PaulJOrchardMD

Pediatric Hematology & Oncology • Minneapolis, MN

Paul J. Orchard, MD Professor, Division of Pediatric Bone Marrow Transplantation University of Minnesota

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Office
660B, Masonic Cancer Research Center
425 E River Parkway
Minneapolis, MN 55455
Phone+1 612-626-2313
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Education & Training

University of MinnesotaFellowship, Pediatric Hematology/Oncology, 1987 - 1990
University of Wisconsin Hospitals and ClinicsResidency, Pediatrics, 1984 - 1987
The Warren Alpert Medical School of Brown UniversityClass of 1984

Certifications & Licensure

MN State Medical License 1988 - 2024
WI State Medical License 1985 - 1987

Awards, Honors, & Recognition

Regional Top Doctor Castle Connolly, 2014-2019
CMS Meaningful Use Stage 1 Certification EpicCare Ambulatory EMR, Epic Systems Corporation, 2012-2013
Super Doctor SuperDoctors.com

Clinical Trials

Stem Cell Transplant for Bone Marrow Failure Syndromes Start of enrollment: 2000 Jun 01
Completed
Phase 2, Phase 3
Stem Cell Transplant w/Laronidase for Hurler Start of enrollment: 2004 Mar 01
Completed
Phase 2
Stem Cell Transplant for Inborn Errors of Metabolism Start of enrollment: 1995 Jan 01
Completed
Phase 2, Phase 3
Stem Cell Transplantation for Hurler Start of enrollment: 1999 May 01
Completed
Phase 2
HSCT for High Risk Inherited Inborn Errors Start of enrollment: 2006 Sep 01
Completed
Phase 2
Intrathecal Enzyme Replacement for Hurler Syndrome Start of enrollment: 2008 Jan 02
Completed
Phase 1
Reduced Intensity AlloTransplant For Osteopetrosis Start of enrollment: 2007 Sep 01
Terminated
Phase 2
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Start of enrollment: 2008 Mar 01
Terminated
Phase 2
Allogeneic Transplantation For Severe Osteopetrosis Start of enrollment: 2008 Aug 01
Completed
Phase 2, Phase 3
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Start of enrollment: 2009 Dec 01
Completed
Phase 2
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler Start of enrollment: 2012 May 29
Completed
Phase 1
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome Start of enrollment: 2012 Mar 28
Terminated
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Start of enrollment: 2013 Aug 21
Completed
Phase 2, Phase 3
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Start of enrollment: 2014 Jul 10
Recruiting
Phase 2
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies Start of enrollment: 2014 Sep 02
Recruiting
Not Applicable
Early Diagnosis Of Childhood Cerebral ALD Start of enrollment: 2018 Nov 01
Withdrawn
Adrenoleukodystrophy National Registry Study Start of enrollment: 2019 May 01
Recruiting
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) Start of enrollment: 2012 Aug 01
Recruiting
Not Applicable
Roles: Contact
MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT Start of enrollment: 2022 Dec 01
Withdrawn
Phase 1, Phase 2
Roles: Principal Investigator, Contact
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Publications & Presentations

PubMed

1 citations
Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10-year prospective study.
Bradley S Miller, Ellen B Fung, Klane K White, Troy C Lund, Paul Harmatz, Paul J Orchard, Chester B Whitley, Lynda E Polgreen> ;Journal of Inherited Metabolic Disease. 2023 Jul 1
17 citations
International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach.
Marc Engelen, Wouter J C van Ballegoij, Eric James Mallack, Keith P Van Haren, Wolfgang Köhler, Ettore Salsano, A S P van Trotsenburg, Fanny Mochel, Caroline Sevin, Mo...> ;Neurology. 2022 Nov 22
12 citations
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy.
Ashish O Gupta, Gerald Raymond, Elizabeth I Pierpont, Stephan Kemp, R Scott McIvor, Arpana Rayannavar, Bradley Miller, Troy C Lund, Paul J Orchard> ;Expert Opinion on Biological Therapy. 2022 Sep 1
1 citations
Glycoprotein nonmetastatic melanoma protein B (GNMPB) as a novel biomarker for cerebral adrenoleukodystrophy.
Leyla A Taghizadeh, Carina J King, David R Nascene, Ashish O Gupta, Paul J Orchard, LeeAnn Higgins, Todd W Markowski, Erin E Nolan, Justin W Furcich, Troy C Lund> ;Scientific Reports. 2022 May 14
2 citations
Primary Adrenal Insufficiency in a Boy with Type I Diabetes: The Importance of Considering X-linked Adrenoleukodystrophy.
Rebecca E Wiersma, Ashish O Gupta, Troy C Lund, Kyriakie Sarafoglou, Elizabeth I Pierpont, Paul J Orchard, Bradley S Miller> ;Journal of the Endocrine Society. 2022 May 1
1 citations
Quantifying medical manifestations in Hurler syndrome with the infant physical symptom score: associations with long-term physical and adaptive outcomes.
Alia Ahmed, Kyle Rudser, Kelly E King, Julie B Eisengart, Paul J Orchard, Elsa Shapiro, Chester B Whitley> ;Molecular Genetics and Metabolism. 2022 May 1
4 citations
Hematopoietic stem cell transplant for Hurler syndrome: does using bone marrow or umbilical cord blood make a difference?
Paul J Orchard, Ashish O Gupta, Julie B Eisengart, Lynda E Polgreen, Laura M Pollard, Elizabeth Braunlin, Marzia Pasquali, Troy C Lund> ;Blood Advances. 2022 Dec 13
12 citations
Association Between the Magnitude of Intravenous Busulfan Exposure and Development of Hepatic Veno-Occlusive Disease in Children and Young Adults Undergoing Myeloablat...
Bognàr, T., Bartelink, I., Egberts, T., Rademaker, C., Versluys, A., Slatter, M., Kletzel, M., Nath, C., Cuvelier, G., Savic, R., Dvorak, C., Long-Boyle, J., Cowan, M....> ;Transplantation and Cellular Therapy. 2022 Apr 1
7 citations
Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral Adrenoleukodystrophy.
Hongge Wang, Matthew D Davison, Martin L Kramer, Weiliang Qiu, Tatiana Gladysheva, Ruby M S Chiang, Can Kayatekin, David R Nascene, Leyla A Taghizadeh, Carina J King, ...> ;Cells. 2022 Mar 7
5 citations
Open-Label Pilot Study of Interferon Gamma-1b in Patients With Non-Infantile Osteopetrosis.
Andrew Nguyen, Weston P Miller, Ashish Gupta, Troy C Lund, Daniel Schiferl, Lok Sze Kelvin Lam, Zorayr Arzumanyan, Paul J Orchard, Lynda E Polgreen> ;JBMR Plus. 2022 Mar 1
Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A BMT Survivor Study Report.
Wadhwa, A., Chen, Y., Hageman, L., Schlichting, E., Funk, E., Hicks, J., Balas, N., Siler, A., Wu, J., Francisco, L., Holmqvist, A., Gupta, A., Lund, T., Orchard, P., ...> ;Transplantation and Cellular Therapy. 2022 Mar 1
2 citations
Hematopoietic cell transplantation for sialidosis type I.
Ashish O Gupta, Marc C Patterson, Tim Wood, Julie B Eisengart, Paul J Orchard, Troy C Lund> ;Molecular Genetics and Metabolism Reports. 2022 Mar 1
4 citations
Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I.
Victor Kovac, Elsa G Shapiro, Kyle D Rudser, Bryon A Mueller, Julie B Eisengart, Kathleen A Delaney, Alia Ahmed, Kelly E King, Brianna D Yund, Morton J Cowan, Julian R...> ;Molecular Genetics and Metabolism. 2022 Feb 1
3 citations
Busulfan dose Recommendation in Inherited Metabolic Disorders: Population Pharmacokinetic Analysis.
Takuto Takahashi, Sílvia M Illamola, Cathryn A Jennissen, Susan E Long, Troy C Lund, Paul J Orchard, Ashish O Gupta, Janel R Long-Boyle> ;Transplantation and Cellular Therapy. 2022 Feb 1
1 citations
Isoprostanoid Plasma Levels Are Relevant to Cerebral Adrenoleukodystrophy Disease.
Cinzia Signorini, Claudio De Felice, Thierry Durand, Jean-Marie Galano, Camille Oger, Silvia Leoncini, Joussef Hayek, Jetty Chung-Yung Lee, Troy C Lund, Paul J Orchard> ;Life. 2022 Jan 20
11 citations
-Acetylcysteine Reverses the Mitochondrial Dysfunction Induced by Very Long-Chain Fatty Acids in Murine Oligodendrocyte Model of Adrenoleukodystrophy.
Jie Zhou, Marcia R Terluk, Paul J Orchard, James C Cloyd, Reena V Kartha> ;Biomedicines. 2021 Dec 3
10 citations
Variables Affecting Outcomes After Allogeneic Hematopoietic Stem Cell Transplant for Cerebral Adrenoleukodystrophy.
, , , , , , , , , , , , , , , , , > ;Blood Advances. 2021 Nov 15
1 citations
Translational and clinical pharmacology considerations in drug repurposing for X-linked adrenoleukodystrophy-A rare peroxisomal disorder.
Julianne H. Tieu, Siddhee A. Sahasrabudhe, Paul J. Orchard, James C. Cloyd, Reena V. Kartha> ;British Journal of Clinical Pharmacology. 2021 Sep 23
2 citations
Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy.
Ashish Gupta, David Nascene, Ryan Shanley, Daniel L. Kenney-Jung, Julie B. Eisengart, Troy C. Lund, Paul J. Orchard, Elizabeth I. Pierpont> ;Journal of Inherited Metabolic Disease. 2021 Sep 17
18 citations
Differences in MPS I and MPS II Disease Manifestations.
Christiane S. Hampe, Brianna Yund, Paul J. Orchard, Troy C. Lund, Jacob Wesley, R. Scott McIvor> ;International Journal of Molecular Sciences. 2021 Jul 23
3 citations
Population Pharmacokinetic Analysis of N-Acetylcysteine in Pediatric Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplant.
Siddhee A. Sahasrabudhe, Reena V. Kartha, Michelle Ng, Lisa Basso, Usha Mishra, James C. Cloyd, Paul J. Orchard, Richard C. Brundage, Lisa D. Coles> ;Journal of Clinical Pharmacology. 2021 Aug 27
19 citations
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Mur...
Lalitha R. Belur, Megan Romero, Junggu Lee, Kelly M. Podetz-Pedersen, Zhenhong Nan, Maureen S. Riedl, Lucy Vulchanova, Kelley F. Kitto, Carolyn A. Fairbanks, Karen Koz...> ;Frontiers in Molecular Neuroscience. 2021 May 10
10 citations
Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I.
Troy C. Lund, Terence M. Doherty, Julie B. Eisengart, Rebecca Freese, Kyle Rudser, Ellen B. Fung, Bradley S. Miller, Klane K. White, Paul J. Orchard, Chester B. Whitle...> ;JIMD Reports. 2021 Mar 1
29 citations
Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.
Christiane S. Hampe, Jacob Wesley, Troy C. Lund, Paul J. Orchard, Lynda E. Polgreen, Julie B. Eisengart, Linda K. McLoon, Sebahattin Cureoglu, Patricia A. Schachern, R...> ;Biomolecules. 2021 Jan 29
7 citations
Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders
Ashish Gupta, Jaap Jan Boelens, Christen L. Ebens, Joanne Kurtzberg, Troy C. Lund, Angela R. Smith, John E. Wagner, Robert Wynn, Bruce R. Blazar, Paul J. Orchard> ;Bone Marrow Transplantation. 2021 Jan 13
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Journal Articles

Successful Hematopoietic Cell Transplantation Following Cardiac Transplantation in Two Pediatric Patients
Paul J Orchard, Angela R Smith, Rachel Phelan, Rebecca K Ameduri, Pediatric Transplantation

Press Mentions

Eli-Cel from Bluebird Gets Accelerated Approval for Boys with CALDSeptember 19th, 2022
Bluebird Bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)September 16th, 2022
Seattle Biotech Company Is the First to Receive Approval to Test B Cell Gene Therapy in HumansSeptember 1st, 2022
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Grant Support

Clinical Trial: Phase II Study Of Combined Laronidase (ALDURAZYME) Enzyme ReplacNational Center For Research Resources2008–2009
Phase II Study Of Combined Laronidase (ALDURAZYME) Enzyme Replacement Therapy (ENational Center For Research Resources2006–2007
Transplantation Of URB T Cells Containing The HSV-TK Suicide GeneNational Center For Research Resources2004–2006
First International Symposium On Osteopetrosis:National Institute Of Arthritis And Musculoskeletal And Skin Diseases2003