Article
Immunology
Zilong Guo, Yirui Zhang, Mingpeng Fu, Liang Zhao, Zhen Wang, Zhuoshuo Xu, Huifen Zhu, Xiaoli Lan, Guanxin Shen, Yong He, Ping Lei
Summary: The transferrin receptor (TfR) is being evaluated as an alternative target for CAR T cell therapy, showing potent cytotoxic effects against hematological malignancies. This suggests TfR could potentially broaden and enhance the therapeutic efficacy of CAR T cells, making it a promising universal target for further research.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Oncology
Jia Xu, Wenjing Luo, Chenggong Li, Heng Mei
Summary: CD19-targeted CAR-T cell therapy has shown remarkable efficacy in treating B-cell malignancies, but CD19-negative relapse remains a significant challenge. CD22 serves as a potential alternative target for CD19 CAR-T cell-resistant patients, with therapies showing acceptable toxicities and promising efficacy.
EXPERIMENTAL HEMATOLOGY & ONCOLOGY
(2023)
Review
Oncology
Bouthaina Dabaja, Michael Spiotto
Summary: Over the past half-century, the role of radiotherapy has shifted from directly killing cancer cells to stimulating anti-tumor immune responses. This interplay between radiation, the tumor microenvironment, and the immune system is crucial in cancer immunology. While most studies have focused on solid tumors, there is growing understanding of this interaction in hematological malignancies. This review highlights recent advances in immunotherapy and adoptive cell therapy and emphasizes the importance of incorporating radiation therapy into the treatment of hematological malignancies.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Martin G. Klatt, Tao Dao, Zhiyuan Yang, Jianying Liu, Sung Soo Mun, Megan M. Dacek, Hanzhi Luo, Thomas J. Gardner, Christopher Bourne, Leila Peraro, Zita E. H. Aretz, Tanya Korontsvit, Michael Lau, Michael G. Kharas, Cheng Liu, David A. Scheinberg
Summary: Mass spectrometry identified a non-immunogenic HLA ligand as a target for CAR T-cell therapy, which showed broad effectiveness against multiple cancer types, particularly hematologic cancers, and had no toxic effects on healthy cells.
Review
Medicine, General & Internal
Lukasz P. Gondek, Vivien A. Sheehan, Courtney D. Fitzhugh
Summary: Sickle cell disease (SCD) is associated with severe morbidity and early mortality. Two recent large studies have found an increased risk of leukemia in individuals with SCD, particularly after graft rejection and gene therapy. Clonal hematopoiesis (CH) is a recognized premalignant condition that may be more common in SCD than in the general population. This review explores the risk factors for CH and progression to leukemia in SCD and suggests the need for individualized benefit/risk assessment for patients undergoing curative therapies.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Cell & Tissue Engineering
Margaret G. Lamb, Hemalatha G. Rangarajan, Brian P. Tullius, Dean A. Lee
Summary: Adoptive transfer of NK cells has shown early evidence of efficacy in hematologic malignancies, where NK cells are dysfunctional and further impaired by chemotherapy, radiation, and immunosuppressants. Improving NK cell therapy may lead to improved therapeutic outcomes in these settings.
STEM CELL RESEARCH & THERAPY
(2021)
Review
Cell Biology
Laura Gragnani, Serena Lorini, Silvia Marri, Anna Linda Zignego
Summary: Notch receptors play critical roles in fundamental cellular processes and are implicated in hematopoiesis, angiogenesis, and various human malignancies. They could serve as potential therapeutic targets for hematological cancers.
Review
Oncology
Zheng Tian, Ming Liu, Ya Zhang, Xin Wang
Summary: Harnessing the power of immune cells, especially T cells, to enhance anti-tumor activities has become a promising strategy in clinical management of hematologic malignancies. Bispecific antibodies such as BiTEs have shown potential in recruiting T cells to tumor cells for immunotherapy, with a focus on improving efficacy and safety in clinical treatments for hematologic malignancies.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Review
Oncology
Hidefumi Hiramatsu
Summary: Acute lymphoblastic leukemia (ALL) is the most common cancer in children, and though the long-term survival rate is high at 90%, about 20% of patients experience relapse and need second-line treatment. The introduction of immunotherapy, including CAR-T cell therapy, has revolutionized the treatment of relapsed and refractory ALL. However, CAR-T cell therapy can cause specific adverse events, and the biggest challenge remains preventing relapse. Overall, the success of CD19 CAR-T cell therapy against B cell malignancies has led to further research into its potential for other hematologic malignancies.
INTERNATIONAL JOURNAL OF CLINICAL ONCOLOGY
(2023)
Review
Oncology
Jun Meng, XiaoQin Wu, Zhen Sun, RenDe Xun, MengSi Liu, Rui Hu, JianChao Huang
Summary: This study summarized the efficacy and safety of three CAR-T cell products in treating hematologic malignancies, showing promising results in patients with different pathological subtypes and clinical characteristics, but also highlighting the potential for severe toxicity in certain situations. Additional data on these products are needed to benefit a broader patient population.
FRONTIERS IN ONCOLOGY
(2021)
Review
Oncology
Jason H. Kurzer, Olga K. Weinberg
Summary: PHF6, an epigenetic regulator, acts as a tumor suppressor protein and is commonly associated with T-lymphoblastic leukemia, playing a critical role in lineage plasticity and disease progression within hematopoietic malignancies.
FRONTIERS IN ONCOLOGY
(2021)
Review
Immunology
Yifan Xu, Jin Jiang, Yutong Wang, Wei Wang, Haokun Li, Wenyu Lai, Zhipeng Zhou, Wei Zhu, Zheng Xiang, Zhiming Wang, Zhe Zhu, Lingfeng Yu, Xiaolan Huang, Hua Zheng, Sha Wu
Summary: Gynecologic malignancies are leading causes of death among women worldwide, and adoptive T cell therapy using engineered T cells has shown promising efficacy in treating tumors. Ongoing research is driving the application of this therapy in the treatment of gynecologic malignancies.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Oncology
Samane Abbasi, Milad Asghari Totmaj, Masoumeh Abbasi, Saba Hajazimian, Pouya Goleij, Javad Behroozi, Behrouz Shademan, Alireza Isazadeh, Behzad Baradaran
Summary: Over the last decade, novel therapeutic approaches like CAR-T-cell immunotherapy have transformed the treatment perspective of human malignancies. However, this therapy is associated with side effects and faces challenges in manufacturing, engineering, applications, and toxicities. Further studies are needed to enhance efficacy and minimize adverse events.
Review
Oncology
Karen Kai-Lin Fang, Jong Bok Lee, Li Zhang
Summary: T-cell malignancies are aggressive and have poor prognoses, but adoptive cell therapy has shown promise as a new treatment option. Overcoming challenges, such as difficulties in applying this therapy to T-cell malignancies, is a focus of research. This review provides an overview of recent progress in adoptive cell therapy for T-cell malignancies, discussing the benefits and drawbacks of different therapy types and emphasizing the potential advantages and current applications of innate immune cell-based approaches.
Article
Hematology
Sarah J. Nagle, Catherine Murphree, Philipp W. Raess, Levanto Schachter, Andy Chen, Brandon Hayes-Lattin, Eneida Nemecek, Richard T. Maziarz
Summary: CAR T-cell therapy is effective in treating patients with R/R DLBCL, but it is associated with significant prolonged hematologic toxicity (PHT) that can affect patients' survival rates. Risk factors associated with PHT include CRS, the use of tocilizumab or steroids, peak levels of ferritin and C-reactive protein. More research is needed to further investigate PHT and establish management standards.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
Correction
Biophysics
Alison Leiper, Maite Houwing, E. Graham Davies, Kanchan Rao, Siobhan Burns, Emma Morris, Joop Laven, Anne-Lotte van der Kooi, Marry van den Heuvel Eibrink, Stephen Nussey
Summary: A revised version of the paper has been published and is accessible through a link at the top of the paper.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Alison Leiper, Maite Houwing, E. Graham Davies, Kanchan Rao, Siobhan Burns, Emma Morris, Joop Laven, Anne-Lotte van der Kooi, Marry van den Heuvel Eibrink, Stephen Nussey
BONE MARROW TRANSPLANTATION
(2020)
Editorial Material
Immunology
Emma C. Morris
Editorial Material
Hematology
Emma C. Morris
Article
Hematology
Siobhan O. Burns, Emma C. Morris
Summary: Inborn errors of immunity (IEIs) are rare disorders caused by germline mutations in immune system regulating genes, primarily manifesting as severe infections and possibly associated with autoimmunity, inflammation, and malignancy. Allogeneic hematopoietic stem cell transplant (HSCT) has become a preferred treatment for early, severely affected IEI children and is now increasingly considered for adult patients as well.
Letter
Hematology
Thomas A. Fox, Amy A. Kirkwood, Louise Enfield, Maeve O'Reilly, Suzanne Arulogun, Shirley D'Sa, Jenny O'Nions, Janki Kavi, Evan Vitsaras, William Townsend, Siobhan O. Burns, Satyen H. Gohil, Kate Cwynarski, Kirsty J. Thomson, Mahdad Noursadeghi, Robert S. Heyderman, Tommy Rampling, Kirit M. Ardeshna, Laura E. McCoy, Emma C. Morris
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Biochemistry & Molecular Biology
A. Magnani, M. Semeraro, F. Adam, C. Booth, L. Dupre, E. C. Morris, A. Gabrion, C. Roudaut, D. Borgel, A. Toubert, E. Clave, C. Abdo, G. Gorochov, R. Petermann, M. Guiot, M. Miyara, D. Moshous, E. Magrin, A. Denis, F. Suarez, C. Lagresle, A. M. Roche, J. Everett, A. Trinquand, M. Guisset, J. Xu Bayford, S. Hacein-Bey-Abina, A. Kauskot, R. Elfeky, C. Rivat, S. Abbas, H. B. Gaspar, E. Macintyre, C. Picard, F. D. Bushman, A. Galy, A. Fischer, E. Six, A. J. Thrasher, M. Cavazzana
Summary: This article presents the long-term follow-up results of gene therapy for patients with Wiskott-Aldrich syndrome, showing that lentiviral gene therapy provides sustained clinical benefits for these patients.
Article
Hematology
Michael H. Albert, Tiarlan Sirait, Dirk-Jan Eikema, Katerina Bakunina, Claudia Wehr, Felipe Suarez, Maria Laura Fox, Nizar Mahlaoui, Andrew R. Gennery, Arjan C. Lankester, Rita Beier, Maria Ester Bernardo, Venetia Bigley, Caroline A. Lindemans, Siobhan O. Burns, Ben Carpenter, Jaroslaw Dybko, Tayfun Gungor, Fabian Hauck, Su Han Lum, Dmitry Balashov, Roland Meisel, Despina Moshous, Ansgar Schulz, Carsten Speckmann, Mary A. Slatter, Brigitte Strahm, Duygu Uckan-Cetinkaya, Isabelle Meyts, Tanja C. Vallee, Robert Wynn, Benedicte Neven, Emma C. Morris
Summary: Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with inborn errors of immunity (IEI), but its adoption for adolescents and adults with IEI has been delayed due to lack of outcome data and perceived high risk of transplant-related mortality. This large retrospective, multicenter study reports on 329 IEI patients who underwent HSCT between 2000 and 2019. The study found that the overall survival (OS) and event-free survival (EFS) rates were 78% and 65% at 1 year post-HSCT, and 71% and 62% at 5 years post-HSCT, respectively. The study also identified risk factors for adverse outcomes, providing important evidence for patient selection.
Correction
Biochemistry & Molecular Biology
A. Magnani, M. Semeraro, F. Adam, C. Booth, L. Dupre, E. C. Morris, A. Gabrion, C. Roudaut, D. Borgel, A. Toubert, E. Clave, C. Abdo, G. Gorochov, R. Petermann, M. Guiot, M. Miyara, D. Moshous, E. Magrin, A. Denis, F. Suarez, C. Lagresle, A. M. Roche, J. Everett, A. Trinquand, M. Guisset, J. Xu Bayford, S. Hacein-Bey-Abina, A. Kauskot, R. Elfeky, C. Rivat, S. Abbas, H. B. Gaspar, E. Macintyre, C. Picard, F. D. Bushman, A. Galy, A. Fischer, E. Six, A. J. Thrasher, M. Cavazzana
Article
Immunology
Alan Kennedy, Erin Waters, Behzad Rowshanravan, Claudia Hinze, Cayman Williams, Daniel Janman, Thomas A. Fox, Claire Booth, Anne M. Pesenacker, Neil Halliday, Blagoje Soskic, Satdip Kaur, Omar S. Qureshi, Emma C. Morris, Shinji Ikemizu, Christopher Paluch, Jiandong Huo, Simon J. Davis, Emmanuel Boucrot, Lucy S. K. Walker, David M. Sansom
Summary: CD28 and CTLA-4 play important roles in regulating T cell immunity. The study found that CTLA-4 captures ligands through transendocytosis, and CD80 leads to ubiquitination and degradation of CTLA-4, while CD86 allows CTLA-4 to detach and recycle back to the cell surface. Clinically relevant mutations disrupt CD86 transendocytosis and are associated with autoimmune diseases.
Article
Cell Biology
Thomas Andrew Fox, Benjamin Christopher Houghton, Lina Petersone, Erin Waters, Natalie Mona Edner, Alex McKenna, Olivier Preham, Claudia Hinze, Cayman Williams, Adriana Silva de Albuquerque, Alan Kennedy, Anne Maria Pesenacker, Pietro Genovese, Lucy Sarah Kate Walker, Siobhan Oisin Burns, David Michael Sansom, Claire Booth, Emma Catherine Morris
Summary: A gene editing strategy was designed to insert the CTLA-4 cDNA into primary human T cells, restoring CTLA-4 expression and demonstrating the feasibility of using T cell gene therapy for CTLA-4 insufficiency.
SCIENCE TRANSLATIONAL MEDICINE
(2022)
Review
Immunology
Emma C. Morris, Sattva S. Neelapu, Theodoros Giavridis, Michel Sadelain
Summary: CAR T cell therapy has revolutionized the field of cancer treatment, but significant toxicities can occur in up to one-third of patients. The most common toxicities include cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. Understanding their pathophysiology is crucial for developing novel therapeutics for prevention and management.
NATURE REVIEWS IMMUNOLOGY
(2022)
Letter
Hematology
Thomas A. Fox, Ethan Troy-Barnes, Amy A. Kirkwood, Wei Y. Chan, James W. Day, Selina J. Chavda, Emil A. Kumar, Kate David, Oliver Tomkins, Emilie Sanchez, Marie Scully, Asim Khwaja, Jonathan Lambert, Mervyn Singer, Claire Roddie, Emma C. Morris, Kwee L. Yong, Kirsty J. Thomson, Kirit M. Ardeshna
BRITISH JOURNAL OF HAEMATOLOGY
(2020)
Article
Allergy
Linlin Yang, Claire Booth, Carsten Speckmann, Markus G. Seidel, Austen J. J. Worth, Gerhard Kindle, Arjan C. Lankester, Bodo Grimbacher, Andrew R. Gennery, Mikko R. J. Seppanen, Emma C. Morris, Siobhan O. Burns
Summary: XIAP deficiency presents with a wide spectrum of clinical features, with no clear genotype-phenotype correlation. Conservative management and hematopoietic stem-cell transplantation (HSCT) have different overall survival probabilities, with potential factors like HLH and myeloablative conditioning regimens impacting outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE
(2022)
Meeting Abstract
Allergy
Tara Saco, Christoph Geier, David Buchbinder, Joseph Hernandez, Svetlana Sharapova, Alexis Cochino, Tomas Milota, Elema Latysheva, Emma Westerman-Clark, Olajumoke Fadugba, Emma Morris, Michael Albert, Dimana Dimitrova, Despina Moshous, Jennifer Kanakry, Steven Holland Md, Jean-Pierre de Villartay, Ravishankar Sargur, Luigi Notarangelo, Jolan Walter
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2021)