Leukemia

Leukemia

Since discovery of the first effective leukemia treatment in the 1940s, researchers have amassed a growing number of therapies to extend survival and cure many different forms of the disease. Research into targeted drugs are also beginning to have a major impact on leukemia treatment.

While certain forms of leukemia can now be cured in up to 95 percent of patients, others are far more challenging and leukemia has proven far more complex than researchers could have imagined in the 1940s. Yet deeper understanding about the disease is fueling the continuous development of new, more effective therapies and is improving survival and quality of life for patients.

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2014

Novel immunotherapy sends rare type of leukemia into remission

Novel immunotherapy sends rare type of leukemia into remission

The FDA grants accelerated approval to the immunotherapy blinatumomab (Blincyto) for a rare and aggressive type of leukemia (Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (ALL)). Blinatumomab is approved for patients whose disease worsened or relapsed despite earlier therapies. The approval follows results from a clinical trial showing that blinatumomab leads to complete remission of cancer in nearly a third of patients. Blinatumomab is an antibody that brings the cancer-destroying immune T cells into contact with leukemia cells.

Four new drugs transform CLL treatment

Four new drugs transform CLL treatment

In a span of eight months, the FDA approves four new treatments for chronic lymphocytic leukemia (CLL), a blood cancer that most commonly develops in older adults. Obinutuzumab (Gazyva) and ofatumumab (Arzerra), approved as initial treatments for CLL, delay cancer worsening by about a year when given with chemotherapy. Targeted drugs idelalisib (Zydelig) and ibrutinib (Imbruvica) were approved for use in patients with CLL that became resistant to or relapsed after standard treatment. In clinical trials, both slowed cancer worsening and ibrutinib also helped patients live longer.  All four new therapies are easier for patients to tolerate than prior therapies, making treatment possible for more patients than ever.  

2010

Adding rituximab extends survival for chronic lymphocytic leukemia

Adding rituximab extends survival for chronic lymphocytic leukemia

A large, long-term trial establishes that adding the targeted drug rituximab (Rituxan) to initial treatment with the standard drug fludarabine (Fludara) slows the progression of chronic lymphocytic leukemia, and improves survival. This is the first drug regimen ever found to significantly extend the lives of patients with the disease. The trial found that more than 10 percent of patients treated with this regimen had no disease progression after ten years.

Tyrosine kinase inhibitors provide additional options for CML

Tyrosine kinase inhibitors provide additional options for CML

Two studies show that dasatinib (Sprycel) and nilotinib (Tasigna) may be more effective than the current standard drug, imatinib (Gleevec), for the initial treatment of chronic myeloid leukemia. Though all three drugs target the same active genetic pathway on the "Philadelphia chromosome," studies suggest the newer drugs possibly elicit faster, stronger responses and cause fewer side effects than imatinib.

2009

Genome of acute myelogenous leukemia is sequenced

Genome of acute myelogenous leukemia is sequenced

Researchers map the genome of a patient with acute myelogenous leukemia (AML), one of the most common forms of leukemia. While they find more than one million tiny genetic differences between the genes of the patient's normal cells and the patient's cancer cells, the researchers are able to isolate 10 key genetic pathways that are most likely to be involved in the cancer's development. Four of these had never before been implicated as having a role in leukemia development and growth, and appear to provide the energy that cancer cells need to grow. These findings provide important information to guide the development of future, more effective targeted therapies.

2007

Bendamustine is effective against chronic lymphocytic leukemia (CLL)

Bendamustine is effective against chronic lymphocytic leukemia (CLL)

Bendamustine (Treanda), a nitrogen mustard-based drug which has been used in Europe for decades, is found to triple the time it takes for cancer to progress and produce more complete remissions than the standard chemotherapy for chronic lymphocytic leukemia. Bendamustine is approved by the FDA as an initial treatment for the disease in early 2008.

Addition of an arsenic compound found to improve survival for rare form of leukemia

Addition of an arsenic compound found to improve survival for rare form of leukemia

A large clinical trial finds that adding arsenic trioxide (Trisenox) to the standard treatment for acute promyelocytic leukemia (APL) improves survival for adults newly diagnosed with the disease. Arsenic has historically been known as a potent poison, but its use in traditional Chinese medicine led to the development of this drug. The drug is approved by the FDA later the same year.

Study supports expanded use of dasatinib for CML

Study supports expanded use of dasatinib for CML

Research shows that dasatinib (Sprycel) – initially approved for use only when treatment with imatinib fails – should also be an option for initial treatment of early-stage chronic myelogenous leukemia. The FDA approves the drug for this broader indication in 2010, giving patients with CML multiple options for controlling their cancer.

2006

New drug helps CML patients who become resistant to imatinib

New drug helps CML patients who become resistant to imatinib

Researchers identify a second targeted treatment, dasatinib (Sprycel), for patients with chronic myelogenous leukemia who cannot tolerate or develop resistance to imatinib (Gleevec). The drug is approved based on study results showing that more than 90 percent of these poor prognosis patients had no evidence of their cancer following dasatinib treatment. Dasatinib targets the same mutated protein as imatinib, but through a different mechanism.

2005

Drug approved to reduce oral sores caused by cancer treatment

2004

Epigenetic drugs approved to prevent cancer in patients with myelodysplastic syndromes

Epigenetic drugs approved to prevent cancer in patients with myelodysplastic syndromes

The FDA approves azacytidine (Vidaza) in 2004 and decitabine (Dacogen) in 2006 for treatment of myelodysplastic syndromes (MDS), a group of serious blood disorders that predispose a person to acute myelogenous leukemia. These targeted drugs interfere with the control of genetic processes that enable cancer cells to develop and multiply. Both drugs are shown to reduce or eliminate the need for blood transfusions – a common MDS treatment that can impair patients' quality of life.

2001

New targeted therapy – imatinib – proven effective against chronic myelogenous leukemia

New targeted therapy – imatinib – proven effective against chronic myelogenous leukemia

The FDA approves imatinib (Gleevec) after just three months of review – the fastest approval in FDA history – based on data showing the drug halted the growth of chronic myelogenous leukemia (CML) in the majority of patients. Imatinib is the first drug proven to shut down a molecular defect known to cause a specific type of cancer – a mutation on the so-called "Philadelphia chromosome." It has since become the standard of care for this disease, and its effectiveness and easily-administered pill formulation enables patients to live with CML as a manageable, chronic disease.

2000

Fludarabine works faster, better for patients with CLL

Fludarabine works faster, better for patients with CLL

A large, long-term clinical trial demonstrates that the drug fludarabine (Fludara) – originally developed as a back-up therapy for patients with chronic lymphocytic leukemia – works in more patients and produces longer-lasting remissions than the previous standard drug, chlorumbucil (Leukeran). However, the drug only works reliably in patients younger than age 65, and stem cell transplantation from a healthy, matched donor remains the only curative therapy for this disease.

1995

Approval of tretinoin leads to 95 percent cure rate for rare leukemia

Approval of tretinoin leads to 95 percent cure rate for rare leukemia

Tretinoin (Vesanoid), a drug related to vitamin A, is found to cause remission in patients with acute promyelocytic leukemia (APL), a rare form of leukemia. It may be given alone or in combination with other drugs in patients whose disease has recurred after previous therapy. The discovery of the drug is fueled by the realization that this form of leukemia develops when cells do not mature properly; tretinoin is the first drug to correct this abnormal process. Prior to the availability of this drug, APL was a rapidly progressing and often fatal illness. Today, patients are treated with a newer drug, called arsenic trioxide, as well as tretinoin, and APL is considered one of the most curable forms of leukemia.

Lymphocyte transfusions re-induce leukemia remissions

Lymphocyte transfusions re-induce leukemia remissions

Researchers discover that giving a "graft" of lymphocytes (a form of white blood cells) from a biologically matched, healthy donor to a patient with chronic myeloid leukemia (CML) can help drive the leukemia back into remission if the cancer returns after a previous stem cell or bone marrow transplant from the same donor. In these patients, the new donor lymphocytes will often recognize the cancer cells as diseased, and work to destroy them.

This phenomenon, known as the "graft-versus-leukemia" or "graft-versus-lymphoma" effect, appears to be most powerful in certain leukemias and in slow-growing lymphomas. Scientists continue to explore ways to capitalize on the graft versus leukemia/lymphoma effect to improve outcomes for patients with both diseases.

1988

Benzene established to cause cancer

Benzene established to cause cancer

Scientists find that occupational exposure to benzene, a chemical commonly used as a solvent and in oil-related products, is associated with increased risk of developing non-lymphocytic leukemia, non-Hodgkin lymphoma, and other diseases. Following this discovery, workers begin taking steps to protect themselves from benzene exposure and reduce their cancer risk.

1986

National bone marrow transplantation registry is established

National bone marrow transplantation registry is established

The National Marrow Donor Program (NMDP) operates the world's largest registry of unrelated adult donors and umbilical cord blood units. (Umbilical cord blood contains stem cells, which can help save the lives of some patients with blood-related cancers.) The registry facilitates and expedites matches between marrow and umbilical cord blood donors and the patients who need these lifesaving transplants.

1982

Daunorubicin proven superior to doxorubicin for AML

Daunorubicin proven superior to doxorubicin for AML

Large trial shows that using the anthracycline drug daunorubicin (Cerubidine, Rubidomycin), in combination with cytarabine (ara-C), is more effective at causing complete remissions of acute myelocytic leukemia – one of the most common leukemias in adults – than the previous standard drug, doxorubicin (Adriamycin). Over the following decade, researchers evaluate similar drugs, such as idarubicin (Idamycin), that provide similar, and possibly better, remission rates.

1977

Chlorambucil induces remissions for chronic lymphocytic leukemia

1975

Era of bone marrow transplantation begins – extending lives of many patients with leukemia and lymphoma

Era of bone marrow transplantation begins – extending lives of many patients with leukemia and lymphoma

The world's first transplant of bone marrow from a sibling donor is successfully performed in Minnesota in an infant with a severe immunodeficiency syndrome. Ten years later, bone marrow transplantation is successfully used to cure lymphoma. These discoveries usher in a new era in which bone marrow transplantation becomes a viable option for treating – and sometimes curing – leukemias, lymphomas and other blood diseases. Much of this progress can be attributed to the research of E. Donnall Thomas, who received the 1990 Nobel Prize in Physiology or Medicine for his pioneering work in the field. Subsequent refinements have enabled doctors to treat leukemias, lymphomas, and myelomas using patients' own marrow (autologous transplants) or with donations of stem cells obtained from umbilical cord blood.

1974

FDA approves doxorubicin

FDA approves doxorubicin

Doxorubicin (Adriamycin), an anticancer antibiotic, is widely used to treat many cancer types, including some leukemias and Hodgkin lymphoma. Together with cytarabine, doxorubicin induces acute myelogenous leukemia remissions by damaging the DNA of cancer cells. However, doxorubicin and similar drugs can cause serious and often long term side effects which require long-term monitoring.

1964

Cytarabine provides major boost to combination chemotherapy for acute myelocytic leukemia

Cytarabine provides major boost to combination chemotherapy for acute myelocytic leukemia

Various studies show that the drug cytarabine (ara-C) has activity against leukemias and is highly effective for treating a type of the disease known as acute myelocytic leukemia. Over time, the drug becomes a critical component of chemotherapy for adults and children with this form of leukemia, and researchers learn that precise timing and dosing of the drug is critical to successful treatment. The drug is still widely used today, often in combination with other chemotherapy drugs.

1961

FDA approves new chemotherapy drugs vinblastine and vincristine

FDA approves new chemotherapy drugs vinblastine and vincristine

Vinblastine (Velban, Velsar) is approved after researchers demonstrate that the drug blocks a key protein involved in cancer cell division and induces some leukemias and lymphomas into remission. Vincristine (Oncovin), a sister drug to vinblastine, is approved in 1963. These drugs are in a family of chemotherapies called "microtubule drugs," and further investigation leads to the development of similar, more effective and sometimes less toxic drugs for leukemia and other cancers, including lymphoma, breast cancer, ovarian cancer and others.

1960

Researchers link "Philadelphia chromosome" to leukemia

Researchers link "Philadelphia chromosome" to leukemia

Investigators in Philadelphia identify a chromosomal abnormality linked to many leukemias. A decade later, researchers discover that this abnormality results when parts of two chromosomes – chromosomes 9 and 22 – switch places in a phenomenon called translocation. It later becomes the target of one of the first-ever targeted cancer treatments, imatinib (Gleevec), which transforms treatment of chronic myelogenous leukemia and other cancers.

1958

US government researchers pioneer use of combination chemotherapy

US government researchers pioneer use of combination chemotherapy

In research sponsored by the National Cancer Institute, scientists find that a combination of the drugs 6-mecaptopurine and methotrexate can reduce or eliminate cancer growth and extend survival in patients with leukemia. The findings herald a new era in the treatment of leukemia, and cancer in general, in which carefully honed drug combinations allow doctors to attack cancer cells from different angles. Combination therapy is now a mainstay of treatment for a wide range of cancers.

1952

Researchers discover ways to protect the body from radiation damage

Researchers discover ways to protect the body from radiation damage

Early studies explore ways to protect healthy tissue in the body from the side effects of intense cancer treatment. Leon Jacobson and others discover that shielding a mouse's spleen from radiation can protect the mouse from otherwise lethal radiation. Eventually, scientists learn that the body recruits stem cells, which are found in the spleen and bone marrow, to protect and heal itself from radiation damage. This finding paves the way for the development and widespread use of stem cell transplantation in patients with lymphoma and leukemia, who often receive intensive radiation therapy and chemotherapy to treat their disease.

1947

First compound discovered to inhibit growth of cancer

First compound discovered to inhibit growth of cancer

Sidney Farber first discovers that a drug, called aminopterin, can induce remissions in acute lymphocytic leukemia in children. Although these remissions were temporary, this finding leads to the development of a new category of chemotherapy drugs, called antimetabolites, that impair the ability of cancer cells to grow and replicate. Soon, further research shows that a similar drug, called methotrexate, is superior to aminopterin for leukemia treatment. Methotrexate remains in use today.