Hematopoiesis and Bone Marrow Failure

Research in the Hematopoiesis and Bone Marrow Failure Laboratory, led by Dr. Neal Young, spans the basic sciences, clinical trials, and epidemiology. Bench work involves methods of cell and molecular biology, immunology, and virology.

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Neal Young

Research Interests

Research Interests

Research in the Cell Biology Section spans the basic sciences, clinical trials, and epidemiology. Bench work involves methods of cell and molecular biology, immunology, and virology. Blood cell production in healthy individuals and especially in patients with bone marrow failure is the main theme. Advanced techniques most recently include single cell RNAseq, CRISPR-Cas9 gene editing, multicolored bar coded flow cytometry, and SomaLogic deep proteomics. Use of patient samples is encouraged, as the Hematology Branch clinic for patients with aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), large granular lymphocytosis (LGL), and constitutional marrow failure is one of the largest in the world. Results in the laboratory have had real impact on the care of patients with these life-threatening illnesses, and access to patient samples over time and with therapeutic perturbation makes the laboratory strategies both innovative and concrete.

Some Current Laboratory Projects

Single cell RNAseq of normal and diseased bone marrow stem and progenitor cells

Gene editing in human cell lines of genes that affect the epigenome

RNAseq and gene editing applied to PNH cells

Murine models of immune mediated marrow failure, potential therapies and mechanisms

Sex hormone action on human marrow cells

Some Current and Planned Clinical Protocals

Eltrombopag in marrow failure, including treatment-naïve SAA, MAA, and MDS

Campath in LGL

Rapamycin to prevent relapse in AA

Sex hormone modulation of telomeres in patients with telomeropathies

Organization and Mentoring

The Cell Biology Section is large and has a nucleus of expert senior staff to provide training and supervision in a wide variety of advanced techniques. The Section is part of the Hematology Branch, and there is close interaction among all the Branch’s members, including sharing of resources and expertise. There is an active program of lectures and group meetings. In addition to knowledge acquisition, clear thinking and crisp verbal and written expression are priorities for development in trainees.

Dr. Young’s former trainees are leaders in hematology in the United States, Asia, Europe, and South America.

Featured Publications

Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia.

Authors: Groarke EM, Patel BA, Gutierrez-Rodrigues F, Rios O, Lotter J, Baldoni D, St Pierre A, Shalhoub R, Wu CO, Townsley DM, Young NS.
Br J Haematol. 2021 Feb 01;192(3):605-514
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HLA associations, somatic loss of HLA expression, and clinical outcomes in immune aplastic anemia.

Authors: Zaimoku Y, Patel BA, Adams SD, Shalhoub R, Groarke EM, Lee AAC, Kajigaya S, Feng X, Rios OJ, Eager H, Alemu L, Quinones Raffo D, Wu CO, Flegel WA, Young NS.
Blood. 2021 Dec 30;138(26):2799-2809
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Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study.

Authors: Patel BA, Groarke EM, Lotter J, Shalhoub R, Gutierrez-Rodrigues F, Rios O, Quinones Raffo D, Wu CO, Young NS.
Blood. 2022 Jan 06;139(1):34-43
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Benign and malignant hematologic manifestations in patients with VEXAS syndrome due to somatic mutations in UBA1.

Authors: Obiorah IE, Patel BA, Groarke EM, Wang W, Trick M, Ombrello AK, Ferrada MA, Wu Z, Gutierrez-Rodrigues F, Lotter J, Wilson L, Hoffmann P, Cardona DO, Patel N, Dulau-Florea A, Kastner DL, Grayson PC, Beck DB, Young NS, Calvo KR.
Blood Adv. 2021 Aug 24;5(16):3203-3215
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Somatic Mutations in "Benign" Disease.

Authors: Mustjoki S, Young NS.
N Engl J Med. 2021 May 27;384(21):2039-2052
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Meet the Team

Neal Young

Neal Young, M.D.

Senior Investigator

Neal Young received a A.B. cum laude from Harvard College in 1967 and his M.D. in 1971 from the Johns Hopkins School of Medicine. He did post-graduate medical training at Massachusetts General Hospital and Barnes Hospital at Washington University in St. Louis before joining the NHLBI in 1981. He is a Master of the American College of Physicians and has accrued many academic honors. His efforts as a US Government employee were recognized with Samuel J. Heyman Service to America Medal in Science and the Environment. Dr. Young has authored over 280 original scientific and medical articles in peer reviewed journals and more than 160 reviews and book chapters. He also is author or editor of 10 medical and scientific books, including a new textbook of hematology. Dr. Young is a member of the American Society for Clinical Investigation, American Association of Physicians, American Federation for Clinical Research, American Society of Hematology, and International Society for Experimental Hematology. He is co-inventor on 7 patents concerning B19 parvovirus. Dr. Young’s trainees head departments of hematology worldwide. He has lectured and published prolifically in hematology. He led a novel training program in Vietnam that transformed that country’s approach to blood diseases. He now heads a successful new trans-NIH initiative in human immunology.

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Kate Roskom

Research Nurse
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