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NHS si prepara a offrire un trattamento contro il cancro rivoluzionario: Il capo del servizio sanitario chiede un accesso accessibile a CAR-T, che modifica il sistema immunitario per distruggere le cellule tumorali

Il NHS is preparing to fast-track a game-changing cancer treatment into hospitals, ha detto il suo amministratore delegato, chiedendo ai produttori di aiutare fissando un prezzo accessibile. Simon Stevens said CAR-T therapy, which has been licensed in the US but not yet in the UK, could be approved for use this year. The treatments, which are hugely expensive, work by genetically engineering the patient’s immune system’s killer T-cells to recognise and destroy cancer cells.

In trials, they have had extraordinary success, putting into remission patients who had only months to live.

The first patient to receive CAR-T therapy was treated in the US in 2011. Da allora, several hundred people have received the treatment.

The cost in the US is $475,000 (£340,000) per patient, which is far in excess of the normal NHS ceiling of £50,000 per year of good-quality life for an end-of-life drug.

But Stevens says this is technology the NHS must embrace. Speaking to the Association of the British Pharmaceutical Industry, he appealed for fair prices.

“The NHS has a proud history of delivering pioneering treatments. As we celebrate the 70th anniversary, the NHS is working harder than ever to save lives and improve care by embracing cutting edge technology like CAR-T therapy and spreading innovation across the whole health service," Egli ha detto.

"Tuttavia, we can’t do this alone, and we need the help of the manufacturers to ensure we can get these treatments to patients as quickly and cost-effectively as possible.

“Preparations are under way to make CAR-T, one of the most innovative treatments that has ever been offered on the NHS, available to patients, but manufacturers need to set fair and affordable prices so treatments can be made available to all who need them.”

Two treatments for children with acute lymphoblastic leukaemia and for adults with lymphoma have been approved in the US and are with European regulators.

In advance of the licensing decision, the Department of Health has referred them to the National Institute for Salute and Care Excellence, which decides whether treatments are cost-effective. If the treatments pass both hurdles, the NHS could adopt the technology this year.

But the manufacturer’s price is not the only cost. Delivering the personalised therapies to patients is also expensive. CAR-T therapies work by taking blood from the patient and extracting the T-cells, which ought to kill off cancer cells but in these advanced cancers fail to recognise them.

The cells must be shipped to a lab where they are genetically engineered to produce receptors on their surface that will recognise cancer cells, allowing the patient’s immune system to destroy them.

“It is a very exciting time,” said Emma Morris, professor of clinical cell and gene therapy at University College London hospital, which has the largest number of trials of these sorts of therapies open in Europe.

“There is a whole body of work being done to draw up service specifications for centres in the UK at NHS hospitals to determine what the requirements are to deliver this kind of therapy to patients, because they are very complex.

“Currently we are only delivering them [in clinical trials] in two or three centres in the UK and run by transplant teams used to giving cell therapies.”

Although there have been spectacular results in the trials, there have also been deaths when the immune system has overreacted to the reintroduced T-cells. Some patients will need intensive care.

The first treatments are for blood cancers. Alasdair Rankin, director of research at the UK blood cancer charity Bloodwise, called the NHS plans a “ really positive step for some people living with cancer whose lives cannot be saved using the treatments doctors can already provide”.

He echoed Stevens’ call for a fair price from manufacturers to allow the therapies to be used and develop. “It’s important to recognise that this is just the beginning for a completely new type of cancer therapy,” Rankin said.

“While it is an exciting time, even once the first therapies are approved, only a small number of people will be treated in this way at first and not unless other forms of effective cancer treatment have been shown not to work. However we can expect to see CAR-T therapy develop significantly over the next decade.”

Researchers are investigating ways to adapt the therapy to other solid tumours.

Prof Charles Swanton, Cancro Research UK’s chief clinician, said it was “very exciting to see that the NHS is exploring the very latest developments in immunotherapy for cancer patients”.

“CAR-T cell therapies have shown great promise in some patients with specific types of blood cancer, but the treatment is complicated, highly personalised and can cause severe side effects in some patients," Egli ha detto.

“The process of genetically altering a patient’s own immune cells is immensely complex and also very expensive. We hope that manufacturers can agree on a deal that ensures affordable access for patients in the UK as soon as possible, to achieve long-term remissions and even cures.”

fonte:, by Sarah Boseley

Di Marie

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