Research into acute lymphoblastic leukaemia (ALL)

Thanks to research, survival for acute lymphoblastic leukaemia (ALL) has improved in the last 40 years. Researchers around the world are looking for new and kinder treatments to improve survival and quality of life for people with ALL. They also want to learn more about:

  • how ALL develops
  • the genetic differences between leukaemia cells in adults and children
  • how these differences might affect treatment 

Go to Cancer Research UK’s clinical trials database if you are looking for a trial for ALL in the UK. You need to talk to your specialist if there are any trials that you think you might be able to take part in.

Some of the trials on this page have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of what's happening in adult ALL.

Research into treatment for ALL

There are some clinical trials looking at treatment for ALL in the UK and around the world. Researchers are mainly looking at:

  • chemotherapy
  • stem cell and bone marrow transplants
  • targeted cancer drugs and immunotherapy


Chemotherapy is the main treatment for ALL. Chemotherapy uses anti cancer (cytotoxic) drugs to destroy cancer cells. The drugs circulate throughout the body in the bloodstream.

Trials are looking into:

  • different combinations of chemotherapy drugs
  • reducing chemotherapy side effects
  • new chemotherapy drugs such as nelarabine
  • how chemotherapy affects quality of life
  • chemotherapy for different age groups
  • combining chemotherapy with other types of treatment
  • reducing the amount of chemotherapy people have
  • what works best to prevent the leukaemia from coming back

Stem cell and bone marrow transplants

Stem cell and bone marrow transplants use high dose chemotherapy. You might also have total body irradiation (TBI) and targeted cancer drugs. The chemotherapy, TBI and targeted drug kills off all the stem cells inside your bone marrow. Stem cells are cells that make your blood cells such as white blood cells. To replace these you usually have someone else's (a donor's) stem cells or bone marrow. 

This high dose treatment can cause severe side effects. Doctors are trying to find ways of improving treatment to reduce these side effects.

Half matched transplants

Doctors can't always find a perfect match for people who need a stem cell transplant. So for these people an option might be to have a half matched or haploidentical stem cell transplant. This transplant uses donor stem cells from a family member who is at least a 50% match. Researchers are looking at:

  • a better way to give half matched transplants
  • reducing the side effects for this type of transplant

Stem cells from umbilical cords

Sometimes doctors use blood stem cells collected from umbilical cords. These are cord transplants. Researchers are looking at adding a supplement to the stem cells collected. Nicotinamide is the name of the supplement and is a form of vitamin B3. Once added to the stem cells its then left to grow in the laboratory. Nicotinamide has shown to increase the number of stem cells available. It has also helped bring forward blood count recovery.

Helping with recovery after a transplant

Other research is looking at improving a person’s immune system after a stem cell transplant. The immune system doesn’t work properly for several months after a transplant. So it is more prone to getting an infection.

Researchers are looking at a way of giving donor immune cells (T cells) after a transplant. This is to help fight infection. They hope this will help people recover more quickly, but without increasing the risk of getting graft versus host disease (GvHD).

GvHD is a complication of donor transplants. The donor’s T cells fight the body’s own tissues, causing problems such as diarrhoea, skin rashes or liver damage.

Targeted cancer drugs and immunotherapy

Targeted cancer drugs work by ‘targeting’ those differences that help a cancer cell to survive and grow. There are different types of targeted cancer drugs including:

  • monoclonal antibodies
  • tyrosine kinase inhibitors

Immunotherapies can boost the body's own immune system to fight off or kill cancer cells.

Monoclonal antibodies

Monoclonal antibodies can seek out cancer cells by looking for particular proteins on the cell’s surface. Some of the drugs currently being looked at in people with ALL are:

  • blinatumomab
  • inotuzumab ozogamicin (INO)
  • rituximab
  • moxetumomab pasudodotox
  • ofatumumab
  • obinutuzumab
  • denintuzumab (SGB-CD19A)
  • daratumumab
  • a new type of monoclonal antibody called AMG 404 in combination with blinatumomab
Cancer growth blockers

Tyrosine kinase inhibitors

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 (Glivec) is a TKI. It's a treatment for people with ALL who have a change in the leukaemia cells called the Philadelphia chromosome Open a glossary item. About 20 to 30 out of every 100 people with ALL (about 20 to 30%) have this change. Research shows that having imatinib as part of induction treatment improves your chances of getting into remission Open a glossary item.

Other TKI's that are being looked at in trials include:

  • ruxolitinib
  • ponatanib
  • selumetinib

Proteasome inhibitors

Proteasomes are substances found in cells. They help break down proteins the cell doesn't need into smaller parts. The cell can then use them to make new proteins that it does need.

Drug treatments that block proteasomes from working are proteasome inhibitors Open a glossary item. They cause a build up of unwanted proteins in the cell, which makes the cancer cells die.

Researchers are looking at some of these drugs in clinical trials for ALL:

  • bortezomib
  • carfilzomib

Other types of targets cancer drugs being looked at include:

  • venetoclax
  • navitoclax

Chimeric antigen receptor T-cell therapy (CAR T-cell) is a new type of immunotherapy treatment. It is being looked at in early phase trials and uses your own immune system to treat leukaemia.

Researchers collect a certain type of white blood cell, called T cells from your body. These cells help fight infections but they can't tell the difference between a normal cell and a cancer cell. They then change the T cells in the laboratory and give them back to you through an infusion. Researchers hope the altered T cells will recognise and attack any leukaemia cells. 

Other treatment

Doctors are looking at a drug called pegylated human arginase (BCT-100). Arginine is a protein (an amino acid) that helps cells to grow and survive. Researchers hope that reducing the amount of arginine starves the cancer cells and stops them from growing.

Research into managing treatment side effects

Research is looking at a drug called tranexamic acid to treat low platelet counts. Usually doctors give platelet transfusions Open a glossary item if you have a low platelet count. This trial hopes to:

  • lower the amount of bleeding people get with cancer of the bone marrow and lymphatic system
  • lower the number of platelet transfusions
  • see how this affects people’s quality of life Open a glossary item

In another study, researchers are looking at how ALL impacts your quality of life. They want to find out how symptoms and treatment affect quality of life. The researchers want to create new quality of life questionnaires for people with blood cancer. 

Researchers are looking at people aged between 10 and 25. They want to know the number of people that develop a condition called osteonecrosis.

Osteonecrosis means death of bone tissue. This happens when the blood supply to part of the bone is reduced or cut off. Meaning the bone cells can’t get oxygen or nutrients. This increases the risk of developing breaks (fractures) in the bones. Doctors already know treatment with steroids can increase this risk of developing osteonecrosis. But this study hopes to help doctors understand more about:

  • how it develops
  • if they can predict who will develop it in the future

Finding out more about leukaemia and how well it responds to treatment

Following the UKALL14 trial researchers created an additional study. This is looking at the gene changes in the leukaemia cells. They also want to find out whether they can predict how well treatment will work based on these changes.

Researchers are looking at a particular type of leukaemia called Philadelphia positive leukaemia. They want to know what gene changes (mutations) people with Philadelphia positive leukaemia have. The researchers are using a technique called Next Generation Sequencing (NSG) to look at the changes.

Next Generation Sequencing is a scientific technique that gives genetic information about a person.

Last reviewed: 
29 Jul 2021
Next review due: 
29 Jul 2024
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  • The information on this page is based on literature searches and specialist checking. We used many references and there are too many to list here. If you need additional references for this information please contact with details of the particular risk or cause you are interested in. 

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