Explorez tous les épisodes du podcast HemaSphere Podcast

Could making CAR T cells directly inside the patient change how treatment is delivered? Scientific Editor Dr Charles De Bock joins Prof Roch Houot, Dr Jaime Fuentealba, François Vilcot, PharmD, and Carole-Anne Brugère, PharmD, to explore the promise and challenges of in vivo CAR therapy. They compare delivery approaches, examine safety and targeting, and discuss potential gains in access alongside uncertainty over costs. Listen for a multidisciplinary perspective on what comes next.

Read the referenced Review Article here: https://bit.ly/4d1mfjP

Key topics

• How in vivo CAR therapy differs from conventional CAR T manufacturing
• Lentiviral vectors and lipid nanoparticles: targeting, persistence, and safety, including the need for long-term monitoring
• Clinical translation, scalability, and why simpler delivery may not mean lower prices
• Europe’s role in development and lessons from writing a multidisciplinary review

Timestamps

00:00 — Why in vivo CAR is attracting attention now

02:37 — What it means to manufacture CAR T cells inside the patient

05:29 — Three layers of specificity for lentiviral delivery

10:08 — Why modern lentiviral vectors are safer than early gene therapy vectors

14:41 — How lipid nanoparticles can target T cells and avoid the liver

16:26 — Whether transient CAR expression can still be therapeutic

19:10 — Choosing between lentiviral vectors and lipid nanoparticles

22:53 — The hardest unsolved problem: balancing efficacy, safety, and scalability

23:18 — Site-specific gene editing and TRAC locus insertion

26:21 — Industry momentum and whether in vivo CAR can lower cost

28:40 — Europe’s opportunity in clinical translation

31:23 — What to expect from upcoming ASH and other conference data

35:05 — How the multidisciplinary review was scoped and organized

37:09 — Why figures were designed early in the writing process

39:57 — Advice for PhD students writing a review in a fast-moving field

This episode explores how chronic myeloid leukemia has been transformed by imatinib and other TKIs, while also showing why major clinical challenges remain for the patients who do not respond well or want to stop therapy. Host Scientific Editor Dr Charles de Bock talks with Dr. Ilaria Pagani about her recent HemaSphere paper on mitochondrial DNA mutations, oxidative phosphorylation, and treatment response in CML, and why mitochondria may be an overlooked source of biomarkers and new targets. We discuss the biology of relapse, why some patients can achieve treatment-free remission, and how mitochondrial metabolism may help explain differences between poor responders and super responders. The conversation also touches on patient advocacy, quality of life, and the need for more collaborative, metabolism-focused research.

Read the article in full, open access: https://hsphere.pub/448

You've read the paper, now hear the story.

In this episode, host Dr Charles de Bock delves into groundbreaking preclinical research exploring the potential of tasquinimod to restore blood cell production and modify the bone marrow microenvironment in MDS with Dr Manja Wobus and Dr Katja Sockel. Join us as we unpack the mechanisms, animal studies, and future clinical prospects for this promising therapy.

Read the accompanying article published in HemaSphere https://bit.ly/3OjuuiS

i4MDS, EHA's Specialized Working Group information https://hsphere.pub/5d4

Discover the latest insights into the multifaceted role of p53 in normal hematopoiesis and its critical involvement in myeloproliferative neoplasms (MPNs). This episode features Dr. Isabelle Plo and Dr. Barbara Monte-Mór sharing their expertise on TP53 mutations, molecular mechanisms driving leukemic transformation, and emerging therapeutic strategies targeting p53.

Article: https://bit.ly/4sZbo01

Comments, Suggestions or Feedback? Please take a few minutes and let us know: https://wkf.ms/4pDYjYb

In this episode, host HemaSphere Scientific Editor Dr Charley de Bock speaks with Associate Professor Dr Matthew Greenwood about the SUBLIME (ALL09) trial, which explores replacing consolidation chemotherapy with blinatumomab in adolescents and young adults with B‑cell ALL. They discuss why immunotherapy may outperform traditional cytotoxic approaches, how MRD‑guided treatment is reshaping risk stratification, and what future protocols might look like as genomic and immune‑response data are integrated in real time. Dr. Greenwood also shares upcoming innovations—including menin inhibitors, venetoclax‑based MRD targeting, and adaptive platform designs—along with honest insights into the challenges of running clinical trials across Australia.

In this extended episode of the HemaSphere podcast, based on a must-read Perspective "Can health inequalities in sickle cell disease be addressed through novel therapies?", our host Scientific Editor Charles de Bock discusses sickle cell disease with co-authors, covering both expert and lived-patient perspectives. They explore the complexities of living with the disease, the current treatment landscape, and highlight the ongoing health inequalities faced by patients, the impact of withdrawn therapies, and the need for improved communication and trust between patients and healthcare providers.

Further information:

No One's Listening Report

Shaniqua's Sickle Chronicles

NICE (National Institute for Health and Care Excellence)

UK NHS blood donation

Please take a few minutes and let us know what you think about this episode! Podcast Feedback

Dr. Anikó Sijs-Szabó & Roger Mulet Lázaro, PhD, join Dr. Charles de Bock to unpack the challenges of diagnosing and treating acute leukemias of ambiguous lineage (ALAL). Discover how RNA sequencing, machine learning, and collaboration are shaping the future of precision medicine in hematology.

🎧 Listen and then read the full open-access article on the HemaSphere website https://bit.ly/4oeJo6w

Join our host HemaSphere Scientific Editor Dr Charles de Bock as he chats with Dr Kai Rejeski, first author of this 2025 HemaSphere Top Cited –Clinical Award-winning paper. The conversation delves into the complexities of CAR-T therapy, particularly focusing on the associated toxicities and the need for a structured grading system.

EHA/EBMT Survey on Grading and Management of ICAHT, is on our website and all major podcast platforms (Spotify, Apple Podcast, and more) and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

Host Dr Charles de Bock speaks with Prof Michel Zwaan and Prof Olaf Heidenreich, co-authors from the 2025 HemaSphere Award - Top Cited: Biology paper, "The MLL-Menin Interaction is a Therapeutic Vulnerability in NUP98-rearranged AML." The conversation delves into the complexities of #AML, particularly focusing on NUP98 rearranged AML and the therapeutic potential of Menin inhibitors. The discussion highlights the challenges in treating this aggressive form of leukemia, the mechanisms of menin inhibitors, and the importance of combining therapies to improve patient outcomes.

Season 3, Episode 5: Tackling NUP98 Rearranged AML in Pediatric Oncology, is on our website and all major podcast platforms. Listen and enjoy casual, insightful discussions about #hematology research.

In this second part of a 2-part episode, Dr Charles De Bock engages with Prof Dr Petya Apostolova and Dr Jörg Büscher to explore the intricate relationship between metabolism and hematology, particularly focusing on #AML. They discuss the challenges and advancements in metabolomics, the importance of integrating multi-omics approaches, and the future directions of research in this field. The conversation highlights the need for a deeper understanding of metabolic pathways and their implications for cancer treatment, emphasizing the significance of context in cellular behavior and the potential of spatial metabolomics.

"Metabolism in hematology, Part 2”, is on our website, all major podcast platforms, and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced Review article, in full and open access, here on the HemaSphere website.

In this 2-part episode of the HemaSphere podcast, host Dr Charles de Bock and guests Dr Jörg Büscher and Prof Dr Petya Apostolova discuss the critical role of metabolism in cellular function, particularly in the context of hematology and cancer research. The discussion highlights the technological advancements in metabolic analysis and their impact on understanding cellular metabolism. They also explore the challenges and future directions in metabolism research and the potential of spatial metabolomics.

"Metabolism in hematology”, is on our website, all major podcast platforms, and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced Review article, in full and open access, here on the HemaSphere website.

In this episode of the HemaSphere podcast, host Charles de Bock and guest Tim Mocking, PhD, discuss the role of machine learning in the assessment of measurable residual disease (MRD) in acute myeloid leukemia (AML). They explore the challenges of MRD detection, the potential of machine learning to improve accuracy and efficiency, and the importance of understanding the human element in computational methods. The conversation also touches on the adoption of AI in hematology, the future of flow cytometry, and the implications of new technologies for patient care.

Episode 3.3 - Machine learning for AML-MRD is based on the recently published Review Article "Applications of machine learning for immunophenotypic measurable residual disease assessment in acute myeloid leukemia”, is on our website, all major podcast platforms, and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

In this episode of the HemaSphere podcast, host Dr Charles De Bock discusses the newly published Guidelines on the treatment of neutropenias with co-authors Dr. Francesca Fioredda and Dr. Eleni Papadaki. They explore the importance of these international guidelines, the challenges faced in gathering evidence, and the consensus-building process among experts. The conversation also delves into treatment options, including gene therapy and stem cell transplants, while highlighting the need for more research in the field.

"European guidelines on treatment and supportive measures in chronic neutropenias: A consensus between the European Hematology Association and the EuNet-INNOCHRON COST Action based on a systematic evidence review”, is on our website, all major podcast platforms, and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

This is a follow up to the neutropenia Guidelines published in 2023, which focused on diagnosis and management. Both the previous Guidelines and the podcast episode can be found here.

Other papers referenced:

Ritter MU, Nasri M, Dannenmann B, et al. Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the ELANE Gene. CRISPR J. 2024 Oct;7(5):258-271. doi: 10.1089/crispr.2024.0006. PMID: 39436283. https://pubmed.ncbi.nlm.nih.gov/39436283/

Nasri M, Ritter MU, Mir P, Dannenmann B, et al. CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia. Mol Ther. 2024 Jun 5;32(6):1628-1642. doi: 10.1016/j.ymthe.2024.03.037. Epub 2024 Mar 30. PMID: 38556793; PMCID: PMC11184331. https://pubmed.ncbi.nlm.nih.gov/38556793/

In this episode of the HemaSphere podcast, host Charles De Bock engages with HemaSphere Editor in Chief Professor Jan Cools, Professor Heidi Segers, and Doctor Margo Aertgeerts to discuss their recent study on acute lymphoblastic leukemia (ALL). They explore the advancements in treatment, particularly for high-hyperdiploid ALL, the challenges of relapse, and the innovative use of single cell sequencing to uncover insights into genetic mutations and clonal composition. The conversation delves into the implications of RAS mutations, the technical challenges of analyzing rare cell populations, and the future of integrating transcriptomics with single cell analysis to enhance understanding and treatment of leukemia.

“Single‐cell DNA and surface protein”, is on our website, all major podcast platforms (Spotify, Apple Podcast, and more) and YouTube. Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

In this episode of the HemaSphere podcast, host Charles de Bock engages with Professor Hélène Cavé and Dr. Marion Strullu to discuss their recent study on juvenile myelomonocytic leukemia (JMML). They delve into the complexities of diagnosing JMML, the genetic mutations associated with the disease, and the prognostic factors that influence patient outcomes. The conversation explores the intriguing biology of JMML, including the role of transcriptional signatures and methylation patterns, as well as the challenges faced in sample collection. The episode concludes with insights into future research directions in understanding JMML. 

"Two distinct fetal-type signatures characterize JMML" is on our website and all major podcast platforms (Spotify, Apple Podcast + more) and YouTube. Listen and enjoy casual,insightful discussions about #hematology research.

You've read the paper, now hear the story.

You can find the referenced article, in full and open access, here on the HemaSphere website.

In this episode of the HemaSphere podcast, host Dr Charles de Bock discusses the critical role of mouse models in understanding leukemogenesis and hematopoiesis with Prof Richard Lock. They explore the significance of patient-derived xenografts (PDXs) in preclinical testing, the challenges faced in generating PDXs for acute myeloid leukemia (AML), and the importance of molecular characterization. The conversation also covers the objective response criteria in drug testing, the innovative single mouse trial approach, and the future of immunotherapy testing in PDX models.

Revolutionizing Pediatric Cancer Research with PDXs is on our website and all major podcast platforms (Spotify, Apple Podcast, and more). Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, The CD123 antibody-drug conjugate pivekimab sunirine exerts profound activity in preclinical models of pediatric acute lymphobloastic leukemia, in full and open access here on the HemaSphere website

URLs mentioned in the recording:

15:09 PedcBioPortal

31:13 The Jackson Laboratory - Mouse Models

In this episode of the HemaSphere podcast, host Charles de Bock discusses the latest research on myelodysplastic syndrome (MDS) with Dr Francesca Tiso and Dr Florentien in 't Hout from Radboud University Medical Center, co-authors of a recent paper published in HemaSphere. The conversation covers the clinical presentation of MDS, treatment options, and the significance of genetic mutations. Delve into their study on the prognostic value of hydroxymethylation levels in patients treated with azacitidine, highlighting the challenges of risk stratification and patient recruitment. They also discuss future research directions and the comparative efficacy of azacitidine and decitabine.

“High 5hmC predicts worse OS” is on our website and all major podcast platforms (Spotify, Apple Podcast, and more) and YouTube.

Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

This podcast episode delves into the complexities of informed consent in healthcare, particularly in the context of hematology. The discussion with host Dr Charles de Bock features insights from Dr Stephen Hibbs and Dr Louise Caldwell, authors of the HemaTopics "Informed consent is almost impossible” and guest Mark Dawson, a patient family member, exploring the emotional, legal, and ethical dimensions of consent. Key themes include the variability of consent across jurisdictions, the impact of time pressures on communication, and the importance of trust in the clinician-patient relationship. The conversation also addresses the challenges posed by genetic testing and the need for ongoing discussions about consent throughout a patient's treatment journey.

“Informed consent is almost impossible”, is on our website and all major podcast platforms (Spotify, Apple Podcast, and more) and our YouTube channel. Listen and enjoy casual, insightful discussions about #hematology research. Hear the stories behind the papers.

You can find the referenced article, in full and open access, on the HemaSphere website.

Publications mentioned in this podcast:

• The Immortal Life of Henrietta Lacks, by Rebecca Skloot
• The Logic of Care: Health and the Problem of Patient Choice, by Annemarie Mol

In the latest episode, Dr Charles de Bock talks with Prof H. Josef Vormoor and Dr Maria Ester Bernardo, Chair and Co-Chair of EHA’s Pediatric Hematology Specialized Working Group. They discuss highlights from the EHA 2024 Congress in Madrid, including key lectures, new insights and what they could mean for clinicians, researchers, patients and their families, along with hopes and expectations for the EHA2025 Congress in Milan.

“Best of Pediatric Hematology from EHA2024”, is on our website and all major podcast platforms (Spotify, Apple Podcast, and more) and our YouTube channel. Listen and enjoy casual, insightful discussions about #hematology research.

We are pleased to offer HemaSphere’s full listing of publications about pediatric hematology, now in a dedicated collection. This online collection will be added to monthly.

You can find the referenced article, in full and open access, on the HemaSphere website.

Authors of this recent HemaSphere published paper discovered a high-risk subgroup of pediatric T-LBL, defined by NOTCH1 gene fusions. This finding will have an important impact on newly diagnosed children with T-LBL globally, listen to two of the co-authors, Dr Jan Loeffen and Professor Roland Kuipers discuss their findings with Dr Charles de Bock, how they discovered them, and dreams of future developments in this HemaSphere podcast episode.

NOTCH1 fusions in T‐LBL, is on our website and all major podcast platforms (Spotify, Apple Podcast, and more) including YouTube

Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, in full and open access, here on the HemaSphere website.

In this newest podcast episode, Dr Charles de Bock hosts Dr Thomas Mercher and Dr Alexandre Fagnan. This episode is based on their recent paper, “The ETO2 transcriptional co-factor maintains acute leukemia by driving a MYB/EP300-dependent stemness program”, highlighting ETO2 activated gene transcription and how this affects erythroleukemia. The discussion traces the journey from part of Dr Fagnan’s PhD project (2022 EHA Junior Grant awardee), the findings in the paper and what may lie beyond.

This episode is also available on our YouTube channel .

Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, “The ETO2 transcriptional co-factor maintains acute leukemia by driving a MYB/EP300-dependent stemness program” in full and open access, here on the HemaSphere website.

Welcome to Season 2 of our podcast! In this episode, Dr. Francesca Vinchi interviews Professor Alessandra Balduini about thrombopoiesis. They explore Prof. Balduini's journey into research, her intuition that led to silk scaffolding, and her hopes on future results.

"Unraveling the secrets of thrombopoiesis", is on our website and all major podcast platforms (Spotify, Apple Podcast, YouTube, etc). Listen and enjoy casual, insightful discussions about #hematology research.

You can find the referenced article, "Targeting Bone Marrow Mechanosensation in Myelofibrosis." in full and open access, here on the HemaSphere website.

Dr Stephen Hibbs invites HemaSphere Associate Editor Prof Robert K Hills to discuss statistics in scientific writing in an entertaining and informative episode: What’s so Great About the P-value? A Statistician’s Point of View. This discussion covers the overlap of statistics and hematology with themes like the importance of P value and the use of statistics in clinical trials.

What’s so Great About the P-value? A Statistician’s Point of View, is on our website , all major podcast platforms (Spotify, Apple Podcast, etc), and YouTube.  

Listen and enjoy casual, insightful discussions about #hematology research. HemaSphere articles are always fully open access.

Recent HemaSphere articles you may find interesting: 

Interim Positron Emission Tomography During Frontline Chemoimmunotherapy for Follicular Lymphoma. 

Merryman R, Michaud L, Redd R, et al.

Definition and Prognostic Value of Ph-like and IKZF1plus Status in Children With Down Syndrome and B-cell Precursor Acute Lymphoblastic Leukemia.

Palmi C, Bresolin S, Junk S, et al.

Dr Stephen Hibbs invites Dr Joseph Taylor to a discussion based on Dr Hibbs popular HemaTopics article, This Is Going to Hurt: Revisiting the Patient Experience of Bone Marrow Biopsies.  The conversation ranges from the type of training providers receive, how to talk to patients about these bone marrow biopsies,  and some of the outstanding questions about bone marrow biopsies and the patient experience.

This Is Going to Hurt: Revisiting the Patient Experience of Bone Marrow Biopsies, is on our website, all major podcast platforms (Spotify, Apple Podcast, etc), and YouTube. You can find the referenced article, in full and open access, on the HemaSphere website.

Listen and enjoy casual, insightful discussions about #hematology research.

HemaTopics are HemaSphere publications written by our Scientific Editors, focusing on hot hematology topics including recent research developments, updates on diagnosis and treatment, policy and guidelines news, and more.

Though many advances have been made in neutropenias, diagnosis and management are still based on physicians’ experience or local practices. Prof Francesca Fioredda and Prof Helen Papadaki, two co-authors of the HemaSphere published article The European Guidelines on Diagnosis and Management of Neutropenia in Adults and Children: A Consensus Between the European Hematology Association and the EuNet-INNOCHRON COST Action, discuss with host Dr Stephen Hibbs the challenges and outcomes in creating a guideline for diagnosis and managing neutropenia in adults and children. Read the referenced article on the HemaSphere website and watch on YouTube.

POEMS syndrome (Polyneuropathy, Organomegaly, Endocrinopathy, M-protein and Skin changes) is rare, and systemic manifestations are commonly overlooked initially. Diagnosis is often delayed 12-16 months, during which patients can be severely disabled. No established treatment guidelines exist for POEMS. A recent HemaSphere published article Comprehensive Diagnosis and Management of POEMS Syndrome offers a clinical blueprint for managing POEMS, from diagnostics to work-up, therapy selection, follow-up, and relapse treatment. Shirley d'Sa & Michael Lunn, two co-authors of the article discuss with host Dr Stephen Hibbs, in this second of two episodes,  why multidisciplinary collaboration is essential.

Catch up with Part 1 here.

Read the referenced article in HemaSphere, and also watch the videos onYouTube: Part 1 https://youtu.be/TE-t-rPztJw  Part 2 https://youtu.be/hBE7KShFRhI

POEMS syndrome (Polyneuropathy Organomegaly, Endocrinopathy, M-protein and Skin changes) is rare, and systemic manifestations are commonly overlooked initially. Diagnosis is often delayed 12-16 months, during which patients can be severely disabled. No established treatment guidelines exist for POEMS. A recent HemaSphere published article, Comprehensive Diagnosis and Management of POEMS Syndrome, offers a clinical blueprint for managing POEMS, from diagnostics to work-up, therapy selection, follow-up, and relapse treatment. Shirley d'Sa & Michael Lunn, two co-authors of the article discuss with host Dr Stephen Hibbs, in this first of 2 episodes, why multidisciplinary collaboration is essential.

Continue with Part 2 here.

This podcast is also available on YouTube: Part 1 and Part 2

Machine Learning is a subsection in the field of Artificial Intelligence. How can this be used as a tool in hematology-oncology for advanced disease prognostification and beyond? Adrián Mosquera-Orgueira and Juan Carlos Hernández-Boluda, two co-authors of the HemaSphere published article Machine Learning Improves Risk Stratification in Myelofibrosis: An Analysis of the Spanish Registry of Myelofibrosis, discuss with host Dr Stephen Hibbs the challenges and outcomes in utilizing a novel myelofibrosis survival model that aids in risk stratification for patients diagnosed with myelofibrosis. Read the referenced article in HemaSphere, also available on our YouTube channel.

As the overall cancer death rate has declined over past decades and cancer survival has increased, unforeseen challenges are emerging. Thrombocytopenia, often associated with cancer disease or anticancer therapy, will be more present in thrombotic cancer patients, but evidence on how to treat these patients is lacking. Prof Anna Falanga discusses these Guidelines with host Dr Stephen Hibbs in this HemaSphere Podcast episode.  You can find the referenced article, in full and open access, here on the HemaSphere website, or watch the video on our YouTube channel.

What to do next for “Long COVID-19” of researchers? A survey from scientists to

scientists led by the Young European Hematology Association (Young EHA)committee led to

this discussion about how COVID-19 impacted researchers, especially earlier in

their career and without a network. What tools did they discover to get through

it? Find it on the HemaSphere website, “Long COVID-19” of Researchers: What to Do Next? , or watch on our YouTube channel.