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Treatment Update: Blood Cancers
Each year, CancerCare publishes a special edition of the CancerCare Connect Booklet Series that presents research highlights from the Annual Meeting of the American Society of Hematology. The information contained in these pages is intended for discussion with your doctor. They can tell you whether these advances in cancer treatment affect your treatment plan and whether a clinical trial is right for you.
Some of the treatments discussed in this booklet are still in the very early stages of research and may not be available to the general public outside of a clinical trial. The advances in treatment that have come about are because of the many people who have taken part in such studies. If current drugs or other types of cancer treatment no longer benefit you, you may wish to explore joining a clinical trial. The members of your health care team will help you fully understand the possible risks and benefits involved.
The Importance of Clinical Trials
Clinical trials are the standard by which we measure the worth of new treatments and the quality of life of patients as they receive those treatments. For this reason, doctors and researchers urge people with cancer to take part in clinical trials.
Your doctor can guide you in making a decision about whether a clinical trial is right for you. Here are a few things that you should know:
- Often, people who take part in clinical trials gain access to and benefit from new treatments.
- Before you participate in a clinical trial, you will be fully informed of the risks and benefits of the trial, including any possible side effects.
- Many clinical trials are designed to test a new treatment against a standard treatment to find out whether the new treatment has any added benefit.
- Participation is voluntary and does not affect your access to treatment in other settings. You can stop taking part in a clinical trial at any time for any reason.
When considering participation in a clinical trial, it’s important to consult with your primary care physician and your oncologist and make sure that all of your questions are answered.
This is a very exciting time in cancer research, and there are clinical trials underway to study newer treatment approaches, such as immunotherapy and targeted therapy. In immunotherapy, the immune system’s ability to seek out and destroy cancer cells is enhanced. Targeted therapies are designed to target the specific cell mechanisms that are important for the growth and survival of tumor cells.
Researchers reported a number of important findings in the treatment of leukemia at the 2024 Annual Meeting of the American Society of Hematology:
Results from the phase lll AMPLIFY trial showed that acalabrutinib, combined with venetoclax, was effective and well tolerated in the treatment of CLL.
The safety and efficacy of ponatinib and blinatumomab was evaluated as a chemotherapy-free frontline treatment for Ph+ ALL.
Follow-up efficacy and safety results from a phase III trial demonstrated the benefit of adding midostaurin to intensive chemotherapy in previously untreated FLT3-mutated AML.
In the pivotal phase lll ASC4FIRST trial, the TKI asciminib demonstrated favorable safety and tolerability compared to standard of care TKIs in newly diagnosed CML-CP.
Acalabrutinib plus venetoclax effective in treatment of CLL
Results from the phase lll AMPLIFY trial showed that acalabrutinib, combined with venetoclax, was effective and well tolerated in the treatment of CLL. Acalabrutinib is a Bruton tyrosine kinase (BTK) inhibitor that blocks signals to the cell’s control center that help tumor cells grow. Venetoclax targets the B-cell lymphoma 2 (BCL-2) protein, leading to the death of CLL cells.
The results could lead to the FDA approval of the first all-oral frontline CLL therapy.
What Patients Need to Know
The trial showed the addition of the monoclonal antibody obinutuzumab improved efficacy (effectiveness) but caused an increased risk of infection.
Ponatinib and blinatumomab safe and effective as frontline treatment for Ph+ ALL
Updated results from the phase III GIMEMA ALL2820 trial showed the safety and efficacy of ponatinib and blinatumomab as a chemotherapy-free frontline treatment for Philadelphia chromosome–positive acute lymphoblastic leukemia (ALL).
What Patients Need to Know
Ponatinib is a tyrosine kinase inhibitor that interferes with the growth of cancer cells. Blinatumomab is a bispecific T-cell engager that targets specific proteins on the surface of cancer cells.
Follow-up results reported on adding midostaurin to chemotherapy in AML treatment
Ten-year follow-up efficacy and safety results from the phase III C10603/RATIFY trial demonstrated the benefit of adding the FLT3 inhibitor midostaurin to intensive chemotherapy in previously untreated FLT3-mutated AML.
What Patients Need to Know
FLT3 inhibitors are drugs that penetrate into the cell and target a specific cellular mechanism to control cancer cell growth.
Asciminib demonstrated favorable results compared to other TKIs in CML-CP
In the pivotal phase lll ASC4FIRST trial, the tyrosine kinase inhibitor (TKI) asciminib demonstrated favorable safety and tolerability compared to standard of care TKIs in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP). TKIs work by blocking the action of an abnormal protein that signals cancer cells to multiply.
What Patients Need to Know
The primary goal of a pivotal trial is to demonstrate that a new treatment is effective and safe enough to be approved for use in the general population.
Researchers reported a number of important findings in the treatment of lymphoma at the 2024 Annual Meeting of the American Society of Hematology:
Adding tafasitamab to lenalidomide and rituximab showed benefit in the treatment of relapsed/refractory CD19/ CD20+ follicular lymphoma.
Data from a phase lll trial showed pirtobrutinib significantly reduced the risk of disease progression or death in relapsed/refractory CLL/small lymphocytic lymphoma.
Compared with maintenance therapy alone, patients with no detectable cancer after initial therapy for MCL did not experience any survival benefit from undergoing an autologous hematopoietic cell transplant.
Primary analysis of a phase II trial showed mosunetuzumab demonstrated benefit when used as first-line therapy for follicular lymphoma.
Adding tafasitamab of benefit in the treatment of CD19/CD20+ follicular lymphoma
Data from the phase lll inMIND trial showed adding tafasitamab to lenalidomide and rituximab reduces the risk of disease progression, relapse or death in patients with relapsed/refractory CD19/CD20+ follicular lymphoma that did not respond to or recurred after prior therapy.
What Patients Need to Know
Tafasitamab is a monoclonal antibody that binds to the CD19 protein, potentially helping the immune system kill cancer cells.
Benefits of pirtobrutinib in the treatment of CLL/small lymphocytic lymphoma
In relapsed/refractory CLL/small lymphocytic lymphoma, interim data from the phase III trial BRUIN CLL- 321 showed pirtobrutinib significantly reduced the risk of disease progression or death compared to idelalisib plus rituximab or bendamustine plus rituximab.
What Patients Need to Know
Pirtobrutinib is a Bruton tyrosine kinase (BTK) inhibitor. Kinase proteins send signals to the cell’s control center to help tumor cells grow. Kinase inhibitors block these proteins.
Evaluation of auto-HCT in MRD-negative mantle cell lymphoma
According to interim data from the phase lll trial EA4151, patients with no detectable cancer after initial therapy for MCL did not experience any survival benefit from undergoing an autologous hematopoietic cell transplantation compared with maintenance therapy alone.
Auto-HCT is a procedure where a patient’s own stem cells are collected, stored and then reinfused after high-dose chemotherapy to restore blood and immune function.
What Patients Need to Know
The findings suggest that patients who test negative for minimal residual disease (MRD) do not need to undergo a stem cell transplant or the high-dose chemotherapy regimen used to prepare for the transplant.
Analysis of mosunetuzumab as first-line therapy for follicular lymphoma
Primary analysis of the phase II MITHIC-FL1 trial of mosunetuzumab monotherapy demonstrated a high overall response rate, a high 1-year overall survival rate and a manageable safety profile when used as first-line therapy for follicular lymphoma.
What Patients Need to Know
Mosunetuzumab is currently approved by the FDA for the treatment of relapsed or refractory follicular lymphoma after two or more therapy regimens. It works by bringing healthy T cells and lymphoma cells close together so the T cells can more effectively destroy the lymphoma cells.
Researchers reported a number of important findings in the treatment of multiple myeloma at the 2024 Annual Meeting of the American Society of Hematology:
Compared with active monitoring, the phase lll AQUILA trial showed significant improvement in progression-free survival with the use of daratumumab in the treatment of smoldering multiple myeloma.
Results from a phase lll trial suggested that daratumumab plus lenalidomide with only 2 cycles of dexamethasone show benefit in the treatment older patients with multiple myeloma.
According to data from a phase lll trial, maintenance therapy consisting of teclistamab alone or in combination with lenalidomide was safe and effective in treating people with multiple myeloma who underwent prior induction therapy and an autologous stem cell transplant.
Results from the phase lll DREAMM-7 trial showed sustained overall survival benefit with the use of belantamab mafodotin compared to daratumumab.
Monoclonal antibody evaluated in the treatment of smoldering multiple myeloma
In the treatment of smoldering (not causing symptoms) multiple myeloma, results from the phase lll AQUILA trial showed significant improvement in progression-free survival from 3 years of subcutaneous use of the monoclonal antibody daratumumab as compared with active monitoring.
What Patients Need to Know
Monoclonal antibodies are lab-generated proteins that target specific substances on the surface of lymphoma cells, triggering an immune response.
Dexamethasone-sparing treatment shows benefit in older patients with MM
Results from the phase lll IFM2017-03 trial suggest that, in older patients with multiple myeloma, daratumumab plus lenalidomide with only 2 cycles of dexamethasone can improve overall survival and progression-free survival when compared to continued treatment with dexamethasone and lenalidomide.
What Patients Need to Know
According to experts, these results could change the treatment approach of newly diagnosed and relapsed or refractory multiple myeloma.
Maintenance therapy evaluated in phase lll trial
According to data from the MajesTEC-4/EMN30 phase lll trial, maintenance therapy consisting of teclistamab alone or in combination with lenalidomide was safe and effective in patients with multiple myeloma who underwent prior induction therapy and an autologous stem cell transplant (ASCT).
What Patients Need to Know
Teclistamab is a first-in-class, bispecific T-cell engager antibody. Bispecific antibodies aim at two cancer targets, increasing the treatment’s potency. Lenalidomide, an immunomodulatory drug, leads to the degradation (reduction) of growth signals in myeloma cells.
Overall survival benefit shown with use of belantamab mafodotin
In the treatment of multiple myeloma, results from the phase lll DREAMM-7 trial showed sustained overall survival benefit with the use of belantamab mafodotin with bortezomib/dexamethasone compared to daratumumab with bortezomib/dexamethasone.
What Patients Need to Know
Both belantamab mafodotin and daratumumab are monoclonal antibodies, lab-generated proteins that target specific substances on the surface of myeloma cells, triggering an immune response.
Researchers reported a number of important findings in the treatment of myeloproliferative neoplasms (MPNs) at the 2024 Annual Meeting of the American Society of Hematology:
Navtemadlin, a novel MDM2 inhibitor, reduced the biomarkers of disease severity in JAK inhibitor-relapsed/ refractory myelofibrosis.
Compared to placebo plus ruxolitinib, the investigational drug pelabresib plus ruxolitinib demonstrated several improvements in patients with JAK inhibitor-naive myelofibrosis.
Results from a phase ll trial showed selinexor, given in combination with ruxolitinib, demonstrated encouraging effectiveness in the treatment of myelofibrosis.
Momelotinib demonstrated improved overall survival vs. best available therapy in patients with myelofibrosis pretreated with ruxolitinib.
Navtemadlin evaluated as treatment for relapsed/refractory myelofibrosis
In the phase III BOREAS trial, navtemadlin given as a single agent was compared to the best available therapy in patients with JAK inhibitor-relapsed/refractory myelofibrosis.
What Patients Need to Know
Navtemadlin, a novel MDM2 inhibitor, reduced the biomarkers of disease severity, suggesting the drug may target the diseasedriving cells and alter its progression.
Pelabresib showed benefit in treating JAK inhibitor-naive myelofibrosis
According to results from the phase lll MANIFEST-2 trial, the investigational drug pelabresib plus ruxolitinib demonstrated improvements in spleen volume, total symptom score, anemia and the bone marrow microenvironment in patients with JAK inhibitor-naive myelofibrosis. The comparison was to placebo plus ruxolitinib.
What Patients Need to Know
Pelabresib is a small-molecule inhibitor targeting the bromodomain and extra-terminal domain (BET). Ruxolitinib, a JAK inhibitor, is considered the standard of care for most people with primary or secondary myelofibrosis.
Selinexor demonstrated effectiveness in treatment of myelofibrosis
Selinexor, given in combination with ruxolitinib, demonstrated encouraging effectiveness with a manageable safety profile in patients with myelofibrosis previously treated with ruxolitinib. The data presented was from a phase ll trial.
What Patients Need to Know
Selinexor is a Selective Inhibitor of Nuclear Export (SINE). It works by inhibiting the action of proteins.
Improved overall survival in myelofibrosis treated with momelotinib
Momelotinib demonstrated improved overall survival vs. best available therapy in patients with myelofibrosis pretreated with ruxolitinib, according to data from a matching-adjusted indirect comparison analysis. Momelotinib is a JAK inhibitor that works by blocking the activity of certain enzymes involved in the development of myelofibrosis.
What Patients Need to Know
A Matching-Adjusted Indirect Comparison (MAIC) is a statistical method used in comparative effectiveness research, particularly in health technology assessments and clinical trials.