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The following treatments are not licensed for use in the UK for AML and so are not generally available outside of research trials. If you take part in a clinical trial| you may be offered one or more of these drugs as part of your treatment.
Clofarabine| is very similar to another drug commonly used to treat people with AML called fludarabine|. It's thought that as clofarabine has fewer side effects than fludarabine it may be more suitable for older people who are less able to have intensive chemotherapy|.
Clofarabine is given by infusion and is only available for AML as part of research trials. Some people taking part in the AML-17| trial will be given it.
Everolimus is a type of drug called a kinase inhibitor. Kinases work as chemical messengers in the body. A kinase called the mTOR kinase sends signals to the cancer cell that tell it to grow. Everolimus blocks these signals and so interferes with the cancer's ability to grow. It can be given in tablet form or by infusion. This drug is only available for AML as part of a research trial. Some people taking part in the AML-17 trial will be given Everolimus.
Gemtuzumab|, also known as Mylotarg®, is given as a drip into a vein (intravenously).
It is made up of a combination of an antibody and a chemotherapy drug. The antibody attaches itself to a protein (CD33) found on the surface of leukaemia cells. In this way the antibody carries the chemotherapy directly to the leukaemia cells. Because CD33 is found mainly on leukaemia cells, it is hoped this drug will target the chemotherapy against leukaemia cells while causing less damage to healthy cells.
Some people taking part in the research trials AML-17 adn AML-16 will be given gemtuzumab as part of their treatment.
Some people diagnosed with AML have a FLT3 mutation (change) in the leukaemia cells. This mutation can increase the risk of the leukaemia coming back in the future.
CEP701 is an experimental new treatment designed to act against cells with this mutation. It is a liquid that is drunk and can be taken as an outpatient. It's hoped that by giving CEP701 between courses of chemotherapy this risk can be reduced.
Some people taking part in the AML-17 trial who have the FLT3 mutation will be offered CEP701.
Arsenic trioxide is licensed to treat people who have acute promyelocytic leukaemia (APL) that has come back after treatment, or has not gone into remission with treatment. This drug is made from the poison arsenic, but is given at low, safe doses.
Although it is licensed to treat APL we don’t yet know how well it might work for other types of AML. Some people taking part in the AML-17 trial will be given arsenic trioxide along with their chemotherapy treatment. It is given as a drip into a vein (intravenously). It may be given as an outpatient.
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