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NICE approves access to new treatments for THREE types of blood cancer

The Bloodwise logo. Bloodwise appears in black text against a white background
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04 May 2018

The National Institute for Health and Care Excellence (NICE) has announced today that three new types of treatment will become routinely available on the NHS for people with blood cancer in England.

Arsenic trioxide (Trisenox) has been approved for patients with a sub-type of acute myeloid leukaemia. Midostaurin has been approved for patients with acute myeloid leukaemia who have mutations to the FLT3 gene and brentuximab has been approved for patients with Hodgkin lymphoma who do not respond to chemotherapy.

Dr Alasdair Rankin, Director of Research and Patient Experience at Bloodwise, said: “There are over 100 different types of blood cancer, which together are the fifth biggest cause of cancer and the third biggest cause of cancer death. Survival rates can vary greatly. Thanks to research some blood cancers have very effective treatments, but where progress has been harder, survival rates can be amongst the poorest of all cancers and many people are still treated with gruelling chemotherapy.

“Today’s news offers hope to patients with three different types of blood cancer – with approvals of targeted therapies that for some will reduce the chances of relapse and for others will offer the chance of a long-term cure or the reduction the severe side-effects connected to successful traditional treatments.”

Arsenic trioxide (Trisenox) in combination with all trans retinoic acid (ATRA) to treat patients with newly diagnosed low-to-intermediate risk acute promyelocytic leukaemia (APL) 

APL is a type of acute myeloid leukaemia diagnosed in around 170 people each year in the UK. It is one of the fastest developing types of blood cancer and before current treatments became available, survival rates were extremely low. Now around 65% of all patients will survive for five years or more after diagnosis and around 80% of patients with lower risk disease will survive.  The majority of deaths occur within three months of diagnosis and most patients alive after one year of treatment can now expect to have achieved a long-term cure.

Currently standard treatment involves all-trans retinoic acid (ATRA) combined with intensive chemotherapy. But chemotherapy can have gruelling short-term side effects including severe nausea, hair loss and reduced immunity - leading to serious infections and long periods in hospital. Longer-term side effects include the risk of developing secondary cancers, effects on fertility and increased risk of heart disease.

Recent clinical trials have shown that patients with APL who have low-risk disease can be successfully treated with ATRA and arsenic trioxide, sparing them from intensive chemotherapy. Arsenic trioxide works in a similar way to ATRA, by targeting the faulty gene that drives APL. Most patients had limited side effects and were even able to continue working throughout treatment. Importantly, relapse and death rates for those treated with arsenic trioxide were far lower than for those patients treated with chemotherapy.

Arsenic trioxide is already used to treat people with APL who have relapsed after chemotherapy, but this is the first time that it has been recommended for ‘first line’ treatment for some patients – sparing them from the side effects of chemotherapy completely.  

Dr Alasdair Rankin said: “This decision is extremely positive news. Chemotherapy can have horrendous side effects and it has been shown that some patients with APL can be spared from this gruelling treatment altogether. The idea of using arsenic to treat cancer may sound strange, given that it is a poison when used in high doses, but it has been used safely in medicine for many years and is a very effective and targeted treatment for this type of leukaemia.

“For people affected by APL, today’s announcement is another important advance. For people with other types of acute myeloid leukaemia, where better treatments remain hard to find, this shows what and ultimately will be achieved through research.”

Today’s announcement by NICE follows the US Food and Drug Administration (FDA) decision in January this year to approve the use of arsenic trioxide and ATRA to treat newly diagnosed APL patients.

Almost all cases of APL are cause by a chromosomal translocation involving the retinoic acid receptor alpha (RARα or RARA) gene. A chromosomal translocation happens when two chromosomes breaking apart and fuse with one another to create an abnormal gene. Translocations that cause cancer are good targets for therapy because they are specific only to cancer cells and drugs that target them should kill cancer cells and leave other tissue unharmed.

ATRA, which targets the RARα translocation, was first used in 1985 to treat APL patients and was the first ever targeted drug used to treat cancer.

Midostaurin in combination with chemotherapy for newly diagnosed patients with acute myeloid leukaemia (AML) who have the FLT3 mutation and as maintenance treatment to prevent relapse after treatment

Around 2,500 people are diagnosed with acute myeloid leukaemia (AML) each year. While the outlook for patients under 50 is significantly better, overall fewer than one in five patients survive for longer than five years.  Developing effective treatments for AML has been hard, in part because there is quite a lot of variation between patients in the type of genetic defects in their cancer. But some advances are being made in sub-groups of patients who have particular genetic defects.

Approximately a third of patients with AML have leukaemia cells that carry a mutation to the FLT3 gene and these patients are known to be more likely to relapse after treatment.

Current treatment for AML uses the high dose chemotherapy drugs, daunorubicin and cytarabin, which while having severe side effects, is effective at putting many patients into remission. Rates of relapse are high, however, particularly in patients with FLT3 positive AML.

Midostaurin, a protein kinase inhibitor targets the FLT3 protein and has been shown to have limited side effects. While midostaurin does not remove the need for chemotherapy - NICE has approved its use in combination with intensive chemotherapy– it has been shown to significantly improve survival and reduce relapse rates. Midostaurin is then also taken as maintenance treatment for a year after chemotherapy treatment has finished to reduce relapse rates.

Alasdair Rankin said: “A diagnosis of AML is devastating, particularly when patients are FLT3 mutation positive, due to a high risk of relapse after treatment. We know that for people treated for AML, living with the fear of relapse can have a severe psychological impact and cause a huge amount of anxiety. Today’s announcement is good news for people living with this particular type of AML. Midostaurin has been shown to have minimal side effects and offers the reassurance to patients that everything possible is being done to reduce their chances of the disease coming back.”

Brentuximab for the treatment of patients with classical Hodgkin lymphoma whose disease returns after, or is resistant to, two types of chemotherapy and who are not able to undergo additional chemotherapy or a stem cell transplant.

People with Hodgkin lymphoma in England whose cancer returns after two previous types of treatment will now be able to routinely access brentuximab vedotin through the NHS. 

Brentuximab is a monoclonal antibody, which targets specific antigens on the surface of cancer cells. Brentuximab targets the CD30 protein, found on Hodgkin lymphoma cells but not healthy cells, selectively killing the cancer cells. 

Today’s decision by NICE makes brentuximab available to the approximately 100 people a year in England with classical Hodgkin lymphoma whose disease returns after, or is resistant to, two types of chemotherapy and who are not able to undergo additional chemotherapy or a stem cell transplant.

Standard treatment for Hodgkin lymphoma typically includes chemotherapy and in some cases radiotherapy, steroids and – if the Hodgkin lymphoma comes back – a stem cell transplant. Treatment is usually very successful but some patients do not respond or become resistant to chemotherapy.

Brentuximab has been available to patients in England in this situation for the past five years, but only through the Cancer Drugs Fund. It was shown to be successfully used as a ‘bridge’ in a large proportion of patients, with many patients going into remission and able to then undergo a stem cell transplant, offering the chance of a long-term cure. 

Dr Alasdair Rankin said: “We are pleased that NICE and the drug’s manufacturers have worked together to secure access to brentuximab for future patients. This is extremely positive news and offers reassurance to people with Hodgkin lymphoma that they will be able to access the most appropriate therapies at every stage of their treatment.  While standard treatments for Hodgkin lymphoma are generally successful, this decision enables doctors to give patients the best chance of a long-term cure if they do not respond to them.” 

Hodgkin lymphoma is diagnosed in over 1,600 people each year in the UK. In 2017 NICE approved the use of brentuximab for the treatment of Hodgkin lymphoma in patients who had relapsed after an autologous stem cell transplant. Brentuximab is already available to patients who do not respond to two types of treatment on the NHS in Wales, Scotland and Northern Ireland.

Read more about today's NICE recommendations.