The following treatments are not licensed for use in the UK for acute myeloid leukaemia 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 is 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-16 trial will be given clofarabine.

Gemtuzumab also known as Mylotarg® is given as a drip into a vein (intravenously).

Gemtuzumab is a treatment 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.

This drug is not licensed in the UK and so is not generally available outside of research trials. Some people taking part in the research trials AML-15 and AML-16, will be given gemtuzumab as part of their treatment.

CEP701 is an experimental new treatment designed to act against cells that have a change called a FLT3 mutation. It is a liquid that is drunk and can be taken as an outpatient.

About 1 in 3 people diagnosed with AML have a FLT3 mutation (change) in the leukaemia cells. This mutation can increase their risk of the leukaemia coming back in the future. It is hoped that by giving CEP701 between courses of chemotherapy this risk can be reduced.

Some people taking part in the AML-15 trial who have the FLT3 mutation will be offered CEP701.

Tipifarnib is designed to block the messages that tell cells to grow, from reaching leukaemia cells. It is a tablet you swallow.

Tipifarnib belongs to a group of drugs called farnesyl transferase inhibitors (FTIs). Farnesyl transferase is a special protein that stimulates acute myeloid leukaemia cells to grow. FTI's block this protein. It has fewer side effects than the high-dose chemotherapy usually used to treat AML, because it is a targeted therapy. Doctors therefore hope that when it is given with lower doses of chemotherapy it will improve the effectiveness of treatment for older people who aren’t able to have intensive chemotherapy.

Some people taking part in the AML-16 trial will be given tipifarnib along with their chemotherapy treatment.

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-16 trial will be given arsenic trioxide along with their chemotherapy treatment. Arsenic trioxide is given as a drip into a vein (intravenously). It may be given as an outpatient.