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SickKids

Andreas Schulze

Title: Medical Director, Newborn Screening Program, Department of Paediatrics
Designations: MD, PhD, FRCPC
Phone: 416-813-7654 Ext. 401480
Email: andreas.schulze@sickkids.ca
Alternate Contact Name: Denese Henry
Alternate Phone: 416-813-7654 Ext. 401480
Alternate Email: denese.henry@sickkids.ca
U of T Positions: Professor, Paediatrics and Biochemistry

Research Positions

Senior Associate Scientist, Research Institute

Biography

Dr. Andreas Schulze is a metabolic paediatrician who works as a clinician-scientist at the Hospital for Sick Children in Toronto. He is Professor in the Departments of Paediatrics and Biochemistry at the University of Toronto. He is Medical Director of the Newborn Screening Program at The Hospital for Sick Children, and he is a Senior Associate Scientist at the SickKids Research Institute.

Dr. Schulze graduated from medical school with a medical diploma and a doctorate in medicine at the University of Leipzig in 1987. From 19871992 and under the supervision of Dr. Hans-Joachim Boehme and Dr. Eberhard Hoffmann, Dr. Schulze attended the PhD program in Physiological Biochemistry and defended his PhD thesis with summa cum laude. That was followed by clinical training in Paediatrics at the University Children’s Hospital in Heidelberg (19921999) under Dr. Hans-Juergen Bremer and Dr. Georg F. Hoffmann. In 2004, Dr. Schulze defended a Professorial Thesis (Habilitation) and received the Venia Legendi from the Ruprecht-Karls University Heidelberg. He got board certified in Physiological Biochemistry (1993) and in Pediatrics (1999).

In 2007, Dr. Schulze joined The Hospital for Sick Children in Toronto where he continued his clinical work in Inborn Errors of Metabolism and Newborn Screening and established a research laboratory at the SickKids Research Institute. As discovery- and translational scientist, Dr. Schulze’s centers his research program around creatine deficiency syndromes and creatine homeostasis, also encompassing arginine-, ornithine-, and guanidino compound metabolism, and small molecule drug discovery.

Research

Dr. Schulze is a Clinician Scientist with 30 years of work in the field of Inborn Errors of Metabolism. As discovery- and translational scientist, Dr. Schulze’s research centers around creatine deficiency syndromes and creatine homeostasis, also encompassing arginine-, ornithine-, and guanidino compound metabolism, small molecule drug discovery, and newborn screening.

He introduced tandem mass spectrometry for Expanded Newborn Screening in Germany and implemented quantitative in vivo MR spectroscopy at SickKids. He further applied and developped HPLC- and LC-MSMS methods for comprehensive guanidino compound analysis. With his research group in the Genetics and Genome Biology program at the SickKids Research Institute he focuses on Creatine Deficiency Syndromes, mechanism of creatine homeostasis, pathophysiology of guanidino compounds, small molecule therapeutics discovery, and genetic therapies.

In 1994, he described the full biochemical spectrum of Guanidinoacetate Methyltransferase Deficiency (GAMT-D). Since then, he became a leader in the field of Creatine Deficiency Syndromes and innovative therapies.

Dr. Schulze’s group has identified druggable treatment targets for GAMT-D and Sanfilippo syndrome and has engaged in drug discovery. The work on GAMT-D has led to the basic question how creatine controls its own homeostasis and thus the cellular energy metabolism. His group has recently uncovered a novel mechanism for the creatine feedback loop that controls creatine homeostasis. The current research is providing proof and elucidating the undepinnings of the mechanism.

 

Education

  • 20012004: Dr. med. habil. (D), Ruprecht Karls Universitat, Heidelberg, Germany. 
  • 19871992: Dr. med. (PhD), Universitat Leipzig, Leipzig, Germany.
  • 19841987: Dipl.-Med. (D), Universitat Leipzig, Leipzig, Germany.
  • 19811987: Medical Doctor, Universitat Leipzig, Leipzig, Germany.

Experience

  • 2017Present: Professor, Faculty of Medicine, University of Toronto, Toronto, Ontario.
  • 2017Present: Professor (Status Only), Department of Biochemistry, University of Toronto, Toronto, Ontario.
  • 2017Present: Senior Associate Scientist, Genetics and Genome Biology, Research Institute, The Hospital for Sick Children, Toronto, Ontario.
  • 20162017: Associate Professor (Status Only), Department of Biochemistry, University of Toronto, Toronto, Ontario.
  • 20072017: Associate Scientist, Genetics and Genome Biology, Research Institute, The Hospital for Sick Children, Toronto, Ontario.
  • 20072017: Associate Professor, Faculty of Medicine, University of Toronto, Toronto, Ontario.
  • 20062006: Assistant Professor, Johann Wolfgang Goethe-University, Frankfurt, Germany.
  • 20062006: Faculty Senior Staff Paediatrician, Paediatrics, Department of Paediatrics I, Johann Wolfgang Goethe-University, Frankfurt, Germany.
  • 20042007: University Lecturer, Ruprecht-Karls-University, Heidelberg, Germany.
  • 20022005: Faculty Senior Staff Paediatrician, Department of Paediatrics I (General Paediatrics, Metabolism, Gastroenterology, and Nephrology), Faculty of Medicine, Ruprecht-Karls-University of Heidelberg, Heidelberg, Germany.
  • 20002000: Head, Clinical Chemistry Laboratory, University Children’s Hospital Heidelberg (until closure because of laboratory centralization), Heidelberg, Germany.
  • 19992002: Faculty Staff Paediatrician, Department of Paediatrics I (General Paediatrics, Metabolism, Gastroenterology, and Nephrology), Faculty of Medicine, Ruprecht-Karls-University of Heidelberg, Heidelberg, Germany.
  • 19942000: Head, Newborn Screening Laboratory and Provincial Newborn Screening Program, University Children’s Hospital Heidelberg, Heidelberg, Germany.
  • 19922004: Academic Associate, Department of Paediatrics, Ruprecht-Karls-University, Heidelberg, Germany.
  • 19921999: Scientific Associate Physician, University Children’s Hospital, Paediatrics & Metabolic Genetics, Ruprecht-Karls-University, Heidelberg, Germany.
  • 19871992: Academic Associate, Institute of Biochemistry, Medical Faculty University of Leipzig, Leipzig, Germany.

Achievements

  • 2025: Leaders Circle Recognition Award. Leader’s Circle, Destination Toronto.
  • 2024: Nursing Excellence Award: Grace Evelyn Simpson Reed Award.
  • 2024Present: Medical Advisory Board Member, Arg1D Foundation, USA.
  • 2022Present: Chair, Inherited Metabolic Diseases Program, Ontario Public Drug Programs, Ministry of Health and Long-term Care.
  • 2022Present: Scientific Medical Advisory Board Member, Association for Creatine Deficiencies, USA.
  • 2022Present: Creatine Deficiency Syndromes Gene Therapy Consortium Member, Association for Creatine Deficiencies, USA.
  • 20202024: Chair, Faculty Appointment Advisory Committee (FAAC), Temerty Faculty of Medicine, University of Toronto. (Member since 2017).
  • 20222024: Chair, Newborn Screening Ontario Advisory Council. (Member since 2015).
  • 20192022: President, Garrod Association, Canada.
  • 20142022: Chair, Provincial Newborn Screening Working Group: Metabolic Disorders.
  • 2015: Student Choice Award, Arts & Science Co-op Programs - sciences category, University of Toronto Scarborough.
  • 2011: Planters Tapestry Award for Cultural Competence - The New Immigrant Support Network, Hospital for Sick Children.
  • 2008: The 2008 Interprofessional Practice Award in Paediatric Hospice Palliative Care - Hospital for Sick Children.

Publications

  1. Lee A, Anderson L, Tkachyova I, Tropak MB, Wang D, Schulze A. Validation and Optimization of a Stable Isotope-Labeled Substrate Assay for Measuring AGAT Activity.Int J Mol Sci. 2024 Nov 21;25(23). doi: 10.3390/ijms252312490. PubMed PMID: 39684202; PubMed Central PMCID: PMC11641458.
  2. Tkachyova I, Tropak MB, Lee A, Datti A, Ito S, Schulze A. Targeting AGAT gene expression - a drug screening approach for the treatment of GAMT deficiency. Expert Opin Drug Discov. 2024 Nov;19(11):1383-1397. doi: 10.1080/17460441.2024.2412994. Epub 2024 Oct 15. PubMed PMID: 39402976.
  3. Koeberl D, Schulze A, Sondheimer N, Lipshutz GS, Geberhiwot T, Li L, Saini R, Luo J, Sikirica V, Jin L, Liang M, Leuchars M, Grunewald S. Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature. 2024 Apr;628(8009):872-877. doi: 10.1038/s41586-024-07266-7. Epub 2024 Apr 3. PubMed PMID: 38570682; PubMed Central PMCID: PMC11156579.
  4. Cameron JM, Osundiji MA, Olson RJ, Olarewaju BA, Schulze A. ACMG/AMP variant classification framework in arginase 1 deficiency: Implications for birth prevalence estimates and diagnostics. Genet Med Open. 2024;2:101815. doi: 10.1016/j.gimo.2024.101815. eCollection 2024. PubMed PMID: 39669610; PubMed Central PMCID: PMC11613747.
  5. Tropak MB, Tkachyova I, Gu R, Lee A, Schulze A. Evidence of an intracellular creatine-sensing mechanism that modulates creatine biosynthesis via AGAT expression in human HAP1 cells. Sci Rep. 2023 Dec 16;13(1):22392. doi: 10.1038/s41598-023-49860-1. PubMed PMID: 38104212; PubMed Central PMCID: PMC10725494.

See a full list of Andreas Schulze's publications.

  • 2022–2028: Site-Principal Investigator. DTX301-CL301 - Phase 3, randomized, double-blind, placebo-controlled study of AAV- mediated gene transfer in patients with late-onset OTC deficiency 12 years of age and older. Ultragenyx Pharmaceutical Inc. (US).
  • 2021–2027: Site-Principal Investigator. mRNA-3927 - A Global, Phase 1/2, Open-label, Dose Optimization and Extension Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia. Moderna, Inc. (US).
  • 2021–2027: Site-Principal Investigator. mRNA-3705 - A Global, Phase 1/2, Open-label, Dose Optimization and Extension Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with isolated Methylmalonic Acidemia due to Methylmalonyl-CoA Mutase deficiency. Moderna, Inc. (US).
  • 2024–2026: Co-Principal Investigator. Combination Therapy for Sanfilippo Disease. Saint Justine & The Hospital for Sick Children. PCHP Catalyst Project Program. Co-Principal Investigator: Pshezhetsky AV.
  • 2014–2025: Site-Principal Investigator. Rare Disease Clinical Research Network - Urea Cycle Disorder Consortium (US).
  • 2021-2023: Principal Investigator. Creatine represses expression of AGAT - The mechanism how creatine regulates its own synthesis (Lee, A.). Association for Creatine Deficiencies (US).
  • 2018–2023: Site-Principal Investigator. Phase 1/2 Open-label Study in Patients with Arginase I, Aeglea Therapeutics (US).
  • 2017–2021: Principal Investigator. New Therapy for Six Mucopolysaccharidoses. Sanfilippo Children's Foundation (Australia) and Cure Sanfilippo (US).
  • 2020–2021: Principal Investigator. Guanidino Compounds Toxicity in Urea Cycle Disorders. Urea Cycle Disorders Consortium (US). Small Career Development Grant (Osundiji, M.)  

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