"featured_media":265482,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[134,527,111,139,69],"class_list":{"0":"post-265481","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-healthcare","8":"tag-health","9":"tag-healthcare","10":"tag-new-zealand","11":"tag-newzealand","12":"tag-nz"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/265481","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/comments?post=265481"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/265481\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media\/265482"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media?parent=265481"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/categories?post=265481"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/tags?post=265481"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}