London Daily

Focus on the big picture.
Saturday, May 31, 2025

Transformational therapy cures haemophilia B

Transformational therapy cures haemophilia B

A "transformational" therapy has effectively cured people with the bleeding disorder haemophilia B, say British doctors.

The treatment corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding.

Elliott Mason, who was part of the trial which tested the therapy, says his life now feels "completely normal".

The medical team says the majority of adults with haemophilia could be cured in the next three years.

From the day Elliott was born, he was unable to make enough of a crucial protein called clotting factor IX. When you cut yourself and it scabs over, clotting factor IX is one of the proteins that stops the bleeding.

It meant Elliott grew up "anxious of getting hurt", and teachers "wrapped me up in bubble wrap". The sport he wanted to play - rugby - was completely off limits.

"I didn't like the fact that I was different and not able to do things," he says.

At one point, Elliott was having injections of factor IX every other day in order to prevent a deadly bleed.

But he managed to stay healthy, unlike many others with haemophilia who face severe damage to their joints from the bleeding.

"We have a lot of young patients in excruciating agony and there's nothing we can do to reverse the joint damage," says Prof Pratima Chowdary, from the Royal Free Hospital and University College London.


Gene therapy


Elliot was given an engineered virus that was filled with the instructions for manufacturing the missing factor IX.

The virus acts like a microscopic postman that delivers the blueprints to the liver, which then starts producing the clotting protein.

It was a one-off infusion that took about an hour to drip into Elliott's body.

He recalls being "astonished" to see the amount of factor IX in his blood go from only 1% of normal levels to normal.

Elliott says the treatment has made his life "completely normal", without him having to worry how haemophilia could affect it


The findings, published in the New England Journal of Medicine, showed nine out of 10 patients given the therapy no longer needed their clotting factor IX injections.

"I've not had any treatment since I had my therapy, it's all a miracle really, well it's science, but it feels quite miraculous to me," says Elliott, who now lives in London.

"My life is completely normal, there's nothing that I have to stop and think 'how might my haemophilia affect this?'."

For Elliott, that includes skiing and riding a motorbike.


'Very excited'


"We're very excited by the results," said Prof Chowdary.

She says there was a "transformational impact" about a year after the therapy, when suddenly people realised: "I don't need to worry about my haemophilia at all."

This trial is just the latest in a series of breakthroughs in treating both haemophilia A and B.

Prof Chowdary told me she's now "looking for my next job", as curing haemophilia "will be a reality for the majority of the adults in the next one to three years."

But there are still questions which need answering:

*  How much will the gene therapies cost? Current clotting factor injections can cost between £150,000 and £200,000 per patient per year

*  How long will the treatment last? Studies suggest it will be at least a decade, but nobody knows for sure

*  How early in life can the therapy be given? Before the age of 12 the liver is still developing, but scientists hope it will be an option from then on

Clive Smith, chairman of the Haemophilia Society, said: "This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments.

"If they are shown to be safe and effective, NICE [National Institute for Health and Care Excellence] and the NHS must work together to make these innovative treatments available."

Newsletter

Related Articles

0:00
0:00
Close
Satirical Sketch Sparks Political Spouse Feud in South Korea
Indonesia Quarry Collapse Leaves Multiple Dead and Missing
South Korean Election Video Pulled Amid Misogyny Outcry
Asian Economies Shift Away from US Dollar Amid Trade Tensions
Netflix Investigates Allegations of On-Set Mistreatment in K-Drama Production
US Defence Chief Reaffirms Strong Ties with Singapore Amid Regional Tensions
Vietnam Faces Strategic Dilemma Over China's Mekong River Projects
Malaysia's First AI Preacher Sparks Debate on Islamic Principles
White House Press Secretary Criticizes Harvard Funding, Advocates for Vocational Training
France to Implement Nationwide Smoking Ban in Outdoor Spaces Frequented by Children
Meta and Anduril Collaborate on AI-Driven Military Augmented Reality Systems
Russia's Fossil Fuel Revenues Approach €900 Billion Since Ukraine Invasion
U.S. Justice Department Reduces American Bar Association's Role in Judicial Nominations
U.S. Department of Energy Unveils 'Doudna' Supercomputer to Advance AI Research
U.S. SEC Dismisses Lawsuit Against Binance Amid Regulatory Shift
Alcohol Industry Faces Increased Scrutiny Amid Health Concerns
Italy Faces Population Decline Amid Youth Emigration
U.S. Goods Imports Plunge Nearly 20% Amid Tariff Disruptions
OpenAI Faces Competition from Cheaper AI Rivals
Foreign Tax Provision in U.S. Budget Bill Alarms Investors
Trump Accuses China of Violating Trade Agreement
Gerry Adams Wins Libel Case Against BBC
Russia Accuses Serbia of Supplying Arms to Ukraine
EU Central Bank Pushes to Replace US Dollar with Euro as World’s Main Currency
Chinese Woman Dies After Being Forced to Visit Bank Despite Critical Illness
President Trump Grants Full Pardons to Reality TV Stars Todd and Julie Chrisley
Texas Enacts App Store Accountability Act Mandating Age Verification
U.S. Health Secretary Ends Select COVID-19 Vaccine Recommendations
Vatican Calls for Sustainable Tourism in 2025 Message
Trump Warns Putin Is 'Playing with Fire' Amid Escalating Ukraine Conflict
India and Pakistan Engage Trump-Linked Lobbyists to Influence U.S. Policy
U.S. Halts New Student Visa Interviews Amid Enhanced Security Measures
Trump Administration Cancels $100 Million in Federal Contracts with Harvard
SpaceX Starship Test Flight Ends in Failure, Mars Mission Timeline Uncertain
King Charles Affirms Canadian Sovereignty Amid U.S. Statehood Pressure
Trump Threatens 25% Tariff on iPhones Amid Dispute with Apple CEO
Putin's Helicopter Reportedly Targeted by Ukrainian Drones
Liverpool Car Ramming Incident Leaves Multiple Injured
Australia Faces Immigration Debate Following Labor Party Victory
Iranian Revolutionary Guard Founder Warns Against Trusting Regime in Nuclear Talks
Macron Dismisses Viral Video of Wife's Gesture as Playful Banter
Cleveland Clinic Study Questions Effectiveness of Recent Flu Vaccine
Netanyahu Accuses Starmer of Siding with Hamas
Junior Doctors Threaten Strike Over 4% Pay Offer
Labour MPs Urge Chancellor to Tax Wealthy Over Cutting Welfare
Publication of UK Child Poverty Strategy Delayed Until Autumn
France Detains UK Fishing Vessel Amid Post-Brexit Tensions
Calls Grow to Resume Syrian Asylum Claims in UK
Nigel Farage Pledges to Reinstate Winter Fuel Payments
Boris and Carrie Johnson Welcome Daughter Poppy
×