{"id":32901,"date":"2024-08-14T13:07:40","date_gmt":"2024-08-14T13:07:40","guid":{"rendered":"https:\/\/cells4life.com\/?p=32901"},"modified":"2025-10-22T14:38:12","modified_gmt":"2025-10-22T14:38:12","slug":"gene-editing-stem-cell-therapy-begins-nhs-rollout","status":"publish","type":"post","link":"https:\/\/cells4life.com\/us\/2024\/08\/gene-editing-stem-cell-therapy-begins-nhs-rollout\/","title":{"rendered":"Gene Editing Stem Cell Therapy for Blood Disorder Begins NHS Rollout"},"content":{"rendered":"[et_pb_section fb_built=”1″ _builder_version=”4.16″ global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]\n

Last week it was announced that a new gene editing stem cell therapy for beta thalassemia would start to be made available on the UK’s National Health Service.\u00a0<\/span><\/p>\n

The therapy, called Casgevy, is developed by Vertex Pharmaceuticals and CRISPR Therapeutics and received approval from the UK regulator in November of last year. <\/span>You can read about that announcement here<\/span><\/a>.<\/span><\/p>\n

It\u2019s estimated that there are approximately 460 patients aged 12 or older with transfusion dependent beta thalassemia in England who are currently eligible for the treatment.\u00a0<\/span><\/p>\n

According to the NHS, the therapy will be offered across seven specialist centers and manufactured in the UK. <\/span>[1]<\/span><\/a>\u00a0<\/span><\/p>\n

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What is beta thalassemia?<\/span><\/h3>\n

Beta thalassemia is an inherited blood disorder which affects the production of hemoglobin, a protein in red blood cells used to carry oxygen around the body.<\/span><\/p>\n

Those affected by beta thalassemia produce little to no hemoglobin and as a result require frequent blood transfusions to supply red blood cells that can carry oxygen to tissues in the body.<\/span><\/p>\n

Symptoms of beta thalassemia are associated with anemia and range from mild – fatigue, weakness, headaches – to severe: heart palpitations, abdominal swelling, and jaundice.\u00a0<\/span><\/p>\n

Beta thalassemia has a debilitating impact on sufferers\u2019 quality of life and it can also dramatically reduce life expectancy. <\/span>[2]<\/span><\/a>\u00a0<\/span><\/p>\n

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What is Casgevy and how does it work?<\/span><\/h3>\n

The new therapy utilizes a combination of the patient\u2019s own stem cells and gene editing technology to reprogram cells so that they produce healthy red blood cells.\u00a0<\/span><\/p>\n

Once the stem cells are harvested from the patient\u2019s bone marrow they are then sent to a lab to undergo modification using the gene-editing tool CRISPR.\u00a0<\/span><\/p>\n

CRISPR effectively works like a pair of highly-targeted scissors, seeking out a specific section of DNA and editing a specific gene within it. In this instance, CRISPR targets the gene BCL11A.<\/span><\/p>\n

During fetal development the body produces a unique form of hemoglobin in order to extract oxygen from the mother\u2019s bloodstream. Once we\u2019re born, BCL11A is responsible for silencing the production of fetal hemoglobin so that beta, or adult, hemoglobin can begin to be produced.<\/span><\/p>\n

By targeting the BCL11A gene, the CRISPR tool is able to reverse the silencing process and re-encourage the production of fetal hemoglobin which, crucially, is left unaffected by beta thalassemia.\u00a0<\/span><\/p>\n

Once the stem cells have been edited to turn off BCL11A, they are then returned to patients via infusion and take residence in the bone marrow. The new, edited stem cells then begin to produce healthy red blood cells. <\/span>[3]<\/span><\/a><\/p>\n

In clinical trials, the new therapy removed the need for blood transfusions for at least a year in 39 of the 42 beta thalassemia sufferers treated. <\/span>[4]<\/span><\/a>\u00a0<\/span><\/p>\n

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What\u2019s next for the new therapy?<\/span><\/h3>\n

While the cost to the NHS of the new therapy is hefty – an estimated \u00a31.6 million per patient ($2.1 million US Dollars) – the treatment is being heralded as a cure, with the added benefit of eradicating the need for frequent blood transfusions, thereby reducing strain on health care services and improving patients\u2019 quality of life outcomes.\u00a0<\/span><\/p>\n

With the new therapy expected to be rolled out in the coming weeks, attention now turns to whether the Casgevy treatment for sickle cell anemia – another blood disorder affecting hemoglobin – will also be offered on the NHS, after its approval in November last year. <\/span>[5]<\/span><\/a><\/p>\n

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What does this mean if I want to store my baby\u2019s stem cells?<\/span><\/h3>\n

If it\u2019s likely your baby could have beta thalassemia, it\u2019s probably worth considering banking their umbilical cord blood.\u00a0<\/span><\/p>\n

Umbilical cord blood is rich in hematopoietic stem cells which are already being used in over 80 approved treatments, including for blood disorders.\u00a0<\/span><\/p>\n

Giving your baby the gift of stem cell storage may mean that they\u2019ll one day be able to use their cord blood stem cells for the Casgevy therapy instead of the more intrusively procured bone marrow stem cells.\u00a0<\/span><\/p>\n

While not currently an approved version of the procedure, using the stem cells from banked umbilical cord blood rather than bone marrow could make for a less invasive procedure overall for your child, should they ever need it.\u00a0\u00a0<\/span><\/p>\n

To find out more about how banking your baby\u2019s stem cells could give them access to future medical advancements, request your free Info Kit below.\u00a0<\/span><\/p>\n

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References<\/b><\/p>\n

[1] (2024, August 8). <\/span>Gene-editing therapy that could cure blood disorder thalassaemia for NHS patients<\/span><\/i>. NHS England. https:\/\/www.england.nhs.uk\/2024\/08\/gene-editing-therapy-that-could-cure-blood-disorder-thalassaemia-for-nhs-patients\/#:~:text=The%20one%2Doff%20gene%20therapy,a%20severe%20form%20of%20thalassaemia.<\/span><\/a><\/p>\n

[2] (2022, July 20). <\/span>Beta Thalassemia<\/span><\/i>. Cleveland Clinic. https:\/\/my.clevelandclinic.org\/health\/diseases\/23574-beta-thalassemia<\/span><\/a><\/p>\n

[3] Gallagher, J. (2024, August 8). <\/span>First gene-editing therapy may cure blood disorder<\/span><\/i>. BBC News. https:\/\/www.bbc.co.uk\/news\/articles\/c4gzldll44lo<\/span><\/a><\/p>\n

[4] (2024, June 14). <\/span>Vertex Presents Positive Long-Term Data On CASGEVY\u2122 (exagamglogene autotemcel) at the 2024 Annual European Hematology Association (EHA) Congress<\/span><\/i>. Vertex. https:\/\/news.vrtx.com\/news-releases\/news-release-details\/vertex-presents-positive-long-term-data-casgevytm-exagamglogene<\/span><\/a><\/p>\n

[5] (2024, August 8). <\/span>World\u2019s first gene editing therapy for blood disorder to be available to hundreds of patients in England<\/span><\/i>. National Institute for Health and Care Excellence. https:\/\/www.nice.org.uk\/news\/articles\/worlds-first-gene-editing-therapy-for-blood-disorder-to-be-available-to-hundreds-of-patients-in-england<\/span><\/a><\/p>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_class=”down-arrow” _builder_version=”4.27.4″ background_color=”#bd144f” custom_padding=”0px|||” locked=”off” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”RWP” module_id=”request-welcome-pack” _builder_version=”4.27.4″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” background_layout=”dark” use_border_color=”off” global_colors_info=”{}”]\n

FIND OUT MORE, REQUEST YOUR FREE INFO KIT TODAY<\/h3>\n

All you need to know to make an informed decision.<\/p>\n

Provide your contact details to request:<\/p>\n

– Complete Info Kit and your Guide to Cord Blood Banking
– Information via email
– Contact from our specialist advisors<\/p>\n

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