Research in acute lymphoblastic leukemia (ALL)

We are always learning more about cancer. Researchers and healthcare professionals use what they learn from research studies to develop better practices that will help find and treat acute lymphoblastic leukemia (ALL). They are also looking for ways to improve the quality of life of people with ALL.

The following is a selection of research showing promise for ALL. We’ve included information from PubMed, which is the research publication database of the National Library of Medicine. Each research article in PubMed has an identity number (called a PMID) that links to a brief overview (called an abstract).


Researchers are trying to find better ways to help doctors predict a prognosis (how likely it is that the cancer can be successfully treated or that it will come back after treatment) for ALL. They are also trying to determine the best treatment options based on certain characteristics of the disease, such as specific biomarkers. Biomarkers are substances, such as proteins, genes or pieces of genetic material like DNA and RNA, that are found naturally in the body. They can be measured in body fluids, including blood, lymph fluid and bone marrow, or on certain types of cells, such as cancer cells. Doctors can look for and measure these biomarkers to check if cancer is present or that it is responding to treatment.

Prognostic and predictive biomarkers for ALL can be used to help plan treatment. Prognostic biomarkers can be used to identify people who have a greater risk that the disease will progress or come back after treatment (recur or relapse). Predictive biomarkers are used to identify people who are more likely to have a favourable or unfavourable effect from treatment compared to people without the biomarker. Researchers are looking at the following biomarkers to see if they can help doctors diagnose, predict a prognosis for and find out which treatments will benefit a person with ALL:


Researchers are looking for new ways to improve treatment for ALL. Advances in cancer treatment and new ways to manage the side effects from treatment have improved the outlook and quality of life for many people with cancer. The following is noteworthy research into treatment for ALL.


The following chemotherapy drugs or combinations are showing promise as treatments for ALL:

Pediatric treatment protocols may work better than adult regimes for adolescents and young adults with ALL. While results are promising, there is a risk of more severe side effects when using pediatric treatment protocols (British Journal of Haematology, PMID 26683485; Journal of Clinical Oncology, PMID 26644537; Leukemia, PMID 25079173).

Targeted therapy

Targeted therapy drugs target specific molecules (usually proteins) that cause cancer cells to grow. Unlike traditional chemotherapy, which targets DNA replication in any actively dividing cells, these treatments are more focused on cancer cells. Targeted therapies can target specific proteins or biomarkers on the surface of cells.

Researchers are studying the following types of targeted therapy drugs in treating ALL.

mTOR inhibitors slow or stop cancer growth by blocking mTOR, which is a protein that regulates cellular metabolism and growth and how quickly cells multiply. This protein can trigger cancer cells to grow and new blood vessels to form, which cancers need to grow. Everolimus (Afinitor) is an mTOR inhibitor that may be a treatment option for refractory or relapsed ALL and T lymphoblastic lymphoma (Blood, PMID 25921059; Clinical Cancer Research, PMID 25724525).

PI3K (phosphoinositide 3-kinase) inhibitors work by switching off PI3K, which is an enzyme in cells that makes them grow and divide. Researchers are studying the PI3K inhibitor buparlisib as a treatment for ALL (American Journal of Hematology, PMID 27673440).

Proteasome inhibitors work by stopping proteasomes, which are a group of enzymes that cancer cells need to grow. Interfering with how proteasomes work may help stop the growth of cancer cells or destroy them. Researchers are studying the proteasome inhibitor carfilzomib (Kyprolis) as a treatment for ALL (Leukemia and Lymphoma, PMID 26674111).

Monoclonal antibodies

Monoclonal antibodies are both a type of immunotherapy and a targeted therapy. They bind to specific antigens on cancer cells to help destroy them. Researchers are looking at using monoclonal antibodies alone and with other drugs to treat ALL. Some monoclonal antibodies that are showing promise in treating ALL include:


Immunotherapy boosts or helps the immune system find and destroy cancer cells. Researchers are studying the following types of immunotherapy for ALL.

Engineered chimeric antigen receptor (CAR) T cells

T cells are part of the immune system. They help fight infection and destroy abnormal cells, including cancer cells. Doctors take T cells from a person’s blood and genetically engineer, or modify, them in the lab so they have chimeric antigen receptors (CARs) on their surface. CARs are proteins that make the T cells recognize cancer cells. Doctors can grow CAR T cells in the lab until they have billions of them. They then infuse the CAR T cells back into the person’s body, where they will multiply, and then target and kill cancer cells. Researchers have found that engineered CAR T cells are effective in treating ALL (Lancet, PMID 25319501; Blood, PMID 28408462; Journal of Clinical Investigation, PMID 27111235; Journal of Hematology and Oncology, PMID 27887660; BMJ Open, PMID 28039295; New England Journal of Medicine, PMID 29385376).

Stem cell transplant

Stem cell transplant replaces a person’s blood-forming (hematopoietic) stem cells. It is used when stem cells or the bone marrow has been damaged by chemotherapy drugs, radiation therapy or disease (such as cancer). The new stem cells make healthy blood cells.

Stem cell transplants are commonly used to treat ALL. Researchers are studying the following types of stem cell transplant to see if they could be safer, easier and more effective for people with ALL.

Reduced-intensity allogeneic transplant uses lower doses of chemotherapy or radiation therapy before the transplant. The lower doses don’t completely destroy the recipient’s bone marrow, so blood cell counts don’t drop as low as they do in standard stem cell transplants. For this reason, there is a lower risk for complications. This may be of great importance for older adults who can’t tolerate the higher doses of chemotherapy and radiation normally used with stem cell transplants (Biology of Blood and Marrow Transplantation, PMID 26260679; Blood, PMID 25878120).

Half-matched stem cell transplant (also called haploidentical transplant) is a new type of stem cell transplant. To do a stem cell transplant, the donor and recipient are matched through a process called human leukocyte antigen (HLA) typing. Usually the antigens on the donor’s and recipient’s stem cells need to be a perfect or nearly perfect match. But a half-match stem cell transplant can be done if only half of the donor’s and recipient’s stem cells match. All parents and children, and about 50% of siblings, are half-matched. Half-matched stem cell transplant may be an option for adults with ALL who do not have a matched donor (Bone Marrow Transplantation, PMID 26039212, PMID 25222501; Journal of Hematology and Oncology, PMID 26208715; Clinical Cancer Research, PMID 26927664).

Cord blood stem cell transplant uses stem cells collected from blood in the placenta and umbilical cord of newborn babies. Researchers have found that cord blood seems to be a good alternate source for stem cells for people who don’t have a suitable related or unrelated donor. Cord blood stem cell transplants are also linked with a lower risk of a life-threatening condition called graft-versus-host disease (GVHD) (New England Journal of Medicine, PMID 27602666; American Journal of Hematology, PMID 26910296; Biology of Blood and Marrow Transplantation, PMID 25498906).

Lowering the risk for relapse or recurrence and GVHD is a significant area of research regarding treatment of ALL. Some studies include using the following:

Supportive care

Living with cancer can be challenging in many different ways. Supportive care can help people cope with cancer, its treatment and possible side effects. The following is noteworthy research into supportive care for ALL.

Taurine is an amino acid that improves kidney and liver function. Taking taurine can help relieve nausea and vomiting from the chemotherapy drugs used to treat ALL (Amino Acids, PMID 25323734; Journal of Cancer Research and Therapeutics, PMID 26148612).

Learn more about cancer research

Researchers continue to try to find out more about ALL. Clinical trials are research studies that test new ways to prevent, detect, treat or manage ALL. Clinical trials provide information about the safety and effectiveness of new approaches to see if they should become widely available. Most of the standard treatments for ALL were first shown to be effective through clinical trials.

Find out more about clinical trials.