'Game Changer' CAR-T Therapy for Adult Cancer Patients

Peter Russell

October 05, 2018

The Health Secretary, Matt Hancock, welcomed an announcement this week that a price deal would allow a gene therapy treatment to be available for lymphoma patients in England.

There was news of a breakthrough in discussions between the National Institute for Health and Care Excellence (NICE) that could potentially lead to a treatment for cystic fibrosis becoming available for routine NHS use in England and Wales.

This week also brought an announcement that NICE would reconsider its guidance on treating and managing adult depression by including up-to-date evidence.

CAR-T Therapy for Adult Lymphoma Patients

Adult cancer patients in England will be eligible to receive the chimeric antigen receptor T-cell (CAR-T) therapy axicabtagene ciloleucel (Yescarta, Gilead) following a confidential deal on the cost of treatment struck with the manufacturer.

NHS England said that axicabtagene ciloleucel could benefit up to 200 patients a year whose large cell lymphoma has returned or has stopped responding to previous treatment.

Matt Hancock, secretary of state for health and social care, said the therapy was "game-changing" and that the decision meant that NHS patients in England would be among the first in the world to benefit.

The treatment works by genetically modifying the patient's own T cells so that they make a protein called chimeric antigen receptor (CAR), which can attach to and kill cancer cells.

Axicabtagene ciloleucel is licenced to treat adult patients with diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma after two or more previous therapies have failed. Trials indicate that the therapy could potentially cure 40% of patients, according to NHS England.

Simon Stevens, NHS England chief executive, said: "CAR-T cell therapy is one of the most promising new treatments in a generation for lymphoma and leukaemia."

The announcement follows a deal NHS England reached last month to make rival CAR-T therapy tisagenlecleucel (Kymriah, Novartis) available for children and young people with B-cell acute lymphoblastic leukaemia.

Lumacaftor–ivacaftor for Cystic Fibrosis

Long-standing uncertainties over lumacaftor–ivacaftor (Orkambi, Vertex Pharmaceuticals) appear to be closer to resolution after NICE announced on Friday that it had held a "productive" meeting with the manufacturer this week.

The health watchdog announced more than 2 years ago that it would not recommend the systemic protein modulator for treating cystic fibrosis in people 12 years and older who were homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

A NICE spokesman said: "Vertex and NICE held a productive meeting on 4th October and have committed to continuing their discussions over the appraisal of treatments for cystic fibrosis."

Rethink on Adult Depression

NICE announced this week that it would reconsider the most up-to-date research on managing depression in adults before issuing treatment guidance.

It follows complaints from experts that its draft proposals were "not fit for purpose".

A position statement, signed by 14 organisations including the Royal College of Psychiatrists (RCP), and six leading doctors, raised serious concerns about "significant flaws in methodology, lack of transparency and several inconsistencies" in the document.

They warned that if published, it would "seriously impede the care of millions of people in the UK suffering from depression, potentially even causing clinical harm".

As a result, NICE has announced it will produce a new draft guideline. Paul Chrisp, director of the centre for guidelines at NICE, said: "It is important that the final recommendations are based on the most up-to-date evidence possible."

Work will begin in December 2018 with final publication of the guideline due in December 2019.

Renal Replacement Therapy

People being treated with dialysis after kidney failure should be offered a choice over where and what type of treatment they have.

New guidance from NICE said patients, in discussion with their clinician, should be able to choose whether to have peritoneal dialysis, haemodialysis, or haemodiafiltration. They should also, subject to local arrangements, be able to choose whether treatment takes place at home or in hospital.

The committee agreed that clinicians should consider offering haemodiafiltration in the first instance to patients who opt for treatment in hospital.

Children under the age of 2 will continue to be offered peritoneal dialysis in the first instance.

Paul Chrisp, director for the centre for guidelines at NICE, said: "Dialysis is a time consuming procedure. Allowing a patient to decide where and when they have their treatment will allow them to lead the life they want."

iFuse Implants for Sacroiliac Joint Pain

The iFuse implant system should be considered for treating people with a confirmed diagnosis of chronic sacroiliac joint pain.

The iFuse implant system (SI-Bone) is a titanium implant for stabilising the joint and correcting misalignment or weakness.

Using iFuse led to improved pain relief, better quality of life, and less disability compared with non-surgical management, NICE said in final guidance.

Tofacitinib for Psoriatic Arthritis

Final guidance recommended tofacitinib (Xeljanz, Pfizer), with methotrexate, as an option for treating active psoriatic arthritis in adults who have not responded adequately or have been intolerant to disease-modifying anti-rheumatic drugs (DMARDs).

An appraisal committee set a number of conditions for treatment.

Tofacitinib is the first of a new class of drugs known as Janus kinase inhibitors. In clinical trial, it was found to be more effective than placebo at treating joint and skin symptoms.

NICE decided that tofacitinib was likely to be cost-effective when used after two conventional DMARDs, or after treatment with a TNF-alpha inhibitor after two conventional DMARDs.

Cabozantinib for Renal Cell Carcinoma

Cabozantinib (Cabometyx, Ipsen) was recommended for routine NHS use in adults with untreated advanced renal cell carcinoma that is intermediate or poor-risk.

In final guidance, NICE said evidence showed that cabozantinib extends the time until cancer progresses compared with usual current treatments pazopanib (Votrient, Novartis) and sunitinib (Sutent, Pfizer).

However, NICE said uncertainties remained about how much extra benefit cabozantinib offered patients.

Mental Capacity

NICE published its guideline this week on decision-making and mental capacity.

It is designed to ensure that practitioners are trained to apply the Mental Capacity Act 2005, which was designed to empower and protect individuals in England and Wales who may lack the capacity to make certain decisions for themselves at some point because of illness, injury or disability.

The Care Quality Commission (CQC) estimates that around 2 million people in England and Wales may be affected in this way at some point in the lives.

In a blog, Nageena Khalique QC, chair of the guideline committee, said: "It applies to a range of decisions including care, treatment, financial matters, day-to-day living and emergencies."


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