A 13-year-old girl is free of leukaemia after the world's first use of what scientists have described as the most sophisticated cell engineering to date.
The teenager, called Alyssa, said she felt that volunteering for the experimental new treatment for the disease would help others. “Of course I’m going to do it,” she said.
Scientists said that without the treatment, which came after chemotherapy and an initial bone marrow transplant failed to clear her cancer, her only alternative would have been palliative care.
Speaking about the therapy, Alyssa said: “Once I do it, people will know what they need to do, one way or another, so doing this will help people.”
The teenager, from Leicester, received base-edited T-cells in the first ever use of a base-edited cell therapy at Great Ormond Street Hospital for Children.
Pre-manufactured cells from a healthy volunteer donor were edited to enable them to hunt down and kill cancerous T-cells without attacking each other.
T-cells are white blood cells that move around the body, finding and destroying defective cells.
Alyssa, who was diagnosed with T-cell acute lymphoblastic leukaemia, or T-All, in 2021, was given all the conventional treatments including chemotherapy and a bone-marrow transplant, but the disease returned.
She then became the first patient enrolled on to a new clinical trial, funded by the Medical Research Council, during which she was given universal Chimeric Antigen Receptor T-cells that had been pre-manufactured from a healthy volunteer donor in May this year.
The researchers described base-editing as chemically converting letters of the DNA code that carry instructions for a specific protein.
The edited Car T-cells can be given to a patient so that they quickly find and destroy T-cells in the body, including cancerous ones, after which the person can have a bone-marrow transplant to restore their depleted immune system.
Twenty-eight days after being given the treatment, Alyssa was in remission, researchers said, and was able to have a second bone marrow transplant.
She is said to be “doing well at home” as she recovers and continues with follow-up monitoring at the hospital.
It is hoped the research, due to be presented for the first time at the American Society of Haematology annual meeting in New Orleans in the US, could lead to new treatments and “ultimately better futures for sick children”.
Scientists aim to recruit up to 10 patients who have T-cell leukaemia and have exhausted all conventional options for the clinical trial into the new treatment.
Medics at Great Ormond Street hope that if it is successful it can be offered to children earlier in their treatment when they are less sick and that it can be used for other types of leukaemia in future.
Potential patients for trials will be referred by NHS specialists.
Prof Waseem Qasim, consultant immunologist at Great Ormond Street, said: “This is a great demonstration of how, with expert teams and infrastructure, we can link cutting-edge technologies in the lab with real results in the hospital for patients. It’s our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children.
“We have a unique and special environment here that allows us to rapidly scale up new technologies and we’re looking forward to continuing our research and bringing it to the patients who need it most.”
Alyssa’s mother Kiona said the family were “on a strange cloud nine” and that it was “amazing to be home”.
She said: “Hopefully this can prove the research works and they can offer it to more children — all of this needs to have been for something.”
Dr Robert Chiesa, consultant in bone marrow transplant and Car T-cell therapy at the hospital, said: “Since Alyssa got sick with her leukaemia in May last year, she never achieved a complete remission — not with chemotherapy and not after her first bone marrow transplant. Only after she received her CD7 Car-T cell therapy and a second bone marrow transplant has she become leukaemia-free.”
He described the outcome as “quite remarkable” but cautioned that it must be monitored and confirmed over the next few months.
What is blockchain?
Blockchain is a form of distributed ledger technology, a digital system in which data is recorded across multiple places at the same time. Unlike traditional databases, DLTs have no central administrator or centralised data storage. They are transparent because the data is visible and, because they are automatically replicated and impossible to be tampered with, they are secure.
The main difference between blockchain and other forms of DLT is the way data is stored as ‘blocks’ – new transactions are added to the existing ‘chain’ of past transactions, hence the name ‘blockchain’. It is impossible to delete or modify information on the chain due to the replication of blocks across various locations.
Blockchain is mostly associated with cryptocurrency Bitcoin. Due to the inability to tamper with transactions, advocates say this makes the currency more secure and safer than traditional systems. It is maintained by a network of people referred to as ‘miners’, who receive rewards for solving complex mathematical equations that enable transactions to go through.
However, one of the major problems that has come to light has been the presence of illicit material buried in the Bitcoin blockchain, linking it to the dark web.
Other blockchain platforms can offer things like smart contracts, which are automatically implemented when specific conditions from all interested parties are reached, cutting the time involved and the risk of mistakes. Another use could be storing medical records, as patients can be confident their information cannot be changed. The technology can also be used in supply chains, voting and has the potential to used for storing property records.
Brief scoreline:
Manchester United 2
Rashford 28', Martial 72'
Watford 1
Doucoure 90'
Full list of Emmy 2020 nominations
LEAD ACTOR IN A COMEDY SERIES
Anthony Anderson, Black-ish
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OUTSTANDING VARIETY/TALK SERIES
The Daily Show with Trevor Noah
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Jeremy Irons, Watchmen
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Cate Blanchett, Mrs. America
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OUTSTANDING LIMITED SERIES
Little Fires Everywhere
Mrs. America
Unbelievable
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OUTSTANDING COMEDY SERIES
Curb Your Enthusiasm
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The Good Place
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The Marvelous Mrs. Maisel
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Better Call Saul
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Succession
Specs
Engine: Electric motor generating 54.2kWh (Cooper SE and Aceman SE), 64.6kW (Countryman All4 SE)
Power: 218hp (Cooper and Aceman), 313hp (Countryman)
Torque: 330Nm (Cooper and Aceman), 494Nm (Countryman)
On sale: Now
Price: From Dh158,000 (Cooper), Dh168,000 (Aceman), Dh190,000 (Countryman)
Profile
Company: Libra Project
Based: Masdar City, ADGM, London and Delaware
Launch year: 2017
Size: A team of 12 with six employed full-time
Sector: Renewable energy
Funding: $500,000 in Series A funding from family and friends in 2018. A Series B round looking to raise $1.5m is now live.
The specs
Engine: 4.0-litre V8 twin-turbocharged and three electric motors
Power: Combined output 920hp
Torque: 730Nm at 4,000-7,000rpm
Transmission: 8-speed dual-clutch automatic
Fuel consumption: 11.2L/100km
On sale: Now, deliveries expected later in 2025
Price: expected to start at Dh1,432,000
EPL's youngest
- Ethan Nwaneri (Arsenal)
15 years, 181 days old
- Max Dowman (Arsenal)
15 years, 235 days old
- Jeremy Monga (Leicester)
15 years, 271 days old
- Harvey Elliott (Fulham)
16 years, 30 days old
- Matthew Briggs (Fulham)
16 years, 68 days old
Where to donate in the UAE
The Emirates Charity Portal
You can donate to several registered charities through a “donation catalogue”. The use of the donation is quite specific, such as buying a fan for a poor family in Niger for Dh130.
The General Authority of Islamic Affairs & Endowments
The site has an e-donation service accepting debit card, credit card or e-Dirham, an electronic payment tool developed by the Ministry of Finance and First Abu Dhabi Bank.
Al Noor Special Needs Centre
You can donate online or order Smiles n’ Stuff products handcrafted by Al Noor students. The centre publishes a wish list of extras needed, starting at Dh500.
Beit Al Khair Society
Beit Al Khair Society has the motto “From – and to – the UAE,” with donations going towards the neediest in the country. Its website has a list of physical donation sites, but people can also contribute money by SMS, bank transfer and through the hotline 800-22554.
Dar Al Ber Society
Dar Al Ber Society, which has charity projects in 39 countries, accept cash payments, money transfers or SMS donations. Its donation hotline is 800-79.
Dubai Cares
Dubai Cares provides several options for individuals and companies to donate, including online, through banks, at retail outlets, via phone and by purchasing Dubai Cares branded merchandise. It is currently running a campaign called Bookings 2030, which allows people to help change the future of six underprivileged children and young people.
Emirates Airline Foundation
Those who travel on Emirates have undoubtedly seen the little donation envelopes in the seat pockets. But the foundation also accepts donations online and in the form of Skywards Miles. Donated miles are used to sponsor travel for doctors, surgeons, engineers and other professionals volunteering on humanitarian missions around the world.
Emirates Red Crescent
On the Emirates Red Crescent website you can choose between 35 different purposes for your donation, such as providing food for fasters, supporting debtors and contributing to a refugee women fund. It also has a list of bank accounts for each donation type.
Gulf for Good
Gulf for Good raises funds for partner charity projects through challenges, like climbing Kilimanjaro and cycling through Thailand. This year’s projects are in partnership with Street Child Nepal, Larchfield Kids, the Foundation for African Empowerment and SOS Children's Villages. Since 2001, the organisation has raised more than $3.5 million (Dh12.8m) in support of over 50 children’s charities.
Noor Dubai Foundation
Sheikh Mohammed bin Rashid Al Maktoum launched the Noor Dubai Foundation a decade ago with the aim of eliminating all forms of preventable blindness globally. You can donate Dh50 to support mobile eye camps by texting the word “Noor” to 4565 (Etisalat) or 4849 (du).
EA Sports FC 26
Publisher: EA Sports
Consoles: PC, PlayStation 4/5, Xbox Series X/S
Rating: 3/5
Groom and Two Brides
Director: Elie Semaan
Starring: Abdullah Boushehri, Laila Abdallah, Lulwa Almulla
Rating: 3/5
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How to watch Ireland v Pakistan in UAE
When: The one-off Test starts on Friday, May 11
What time: Each day’s play is scheduled to start at 2pm UAE time.
TV: The match will be broadcast on OSN Sports Cricket HD. Subscribers to the channel can also stream the action live on OSN Play.
