World first use of gene-edited immune cells to treat ‘incurable’ leukaemia

A new treatment that uses ‘molecular scissors’ to edit genes and create designer immune cells programmed to hunt out and kill drug resistant leukaemia has been used at Great Ormond Street Hospital (GOSH).

The treatment, previously only tested in the laboratory, was used in one-year-old, Layla, who had relapsed acute lymphoblastic leukaemia (ALL). She is now cancer free and doing well.

This breakthrough comes from GOSH and UCL Institute of Child Health’s (ICH) pioneering research teams with support from the National Institute for Health Research (NIHR) Great Ormond Street Biomedical Research Centre, who together are developing treatments and cures for some of the rarest childhood diseases.

Chemotherapy successfully treats many patients with leukaemia but it can be ineffective in patients with particularly aggressive forms of the disease where cancer cells can remain hidden or resistant to drug therapy. Recent developments have led to treatments where immune cells, known as T-cells, are gathered from patients and programmed using gene therapy to recognise and kill cancerous cells. Multiple clinical trials are underway, but individuals with leukaemia, or those who have had several rounds of chemotherapy, often don’t have enough healthy T-cells to collect and modify meaning this type of treatment is not appropriate.

Continue reading “World first use of gene-edited immune cells to treat ‘incurable’ leukaemia”


Trial leukaemia drug ‘saved my life’

A grandfather, who was given two years to live after being diagnosed with leukaemia, is in remission after taking a new trial drug.

Douglas Clarke, from Stoke-On-Trent, was one of 100 patients chosen by The Christie Hospital to trial the drug ABT-199, while he was waiting for a life-saving bone marrow transplant.

Just three weeks after taking the tablets, consultant haematologist Dr Adrian Bloor told Mr Clarke that he was in remission and could live for another 30 years. Continue reading “Trial leukaemia drug ‘saved my life’”

Research trial saved my son’s life

A 14-year-old who was the first child in the country to take part in a global drug trial has returned to school after being in remission for over a year – a fact his mum puts down to his participation in the trial.

At the age of 11 James Morley started seeing red wherever he looked. Knowing something was wrong his parents took him to see an optician, who could see blood cells bursting in his eyes. James, from Sutton-in-Ashfield, was then rushed to the Queen’s Medical Centre in April 2012 where he was diagnosed with Philadelphia positive acute lymphoblastic leukaemia – a rare illness in children. Continue reading “Research trial saved my son’s life”

The cancer girl cured by the HIV virus: Seven-year-old makes extraordinary recovery after U.S. doctors re-wire her immune system to destroy leukaemia cells

In April this year, Emily Whitehead’s family had almost given up hope.

The brave six-year-old had been fighting leukaemia for two years, only to relapse for a second time during intensive chemotherapy treatment in February.

Doctors had exhausted all the traditional treatments as Emily could not remain in remission for long enough to attempt a bone marrow transplant. So her desperate parents, Kari and Tom, started looking at more radical options. Continue reading “The cancer girl cured by the HIV virus: Seven-year-old makes extraordinary recovery after U.S. doctors re-wire her immune system to destroy leukaemia cells”

Cancer Survivor Applauds Clinical Trial ‘Miracles’

“Thank you for your efforts to make miracles happen,” Keith Eaton, a leukemia cancer survivor and physician, told attendees at the Association of Clinical Research Professionals (ACRP) 2017 Meeting & Expo in Seattle this morning.

Clinical trial professionals follow a higher calling and should be proud of their industry and their achievements, he affirmed. “Clinical trials are people’s hope when there are no standard options.” Continue reading “Cancer Survivor Applauds Clinical Trial ‘Miracles’”

Taking part in a trial to transform treatment

Deciding to take part in a clinical trial

“Alice was diagnosed with sJIA when she was four,” says Nerys. “She initially went to our local hospital who thought she had something called Kawasaki disease and we were then sent straight up to GOSH.  After lots of tests we were told it wasn’t Kawasaki disease but arthritis. Alice needed to take methotrexate and steroids, but these aren’t meant to be taken long-term.

“We had a call [from the Clinical Research Facility at GOSH] asking if we would be interested in taking part in a clinical trial, which we definitely were. Taking part in a trial was obviously a little bit daunting as you want to do your best for your child. It does have a little bit of frightfulness but we had all the information from the Clinical Research Facility that we could take away and take the time to make an informed decision.

“Initially we were on the placebo part of the trial but Alice’s stats were carefully monitored and after a short amount of time we were given the tocilizumab drug, which ever since has been wonderful for her.”

At the time, the trial was the largest randomised placebo controlled study into this relatively rare disease group, taking place in 43 centres around the world. At GOSH, the trial was led by Professor Pat Woo and the Rheumatology team.

Alice’s trial was to discover if tocilizumab, which was already licensed for adults, could work for children. The drug has now been licensed for children and has become the standard of care for sJIA, offering an alternative treatment where other drugs fail to work.

Being on a clinical trial at GOSH

“Being on the trial was an innovating experience, we enjoyed it whole heartedly and the level of care that the facility gave was amazing,” says Nerys. “The attention for the child going through a trial was amazing. Alice is not particularly fond of needles but the time taken and patience with Alice was amazing.

“GOSH is a great place. I always grew up thinking it was the best place to go and still think it is. We have been provided with the best care.

“I am absolutely thankful that they caught Alice’s disease when they did and for bringing her onto the trial when they did. Things could have been a whole lot different for Alice than they are now.  And so I will always be grateful for Alice’s life as it is at the minute. It could have been a whole different scenario.”

Now the trial has ended, Alice is still followed up at GOSH but receives tocilizumab at her local hospital.

What the trial means to Alice

“I have sJIA which is systemic Juvenile Arthritis, an autoimmune disease which means my white blood cells attack my joints instead of doing the job they should, so it affects my joints,” says Alice. “I am quite stiff in the morning. I can’t run as fast as other people at school. But now I don’t really think about my condition day to day, as it doesn’t hurt anymore.

“I think other children should consider taking part in clinical trials because you could get better and you could help a lot of other people get better.”

World first drug trial at The Christie makes Bob’s cancer disappear without a trace

A patient, whose cancer spread from his lungs to his lymph nodes has been left with no trace of the disease after taking part in a first in world drug trial at The Christie in Manchester.

Bob Berry, 60 from Hazel Grove, Stockport started the first in human and first in class drug trial when he was referred to the clinical trials unit at the specialist cancer centre 12 months ago, because he wasn’t responding to standard treatment. Continue reading “World first drug trial at The Christie makes Bob’s cancer disappear without a trace”