Leukemia Research Results and Study Updates
See Advances in Leukemia Research for an overview of recent findings and progress, plus ongoing projects supported by NCI.
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Whole-Genome Sequencing Could Help Guide AML TreatmentPosted:
For people with acute myeloid leukemia and related cancers, a new study shows whole-genome sequencing could replace a series of conventional tests used to help guide decisions about treatment.
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Blinatumomab Improves Survival in Children with Relapsed LeukemiaPosted:
The results of two trials establish blinatumomab (Blincyto) as a new standard treatment for children and young adults with high-risk relapsed B-cell acute lymphoblastic leukemia after remission has been achieved and before a stem cell transplant.
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Stopping TKI Treatment Is Safe, Improves Quality of Life for Some with CMLPosted:
For adults with CML who are in a sustained deep molecular remission, stopping treatment with a tyrosine kinase inhibitor is safe and improves their quality of life, a study shows. But researchers cautioned that these patients must be closely monitored.
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Rediscovered Drugs Hit Leukemia from Two Different AnglesPosted:
Two rediscovered drugs, bisantrene and brequinar, slowed the growth of acute myeloid leukemia in studies of mice. The drugs blocked the activity of a protein called FTO, killing cancer stem cells and helping the immune system attack the cancer.
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Maintenance Therapy with CC-486 Extends Survival of Adults with AMLPosted:
Maintenance therapy with CC-486 extended overall survival of adults with the blood cancer acute myeloid leukemia (AML) in a large clinical trial. CC-486 is a pill form of another cancer therapy called azacitidine (Vidaza).
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Immunotherapy drug improves outcomes for some children with relapsed leukemiaPosted:
For children and young adults with certain relapsed B-cell acute lymphoblastic leukemia (B-ALL), the immunotherapy drug blinatumomab is superior to standard chemotherapy, an NCI-sponsored Children’s Oncology Group trial shows.
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Gilteritinib Improves Survival in AML with FLT3 MutationsPosted:
People with relapsed or refractory acute myeloid leukemia (AML) with FLT3 gene mutations treated with gilteritinib had improved survival, higher rates of remission, and fewer side effects than those treated with chemotherapy, a recent trial found.
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Children with Acute Lymphoblastic Leukemia Can Skip Radiation to the BrainPosted:
Only 1.5% of children with acute lymphoblastic leukemia who skipped radiation had a recurrence in the central nervous system, according to a recent trial. The therapy, which is intended to prevent such a recurrence, can have devastating side effects.
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Combination Therapy with Venetoclax Approved for Chronic Lymphocytic LeukemiaPosted:
The Food and Drug Administration has approved venetoclax (Venclexta) in combination with obinutuzumab (Gazyva) for the initial treatment of adults with chronic lymphocytic leukemia or small lymphocytic lymphoma.
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Tagraxofusp Proves Effective for BPDCN, A Rare Blood CancerPosted:
New findings from a clinical trial of the drug tagraxofusp confirm its efficacy against the rare blood cancer blastic plasmacytoid dendritic cell neoplasm (BPDCN).
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Eliminating Hairy Cell Leukemia Minimal Residual DiseaseReviewed:
In this trial, patients with hairy cell leukemia who have disease-related symptoms that require treatment, and who have not been treated or have had only one prior treatment with cladribine, will be randomly assigned to receive cladribine with either concurrent rituximab or rituximab at least 6 months after completing cladribine therapy.
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Treating Multiply Relapsed or Refractory Hairy Cell LeukemiaReviewed:
In this trial, patients with hairy cell leukemia who have not responded to initial chemotherapy followed by second-line treatment with rituximab, or who have relapsed following two courses of chemotherapy, will be randomly assigned to receive rituximab combined with either pentostatin or bendamustine.
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Treatment for Children with Leukemia Also Effective for Adolescents, Young AdultsPosted:
A clinical trial found that an intensive treatment regimen developed specifically for children with acute lymphoblastic leukemia is also effective for older adolescents and young adults with the disease.
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FDA Approvals Bring New Options for Older Patients with AMLPosted:
FDA has approved venetoclax (Venclexta) and glasdegib (Daurismo) for use in people with acute myeloid leukemia aged 75 and older and those with health conditions that prevent them from receiving the intensive chemotherapy regimen that is the standard initial treatment.
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Drug Combination May Target the Unique Metabolism of Leukemia Stem CellsPosted:
Two new studies show how the drugs venetoclax (Venclexta) and azacitidine (Vidaza) team up to block the unique metabolism of leukemia stem cells and may explain why the drug combination is effective against acute myeloid leukemia.
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Ibrutinib plus rituximab superior to standard treatment for some patients with chronic leukemiaPosted:
A clinical trial showed that ibrutinib plus rituximab is superior to standard treatment for patients age 70 and younger with untreated chronic lymphocytic leukemia (CLL). Findings were announced at the American Society of Hematology annual meeting.
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Moxetumomab Approved by FDA for Hairy Cell LeukemiaPosted:
The FDA has approved moxetumomab pasudotox (Lumoxiti), a bacterial toxin–based drug, for the treatment of some patients with hairy cell leukemia (HCL). Moxetumomab is approved to treat patients with HCL who have already undergone at least two lines of standard treatments.
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Targeted Drug Approved for Acute Myeloid Leukemia with IDH1 Gene MutationsPosted:
The FDA has approved ivosidenib (Tibsovo) for the treatment of adults with acute myeloid leukemia (AML) that has a specific mutation in a gene called IDH1. Ivosidenib becomes the first FDA-approved IDH1-targeted treatment.
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Approval Expanded for Venetoclax in Chronic Lymphocytic LeukemiaPosted:
FDA expanded the approval of venetoclax (Venclexta) for people with chronic lymphocytic leukemia (CLL) to include those whose cancer has progressed after previous treatment, regardless of whether their cancer cells have the deletion 17p gene alteration.
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Trial Produces Practice-Changing Findings for Some Children, Young Adults with LeukemiaPosted:
This NCI-funded Children’s Oncology Group trial tested the addition of nelarabine (Arranon) to standard treatment for children and young adults with T-cell acute lymphoblastic leukemia (T-ALL).
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Toxin-Based Drug Moxetumomab Pasudotox May Be New Option for Rare LeukemiaPosted:
People diagnosed with hairy cell leukemia (HCL) may have an effective new treatment option, a type of drug called an immunotoxin. Read more about how this treatment, moxetumomab pasudotox, fared in a phase 3 clinical trial in patients with advanced HCL.
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Drug May Help Prevent Resistance to Toxin-Based Leukemia TherapyPosted:
A new study has identified a possible strategy for improving the efficacy of a toxin-based cancer treatment, moxetumomab pasudotox, in some patients with acute lymphoblastic leukemia (ALL).
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TARGET Study Finds Major Differences between Childhood and Adult AMLPosted:
An NCI-funded study has found significant differences in the genetics of acute myeloid leukemia in younger and older patients. The findings could help guide the development of treatments tailored specifically for childhood AML.
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Nilotinib Can Be Discontinued in Some Patients with Chronic Myelogenous LeukemiaPosted:
On December 22, FDA approved an update to the label of nilotinib (Tasignia) that states that some patients with CML who are taking nilotinib and whose cancer has been in remission for an extended period can safely stop taking it.
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Drug Combination Improves Outlook for Some Patients with Chronic Lymphocytic LeukemiaPosted:
Interim results from an ongoing clinical trial show that patients with relapsed or refractory chronic lymphocytic leukemia treated with rituximab plus venetoclax have longer progression-free survival compared with patients treated with chemotherapy.
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Unique Trial Aims to Decrease Early Deaths in Patients with Rare LeukemiaPosted:
In a unique clinical trial, a group of oncologists with experience treating acute promyelocytic leukemia are making themselves available around the clock to help clinicians at hospitals across the country treat their APL patients.
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Dasatinib Approved by FDA for Some Children with Chronic Myelogenous LeukemiaPosted:
On November 9, the FDA approved dasatinib (Sprycel®) for the treatment of children with chronic myelogenous leukemia (CML) whose cancer cells express the Philadelphia chromosome and whose disease is in a relatively early stage, known as the chronic phase.
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Gemtuzumab Receives New FDA Approval for Acute Myeloid LeukemiaPosted:
FDA has approved gemtuzumab ozogamicin (Mylotarg™) for adults with newly diagnosed CD33-positive AML and patients 2 years and older with CD33-positive AML who have experienced a relapse or whose disease has not responded to initial treatment.
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FDA Approves Inotuzumab for Adults with B-Cell Acute Lymphoblastic LeukemiaPosted:
FDA has approved inotuzumab (Besponsa®) for some adults with B-cell acute lymphoblastic leukemia (ALL). The approval covers patients with B-cell ALL whose disease has relapsed or is refractory to standard chemotherapy.
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CAR T-Cell Therapy Approved for Some Children and Young Adults with LeukemiaPosted:
FDA has approved tisagenlecleucel (Kymriah™), a type of immunotherapy called CAR T-cell therapy, for some children and young adults with advanced acute lymphoblastic leukemia (ALL).
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Two New Therapies Approved for Acute Myeloid LeukemiaPosted:
FDA has approved two new treatments for some adult patients with acute myeloid leukemia (AML): enasidenib (Idhifa®), which targets the IDH2 protein; and liposomal cytarabine-daunorubicin CPX-351 (Vyxeos®), a two-drug chemotherapy combination encapsulated in liposomes.
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FDA Grants Full Approval to Blinatumomab for Acute Lymphoblastic LeukemiaPosted:
FDA changed its accelerated approval of blinatumomab (Blincyto) for some patients with acute lymphoblastic leukemia to a full approval and expanded the approved indications for its use.
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New Rituximab Formulation Approved for Some Lymphomas, LeukemiaPosted:
The FDA has approved a new formulation of rituximab, Rituxan Hycela, that reduces treatment administration time for patients with several types of blood cancer.
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Midostaurin Approved by FDA for Acute Myeloid LeukemiaPosted:
The FDA has approved midostaurin for patients with newly diagnosed acute myeloid leukemia (AML) with mutations in the FLT3 gene. The approval also covers several rare conditions.
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Intensive Pre-Stem Cell Transplant Regimen May be Best for Younger Patients with AML, MDSPosted:
Results from a large phase III clinical trial suggest that a highly intensive preparatory regimen should be used for younger patients with acute myeloid leukemia or myelodysplastic syndromes preparing to undergo an allogeneic stem cell transplant.
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Blinatumomab Extends Survival for Patients with Advanced ALLPosted:
Patients with previously treated acute lymphoblastic leukemia who received blinatumomab, which encourages the immune system to kill cancer cells, lived longer and experienced fewer side effects than patients given standard chemotherapy.
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New Mouse Model Closely Mimics Most Common Leukemia in InfantsPosted:
Researchers have created a long-sought-after mouse model for an aggressive form of acute lymphoblastic leukemia that occurs commonly in infants and that the researchers believe can accelerate the development of new therapies for the disease.
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Modified Immunotherapy Approach Shows Promise for LeukemiaPosted:
Researchers have developed an immunotherapy approach that uses immune cells whose natural cancer killing abilities have been enhanced in the lab. In a small clinical trial, the treatment led to remissions in about half of patients with acute myeloid leukemia.
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TARGET Initiative Fueling Progress against Childhood LeukemiaPosted:
Research from TARGET, an NCI-funded program supporting genomic analyses of childhood cancers, has led to the launch of several clinical trials of targeted therapies to treat children with acute lymphoblastic leukemia.
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Venetoclax Approved for Some Patients with Chronic Lymphocytic LeukemiaPosted:
The FDA has approved venetoclax for patients with chronic lymphocytic leukemia whose tumors have a specific genetic alteration.
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Two Drugs Show Efficacy against Common Form of LeukemiaPosted:
Reports from early-stage trials of two new oral drugs provide hope for patients with high-risk chronic lymphocytic leukemia.
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Persistence of Genetic Mutations after Chemotherapy Linked to Poor Outcomes in Some Patients with AMLPosted:
The persistence of genetic mutations in some patients with acute myeloid leukemia may allow physicians to better classify their risk of recurrence.