Leukemia Research Results and Study Updates

Treatment options for people with acute myeloid leukemia (AML) have expanded yet again. On July 20, FDA approved quizartinib (Vanflyta) combined with chemotherapy as a first-line treatment for AML with a specific change in the FLT3 gene.

Giving the drug blinatumomab (Blincyto) after standard chemotherapy substantially increased survival for infants with an aggressive form of acute lymphoblastic leukemia (ALL), a recent study showed. If confirmed in larger studies, the treatment may become standard therapy for infants with ALL caused by KMT2A rearrangements.

Treatment with revumenib caused complete remission in about one-third of participants in an early-phase clinical trial involving patients who’d had many prior treatments. Revumenib is part of a new class of targeted drugs known as menin inhibitors.

An NCI-funded clinical trial has shown that treatment-related early deaths in people with a rare leukemia can be dramatically reduced. How did they do it? In part, by establishing a help desk staffed by experts in treating APL.

FDA has approved zanubrutinib (Brukinsa) for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) based on results from two clinical trials. In both trials, the drug, which blocks a protein called BTK, was more effective and caused fewer side effects than other treatments.

The immunotherapy drug blinatumomab (Blincyto) extends life for people with acute lymphoblastic leukemia who are in remission, even those with no signs of disease after initial treatment, a trial has found.

For some people with acute myeloid leukemia (AML) that has a mutation in the IDH1 gene, combining ivosidenib (Tibsovo) with the chemotherapy drug azacitidine may be a new treatment option, according to results from a large clinical trial.

Removing immune cells called naive T cells from donated stem cells before they are transplanted may prevent chronic graft-versus-host disease (GVHD) in people with leukemia, a new study reports. The procedure did not appear to increase the likelihood of patients’ cancer returning.

A Children’s Oncology Group trial shows that the combination of all-trans retinoic acid and arsenic trioxide is highly effective in children with acute promyelocytic leukemia. The therapy avoids or minimizes the use of conventional chemotherapy.

The CAR T-cell therapy Tecartus has become the first such treatment approved by FDA to treat adults with acute lymphoblastic leukemia (ALL). The approval is for patients whose cancer has not responded to treatment or returned after treatment.

FDA has approved belumosudil (Rezurock) for the treatment of chronic graft-versus-host disease (GVHD). The approval covers the use of belumosudil for people 12 years and older who have already tried at least two other therapies.

FDA has approved a new form of asparaginase called Rylaze. The drug was developed to help alleviate shortages of Erwinia asparaginase, a key part of treatment for children and adults with acute lymphoblastic leukemia.

In a small study, vemurafenib (Zelboraf) and rituximab (Rituxan) helped 85% of participants stay in remission for nearly 3 years. The study involved 30 people with hairy cell leukemia that had come back after or had not responded to previous treatment.

People with blood cancers seem to be less protected by COVID-19 vaccines than those with other cancers and people without cancer, three new studies suggest. Experts believe this limited effectiveness is likely due to patients’ weakened immune systems.

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.

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.

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.

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.

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).

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.

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.

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.

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.

New findings from a clinical trial of the drug tagraxofusp confirm its efficacy against the rare blood cancer blastic plasmacytoid dendritic cell neoplasm (BPDCN).

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.

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.

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.

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.

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.

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.

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.

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.

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).

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.

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).

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.

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.

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.

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.

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.

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.

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.

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).

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.

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.

The FDA has approved a new formulation of rituximab, Rituxan Hycela, that reduces treatment administration time for patients with several types of blood cancer.

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.

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.

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.