UNLOCK DARA-CYBORD: CHARITY FIGHTS BACK AFTER PATIENTS DENIED FIRST TREATMENT FOR RARE INCURABLE DISEASE

Patients have been dealt a major blow after the first ever treatment for AL amyloidosis, a rare incurable condition, was rejected by the National Institute for Health and Care Excellence (NICE). 

This is a significant setback for patients, who currently have to rely on treatments designed for other conditions. 

Up to 600 patients would have benefitted from the life-extending treatment, known as Dara-CyBorD, each year. 

The news comes three months after the same treatment was approved and rolled out to newly-diagnosed patients in Scotland on the NHS.  

Blood cancer charity Myeloma UK, which supports patients with the condition, is now fighting back with the launch of #UnlockDaraCyBorD. 

As part of the campaign, the charity is calling on NICE to make the pioneering drug combination Dara-CyBorD available to newly-diagnosed patients in England and Wales before it’s too late. 

Myeloma UK is also urging MPs to back the campaign and give patients a chance at a life worth living post-diagnosis. 

As it stands, around a quarter of patients die within six months of being diagnosed. 

A pioneering four-drug combination, Dara-CyBorD has been shown to be effective in clinical trials with patients five times more likely to have a complete response to treatment. 

59% of patients who received Dara-CyBorD also had no sign of AL amyloidosis following treatment and longer remission times. 

Yet it has now been rejected by The National Institute for Health and Care Excellence (NICE) for patients in England and Wales on the grounds it is not cost-effective. 

NICE also argued there was insufficient data to show how much longer patients live for post-treatment. 

AL amyloidosis is a rare incurable condition which affects multiple parts of the body and can severely damage the kidney and heart. 

Between 3,000 and 4,000 people have AL amyloidosis in the UK. 

The new quadruplet treatment would have seen daratumumab (Darzalex®) used in combination with cyclophosphamide, bortezomib (Velcade®) and dexamethasone. 

All four drugs are already individually available on the NHS. 

Dr Sophie Castell, Chief Executive of Myeloma UK, a charity advocating for patients with both AL amyloidosis and the incurable blood cancer myeloma, said: “This decision is a huge setback for the AL amyloidosis community and, quite frankly, utterly unacceptable. We know this new drug combination works and helps to improve people’s quality of life and remission times. 

“Patients have told us time and time again how hopeless they feel knowing that they’ve been diagnosed with a condition which has no dedicated treatment option. To keep this game-changing treatment out of their reach is devastating. Particularly when fellow patients in Scotland have benefitted from Dara-CyBorD on the NHS for months now. 

“Access to vital treatments should not come down to a postcode lottery. We will appeal this decision and we’re calling on MPs to join the fight for Dara-CyBorD. We won’t rest until Dara-CyBorD is available to every last patient who needs it, no matter where they live.” 

Overall, between 500 and 600 people are diagnosed with AL amyloidosis in the UK every year. 

It is a relapsing-remitting condition. This means patients have periods of remission after treatment, when the AL amyloidosis is not active or causing symptoms, but it will become active again after a period of time. 

Life expectancy is highly variable depending on the level of organ damage, how the condition is treated and how well patients respond to treatment. 

Those who respond well to treatment have a life expectancy of ten years or more. 

Huw Stiley, 55, from London, served as a patient expert, championing the voice of the AL amyloidosis community at NICE’s appraisal committee. He said: “Any newly-diagnosed amyloidosis patient in England and Wales will be devastated to learn a treatment pathway available in Scotland has now been denied to them. The proposal issued to Scotland was broadly identical, one would assume, to the one issued to NICE. How can two different panels arrive at two completely different conclusions? If Scotland has approved Dara-CyBorD, that implies the treatment delivers tangible clinical results and an improved quality of life for patients.” 

Huw, who was diagnosed in 2016, added: “It feels like there is a monetary dimension to the decision and I sincerely hope the drug company and NICE can find a way forward. Amyloidosis patients in England and Wales deserve a treatment pathway just like Scotland.” 

Patrick Smyth, from Sunbury-on-Thames, was diagnosed with a rare type of amyloidosis back in 2010 – the day before his 50th birthday. After being “pushed from pillar to post and getting nowhere” on the NHS, he went private. His decision to go private meant he was able to receive treatments unavailable to many on the NHS and which have “held the disease at bay.” 

“I’ve often felt guilty about the fact that I’ve been able to have this treatment when other people, who felt a lot sicker than me, have not been able to have the same kind of treatment,” said the 62-year-old, who is now on a mixture of NHS and private care. “It needs to be evened up. I see people being very ill. It’s an illness that’s growing and treatment needs to be available to everybody.” 

Charles Montlake, 60, from Greenwich, who was diagnosed with both AL amyloidosis and myeloma, said: “What about the newly-diagnosed patients who could have benefitted from the drug combination and now will have missed out? They could have had a few more years with this drug combination.” 

Ross Cunliffe, from Bedfordshire, was 56 when he was diagnosed with cardiac amyloidosis and myeloma, back in 2014. The father-of-two said: “I’ve been lucky enough to get private healthcare through my work and it’s been invaluable. I’ve been able to receive eight different types of treatment, very often these drugs are drugs that aren’t available on the NHS.” 

The 64-year-old added: “It’s such an incredible shame that so many people are forced to go privately to save their own lives. I’ve had about six or seven ‘sliding-door moments’ when I had certain drugs that made me better for a period that would not have been available to me on the NHS. But people on the NHS still can’t get what I was able to have.” 

Prof Graham Jackson, consultant haematologist at Newcastle Hospitals NHS Foundation Trust and professor of clinical haematology at Newcastle University, said: “This is a huge blow to AL amyloidosis patients, especially given the international Andromeda trial shows a significant benefit for this often forgotten, frequently ignored cohort of patients with a poorly understood life-threatening condition. Indeed, this decision leaves these patients in England and Wales without a single approved therapy. This will be an especially bitter pill to swallow when this treatment is available in Scotland. Not to mention that these four individual drugs are all already available on the NHS.” 

For more information about AL amyloidosis and myeloma, or to get in touch with Myeloma UK go to www.myeloma.org.uk. Myeloma UK runs an Infoline on 0800 980 3332. 

WHAT IS AL AMYLOIDOSIS? 

• ‘Amyloidosis’ is a general term used for a group of conditions where an abnormal protein, called amyloid, accumulates in the tissues. 
• AL amyloidosis is the most common type of amyloidosis. It is caused by abnormal plasma cells in the bone marrow. Amyloid can build up in the kidneys, heart, liver, spleen, nerves, or digestive system. 
• AL amyloidosis is a relatively rare condition, with approximately 500-600 people diagnosed in the UK each year. 
• Between 3,000 and 4,000 people have AL amyloidosis in the UK. 
• It can be treated but it is a relapsing-remitting condition. This means patients have periods of remission after treatment, when the AL amyloidosis is not active or causing symptoms, but it will become active again after a period of time. 
• 15% of myeloma patients also have AL amyloidosis.