Great Ormond Street’s been put on a pedestal it doesn’t deserve, says father of disabled girl treated there

The lonely death of Baby P was one of those moments that jolted the entire nation. That picture of the blond-haired infant in his blue jumper looking pleadingly into the camera became an iconic image, symbolising the horrors visited on those who fall through the fissures in society.

The death led to a series of inquiries, with social workers sacked and a doctor struck off. Four years on, the shockwaves still reverberate. Now they have rocked one of Britain’s most revered institutions. Continue reading “Great Ormond Street’s been put on a pedestal it doesn’t deserve, says father of disabled girl treated there”

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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”

NHS pays out more than £100MILLION in compensation to families of patients who died in hospital because of medics’ blunders

The NHS has paid out more than £100million in compensation to the loved ones of patients who died as a result of medics’ blunders.

More than 20 families each week receive huge settlements as a result of negligent care which has ended in death.

New figures from the NHS show there have been more than 2,300 successful cases in the last two years where the family of patients have sued, winning a total of £108million in compensation. Continue reading “NHS pays out more than £100MILLION in compensation to families of patients who died in hospital because of medics’ blunders”

Boy left brain damaged after Great Ormond Street ‘ignored advice of his medical scientist mother’

Gabrielle Ali says her son’s disabilities – which mean he cannot walk or talk – are the result of a series of blunders by Great Ormond Street.

An 11-year old boy has been left brain damaged after a leading hospital ignored the advice of his medical scientist mother, she claims.

Gabrielle Ali says her son’s disabilities – which mean he cannot walk or talk – are the result of a series of blunders by Great Ormond Street.

After her son developed a complication following routine surgery at the world famous hospital, she was told to take son Elijah to her local hospital.

Continue reading “Boy left brain damaged after Great Ormond Street ‘ignored advice of his medical scientist mother’”

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.”

 

http://www.gosh.nhs.uk/research-and-innovation/taking-part-research-0/taking-part-trial-transform-treatment