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Acute lymphoblastic leukaemia research

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This page of the acute lymphoblastic leukaemia (ALL) section is about research into the causes, prevention and treatments of acute lymphoblastic leukaemia. You can find information about


A quick guide to what's on this page

Acute lymphoblastic leukaemia (ALL) research

All treatments must be fully researched before they can be adopted as standard treatment for everyone. This is so that we can be sure they work better than the treatments we already use. And so we know that they are safe.

First of all, treatments are developed and tested in laboratories. Only after we know that they are safe are they tested in people, in clinical trials.

Research is looking into new chemotherapy drugs for ALL, resistance to chemotherapy, biological therapies, and managing problems during treatment. Cancer Research UK supports a lot of UK laboratory research into cancer and also supports many clinical trials.


CR PDF Icon You can view and print the quick guides for all the pages in the Treating ALL section.



Why we need research

We must fully research all potential treatments before we can adopt them as standard treatment for everyone. This is so that

  • We can be sure they work
  • We can be sure they work better than the treatments that are available at the moment
  • They are known to be safe

First of all, treatments are developed and tested in laboratories. For ethical and safety reasons, experimental treatments must be tested in the laboratory before they can be tried in people. If a treatment described here is said to be at the laboratory stage of research, it is not ready for patients and is not available either within or outside the NHS.

Cancer Research UK supports a lot of UK laboratory research into cancer and also supports many clinical trials. Clinical trials test treatments in patients and there are 4 phases. This is fully explained in the trials and research section

If you are interested in taking part in a clinical trial for acute leukaemia, visit our searchable database of clinical trials in the UK. If you are interested in joining one of the open trials, print it off and take it to your own specialist. If the trial is suitable for you, your doctor will need to refer you to the research team. The database also has information about closed trials and trial results. Most major leukaemia treatment centres are continually involved in clinical trials.

Here is a video on what it's like to take part in a clinical trial:

View a transcript of the video (Opens in a new window) 


Chemotherapy drugs

New drugs are being developed all the time. It is most important that we know that these drugs work and whether they work better than the treatments we currently use. For this reason, drugs have to go through a long testing process. You will often only be able to have new drugs within clinical trials. Early trials are often carried out with patients who have had all available treatment.

Clofarabine (Evoltra) is similar to another leukaemia drug already in use, fludarabine. It has been researched for children with ALL. It is licensed in the UK for treating people aged between 1 and 21, if they have already had at least 2 different courses of treatment, and the ALL has not responded or has come back. Clofarabine is now also being looked at in studies to see if it helps adults with acute leukaemia.

Doctors also investigate different combinations of chemotherapy drugs and different doses. Their aim is to get good results with treatment, but at the same time, not cause too many side effects. Trials are looking at different combinations and doses of chemotherapy.

The UKALL 2011 trial is looking at treatment for children and young adults (up to age 24) with ALL. The aims of this trial are to see if changing the standard treatment will reduce side effects and help stop the disease from coming back. 

The UKALL 14 trial is comparing treatments for adults between 25 and 65 years old who have recently been diagnosed with ALL. Doctors usually treat ALL with several different chemotherapy drugs, often in high doses, and sometimes with a stem cell transplant. The researchers are looking at changing the treatment in several different ways with the aim of improving treatment for adults with ALL. 

The aim of the UKALL60+ trial is to find a standard treatment for people over the age of 55 who are diagnosed with ALL. Researchers can then use this in the future as a comparison when looking at any new treatments in clinical trials. 

Trials for ALL are listed on our clinical trials database.


Chemotherapy side effects

Chemotherapy can affect your immune system, which helps to fight infection. Doctors know that children having chemotherapy for ALL are at increased risk of an infection called pneumococcus. A trial is looking at a vaccine to help protect against this infection in children who are having or have recently finished treatment for ALL. This trial has now closed and we are waiting for the results.


Resistance to chemotherapy

One difficulty in treating leukaemia with chemotherapy is that the cancer gradually becomes resistant to the chemotherapy. In other words, treatment that worked before no longer does. This is because the cells of many cancers have an overactive gene called mdr1. This stands for multi drug resistance.

Cells that have this overactive gene create a protein that makes the cancer cell pump out any chemotherapy drug that gets inside. Some drugs have been tried that can overturn this, but they cause a lot of side effects. A new drug is now available. This is a slightly changed version of a drug called ciclosporin. This drug (known as PSC833) has had some success in trials. But it caused serious side effects in a phase 3 trial, so the trial was stopped early. Researchers are still trying to find better ways to use this and other drugs.

A chemotherapy drug called nelarabine (Atriance) may be used to treat people with T cell acute lymphoblastic leukaemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL). The disease must be resistant to standard treatment, or have come back (relapsed) after treatment with at least 2 chemotherapy combinations.

The ALL R3 trial has looked at different types of treatment (including bone marrow transplants and chemotherapy) for children who had ALL that was not responding to treatment, or had come back (relapsed). You can read the results of the ALL R3 trial on our clinical trials database.


Stem cell transplant

The ICAT trial is looking at a treatment to help your immune system recover after a stem cell transplant, using cells from an unrelated donor. In this trial, researchers are looking at a way of giving you T cells from your donor to help fight infection. 


Cord blood stem cell transplants

Bone marrow and stem cell transplants for ALL use high dose chemotherapy, and sometimes total body irradiation (TBI). This treatment kills off all the stem cells inside your bone marrow that make all your blood cells. You have the stem cells replaced by a drip of stem cells or bone marrow. You can have someone else’s bone marrow or stem cells, or your own bone marrow. There is detailed information about bone marrow and stem cell transplants in the ALL treatment section. Bone marrow or stem cell transplants give the best chance of controlling the ALL for a long time in some people. But the high dose treatment can also cause severe side effects. Doctors are trying to find ways of improving these treatments. 

The RIC UCBT trial is looking at using stem cells collected from the umbilical cords of newborn babies. The cells are given to people after a transplant that uses lower doses of chemotherapy than usual (reduced intensity conditioning). These transplants are for people who don't have a relative or unrelated donor who can be their stem cell donor. Doctors hope that the umbilical cord stem cells will cause fewer side effects than adult stem cells. This trial has now closed and we are waiting for the results to become available. 

The MAC UCBT trial is also looking at using umbilical cord blood from unrelated donors after high dose chemotherapy. This trial has now closed and we are waiting for the results.


Half matched stem cell transplant

The UK Haplo study is looking at using donor stem cells from a family member who is at least a 50% match with the person having the stem cell transplant.

In most allogeneic transplants, the donor is someone who is a very close match to the person having the transplant. This is usually a brother or sister. It could also be from a donor who isn't a relative. But doctors can't find a match for about a third of those needing a transplant. An option for these people is a half matched transplant, where the donor is at least 50% match with the person having the transplant. In the past these transplants have been difficult to do. You can react to the donor cells, causing a severe immune response. But researchers think they have found a way to deal with this by using a drug called cyclophosphamide. The aims of the UK Haplo study are to find out how well high dose cyclophosphamide works with a half matched stem cell transplant, how safe it is, and to learn more about the side effects and quality of life for people having this type of transplant.


Biological therapies

Biological therapies use substances that the body makes naturally to treat cancer. There are different types of biological therapy including

Tyrosine kinases

Tyrosine kinase inhibitors (TKIs) are one type of biological therapy for ALL that researchers are looking at.

Tyrosine kinases are chemicals that cells use to signal to each other. Some of these signalling systems tell cancer cells to grow and divide. Scientists have been working on drugs called tyrosine kinase inhibitors (TKIs) that block these signals.

Imatinib mesilate (Glivec) is a TKI drug licensed in the UK to treat chronic myeloid leukaemia (CML). It is now also licensed to treat  people with ALL who have a particular chromosome change called the Philadelphia chromosome. In ALL, about 1 in 5 people (20%) have the Philadelphia chromosome. Research studies show that having imatinib as part of your first phase of treatment (remission induction therapy) improves your chances of getting into remission. Researchers continue to look at imatinib, as well as other TKI's.

The EsPhALL trial is looking at imatinib for children aged between 1 and 17 with Philadelphia chromosome positive leukaemia. This trial has now closed and we are waiting for the results.

Another TKI researchers are looking at is dasatinib. The CA180226 trial is looking at dasatinib to treat children and young people (20 or younger) who have Philadelphia chromosome positive ALL that is not responding to imatinib (Glivec). The researchers want to find out how well dasatinib works for this group and to learn more about the side effects. The CA180372 trial is looking at dasatinib for children and young people who have just been diagnosed with ALL that has the Philadelphia chromosome. Both these trials have now closed and we are waiting for the results. 

Another biological therapy being investigated is called ponatinib. It is a new type of tyrosine kinase inhibitor. It is different to previous TKIs in that it affects a mutation called T3151 found in some people with CML. This genetic mutation is very resistant to treatment and other drugs available do not really work in patients with this mutation. Ponatinib blocks cells that have this mutation, and in early lab tests it appeared to prevent T3151 or other mutations forming. 

The PACE trial found that ponatinib was helpful for Philadelphia positive ALL. This was a phase 2 trial, so larger trials are needed to see how helpful ponatinib is for this group of people. You can read a summary of the results for the PACE trial on our clinical trials database.

The Scottish Medicines Consortium (SMC) have recommended ponatinib for some people with ALL in Scotland. They recommend it for people with Philadelphia positive ALL, who are resistant to, or cannot have dasatinib and imatinib, or who have the T3151 mutation.

Monoclonal antibodies

Other types of biological therapy include monoclonal antibodies. Monoclonal antibodies can seek out cancer cells by looking for particular proteins on the cell’s surface. The MARALL study is looking at adding the monoclonal antibodies veltuzumab and epratuzumab to chemotherapy to treat acute lymphoblastic leukaemia (ALL) that has come back. The main aim of this study is to see if adding veltuzumab and epratuzumab to standard treatment for ALL that has come back is safe. This study has now closed and we are waiting for the results.

A trial is looking at blinatumomab for people who have pre B cell ALL. When you finish chemotherapy you have tests to see how it has worked. Although these may show that your ALL has gone away (in remission), it may start to grow again. Doctors hope blinatumomab will stop ALL from starting to grow again.

The TOWER trial is looking at blinatumomab with chemotherapy for pre B cell ALL that has not responded to treatment or has come back. This trial has now closed and we are waiting for the results.

The Alcantara study is also looking at blinatumomab for ALL that has not responded to treatment or has come back after treatment. The people taking part have ALL that has the Philadelphia chromosome. The researchers want to find out how well this drug works and to learn more about the side effects.

The Scottish Medicines Cosortium (SMC) have recommended blinatumomab for use within NHS Scotland. It is for adults with Philadelphia chromosome negative ALL that has come back, or pre B cell ALL that has not responded to treatment. The National Institute for Health and Clinical Excellence (NICE) have not made a decision about blinatumomab yet. So it may not be available in England and Wales. 

You can find out about all these trials on our clinical trials database.


Treatment for babies with ALL

Babies are sometimes diagnosed with ALL. Trials have shown that treatment with chemotherapy and steroids works quite well, but sometimes the leukaemia comes back. 

Another trial is now looking into whether some babies need more intensive treatment. Babies may join the INTERFANT-06 trial if they have ALL or mixed type (biphenotypic) leukaemia, and are less than one year old. After their first round of chemotherapy treatment, doctors will decide if the babies have a low, medium or high risk of their leukaemia coming back. They will do this by looking at the baby's age, their white cell count, how well they have responded to treatment so far, and whether there is a particular genetic change in their leukaemia cells. Babies who are in the low risk group will continue to have standard treatment. But those at medium and high risk will be put into different groups to look at how well different treatments work at stopping the leukaemia coming back. Some babies will have more intensive chemotherapy, and some will have a transplant of their own stem cells (autologous stem cell transplant). This trial has now closed and we are waiting for the results.

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Updated: 19 May 2015