For the first time in two decades, a new treatment is available on the NHS for people with sickle cell disease.
Approximately 15,000 people in the UK have the inherited blood disorder, a lifelong genetic condition which the NHS says can only be totally cured by a stem cell or bone marrow transplant but these are not done often due to the risks involved.
As a result, those with sickle cell disease, which is more common in those with African or Caribbean background, are left to live with often-debilitating pain that flares up in periods known as a sickle cell crisis and can require hospital admission for pain management and treatment, as well as possible emergency blood transfusions.
The new drug, crizanlizumab, announced on Tuesday, will be offered to over 16s and injected via transfusion drip. It works by binding to a protein in the red blood cells to prevent the restriction of oxygen supply caused by the sickle-shaped cells that block small vessels.
The National Institute for Health and Care Excellence (NICE) says clinical evidence suggests those treated with crizanlizumab have significantly fewer sickle cell crises in a year.
These crises can take days or weeks for patients to recover from. NHS chief executive Amanda Pritchard said the drug would help as many as 5,000 people in the next three years.
Kehinde Salami, 40, from Sydenham, was diagnosed with sickle cell disease in his mid-20s when he was a university student in Manchester but had struggled with symptoms since childhood. I struggled [as] a kid, waking up and going to school, with my fitness, running, he says.
As an adult he now experiences a range of issues, including fatigue, joint pain, organ pain, swelling of the feet after walking, and even on one occasion blindness.
No day is the same, he says. A [sickle cell] crisis can happen wherever blood flows in the body, so I went blind in my left eye due to a crisis and had to have surgery to correct it. My vision is at 70 per cent now.
Last year, Mr Salami, who works for the NHS, contracted Covid-19 and says he was on the verge of death before being hospitalised. It caused me severe organ shutdown, I couldnt breathe, vomiting, blackouts. Post-Covid his energy levels have diminished further. It has been a battle.
Responding to the news of the treatment, Mr Salami says it is an amazing breakthrough and he cannot wait to see the benefits, particularly for younger sickle cell warriors like his nine-year-old daughter, or babies and toddlers.
Weve been neglected when it comes to new treatments, and we needed a win, and for me this is it, he says, adding that he would like to try it himself but [doesnt] want to take priority or take the place of someone that needs it more.
Shianne-Rose Samuels, 20, from west London, was diagnosed with sickle cell disease at birth. Both her parents have the trait.
Since she was in secondary school she has been in hospital on a near-monthly basis, sometimes more regularly, with painful crises. She also had her spleen and gallbladder removed at 17 and, as a result, is now at higher risk of infections.
Ms Samuels says: Its an invisible illness, youre not in a wheelchair or anything so [people] dont really think we need assistance and when we are well, we look well my insides are deteriorating [but] you cant see that.
Although she is extremely excited about the treatment it gives me hope for the future, it really does and would put her name forward, she worries about the accessibility of transfusion-only as she has problems with cannulation.
It is also notable that crizanlizumab is the first new treatment for the illness in 20 years despite 1 in 79 babies born in the UK carrying the sickle cell trait.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: Sickle cell disease can be a debilitating condition that has a huge impact on patients quality of life. Yet treatment for sickle cell disease has been limited for years, and there has been a lack of treatments.
Both Ms Samuels and Mr Salami suggest more could be done for the community, including awareness and support. Mr Salami says: I just think people are ignoring it, maybe because it predominantly affects black people but its not just a black disease I think people need to start taking heed.
Ms Samuels thinks people black people are often perceived as strong, but we do suffer, she adds.
Jabeer Butt, CEO of the Race Equality Foundation, says that although the treatment is life-changing, people with sickle cell continue to face patchy care across the UK, so crizanlizumab must go hand in hand with accessible support and regular screening of at-risk populations.
Mr Salami wants to use the moment to encourage black Britons to donate blood. In July, i reported, the NHS was calling for donors because of rising demand. There are currently 12,633 black and mixed black blood donors, which is about 1.5 per cent of the total donor base.
Mr Salami is currently helping to run a blood drive, partnered with Evidence Joel, mother of Richard Okorogheye who died in April 2021 and also suffered with sickle cell disease.
See the rest here:
- BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -... - October 17th, 2021
- Misinformation on stem cell treatments for COVID-19 linked to overhyped science, researchers argue - EurekAlert - October 17th, 2021
- FDA Approves Genentech's Tecentriq as Adjuvant Treatment for Certain People With Early Non-Small Cell Lung Cancer - BioSpace - October 17th, 2021
- Nic Chapman: my MS battle and the treatment that's given me hope - The Times - October 17th, 2021
- Dream Body Clinic Stem Cell Therapy Stem Cells HGH - October 13th, 2021
- Hematopoietic Stem Cells - October 13th, 2021
- 3D printing: Healthcare insiders' view on the tech - Verdict - October 13th, 2021
- Top 10 Best Stem Cell Supplement Brands - Healthtrends - October 13th, 2021
- Mitochondrial superoxide dismutase overexpression and low oxygen conditioning hormesis improve the performance of irradiated sterile males |... - October 13th, 2021
- Researchers uncover how cancer cells use sugar residue to evade the immune system - News-Medical.net - October 13th, 2021
- The science and practicality of cultured meat The Stute - The Stute - October 11th, 2021
- Vitro Biopharma Inc. July 31st 2021; 3rd Quarter ended Financial Results of Operations and Shareholder Le - Benzinga - October 11th, 2021
- Want To Support Healthy Bones? It's Time To Get Your Vitamin D In Check - mindbodygreen.com - October 11th, 2021
- Joint : Why Breast Cancer Awareness Matters, and How to Get It Done - marketscreener.com - October 11th, 2021
- Faster healing of wounds can decrease pain and suffering and save lives - KY3 - October 7th, 2021
- Fasting boosts stem cells regenerative capacity | MIT ... - October 7th, 2021
- Adventist Review Online | Vaxed, Vexed, or Other? - Adventist Review - October 7th, 2021
- StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WCAX - October 7th, 2021
- New Australian-first gene therapy gives Rylee and Saman sight at night and the chance at a new life - ABC News - October 7th, 2021
- Is Stathmin-2 the Next Piece of the ALS Jigsaw Puzzle? - BioSpace - October 7th, 2021
- Ashley Black, Entrepreneur Of The Year For Health And Beauty, To Launch Best-Selling FasciaBlaster System - LatestLY - October 5th, 2021
- 25 Best Body Scrubs and Exfoliators in 2021 - the Trend Spotter - The Trend Spotter - October 5th, 2021
- Potential of exosomes as cell-free therapy | IJN - Dove Medical Press - October 2nd, 2021
- Poseida Therapeutics to Present at Society for Immunotherapy of Cancer 2021 Annual Meeting - PRNewswire - October 2nd, 2021
- 12 of the best face serums 2021 - Medical News Today - October 2nd, 2021
- Humanigen Submits All Planned Modules for Potential Conditional Marketing Authorization from the UK's MHRA - Business Wire - October 2nd, 2021
- Envigo Announces Collaboration With Biocytogen to Distribute the B-NDG Triple Immunodeficient and Humanizable Mouse Model - StreetInsider.com - September 29th, 2021
- Large-Scale Bioproduction of Adherent Cells To Support Advanced Therapy Workflows - Technology Networks - September 29th, 2021
- Sidney author warns against the abuses of technology Saanich News - Saanich News - September 27th, 2021
- Jasper Therapeutics Closes Transaction With Amplitude Healthcare Acquisition Corporation, Creating a Publicly Traded Biotechnology Company Dedicated... - September 25th, 2021
- Dr. Sean Kelishadi provides stem cell therapy for facial procedures - Digital Journal - September 25th, 2021
- Pipeline of cell and gene therapies holds promise for repairing the Parkinsonian brain - EurekAlert - September 25th, 2021
- National Institutes of Health awards Children - EurekAlert - September 25th, 2021
- Amplitude Healthcare Acquisition : Announces Shareholder Approval of Business Combination with Jasper Therapeutics, Inc (Form 8-K) -... - September 25th, 2021
- Can you mix and match COVID vaccines or boosters? What scientists and doctors say - CNET - September 22nd, 2021
- All The Reasons Why You Should Add Nutrient-Dense Mushrooms To Your Diet - NDTV Doctor - September 22nd, 2021
- My children know I have a terminal illness talking openly about cancer benefits all of us - iNews - September 22nd, 2021
- Orthopedic Regenerative Surgical Products Market Size to Expand Significantly by the End of 2031 - BioSpace - September 22nd, 2021
- Health tips from Dr. Oz and Dr. Roizen for 9-21-21 - The Commercial Dispatch - September 22nd, 2021
- Prescription Drugs Can Be Affordable and Innovative - Center For American Progress - September 18th, 2021
- The Promise of Regenerative Medicine to Treat Chronic Pain - Mega Doctor News - September 18th, 2021
- Precise CRISPR gene editing can correct the mutation that causes cystic fibrosis in mini-intestines - Massive Science - September 18th, 2021
- Fate Therapeutics Announces Publication of FT538 Preclinical Data Demonstrating Enhanced ... - The Bakersfield Californian - September 18th, 2021
- A New Company With a Wild Mission: Bring Back the Woolly Mammoth - The New York Times - September 14th, 2021
- TreeFrog Therapeutics Secures $75m In Series B Financing To Advance A Pipeline Of Stem Cell-Derived Cell Therapies And Deploy Proprietary C-Stem... - September 14th, 2021
- Therapeutic inhibition of USP9x-mediated Notch signaling in triple-negative breast cancer - pnas.org - September 14th, 2021
- What are the health benefits of nectarines? - Medical News Today - September 10th, 2021
- Time to humanize European health research with an action plan - POLITICO.eu - September 10th, 2021
- Tiny radio tags reveal the lives of Neotropical stingless bees - Massive Science - September 10th, 2021
- Bristol Myers Squibb Research at ESMO 2021 Demonstrates Clinical Benefits of Immunotherapies in Multiple Hard-to-Treat Advanced or Metastatic Cancers... - September 8th, 2021
- Opdivo: Use with Yervoy, Side Effects, Cost, and More - Healthline - September 8th, 2021
- The Deal in Duluth: Catholics here on their own in seeking a religious exemption to being vaccinated - Duluth News Tribune - September 8th, 2021
- Diagnosis and Treatment of Three Primary Malignant Tumors | BCTT - Dove Medical Press - September 8th, 2021
- Jeff Bezos is reportedly funding Altos Labs, a new anti-ageing venture aiming to cheat death - Screen Shot - September 7th, 2021
- Sorrento Announces Enrollment of First Subject in Brazil Phase 2 Study of COVI-MSC for Treatment of COVID-19 Associated Acute Respiratory Distress in... - September 2nd, 2021
- 11 of the best vitamin C serums 2021 - Medical News Today - September 2nd, 2021
- Joint Committee on Vaccination and Immunisation (JCVI) advice on third primary dose vaccination - GOV.UK - September 2nd, 2021
- Japanese scientists have 3D printed a square of lab-grown wagyu beef - The A.V. Club - September 2nd, 2021
- This Company is Producing Bioprinted Fur, Wool & Eyelashes Which Could Make Fur Farms Obsolete - vegconomist - the vegan business magazine -... - September 2nd, 2021
- Poseida Therapeutics Presents Preliminary Results from Phase 1 Trial of P-PSMA-101 at the 6th Annual CAR- - Benzinga - September 1st, 2021
- Operational Highlights and Financial Results for the Year Ended June 30, 2021 - GlobeNewswire - September 1st, 2021
- KNUCKLES Role in Flower Clock; This Tiny Multi-Functional Protein Helps Complete Floral Development Properly at the Right Time - Science Times - September 1st, 2021
- Outlook on the Multiple Myeloma Drugs Global Market to 2026 - by Therapy, Drug Type, End-user, Distribution Channel and Region - Yahoo Finance - August 30th, 2021
- 6 Benefits of Not Eating Meat (or at Least Less of It) - Healthline - August 30th, 2021
- Cell Therapy Manufacturing Market by Type of Cell Manufactured, Source of Cell, Scale of Operation, Purpose of Manufacturing and Key Geographical... - August 25th, 2021
- The world's first 3D-printed Wagyu beef is revealed-it's marbling like the real thing - Texasnewstoday.com - August 25th, 2021
- Science-Backed Benefits of Prickly Pear Cactus - Healthline - August 25th, 2021
- AdAlta and Carina Biotech to develop next-generation CAR-T cancer therapeutics | More News | News Channels - PipelineReview.com - August 25th, 2021
- Educating California and the world on the benefits of hydrogen-powered transportation - H2 View - August 25th, 2021
- The Future is Fat: The Startups Looking to Fatten up Vegan Food - vegconomist - the vegan business magazine - August 25th, 2021
- This tech investor believes well soon live to 150. Here are his seven longevity hacks - The Irish Times - August 25th, 2021
- The Best New CBD Skincare Products Of 2021 (So Far) - Forbes - August 25th, 2021
- FDA Approval, Efficacy, Boosters and More: Which COVID Vaccine is Best for You? - NBC Chicago - August 25th, 2021
- Plant Stem Cell Market Technology Research and Global Outlook By Our Experts The Manomet Current - The Manomet Current - August 22nd, 2021
- AAPI's National Blood Donation Drive and Stem Cell Registry Launched, Honoring Fallen Covid Warriors The Indian Panorama - The Indian Panorama - August 22nd, 2021
- Stem Cells Facts and Quiz - WebMD - August 20th, 2021
- Stem cells | healthdirect - August 20th, 2021
- Research Study Published in Functional Foods Highlights Benefits of a Proprietary CBD Formula for Neurological Health - The Free Press Tampa - August 20th, 2021
- Leukemia Stem Cell Transplant: Benefits, Procedure, Side ... - August 20th, 2021
- Fate Therapeutics Announces Positive Interim Clinical Data from its FT596 and FT516 Off-the-shelf, iPSC - GlobeNewswire - August 20th, 2021