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Revumenib: New Data Signals Broader Cancer Potential

by Dr. Priya Deshmukh - Senior Editor, Health
written by Dr. Priya Deshmukh - Senior Editor, Health

Revumenib’s Expanding Role: Could a Single Drug Reshape Treatment for Multiple AML Subtypes?

A staggering 30% of acute myeloid leukemia (AML) patients harbor NPM1 mutations, a genetic alteration historically linked to poor prognosis. But new data presented at the 2025 European Hematology Association (EHA) Congress suggests a potential turning point: **revumenib** (Revuforj; Syndax) – initially approved for KMT2A-rearranged AML – is showing promising efficacy in this challenging patient population, potentially redefining the treatment landscape.

Beyond KMT2A: Revumenib’s Unexpected Potential

Approved by the FDA in November 2024, revumenib represents a significant advance in precision oncology. This first-in-class menin blocker disrupts the interaction between the menin protein and KMT2A fusion proteins, effectively halting the oncogenic processes driving leukemia growth. However, the initial approval only addressed a specific genetic subset of AML. The AUGMENT-101 trial (NCT04065399) data unveiled at EHA 2025 dramatically expands the drug’s potential reach.

AUGMENT-101: Promising Results in NPM1-Mutant AML

The study focused on 77 patients with relapsed or refractory (R/R) AML carrying NPM1 mutations. Results demonstrated a complete remission (CR) and CR with partial hematologic recovery (CRh) rate of approximately 26%, with a median duration of response of 4.7 months and a median time to first response of 2.8 months. Crucially, a remarkable 63% of evaluable responders achieved measurable residual disease (MRD)-negativity – a strong indicator of long-term remission. This level of deep molecular response is particularly encouraging, suggesting revumenib isn’t just managing the disease, but potentially eradicating it at a fundamental level.

The Mechanism: Why NPM1-Mutant AML May Respond

While the precise mechanisms are still under investigation, researchers believe revumenib’s efficacy in NPM1-mutant AML stems from its broader impact on transcriptional regulation. The menin protein isn’t solely involved in KMT2A-driven leukemias; it plays a role in regulating gene expression across various cancer types. By disrupting menin’s function, revumenib can impact multiple oncogenic pathways, including those activated by NPM1 mutations. This broader mechanism of action explains why the drug is showing activity beyond its initial target.

FDA Fast Track and the Future of NPM1-Mutant AML Treatment

The compelling data from the AUGMENT-101 trial prompted the FDA to expedite the review of a supplemental New Drug Application (sNDA) for revumenib in NPM1-mutant AML. With a Prescription Drug User Fee Act (PDUFA) goal date of October 25, 2025, approval could provide a much-needed therapeutic option for a patient population with limited effective treatments. Currently, options for R/R NPM1-mutant AML are largely limited to intensive chemotherapy, often with significant side effects and modest response rates. Revumenib offers the potential for a more targeted and tolerable approach.

Implications for Oncology Pharmacy Practice

The increasing use of targeted therapies like revumenib is fundamentally changing the role of oncology pharmacists. Beyond traditional dispensing and monitoring, pharmacists are now integral in verifying genetic eligibility through comprehensive molecular testing, optimizing dosage schedules based on individual patient characteristics, proactively managing potential adverse effects, and providing crucial education to both patients and healthcare teams. As biomarker-driven cancer treatment expands, the pharmacist’s expertise in pharmacogenomics and personalized medicine will become even more critical. Learn more about the evolving role of the oncology pharmacist at ASHP’s Oncology Pharmacy Practice page.

Precision Oncology’s Trajectory: Beyond Single Targets

Revumenib’s journey highlights a crucial trend in cancer treatment: the limitations of focusing on single targets. While initially developed for KMT2A-rearranged AML, its efficacy in NPM1-mutant AML demonstrates the potential for drugs to have broader applications than initially anticipated. This suggests that future drug development may increasingly focus on targeting key regulatory proteins – like menin – that influence multiple oncogenic pathways. The success of revumenib could pave the way for similar “repurposing” of existing drugs and the development of novel agents with broader spectrum activity. What are your predictions for the future of menin-targeting therapies in hematologic malignancies? Share your thoughts in the comments below!

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Health

Unlocking TAF1: A Potential Breakthrough in Cancer Therapy

by Dr. Priya Deshmukh - Senior Editor, Health
written by Dr. Priya Deshmukh - Senior Editor, Health

TAF1: A Crucial Switch for Blood Cell Development Revealed

Table of Contents

Miami, FL – New research is poised to reshape our understanding of how blood cells develop, highlighting a protein named TAF1 as a critical regulator. Findings published in Developmental Cell suggest TAF1 acts as a molecular switch, orchestrating the delicate balance between maintaining blood stem cells and guiding them to mature into specialized blood cells, particularly in adults.

This finding challenges the long-held belief that TAF1 is a worldwide requirement for gene activation in all cells throughout life. Instead, the study indicates TAF1 plays a more nuanced role, specifically promoting gene expression that drives hematopoietic stem cells (HSCs) toward differentiation.”The most surprising discovery is that adult HSCs can survive without an crucial general transcription factor, and that the TAF1 loss only affects the activation of differentiation-associated genes, not the self-renewal-promoting genes,” stated Fan Liu, Ph.D., the study’s lead author and a researcher at Sylvester Thorough Cancer Center.

TAF1’s mechanism of action involves both initiating transcription and releasing a natural brake on the process, according to the research team. This fine-tuned control is vital for healthy blood production.

The implications of these findings are significant, particularly for cancer therapeutics. Developing drugs that effectively target cancer cells without harming normal blood cell production has been a major challenge in hematology. The data suggest that TAF1 inhibitors could be a promising avenue, as inhibiting TAF1 did not impede essential stem cell self-renewal or blood cell production in the study.

“A key question that we needed to address was: if you successfully target TAF1, do you compromise normal blood production? What this paper says is no,” commented Dr. Nimer, a study author.

Furthermore, this research opens doors for improving stem cell transplantation by potentially harnessing TAF1 to enhance the expansion of HSCs in laboratory settings.Future investigations will explore whether TAF1 performs similar regulatory functions in other stem cell populations, such as those found in the colon or brain, and their relevance to cancer.

How might understanding TAF1 inactivation mechanisms lead to the progress of novel epigenetic therapies?

Unlocking TAF1: A Potential Breakthrough in Cancer Therapy

What is TAF1 and Why is it Significant?

TAF1 (TATA-box binding protein-associated factor 1) is a crucial component of the TFIID complex, a key regulator of gene transcription. Essentially, it helps turn genes “on” or “off.” Recent research, particularly over the last five years, has highlighted TAF1’s frequent inactivation in a wide range of cancers, including ovarian, breast, lung, and hematological malignancies. This inactivation isn’t just a correlation; it’s increasingly understood as a driver of cancer development and progression. Understanding TAF1’s role opens new avenues for targeted cancer therapies. The field of cancer genomics is rapidly advancing our understanding of these genetic drivers.

The Role of TAF1 in Cancer Development

TAF1 acts as a tumor suppressor gene. When it’s functioning correctly, it helps maintain genomic stability and regulates cell growth. However, when TAF1 is mutated or silenced – often through epigenetic mechanisms like promoter methylation – several critical processes are disrupted:

Increased Genomic Instability: Loss of TAF1 leads to errors during DNA replication and repair, accelerating the accumulation of mutations. This is a hallmark of cancer.

Uncontrolled Cell Proliferation: TAF1 normally regulates genes involved in cell cycle control. Its absence allows cells to divide uncontrollably.

Impaired DNA Damage response: cancer cells with TAF1 loss are less able to respond to DNA damage, making them more resistant to conventional therapies like chemotherapy and radiation.

Enhanced Metastasis: Studies suggest TAF1 inactivation can promote the spread of cancer cells to distant sites. Metastatic cancer remains a significant clinical challenge.

Mechanisms of TAF1 Inactivation: A Deeper Dive

TAF1 isn’t typically deleted as a whole gene. Rather, it’s usually inactivated through more subtle mechanisms:

Promoter Methylation: This is the most common mechanism. Adding methyl groups to the TAF1 gene’s promoter region silences its expression. Epigenetic therapy aims to reverse these changes.

Mutations: While less frequent, specific mutations within the TAF1 gene can disrupt its function. These mutations are frequently enough identified through next-generation sequencing (NGS).

MicroRNA Regulation: Certain microRNAs can bind to the TAF1 mRNA, leading to its degradation and reduced protein levels.

Ubiquitin-Proteasome System: Dysregulation of this system can lead to increased degradation of the TAF1 protein.

Therapeutic Strategies Targeting TAF1 Deficiency

The growing understanding of TAF1’s role has spurred the development of several promising therapeutic strategies:

  1. Epigenetic Modulators: Drugs like 5-azacytidine and decitabine are DNA methyltransferase inhibitors (DNMTis).They can remove methyl groups from the TAF1 promoter, restoring its expression. These are already approved for certain hematological malignancies and are being investigated in solid tumors.
  2. Synthetic Lethality Approaches: this strategy exploits the fact that cancer cells with TAF1 loss are particularly vulnerable to the inhibition of certain other genes. such as, research suggests that inhibiting certain histone deacetylases (HDACs) can selectively kill TAF1-deficient cells. Synthetic lethality is a hot area of cancer research.
  3. TAF1 Gene Therapy: While still in early stages, researchers are exploring the possibility of delivering a functional copy of the TAF1 gene directly into cancer cells using viral vectors.
  4. PARP Inhibitors: TAF1 deficient cancers often exhibit defects in DNA repair pathways. PARP inhibitors, already used in BRCA-mutated cancers, show promise in TAF1-deficient tumors. PARP inhibition is a well-established therapeutic strategy.
  5. Immunotherapy Combinations: restoring TAF1 expression can enhance the immunogenicity of cancer cells, making them more susceptible to immunotherapy. combining epigenetic modulators with checkpoint inhibitors is an area of active investigation.

Biomarkers and Patient Selection

identifying patients who will benefit from TAF1-targeted therapies is crucial. Several biomarkers are being investigated:

TAF1 mRNA Expression Levels: Low TAF1 mRNA levels, measured through quantitative PCR (qPCR), can indicate TAF1 inactivation.

TAF1 Protein Levels: Immunohistochemistry (IHC) can assess TAF1 protein expression in tumor samples.

Promoter methylation Status: Bisulfite sequencing can determine the methylation status of the TAF1 promoter.

Genomic Sequencing: Identifying mutations within the TAF1 gene through NGS.

Precision oncology relies heavily on identifying these biomarkers to personalize treatment.

Case Studies & Clinical Trials (as of 2024)

While widespread clinical request is still developing,several early-phase clinical trials have shown encouraging results:

Ovarian Cancer: A Phase I trial evaluating decitabine in combination with carboplatin and paclitaxel in patients with recurrent ovarian cancer showed a significant improvement in progression-free survival in patients with TAF1 promoter methylation.

Acute Myeloid Leukemia (AML): Studies have demonstrated that AML patients with TAF1 inactivation are more sensitive to DNMT

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Health

C-Section & Childhood Cancer Risk: What Parents Need to Know

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Breaking: Planned C-Sections Linked to Slightly Higher Leukemia Risk in Children, study Finds

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Stockholm, Sweden – A groundbreaking study has revealed a potential correlation between planned Cesarean sections (C-sections) and a slightly increased risk of childhood cancers, specifically acute lymphoblastic leukemia (ALL). The research, conducted at Sweden’s Karolinska Institutet, analyzed the health records of nearly 2.5 million children born between 1982 and 2015. This examination brings to light important considerations regarding delivery methods and their potential long-term health impacts.

The Link Between Planned C-Sections and Acute Lymphoblastic Leukemia

The study’s findings indicate that children born via planned C-section – those scheduled before labor begins – appear to have a 21% higher risk of developing ALL compared to those born vaginally. The risk was even more pronounced, at 29%, for B-cell ALL (B-ALL), the most common subtype. While researchers emphasize that the overall risk remains low, the study underscores the need for informed discussions about elective surgical births.

Lead researcher Christina-Evmorfia Kampitsi from the Institute of Environmental Medicine at Karolinska Institutet, stated, “C-sections are an critically important and frequently enough life-saving part of obstetric care. We don’t want mothers to feel anxious about medically indicated C-sections.”

Though, she added that the findings, combined with other studies linking planned C-sections to conditions like asthma, allergies, and type 1 diabetes, warrant a closer look at non-medically necessary procedures.

Emergency C-Sections: A Different Story

Interestingly, children born via emergency C-section, typically after labor has already begun, did not exhibit the same elevated risk. Researchers hypothesize this is due to the exposure of infants to natural stress hormones and maternal vaginal bacteria during emergency deliveries, factors that are crucial for immune system development.

Planned C-sections, ofen performed before labor, bypass these natural exposures, potentially affecting the infant’s immune system maturation.

The increased risk appeared more prominent in boys and younger children, even though researchers acknowledge that some results did not achieve complete statistical meaning.

Did You Know? Acute Lymphoblastic Leukemia (ALL) accounts for approximately 25% of cancer diagnoses among children under 15 years old, according to the American Cancer Society.

Key Findings at a Glance

Delivery Method ALL Risk Increase Potential Explanation
Planned C-Section 21% bypasses natural exposure to maternal bacteria and stress hormones.
Emergency C-Section No significant increase Infants are exposed to beneficial bacteria and hormones during labor.

Expert Perspectives and Future Research

While ALL is rare,

How can parents best discuss potential risks associated with C-sections during prenatal consultations?

C-Section & Childhood Cancer Risk: Understanding the Connection

The topic of childhood cancer is incredibly sensitive, and parents naturally want to understand all potential factors affecting their child’s health. While research is ongoing, several studies have explored the possibility of a link between Cesarean sections (C-sections) and a slightly increased risk of certain childhood cancers. This article delves into the current understanding of this complex issue, providing information and insights for expectant parents and families.

the research: What Does the Science Say?

Several observational studies have investigated the association between C-sections and the risk of childhood cancers. It’s important to understand that *association* does not equal *causation*. These studies look for correlations,but they don’t definitively prove that C-sections *cause* cancer. Instead,they highlight potential areas where more research is needed. The research focuses on various types of childhood cancers, including but not limited to, childhood leukemia and lymphoma, as well as brain tumors.

Key Findings from Studies

  • Slightly Elevated Risk: Some studies have suggested a small but statistically important increase in the risk of certain childhood cancers in children born via C-section compared to those born vaginally.
  • Types of Cancer: The elevated risks have been most often associated with hematological malignancies (cancers of the blood), specifically acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Further investigating this is a *key step.*
  • Vaginal Delivery: research consistently shows that a vaginal delivery, when medically safe and possible, remains the optimal choice for both mother and child. This delivers the baby through the body’s natural process,which naturally exposes the newborn to beneficial bacteria.

Possible Explanations and Contributing Factors

The exact reasons for any potential link are still under investigation, but several theories are being explored. These explanations often link to differences between vaginal and cesarean births. Prenatal care and early childhood development are crucial elements.

The Microbiome and Immune System

One prominent area of research focuses on the role of the infant’s microbiome. During vaginal birth,the baby is exposed to beneficial bacteria from the mother’s birth canal. This is a critical step in the development of a healthy immune system. C-sections, on the other hand, bypass this process. Without that exposure, babies born via C-section may have different gut microbiota profiles, possibly impacting their immune development and, by extension, cancer risk. Research continuously highlights the importance of early exposure to beneficial microbes on infants.

Other Potential Contributing Factors

Beyond the microbiome, other factors may play a role:

  • Immune system development: C-sections may alter the development of the immune system, which may impact cancer susceptibility.
  • Exposure to medication: The use of antibiotics during C-sections may also impact the development of the gut flora and immune system in the newborn.
  • Maternal health factors: Sometimes there may be health conditions or circumstances for mothers with pre-existing health conditions.

What Parents Can Do: Practical Advice

Navigating this information can be overwhelming. Here’s practical advice for parents:

Discuss with your doctor

talk openly with your healthcare provider about the potential risks and benefits of both vaginal delivery and C-section. This open dialog will guide you on how to maintain a healthy pregnancy.

C-section only if medically necessary

Unless there are medical reasons, such as the baby being in a breech position, consult your doctor on the best methods for delivery.

Critically important Considerations

It’s crucial to keep the following points in mind:

  • Rarity of Childhood Cancer: The overall risk of childhood cancer is relatively low. Even if a slightly increased risk is associated with C-sections, the absolute chances remain small.
  • Medical Necessity: C-sections are often vital for saving lives. If medical reasons necessitate a C-section, it is indeed still absolutely the correct course of action to protect your child’s and your own health.
  • Holistic Approach: Focus on other critically important ways to reduce any increased risks,such as focusing on the baby’s diet,and overall lifestyle.

Please consult with your doctor to receive any personalized medical advice or recommendations.

Disclaimer: The information provided in this article is intended for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare professional for any health concerns or before making any decisions related to your health or treatment.

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Health

EHA Plenary: Investigational Drugs & Molecular Signatures

by Dr. Priya Deshmukh - Senior Editor, Health
written by Dr. Priya Deshmukh - Senior Editor, Health

The Future of Blood Cancer Therapy: Molecular Insights and Clinical Breakthroughs



The landscape of blood cancer treatment is undergoing a dramatic transformation. Forget incremental improvements; we’re witnessing a revolution driven by the deepest dives into our very biology. The 2025 European Hematology Association (EHA) Congress showcased this paradigm shift, with researchers presenting groundbreaking findings that promise to redefine how we tackle diseases like multiple myeloma, lymphoma, and leukemia. The advancements center around the power of understanding diseases at their **molecular and genetic levels**, unlocking doors to more effective, targeted therapies.

Targeting Myeloma: A New Hope with Trispecific Antibodies

One of the most exciting developments presented at the EHA Congress was the early data on JNJ-5332, a novel trispecific antibody targeting multiple myeloma. Rakesh Popat, MBBS, PhD, from University College London, highlighted the impressive efficacy of this off-the-shelf agent, achieving complete responses in over 70% of patients who hadn’t been exposed to similar treatments. This type of targeted therapy has the potential to provide similar results to CAR T-cell therapy, but potentially with easier administration in an outpatient setting, making treatment far more accessible. Prophylactic tocilizumab helped to keep adverse effects manageable, paving the way for broader use.

DLBCL and Beyond: Polatuzumab Vedotin’s Promising Role

For patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), the POLARGO trial of polatuzumab vedotin (Pola-R-GemOx) delivered encouraging results. The trial showed a significant improvement in overall survival. While increased toxicity, including peripheral neuropathy, was noted, the potential to avoid bendamustine, a commonly used chemotherapy, offers a valuable treatment alternative. As Dr. Matthew Matasar of Rutgers Cancer Institute points out, the availability of multiple treatment options becomes increasingly important in countries that heavily use CAR T-cell therapy and bispecific antibodies, providing a broader therapeutic arsenal.

Essential Thrombocythemia: Redefining Second-Line Treatment

The EHA Congress also shed light on new avenues for myeloproliferative neoplasms. The phase 3 SURPASS-ET trial offered promising results for ropeginterferon alfa-2b (ropeg) in treating essential thrombocythemia (ET). In a head-to-head comparison with anagrelide, ropeg demonstrated superior efficacy, including higher modified European LeukemiaNet response rates. The potential for disease modification, as evidenced by the achievement of partial molecular responses, underscores the therapeutic promise of this new agent.

Molecular Insights: Decoding the Secrets of Blood Cancers

Beyond clinical trials, the EHA Congress highlighted the crucial role of molecular research in driving these clinical advances. Researchers are using advanced techniques to understand the underlying biology of blood cancers, paving the way for more targeted and effective treatments. The future will undoubtedly see a greater emphasis on personalized medicine tailored to individual patients’ genetic profiles.

CLL Clonal Dynamics: Unveiling Disease Origins

Ian Márquez-López’s work on chronic lymphocytic leukemia (CLL) offered crucial insights into the genesis of the disease. His research suggests that CLL clones may arise from distinct cells of origin, decades before diagnosis. Understanding these early-stage mechanisms will be critical for developing preventive strategies and early interventions.

AML Classification: Refining Outcome Predictions

Amin Turki’s work, which used machine learning to refine the classification of acute myeloid leukemia (AML), has the potential to transform how we approach this disease. His team identified 17 distinct biological clusters, each with varying overall survival rates. By incorporating these refined classifications into existing European LeukemiaNet categorization schemes, we can enhance the accuracy of outcome predictions, enabling more personalized treatment strategies.

Looking Ahead: The Convergence of Science and Patient Care

The advances presented at the 2025 EHA Congress represent a pivotal moment in blood cancer treatment. From novel antibody therapies to refined diagnostic tools, we’re moving closer to a future where treatment is not only more effective but also more targeted and personalized. This is a time of tremendous innovation, and by incorporating these insights, clinicians have the ability to provide a better standard of care for patients around the world. It’s a journey of continuous improvement, with whole genome sequencing and machine learning leading the way forward.

The key to unlocking the next breakthroughs lies in continued investment in both basic research and innovative clinical trials. As we gain a deeper understanding of blood cancer at its molecular and genetic core, we can anticipate even more exciting advances in the years to come.

Want to learn more about the future of cancer treatment? Read this report on the National Cancer Institute’s latest research on cancer treatments.

What do you see as the most significant advancement on the horizon for blood cancer treatment? Share your thoughts in the comments below!

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