World first innovation in cancer therapy at Great Ormond Street

The twinkling eyes and smile of Layla Richards shine out from many newspaper front pages today – and for a newsworthy reason. A world first treatment for her Acute Lymphoblastic Leukaemia was tried out by doctors at London’s Great Ormond Street Hospital. 

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All of this went on without the Medical Innovation Bill or Access to Medical Treatments (Innovation) Bill.

Photo: Great Ormond Street Hospital

Lord Saatchi has consistently framed the need for his legislation with tales of denials of a potential cure, ‘wasted lives’, ‘cut and drilled’ on traumatic trajectories to death, as an uncaring and purely self-interested medical profession looks on.    

‘Current law in relation to medical negligence contributes to this failure. It defines medical negligence as deviation from standard procedure. But as innovation = deviation, then non-deviation = non- innovation.

The result is that all cancer deaths are wasted lives. The deaths, and their attendant tragedies, have not advanced scientific knowledge by one centimetre.

Why?

Because all the deceased have received is the standard procedure – the endless repetition of a failed experiment.’
–  Lord Maurice Saatchi

The rationale for the legislation was, apparently, and as has been written about extensively, to change the law and in turn encourage more doctors to innovate. 

The ‘current law obliges the doctor to follow the status quo, even though he/she knows it leads only to poor life quality followed by death.’

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His claims have been repeated in briefing documents, in the press, at meetings, in the House of Lords and the House of Commons.

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There is no need to change the law of negligence – as this already permits innovative practice. Nor is there any evidence to suggest any case in negligence has arisen due to responsible innovation in treatment, regardless of the outcome. Indeed there are many examples where the legal position is used expressly to favour an innovative approach. 

Those responding to the consultation and responding in an unwaveringly loud chorus against the proposals of any variation of the Bill have made it clear that if there are barriers to innovative treatment, the law of negligence is not the issue. Changing it will not help doctors, and will not help patients. Nor will it be inert. It is a deeply misguided attempt that is not based on evidence – ‘a solution in search of a problem and not  very sound solution at that’, surmised the National Assembly for Wales. The interim report of the Accelerated Access Review led by Sir Hugh Taylor makes no mention of the law of negligence or the fear of it as an obstruction to accessing innovative treatments. No legal, medical or research organisation supports this assertion either. The proposals would destroy the law that operates responsively and permissively to allow responsible innovation to happen today. 

It is time for Chris Heaton-Harris, Lord Saatchi and the Department of Health to recognise when it’s time to stop and- if truly concerned about patients and access to treatments-  assess any of the genuine barriers to access and support a change that would help, rather than harm doctors and patients – children like Layla in future. 

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Photo: Great Ormond Street Hospital

Layla’s case

Read: Full account of Layla’s experience at GOSH

Baby Layla was diagnosed with ALL at 14 weeks. She received a bone marrow transplant and chemotherapy but they were not successful.  Shortly before her first birthday all normal treatments were exhausted, and her cancer was incurable. Her parents asked if anything else could be done.

A highly experimental therapy – tried only in mice- used TALEN gene editing technology to enzymatically deactivate genes in healthy donor T cells and precisely edit DNA to create ‘designer ‘cells targeted to the cancer that would withstand the necessary medication. After a 1ml infusion of 50m engineered cells over 10 minutes, a second bone marrow transplant 2 months later and now just 3 months down the line, baby Layla has a restored immune response and has been declared ‘cancer free’.   This is the first time- ever- that human cells engineered using this technology have been given back to a patient. The first trial of gene editing therapy was published last year, where an approach using a different DNA editing enzyme was used for HIV. 

Layla’s case will require very close follow-up and monitoring to ensure that the therapy is as effective as it appears to be, and a trial is planned for the approach next year. 

As Professor Waseem Qasim said: “We have only used this treatment on one very strong little girl and we have to be cautious about claiming this will be suitable for all children. But this is a landmark in the use of new gene engineering technology and the effects on this child have been staggering. If replicated, it could represent a huge step forward in treating leukaemia and other cancers.”

Here’s to Layla and her family – fantastic and all the very best!

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I’ve been somewhat confused, says MP

Friday morning saw the second reading of the Access to Medical Treatments (Innovation) Bill – the latest incarnation of the Saatchi Bill – take place in the House of Commons.

Now, the list of those opposing the latest rehash of the Saatchi theme includes the Medical Royal Colleges, the BMA, the Association of Medical Research Charities and its constituent members such as Cancer Research UK, and many more. This was pointed out clearly and indefatigably. 

Dr Sarah Wollaston (Totnes) (Con): As with any book, we should not judge a Bill by its cover. All Members want to improve access to innovative medical treatments, but I sincerely believe that the Bill is not the right way forward. My hon. Friend the Member for Daventry (Chris Heaton-Harris) referred to a number of organisations, implying that they are in favour of the Bill, but he knows that the overwhelming majority of research and charitable organisations are ranged against it. It is opposed by the Association of Medical Research Charities, whose membership reads like an “A to Z” of expertise, including bodies such as Cancer Research UK, the Wellcome Trust—the list is very long, so I will not detain the House by reading it out. The Academy of Medical Sciences opposes the Bill, as does the Academy of Medical Royal Colleges, including all those he quoted in his speech.

The General Medical Council, the British Medical Association and the Patients Association oppose the Bill, and I direct my hon. Friend to their article in The Guardian. Action against Medical Accidents, and even the Association of the British Pharmaceutical Industry, oppose the Bill because of its unintended consequences. Legal experts, including Sir Robert Francis, firmly oppose the Bill. All those organisations oppose the Bill because it is unnecessary, it is unworkable, it would unravel important patient protections and, most importantly, it would have unintended and dangerous consequences for research.

I pay tribute to all the Bill’s sponsors and absolutely understand that they are motivated by very good intentions. I would love to sit down and work with them on how we genuinely improve access to innovative treatments. I hope they understand that I oppose the Bill because I sincerely believe that it is the wrong way forward.

Both Dr Wollaston and Labour Shadow Secretary of State for Health Heidi Alexander spoke with real and carefully applied insight to explain why there’s such concern about the baseless and ‘irresponsible’ proposals. 

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Unfortunately, with only 51 MPs voting – despite the unwavering censure toward the latest version from expert medical organisations, research and patient safety charities –  it passed its second reading at 32 votes to 19. 

Mr Heaton-Harris  set about spinning support for the premise behind the Bill – despite his quoted organisations having reiterated their “unequivocal opposition” in new briefing notes. 

Dr Wollaston: My hon. Friend has quoted a number of organisations. Does he accept that all those organisations oppose the Bill? He needs to make that explicit to the House. It is not fair to quote the Royal College of Surgeons, for example, without making it clear that it has explicitly opposed this Bill.

This was nothing compared to the great lashings of nonsense brought forth by Philip Davies MP,  who selectively quoted snippets of consultation responses predating April 2014, out of context, defended by announcing “I am perfectly happy for people to put their own gloss on what others are saying” – when it was pointed out that others aren’t even saying anything like that, Davies continued to derail proceedings, talk in circles, and throw in half sentences, historic quotations, and confabulatory haze for over an hour.

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Seems he was at his filibustering best – most likely because of the next slot.

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The Homes (Fitness for Human Habitation) Bill was an inconvenience to him that he succeeded in talking out.

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Next up David Nuttall MP who managed to speak for many minutes without pausing to let in fact, and George Freeman MP- setting out the government’s position- conflating, again,the Early Access to Medicines Scheme with the Saatchi monster, and fudging plentifully.  Both denied that it would affect the law of negligence; so too did Chris Heaton-Harris MP. This was very interesting, seeing as its operative effect is to change the law of negligence.

Nuttall demonstrated how the Saatchi defence to negligent decisions operates:

‘I have to admit at the outset that an impressive array of bodies have lined up either in outright opposition to the Bill or with at least some reservations about it. When I was considering the evidence, I had to take that into account. I had to decide whether in the light of that evidence I should simply go with the flow and decide that if all those people said that it is a bad thing, it must be a bad thing, or whether I should think about the other side of the coin. I did that, and on balance, I came down on the side of what I like to think of as my constituents’ view. I believe that the Bill has the potential to improve the lives of my constituents if they are struck down by a rare disease that means that they require innovative medical treatment.’

Both Nuttall and Freeman mentioned litigation costs – but omitted that the known figure for litigation relating to innovation is … £0.

Dr Wollaston: How much of the litigation cost is related to complaints about innovative treatments?

George Freeman: Most of the cases are a result of other contexts – as my hon. Friend will know, obstetrics is a big part of that – rather than innovation. I am happy to write to her with the actual figure as I do not have it to hand…

All other pertinent questions were similarly obfuscated around.

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Staggeringly the Access to Medical Treatments (Innovation) Bill edition of the Saatchi Bill was voted through to committee stage in the House of Commons, and while claiming it’s been ‘handed over’ to Heaton-Harris, Lord Saatchi’s Medical Innovation Bill remains in the House of Lords- alongside his motion to suspend SO46. 

HA.

Professor José Miola on the nonsense: ‘It’s like déjà vu all over again…’

What can be done?  Despair, despair some more – then contact your MP  ‘Call to action!’