Michael Henry

Dr. Michael Henry is the Section Chief of the Liquid Tumor Program in the Center for Cancer and Blood Disorders at Phoenix Children’s in Phoenix, Arizona/ He serves as the Director of the Early Drug Development Program and the Director of the Histiocytosis Program at Phoenix Children’s. Dr. Henry also is a founding Board Member and Chair of the Langerhans Cell Histiocytosis Working Group in the North American Consortium for Histiocytosis (NACHO). He is the Chair of the Education Committee in the Histiocyte Society, and he championed the inception of the Mentorship Program within the Society. In addition, Dr. Henry serves on the Non-Hodgkin Lymphoma Committee of the Children’s Oncology Group. His clinical and research interests are in novel therapeutics and clinical trials for histiocytic disorders and mentorship of learners of all levels.

Ashish Kumar

Scott Canna

Dr. Scott Canna has been committed to translational research in autoinflammatory diseases and cytokine storm syndromes since 2005. This began with training in human translational autoinflammation at the NIH, where he contributed to the first trials of Interleukin-1 blockade in patients with mutations of the NLRP3 inflammasome. It continued through work characterizing murine models of Macrophage Activation Syndrome (MAS). It extended to the discovery that NLRC4 inflammasome hyperactivity drove both elevated IL-18 (the other inflammasome-activated cytokine) and life-threatening MAS. His independent research program began in 2017, working to elucidate the contexts and mechanisms by which excess IL-18 promotes hyperinflammatory responses. Along the way, his group’s biomarker work has identified the specific human contexts in which IL-18 rises above the normal suppression by IL-18 binding protein and IL-18 is “free” to drive inflammation. Translating this work, his group demonstrated that IL-18 blockade may be a rational and feasible treatment strategy in NLRC4-MAS, and they are helping drive the development of several clinical trials of IL-18 blockade in autoinflammatory diseases. Follow them at www.labcanna.org or @canna_lab.

Shan Chandrakasan

Dr. Shan Chandrakasan is an associate professor and the director of the immune dysregulation/ immunohematology and immune defects transplant programs at Children's Healthcare of Atlanta, Emory University. His clinical and research interests focus on the early identification and development of definitive curative strategies for HLH and immune dysregulation disorders. His lab works on identifying early biomarkers and a deeper immunobiological understanding of different forms of HLH, global and organ-directed hyperinflammatory, and immune dysregulation disorders through high dimensional immune phenotyping and other next-generation technologies. In addition, utilizing murine models of HLH, his lab is developing definitive treatment strategies such as non-genotoxic conditioning approaches for HSCT and cell and gene therapies in primary HLH.

Julien Haroche

Dr. Julien Haroche is a professor in internal medicine, at Pitié-Salpêtrière hospital, Paris, France. Since 2003, his main research field is Erdheim-Chester disease (ECD) upon which he has acquired a word-renowned experience. To date, he has seen more than 380 patients followed at his institution. His other research fields are the other histiocytoses, such as Langerhans cell histiocytosis, mixed histiocytoses (LCH & ECD) and Rosai-Dorfman disease. He is also interested in vasculitis, systemic lupus and antiphospholipid syndrome.

During the past 15 years, he has described most relevant clinical and radiological aspects and increased the awareness of ECD; he has showed that interferon α was a first line efficient therapy. His team was the first to used targeted therapies in adult patients with histiocytoses in 2012. Since then more than 175 patients have received BRAF and/or MEK inhibitors at his institution.

Karen Rech

Dr. Karen Rech is an Associate Professor of Hematopathology at the Mayo Clinic in Rochester, MN, USA. She received her MD from the University of North Carolina, followed by residency and fellowships in Surgical Pathology and Hematopathology at Mayo Clinic Graduate School of Medicine. Inspired by a busy clinical practice, her research interests focus on novel diagnostic markers in in lymphoma, amyloidosis, and histiocytic neoplasms. As a member of the multidisciplinary University of Alabama Birmingham-Mayo Clinic Histiocytosis Working Group, she seeks to expand medical knowledge and improve the outcome of patients with Erdheim-Chester disease, Rosai-Dorfman disease, Langerhans cell histiocytosis and Malignant Histiocytosis. 

Jonathan Kipnis

Dr. Jonathan (Jony) Kipnis is BJC Investigator, Alan A. and Edith L. Wolff Distinguished Professor of Pathology and Immunology and Professor of Neurology, Neuroscience, and Neurosurgery at Washington University in St. Louis, School of Medicine. He is also the inaugural Director of Brain immunology and Glia (BIG) Center at Washington University. Jony graduated from the Weizmann Institute of Science in Israel, where he was a Sir Charles Clore scholar and a recipient of distinguished prize for scientific achievements awarded by the Israeli Parliament, The Knesset.

Kipnis lab focuses on the complex interactions between the immune system and the central nervous system (CNS). The goal is to elucidate the cellular and molecular mechanisms underlying these interactions in neurodegenerative, neurodevelopmental, and mental disorders as well as in physiology (including healthy aging). They showed that the brain function is dependent, in part, on the function and integrity of the immune system and that immune molecules (cytokines) can play neuromodulatory roles. The fascination with immunity and its role in neurophysiology is what brought the lab to a breakthrough discovery of meningeal lymphatic vessels that drain the CNS into the peripheral lymph nodes and thus serve as a physical connection between the brain and the immune system. This finding challenged the prevailing mechanisms underlying CNS “immune privilege” and opened new avenues to mechanistically study the nature of neuroimmune interactions under physiological and pathological conditions. The implications of this work are broad and range from Autism to Alzheimer’s disease through neuroinflammatory conditions, such as Multiple Sclerosis. Recently Kipnis lab has also identified the skull and vertebrae bone marrow niches as local immune reservoirs for the brain and the spinal cord, whose role in neurological disorders is yet unknown.

Dr. Kipnis is a member of National Academy of Medicine and among other awards, he is a recipient NIH Director’s Pioneer award for 2018 to explore in more depth neuro-immune interactions in healthy and diseased brain and National Institute on Aging MERIT award to study the role of meningeal immunity and lymphatics in aging and Alzheimer’s disease.

Claire Booth

Prof Claire Booth, MBBS PhD is a Gene Therapist and Paediatric Immunologist at UCL Great Ormond Street Hospital Institute of Child Health in London and leads the clinical stem cell gene therapy programme. She graduated from Guy’s, King’s and St. Thomas’ School of Medicine in 2001 and then trained in Paediatrics, subspecialising in Paediatric Immunology and Immunodeficiency. She undertook a Wellcome Trust funded PhD at UCL developing haematopoietic stem cell gene therapy, with continued NIHR and Wellcome Trust post-doctoral support to establish her own research group. She was appointed as a Consultant in Paediatric Immunology at Great Ormond Street Hospital in 2014.

Claire now works as a clinical academic leading an expanding number of gene therapy clinical trials at Great Ormond Street Hospital which treats patients with immune deficiencies, haematological and metabolic disorders. Her lab group is focused on developing novel therapies for immune system disorders using both gene therapy/gene editing and targeted small molecules. She has extensive experience of translating, leading, and delivering first in human clinical trials and the commercialisation pathway. As an attending physician she oversees the clinical management of patients with immune deficiencies, including hematopoietic stem cell transplantation and maintains a strong interest in HLH disorders.

Claire is an internationally recognized expert in gene therapy and immunology, an elected board member of the European Society of Gene and Cell Therapy, Chair of the International Committee of the American Society of Gene and Cell Therapy and previously served two terms on the board of the British Society. She serves on the editorial board of several journals and grant review committees and holds an honorary position at Boston Children’s Hospital/Dana Farber Cancer Institute and Harvard Medical School.

She is also the co-founder of the AGORA initiative (Access to Gene therapies fOr Rare disease) which has founding members across 6 European countries and brings together clinicians and scientist with direct experience of developing and delivering ex vivo gene therapies for rare diseases, aiming to facilitate access to effective gene therapies for treatment of patients with ultra-rare diseases.

Toni Cathomen

Shengdar Tsai

Dr. Shengdar Tsai is an Associate Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on developing genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T-cells for cancer immunotherapy. In 2020, he was chosen as one of the American Society for Gene and Cell Therapy Outstanding New Investigators.

​His group has recently developed CHANGE-seq, a state-of-the-art, sensitive, unbiased, high-throughput method for defining the genome-wide activity of genome editors (Lazzarotto et al. Nature Biotechnology 2020). Previously, he has led the development of methods for high-throughput genome editing with TALENs (Reyon and Tsai et al. Nature Biotechnology 2012), CRISPR-Cas genome editors with improved specificity by dimerization (Tsai et al. Nature Biotechnology 2014), widely adopted methods to define the genome-wide specificity of CRISPR-Cas nucleases such as GUIDE-seq (Tsai and Zheng et al. Nature Biotechnology 2015) and CIRCLE-seq (Tsai et al. Nature Methods 2017 and Lazzarotto et al. Nature Protocols 2018).

Dr. Tsai completed a postdoctoral fellowship at Massachusetts General Hospital & Harvard Medical School, Ph.D. in Functional Genomics and M.S. in Bioinformatics from North Carolina State University, and B.S. from the University of Michigan.

Ed Behrens

Dr. Ed Behrens is the Chief, Division of Rheumatology, Co-Leader, Immune Dysregulation Frontier Program, and Joseph Lee Hollander Endowed Chair in Pediatric Rheumatology at The Children’s Hospital of Philadelphia and The Perelman School of Medicine at the University of Pennsylvania.  He is an expert in systemic hyperinflammatory diseases and in particular hemophagocytic syndromes.  He helped to found and lead the Immune Dysregulation program at CHOP, a multidisciplinary team of physicians, nurse practitioners, pharmacists, and genetic counselors from across all subspecialties, which provides diagnostic and therapeutic solutions for children with undiagnosed immune conditions.

Dr. Behrens' research program centers on the pathogenesis of Cytokine Storm Syndrome, a common pathologic endpoint of a heterogeneous group of initiating diseases. Cytokine Storm Syndrome results in multi-organ failure and a stereotypical immune response including increased serum ferritin and Interferon gamma that leads to the organ failure itself. He is specifically interested in dissecting the initiating, propagating, and regulatory factors that determine the pace and outcome of cytokine storm. This is accomplished using a number of different murine models including perforin deficient mice, repeated TLR9 agonist injections, and other murine systems of systemic inflammation. The ultimate goal of these investigations is to improve the lives of patients that suffer from Cytokine Storm Syndrome by developing strategies for treatment and prevention.

Sabrin Albetuni

 Dr. Sabrin Albeituni is a Scientist in the Department of Oncology at St. Jude Children’s Research Hospital. Dr. Albeituni completed her undergraduate studies in Biology at Birzeit Univiersity in Palestine and earned her PhD in Microbiology and Immunology in 2015 from the University of Louisville, KY. Following the completion of her doctoral degree, she pursued a postdoctoral training under the guidance of Dr. Kim Nichols at St. Jude Children’s Research Hospital in Memphis, TN.

Her research aims to investigate the cellular and molecular mechanisms underlying the pathogenesis of hemophagocytic lymphohistiocytosis (HLH). HLH is a life-threatening hyperinflammatory syndrome characterized by excessive immune cell activation and multi-system organ failure. Through her research, Dr. Albeituni aims to further develop novel therapeutic strategies to dampen hyperinflammation and improve clinical outcomes.

Dr. Albeituni demonstrated in preclinical murine models of HLH that the JAK1/2 inhibitor, ruxolitinib, ameliorates the clinical manifestations of disease through IFN-gamma dependent and independent mechanisms. She published several research articles demonstrating the efficacy of JAK1/2 inhibition in ameliorating the cytokine storm in HLH. Her work set the ground for a clinical trial that is being sponsored by St. Jude for the use of a response-adapted ruxolitinib-containing regimen for the treatment of HLH.  Currently, she has a strong interest in understanding the regulation of T cell responses by myeloid cells during normal immune homeostasis and in disorders associated with hyperinflammation.

Sabrin will be presenting the abstract entitled DIFFERENTIAL EFFECTS OF JAK1 VS. JAK2 INHIBITION IN MURINE MODELS OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS during the Presidential Symposium on Monday, October 23, 2023.

Oshrat Hershkovitz-Rokah

Dr. Oshrat Rokah is the head of the Molecular Research Lab at Assuta Medical Center located in Tel-Aviv, Israel. Dr. Rokah holds a PhD and MSc from the Department of Genetics in the Faculty of Medicine at Tel Aviv University.

During her studies, Dr. Rokah received several honorable awards and scholarships, among them, the David and Paulina Trotsky Award for Outstanding Students. Dr. Rokah was one of the pioneers in studying microRNA expression in chronic myeloid leukemia, this contributed to better understand the molecular mechanism underlying the development of the disease, and new avenues for therapeutic treatment.

Dr. Rokah has extensive knowledge in complex, advanced, and diverse molecular research methods and today she is the mentor of five PhD students from Ariel University. She works in full and intensive cooperation with the clinical field, physicians and scientists, in order to target real clinical needs and deficiencies. The research in the lab focuses on the role of non-coding RNAs in hematological malignancies and the aberrant molecular mechanisms associated with deregulated expression of these molecules.

As a part of the dedicated clinic at Assuta Medical Center for histiocytoses neoplasms, her lab is investigating the functional roles of these non-coding molecules in histiocytic neoplasms.

Oshrat will be presenting the abstract entitled UNDERSTANDING THE EPIGENETIC LANDSCAPE IN HISTIOCYTIC NEOPLASMS during the Presidential Symposium on Monday, October 23, 2023.

Lauren Meyer

Dr. Lauren Meyer graduated from the MD/PhD program at the University of California, San Francisco. Her graduate thesis work focused on understanding the relationship between cytokine receptor signaling and sensitivity to glucocorticoid therapy in T-cell acute lymphoblastic leukemia (T-ALL) and hemophagocytic lymphohistiocytosis (HLH) as well as strategies to improve glucocorticoid efficacy in these diseases. She is currently a pediatrics resident at the University of Washington, where she has continued basic and translational research in HLH. In particular, she is interested in the optimization of diagnostic algorithms for patients with suspected HLH and in the development of tools to aid in the interpretation of variants of unknown significance in genes associated with familial HLH and related immune dysregulation syndromes. 

Lauren will be presenting the abstract entitled A+A2:K5 TOOL FOR PREDICTING PATHOGENICITY OF VARIANTS OF UNKNOWN SIGNIFICANCE IN FAMILIAL HLH GENES during the Presidential Symposium on Monday, October 23, 2023.

Eli Diamond

Dr. Eli Diamond is an Associate Attending Neuro-Oncologist at Memorial Sloan Kettering Cancer Center. He is the Chair of the Rare Histiocyic Disorders Steering Committee of the Histiocyte Society and a member of the Board of Trustees of the Histiocytosis Association and Chair of the Science Committee of this Board. His clinical practice and research focus on the treatment of adults with various histiocytic neoplasms, with additional focus upon neurologic manifestations of disease. He is an NIH-funded principal investigator whose research spans molecular pathogenesis, therapeutics, symptomatology, and caregiver needs.

Eli will be presenting the abstract entitled INTRA-ARTERIAL MELPHALAN FOR HISTIOCYTIC NEOPLASMS INVOLVING NEUROLOGIC, OCULAR, AND HEAD AND NECK STRUCTURES during the Presidential Symposium on Monday, October 23, 2023.

Jean Donadieu

Jean will be presenting the abstract entitled CHILDREN WITH REFRACTORY HISTIOCYTOSIS TREATED BY MAPKI HAVE A HIGH RISK OF NEURO DEGENERATION: AN INTERNATIONAL STUDY during the Presidential Symposium on Monday, October 23, 2023.

Abishek Maiti

Dr. Abhishek Maiti is an Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston, Texas. Dr. Maiti's patient care and research focuses on developing novel therapies for leukemias and associated hematological disorders. He has authored several peer-reviewed publications in leading medical journals and presented his research at national and international meetings.

Abhishek will be presenting the abstract entitled A PHASE 1B STUDY OF ELA026, A MONOCLONAL ANTIBODY TARGETING SIGNAL REGULATORY PROTEIN-A/B1/G, IN PATIENTS WITH NEWLY DIAGNOSED AND PREVIOUSLY TREATED SECONDARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS during the Presidential Symposium on Monday, October 23, 2023.

Astrid van Halteren

Dr. Astrid van Halteren completed her master degree in Biomedical Sciences at the University of Amsterdam in 1991.  After defending her PhD thesis in 1996 (Vrije Universiteit Amsterdam), she moved to the Leiden University Medical Center (LUMC), where she started to work in the hemato-oncology field before being introduced into the histiocytosis field in 2008. As a certified specialist in immunology, she became intrigued by the inflammatory component of histiocytosis lesions. Her research team has investigated characteristics of lesional T-cells, co-published on newly identified driver mutations, studied intracellular processing of neoantigens encoded by the most prevalent molecular alteration (BRAF^V600E) and recently completed a multi-centre study addressing associations between the three most common mutations and clinical presentation of pediatric LCH. Two abstracts presenting the results of these studies have been nominated by the Histiocyte Society for the Nezelof Prize for best Basic Science (2015) and the Nesbit Prize for Best Clinical Science (2019). As off August 2022, she will be affiliated with the Centre for Histiocytic Disorders established at the Erasmus University Medical Center in Rotterdam, which is acknowledged by the ECD Global Alliance and the Dutch Federation of University Medical Centers. In this new position, she will implement molecular monitoring and central biobanking in conjunction to the diagnosis and treatment of adult histiocytosis patients. In addition, she will continue her research on the hematopoietic ‘cell-of-origin’, which is performed in close collaboration with the Princess Máxima Centre for Pediatric Oncology where she is appointed as co-PI. Her research aims at finding alternative ways to eradicate neoplastic stem/progenitor cells beyond conventional chemotherapy.

Astrid will be presenting an update on the proceedings from the 2023 Nikolas Symposium.

Mimi Bandopadhayay

Pratiti (Mimi) Bandopadhayay, MBBS, PhD, is a pediatric neuro-oncologist and scientist within the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, an Assistant Professor of pediatrics at the Harvard Medical School, and an Associate Member of the Broad Institute of MIT and Harvard. She also serves as a faculty member for the Harvard Medical School PhD Program in Biological and Biomedical Sciences (BBS) and as the Director of the DFCI Pediatric Low-Grade Glioma Program.

Mimi was born and raised in Australia, where she completed her medical studies (Monash University), PhD (University of Melbourne), and specialty training in pediatrics and hematology/oncology (Royal Children’s Hospital and Monash Medical Center, Melbourne, and the Australasian College of Physicians), followed by further training in pediatric neuro-oncology under the mentorship of Dr. David Ashley.

In 2011, Mimi joined the team at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, where she completed subspecialty training in pediatric neuro-oncology under the guidance of Dr. Mark Kieran, followed by post-doctoral training  at Dana-Farber Cancer Institute and the Broad Institute under the mentorship of Dr. Rameen Beroukhim. 

In addition to her efforts in the Lab, Mimi is a member of the clinical pediatric neuro-oncology team in the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, contributing to the care of children with brain tumors. These children are a constant motivation for Mimi (and all members of the Bandopadhayay Lab) to undertake translational research that is dedicated to improving the outcomes of children diagnosed with pediatric brain tumors.

Gaurav Goyal

Dr. Gaurav Goyal was born and raised in a small town in India. He obtained his medical school diploma from Smt. N.H.L. Municipal Medical College in Ahmedabad, India, in 2011. Subsequently, he moved to the United States to complete a residency in Internal Medicine from Creighton University Medical Center, Omaha, Nebraska in 2016. During his residency, he developed special interest in hematology-oncology and went on to pursue a fellowship in the same from Mayo Clinic, Rochester, Minnesota from 2016-2019.  

During his fellowship, he developed a unique focus in a rare group of blood diseases called as histiocytic disorders, including Erdheim-Chester disease, Langerhans cell histiocytosis, and Rosai-Dorfman disease. He conducted multiple studies describing the epidemiology, treatments, and outcomes of patients with histiocytosis and led to the establishment of first of its kind multidisciplinary Histiocytosis Working Group. He has led national and international guidelines on the diagnosis and management of these rare disease entities. He was subsequently recruited to join the Hematology-Oncology division at University of Alabama at Birmingham as an Assistant Professor in 2019.  

Dr. Goyal established the histiocytosis program after moving to UAB and expanded the Histiocytosis Working Group to include members from UAB. He continues to conduct clinical, translational, and outcomes research in histiocytosis at his current position. He has additionally developed special interest in survivorship research with ongoing projects evaluating burden of morbidity and mortality among individuals with histiocytic disorders.  

Andy Pearson

Professor Andy Pearson, formerly Cancer Research UK Professor of Paediatric Oncology, at the Institute of Cancer Research and the Royal Marsden Hospital, retired due to ill health in 2014. Previously, he was Professor of Paediatric Oncology and Dean of Postgraduate Medical Studies, University of Newcastle upon Tyne.

Professor Pearson has over 45 years’ experience in drug development and neuroblastoma.

He was the founding chair of International Society of Paediatric Oncology Europe Neuroblastoma Group. He and Professor Sue Cohn created the International Neuroblastoma Risk Group Consortium. Professor Pearson was Chief Investigator of ENSG 5 which changed therapy of high-risk neuroblastoma in Europe. He was the first Chief Investigator of the first randomized European trial (BEACON) for refractory/relapsed neuroblastoma.

Professor Pearson led the early clinical development of many anti-cancer agents. He is on the Innovative Therapy for Children with Cancer Consortium Executive. He is Chair of ACCELERATE, EMA and FDA Paediatric Strategy Forum Oversight Committee, Senior Advisor to ACCELERATE. He was Chair of UK Children’s Cancer and Leukaemia Clinical Studies Group Novel Agents Subgroup. He led the paediatric drug development program at the Royal Marsden, with one of the largest portfolios in Europe.

He was Chairman of the United Kingdom Children’s Cancer Study Group from 2003 - 2006 and has published over 400 manuscripts.

Professor Pearson is chair of Solving Kids Cancer Scientific Advisory Board, Trustee of Neuroblastoma UK andProgramme Committee member of Fight Kids Cancer.

He was awarded a Life Time Achievement Award from Advances in Neuroblastoma Research in 2016.

Patrick Campbell

Patrick Campbell received his MD and a PhD in Genetics and Molecular Biology at Emory University in Atlanta, Georgia. He subsequently completed his Pediatric Residency at Emory and his Fellowship in Pediatric Hematology/Oncology at St. Jude Children’s Research Hospital in Memphis, TN. Patrick is currently an Associate Faculty Member at St. Jude in the department of Oncology. At St. Jude, he has served for almost 15 years as the lead of the Histiocytosis Service, and in this role he acts as the primary attending all patients with LCH and rare non-Langerhans histiocytoses. He has served as member and as the Chair of the Histiocyte Society Education Committee, as a member of the HS Steering Committee, and he is a member of both the Executive and Clinical Trials Committees for the North American Consortium for Histiocytosis. Patrick is also boarded in Clinical Informatics and is the Associate Chief Medical Information Officer for St. Jude, and he is slowly recovering from a recent implementation of an Epic EHR.

Eli Diamond

Dr. Eli Diamond is an Associate Attending Neuro-Oncologist at Memorial Sloan Kettering Cancer Center. He is the Chair of the Rare Histiocyic Disorders Steering Committee of the Histiocyte Society and a member of the Board of Trustees of the Histiocytosis Association and Chair of the Science Committee of this Board. His clinical practice and research focus on the treatment of adults with various histiocytic neoplasms, with additional focus upon neurologic manifestations of disease. He is an NIH-funded principal investigator whose research spans molecular pathogenesis, therapeutics, symptomatology, and caregiver needs.