Advances in Leukemia Research

Written by: National Cancer Institute 

Summarized by: Janya Kumar

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Introduction 

Have you ever wondered what it feels like to hear the words, “You have leukemia”? For thousands of families every year, this diagnosis changes everything—bringing fear, uncertainty, and a desperate search for hope. Yet, thanks to decades of research, what was once a certain death sentence is now, for many, a treatable disease with real chances for survival. 

Leukemia is a cancer of the blood and bone marrow that affects both children and adults. Over the past two decades, research funded by the National Cancer Institute (NCI) has led to significant advances in how leukemia is treated. Scientists are now focusing on making treatments more effective and less harmful, especially for older adults and children. This paper summarizes recent progress in leukemia research, including new therapies, clinical trials, and ongoing challenges.  Current Treatments for Leukemia 

Traditionally, leukemia has been treated with chemotherapy, radiation 

therapy, and stem cell transplantation. These methods can be very 

effective, but they often come with serious side effects. In recent years, 

targeted therapies and immunotherapies have become important new 

options. These treatments aim to attack cancer cells more precisely, 

causing less harm to healthy cells.  Targeted Therapies

Targeted therapies work by focusing on specific proteins or genetic changes in leukemia cells. For example, drugs like imatinib (Gleevec) and dasatinib (Sprycel) are used to treat chronic myelogenous leukemia (CML) by blocking an abnormal protein that causes the cancer to grow. Other targeted drugs, such as venetoclax (Venclexta) and ibrutinib (Imbruvica), are used for chronic lymphocytic leukemia (CLL).

Immunotherapy

Immunotherapy helps the body’s own immune system recognize and destroy cancer cells. One of the most exciting developments is CAR T-cell therapy, which involves modifying a patient’s immune cells to enhance their ability to fight leukemia. This therapy has been approved for certain types of acute lymphoblastic leukemia (ALL) in children and young adults and is being studied for use in adults and other types of leukemia.

Acute Lymphoblastic Leukemia (ALL)

ALL is a fast-growing cancer that requires immediate treatment. Researchers are working to make treatments less toxic, especially for older adults who may not handle standard chemotherapy well. Clinical trials are testing whether targeted therapies can replace or be combined with chemotherapy for better results.

Immunotherapies, such as CAR T-cell therapy and bispecific T-cell engagers (BiTEs), are also being used. For example, the drug blinatumomab (Blincyto) helps immune cells identify and target leukemia cells, and has been approved for use in both adults and children with certain types of acute lymphoblastic leukemia (ALL). While ALL primarily affects children and responds well to recent advances in immunotherapy, another major type of acute leukemia—Acute Myeloid Leukemia (AML)—poses unique challenges, especially in adults.

Acute Myeloid Leukemia (AML)

AML is the most common acute leukemia in adults and is often harder to treat. Researchers are using genomic sequencing to match patients with the most effective targeted therapies. Several new drugs have been approved for AML that target specific genetic mutations, and clinical trials are ongoing to test even more options.

Chronic Myelogenous Leukemia (CML)

CML is usually a slow-growing cancer. Targeted therapies have dramatically improved survival rates, and many patients now live close to a normal lifespan. Researchers are now studying whether some patients can safely stop taking their medication after long-term treatment.

Chronic Lymphocytic Leukemia (CLL)

CLL is a slow-growing leukemia that mostly affects older adults. Targeted therapies like ibrutinib and venetoclax have replaced chemotherapy for many patients, and new drug combinations are being tested to improve outcomes and reduce side effects. CAR T-cell therapy is also being studied for CLL.

Hairy Cell Leukemia (HCL)

HCL is a rare type of leukemia. Researchers are testing combinations of targeted therapies, such as vemurafenib and rituximab, which have shown promising results in clinical trials.

Leukemia Treatment in Children

Children with leukemia are often treated with chemotherapy, radiation, and stem cell transplantation. While survival rates have improved, some children experience relapses or long-term side effects. Researchers are now focusing on targeted therapies and immunotherapies to provide safer and more effective treatments for children. For example, CAR T-cell therapy has been approved for children with relapsed ALL, and new drugs are being tested for other types of childhood leukemia.

Ongoing Challenges and Future Directions

Despite major progress, some types of leukemia remain difficult to treat, and not all patients respond to new therapies. Researchers are working to:

• Develop treatments that are more effective and less toxic, especially for older adults and children.

• Use genomic testing to personalize therapy for each patient.

• Find new ways to help the immune system fight leukemia.

• Reduce the long-term side effects of treatment.

The NCI supports many clinical trials and research programs to keep advancing leukemia care. As more is learned about the biology of leukemia, new treatments will continue to be developed and tested.

Conclusion

Leukemia research has made significant strides in recent years, especially with the development of targeted therapies and immunotherapies. These advances are helping patients live longer, healthier lives with fewer side effects. However, continued research is needed to improve outcomes for all patients and to make treatments as safe and effective as possible, to make sure that by 2060, no more cancer patients will die, as stated in the article.         

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Works Cited

National Cancer Institute. “Advances in Leukemia Research.” National Cancer Institute, Cancer.gov, 25 June 2019, www.cancer.gov/types/leukemia/research.