Patients with previously untreatable blood cancer now disease-free after world-first gene therapy
Patients with a previously untreatable form of blood cancer are now living disease-free after a world-first gene therapy.
Alyssa Tapley, 16, became the first patient with an aggressive form of leukaemia to have the experimental treatment in 2022. She was considering options for palliative care, but is now healthy.
New results have been published of a clinical trial on a further eight children and two adults at Great Ormond Street Hospital (GOSH) and King’s College Hospital in London.
Two-thirds have been disease-free for up to three years.
Dr Deborah Yallop, consultant haematologist at King’s, said: “We’ve seen impressive responses in clearing leukaemia that seemed incurable – it’s a very powerful approach.”
The patients in the study had T-cell acute lymphoblastic leukaemia, a rare form of blood cancer that results from T-cells in the immune system growing out of control.
They had all failed to respond to existing treatment.
So the medical team tried an experimental technique to turn T-cells taken from a donor into fighting machines that beat the cancer.
The technique, called BE-CAR7, is a highly precise tool for changing individual letters in the genetic code, the instruction manual for all cells in the body.
Changing just one letter – what scientists call DNA “bases” – can alter the function of a gene in much the same way as substituting a single key letter in a text message can change its meaning.
The scientists made three individual “base-edits” to the donor T-cells.
The DNA tweaks made the cells an “off-the-shelf” treatment that didn’t need to be matched to patients like other transplants. And they also instructed the donor T-cells to destroy every one of a patient’s own T-cells – whether they were cancerous or not.
If all the T-cells were eradicated within four weeks of treatment, patients were able to go on and have a bone-marrow transplant to rebuild a healthy immune system.
Alyssa ‘doing really well’
According to results published in the New England Journal of Medicine, 82% of the patients in the trial were in “very deep remission” after treatment and were able to have a transplant.
And 64% remain disease-free.
Alyssa said she was now “doing really well”.
“I’ve gone sailing, spent time away from home doing my Duke of Edinburgh Award but even just going to school is something I dreamed of when I was ill,” she said.
“I’m not taking anything for granted.
“Next on my list is learning to drive, but my ultimate goal is to become a research scientist and be part of the next big discovery that can help people like me.”
Hospital charity agrees to support treatment of 10 more patients
The Great Ormond Street Hospital Charity has now agreed to support treatment for another 10 patients.
Dr Rob Chiesa, study investigator and bone marrow transplant consultant at GOSH, said: “Although most children with T-cell leukaemia will respond well to standard treatments, around 20% may not.
“It’s 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.”
Results ‘bring hope’
UK stem cell charity Anthony Nolan worked with the research team to provide T-cell donors.
Dr Tania Dexter, senior medical officer at the charity, said: “Considering these patients had a low chance of survival before the trial, these results bring hope that treatments like this will continue to advance and become available to more patients.
“As with any novel cellular therapy, this phase 1 trial is just an initial indication of the effectiveness and safety of the treatment, and more work must be done to determine its wider clinical application.
“Yet the results are encouraging and demonstrate the recent leaps in technology that are allowing us to take on even greater challenges in the treatment of blood cancers and blood disorders.”
NHS England has warned the health service is facing a “worst case scenario” December with a surge of “super flu”.
The warning comes as new figures revealed the number of people in hospital with flu have increased by more than half in just one week.
Catch up as it happened: NHS warns of ‘worst-case scenario’ as ‘super flu’ surges
Latest figures show:
• An average of 2,660 patients were in hospital per day with flu last week
• This is the highest ever for this time of year and up 55% on last week
• At this point last year the number stood at 1,861 patients, while in 2023 it was just 402
Health service bosses are warning the number of flu patients in hospital has already increased sharply since the week covered by this data – with no peak in sight.
Read more: Why is flu season worse this year?
Virus outbreaks coincide with doctors’ strikes
Weekly flu numbers in England peaked at 5,408 patients last winter and reached 5,441 over the winter of 2022/23, the highest level since the pandemic.
Alongside rocketing flu, the number of norovirus patients in hospital has also risen by 35%.
The NHS is now warning winter viruses are starting to “engulf hospitals”.
Demand for A&Es and ambulance services is also soaring.
New monthly figures show A&E attendances were a record for November at 2.35 million – more than 30,000 higher than November 2024.
In addition, there were 48,814 more ambulance incidents (802,525) compared with last year (753,711).
Some hospitals across the country have asked staff, patients and visitors to wear face masks to cut the spread of flu, while others have gone in and out of critical incident status due to the high number of people attending A&E.
The record-breaking demand on the NHS coincides with a resident doctors’ strike from 17 to 22 December over pay and jobs – sparking fears of major disruption for patients in the run up to Christmas.
People are being advised to attend any planned appointments scheduled during the strikes unless they have been contacted to reschedule.
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Will doctors accept late deal to avoid strikes?
Flu vaccinations on the up… who can get one?
The NHS is urging anyone eligible to get their flu vaccination to help prevent them getting seriously ill.
Latest figures show more than 17.4 million people have been vaccinated so far this year, more than 381,000 higher than last year.
You can get it if you:
• Are 65 or over in England, Scotland, Wales and Northern Ireland
• Are pregnant
• Live in a care home
• Are the main carer for an older or disabled person, or receive carer’s allowance
• Live with someone who has a weakened immune system
• Are a frontline health and social care worker
• Are of school age
• Have certain medical conditions (the NHS has a full list)
Wes Streeting, the health secretary, warned of a “tidal wave of flu tearing through our hospitals”.
“We are working with the NHS to make sure it is able to cope with this as best as possible,” he said.
Warning ‘extremely challenging few weeks ahead’
Professor Meghana Pandit, NHS national medical director, warned the health service faces “an extremely challenging few weeks ahead” with “staff being pushed to the limit”.
She said: “With record demand for A&E and ambulances and an impending resident doctors’ strike, this unprecedented wave of super flu is leaving the NHS facing a worst-case scenario for this time of year – with staff being pushed to the limit to keep providing the best possible care for patients.
“The numbers of patients in hospital with flu is extremely high for this time of year. Even worse, it continues to rise and the peak is not in sight yet, so the NHS faces an extremely challenging few weeks ahead.”
She added: “We have prepared earlier for winter than ever before, and stress-tested services to ensure people have a range of ways to get the help they need and avoid needing to go to A&E.
“For non-life-threatening care, people should call NHS 111 or use 111 online, which can direct you to the most appropriate place, and use A&E and 999 for life threatening conditions and serious injuries.”
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Mr Streeting has offered the British Medical Association (BMA) a last-minute deal in the hope doctors will call off the walkout, which starts next Wednesday.
The doctors’ union has agreed to put the offer to members over the coming day, and is expected to announced a decision on Monday, just two days before the planned strike.
The offer includes a fast expansion of specialist training posts as well as covering out-of-pocket expenses such as exam fees, but does not include extra pay.
The spread of subclade K, the super flu, is picking up – and it’s not looking good for the NHS.
An average of just under 2,700 hospital beds a day in England were occupied by patients with flu last week.
There are fears that could jump to somewhere between 5,000 and 8,000 by the end of this week.
That’s on top of COVID and RSV.
And with the possibility of a five-day strike by resident (junior) doctors next week, it’s a perfect storm for hospitals.
An NHS hospital ward at Ealing Hospital in London. Pic: PA
Christmas flu
Children are the super-spreaders of flu. It races around classrooms and some schools have temporarily shut because of the impact.
The Christmas holidays aren’t far off. They are likely to put the brakes on children passing around the virus.
But it’s also a time of year when families mix with elderly relatives, who are more likely to be hit hard by the infection – perhaps even needing hospital care.
So while the holidays may temporarily slow the overall rise in infections, the impact on hospitals could get much worse.
File pic: PA
Subclade K
Flu is spreading so rapidly at the moment because immunity to subclade K from previous infections and vaccinations is low.
The virus – a variant of the H3N2 flu strain – suddenly acquired seven new mutations in late summer.
Every 100 people infected with seasonal flu would typically pass the virus on to 120 others.
With subclade K, it’s 140.
And that’s why cases are rising so quickly on the charts.
At the moment, 18 in every 100,000 patients in England are consulting their GP with flu-like symptoms. That’s still well short of the peak of around 50 in every 100,000 in 2017/18, the worst flu outbreak in recent years.
File pic: PA
The grim reality of flu
Flu is a really unpleasant disease, nothing like a cold. I’ve had it twice in my life and it physically hurt to get out of bed. It’s grim.
Most people get over it with a few days’ rest and paracetamol to take the edge off the fever.
But vulnerable people can become seriously ill. In the outbreak of 2017/18, around 22,000 died.
Read more:
Nurses ‘deeply concerned about what lies ahead’
NHS braced for ‘toughest winter’ after record-breaking month
That’s why the NHS is urging people in certain groups – the over 65s, those with underlying health conditions, pregnant women, carers and children – to get the jab.
The vaccine isn’t a great match for subclade K, but still reduces the chance of hospital admission by 30-40% in adults.
It’s impossible to say when the spread will peak, but the latest figures suggest the outbreak is far from over.
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