Evidence generation and reproducibility in cell and gene therapy research: A call to action

Mohamed Abou-El-Enein^{1

2}, Aris Angelis^{3

4}, Frederick R Appelbaum^{5

6}, Nancy C Andrews⁷, Susan E Bates⁸, Arlene S Bierman⁹, Malcolm K Brenner¹⁰, Marina Cavazzana^{11

12}, Michael A Caligiuri¹³, Hans Clevers^{14

15}, Emer Cooke¹⁶, George Q Daley^{17

18}, Victor J Dzau¹⁹, Lee M Ellis²⁰, Harvey V Fineberg²¹, Lawrence S B Goldstein^{22

23}, Stephen Gottschalk²⁴, Margaret A Hamburg^{25

26}, Donald E Ingber^{17

18

27

28}, Donald B Kohn^{29

30}, Adrian R Krainer³¹, Marcela V Maus^{18

32}, Peter Marks³³, Christine L Mummery³⁴, Roderic I Pettigrew³⁵, Joni L Rutter³⁶, Sarah A Teichmann^{37

38}, Andre Terzic^{39

40}, Fyodor D Urnov⁴¹, David A Williams^{17

18

42}, Jedd D Wolchok^{43

44}, Mark Lawler⁴⁵, Cameron J Turtle^{5

6}, Gerhard Bauer⁴⁶, John P A Ioannidis^{47

48}

Affiliations

¹ Division of Medical Oncology, Department of Medicine and Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
² Joint USC/CHLA Cell Therapy Program, University of Southern California and Children's Hospital Los Angeles, Los Angeles, CA, USA.
³ Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, UK.
⁴ Department of Health Policy and LSE Health, London School of Economics and Political Science, London, UK.
⁵ Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
⁶ Division of Medical Oncology, Department of Medicine, University of Washington, Seattle, WA, USA.
⁷ Department of Pharmacology and Cancer Biology and Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
⁸ Department of Medicine, Division of Hematology/Oncology, Columbia University Irving Medical Center, New York, NY, USA.
⁹ Center for Evidence and Practice Improvement, Agency for Healthcare Research and Quality, Rockville, MD, USA.
¹⁰ Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
¹¹ Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France.
¹² Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Quest, INSERM, Paris, France.
¹³ Hematologic Malignancies and Stem Cell Transplantation Institute, City of Hope National Medical Center, Duarte, CA, USA.
¹⁴ Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW), Utrecht, the Netherlands.
¹⁵ University Medical Center Utrecht, Utrecht, the Netherlands.
¹⁶ European Medicines Agency, Amsterdam, the Netherlands.
¹⁷ Boston Children's Hospital, Boston, MA, USA.
¹⁸ Harvard Medical School, Boston, MA, USA.
¹⁹ US National Academy of Medicine, Washington, DC, USA.
²⁰ Department of Surgical Oncology and Molecular & Cellular Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
²¹ Gordon and Betty Moore Foundation, Palo Alto, CA, USA.
²² Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA, USA.
²³ Sanford Consortium for Regenerative Medicine, La Jolla, CA, USA.
²⁴ Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.
²⁵ American Association for the Advancement of Science (AAAS), Washington, DC, USA.
²⁶ National Academy of Medicine, Washington, DC, USA.
²⁷ Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
²⁸ Harvard John A. Paulson School of Engineering and Applied Sciences, Cambridge, MA, USA.
²⁹ Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.
³⁰ The Eli & Edith Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA.
³¹ Cold Spring Harbor Laboratory, Cold Spring Harbor, NY, USA.
³² Massachusetts General Hospital Cancer Center, Charlestown, MA, USA.
³³ Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.
³⁴ Department of Anatomy and Embryology, Leiden University Medical Center, Leiden, the Netherlands.
³⁵ ENMED, Colleges of Medicine and Engineering, Texas A&M University, Houston, TX, USA.
³⁶ National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
³⁷ Wellcome Sanger Institute, Wellcome Genome Campus, Cambridge, UK.
³⁸ Theory of Condensed Matter, Cavendish Laboratory, University of Cambridge, JJ Thomson Ave, Cambridge, UK.
³⁹ Center for Regenerative Medicine, Mayo Clinic, Rochester, MN, USA.
⁴⁰ Department of Cardiovascular Medicine, Mayo Clinic, Rochester, MN, USA.
⁴¹ Innovative Genomics Institute, University of California, Berkeley, CA, USA.
⁴² Division of Hematology/Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
⁴³ Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
⁴⁴ Parker Institute for Cancer Immunotherapy, San Francisco, CA, USA.
⁴⁵ Patrick G Johnston Centre for Cancer Research, Faculty of Medicine, Health and Life Sciences, Queen's University Belfast, Belfast, UK.
⁴⁶ Institute for Regenerative Cures, University of California, Davis, Sacramento, CA, USA.
⁴⁷ Stanford Prevention Research Center, Department of Medicine, Stanford University, Stanford, CA, USA.
⁴⁸ Department of Epidemiology and Population Health and Department of Biomedical Data Sciences, Stanford University, Stanford, CA, USA.

PMID: 34377737
PMCID: PMC8322039
DOI: 10.1016/j.omtm.2021.06.012

Editorial

Evidence generation and reproducibility in cell and gene therapy research: A call to action

Mohamed Abou-El-Enein et al. Mol Ther Methods Clin Dev. 2021.

. 2021 Jul 21:22:11-14.

doi: 10.1016/j.omtm.2021.06.012. eCollection 2021 Sep 10.

Authors

Affiliations

¹ Division of Medical Oncology, Department of Medicine and Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
² Joint USC/CHLA Cell Therapy Program, University of Southern California and Children's Hospital Los Angeles, Los Angeles, CA, USA.
³ Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, UK.
⁴ Department of Health Policy and LSE Health, London School of Economics and Political Science, London, UK.
⁵ Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
⁶ Division of Medical Oncology, Department of Medicine, University of Washington, Seattle, WA, USA.
⁷ Department of Pharmacology and Cancer Biology and Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
⁸ Department of Medicine, Division of Hematology/Oncology, Columbia University Irving Medical Center, New York, NY, USA.
⁹ Center for Evidence and Practice Improvement, Agency for Healthcare Research and Quality, Rockville, MD, USA.
¹⁰ Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
¹¹ Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France.
¹² Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Quest, INSERM, Paris, France.
¹³ Hematologic Malignancies and Stem Cell Transplantation Institute, City of Hope National Medical Center, Duarte, CA, USA.
¹⁴ Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW), Utrecht, the Netherlands.
¹⁵ University Medical Center Utrecht, Utrecht, the Netherlands.
¹⁶ European Medicines Agency, Amsterdam, the Netherlands.
¹⁷ Boston Children's Hospital, Boston, MA, USA.
¹⁸ Harvard Medical School, Boston, MA, USA.
¹⁹ US National Academy of Medicine, Washington, DC, USA.
²⁰ Department of Surgical Oncology and Molecular & Cellular Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
²¹ Gordon and Betty Moore Foundation, Palo Alto, CA, USA.
²² Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA, USA.
²³ Sanford Consortium for Regenerative Medicine, La Jolla, CA, USA.
²⁴ Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.
²⁵ American Association for the Advancement of Science (AAAS), Washington, DC, USA.
²⁶ National Academy of Medicine, Washington, DC, USA.
²⁷ Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
²⁸ Harvard John A. Paulson School of Engineering and Applied Sciences, Cambridge, MA, USA.
²⁹ Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.
³⁰ The Eli & Edith Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA.
³¹ Cold Spring Harbor Laboratory, Cold Spring Harbor, NY, USA.
³² Massachusetts General Hospital Cancer Center, Charlestown, MA, USA.
³³ Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.
³⁴ Department of Anatomy and Embryology, Leiden University Medical Center, Leiden, the Netherlands.
³⁵ ENMED, Colleges of Medicine and Engineering, Texas A&M University, Houston, TX, USA.
³⁶ National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
³⁷ Wellcome Sanger Institute, Wellcome Genome Campus, Cambridge, UK.
³⁸ Theory of Condensed Matter, Cavendish Laboratory, University of Cambridge, JJ Thomson Ave, Cambridge, UK.
³⁹ Center for Regenerative Medicine, Mayo Clinic, Rochester, MN, USA.
⁴⁰ Department of Cardiovascular Medicine, Mayo Clinic, Rochester, MN, USA.
⁴¹ Innovative Genomics Institute, University of California, Berkeley, CA, USA.
⁴² Division of Hematology/Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
⁴³ Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
⁴⁴ Parker Institute for Cancer Immunotherapy, San Francisco, CA, USA.
⁴⁵ Patrick G Johnston Centre for Cancer Research, Faculty of Medicine, Health and Life Sciences, Queen's University Belfast, Belfast, UK.
⁴⁶ Institute for Regenerative Cures, University of California, Davis, Sacramento, CA, USA.
⁴⁷ Stanford Prevention Research Center, Department of Medicine, Stanford University, Stanford, CA, USA.
⁴⁸ Department of Epidemiology and Population Health and Department of Biomedical Data Sciences, Stanford University, Stanford, CA, USA.

PMID: 34377737
PMCID: PMC8322039
DOI: 10.1016/j.omtm.2021.06.012

No abstract available

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References

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1. Tambuyzer E., Vandendriessche B., Austin C.P., Brooks P.J., Larsson K., Miller Needleman K.I., Valentine J., Davies K., Groft S.C., Preti R. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead. Nat. Rev. Drug Discov. 2020;19:93–111. - PubMed
1. Cavazzana M., Bushman F.D., Miccio A., André-Schmutz I., Six E. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges. Nat. Rev. Drug Discov. 2019;18:447–462. - PubMed
1. Feucht J., Abou-El-Enein M. Senolytic CAR T Cells in Solid Tumors and Age-Related Pathologies. Mol. Ther. 2020;28:2108–2110. - PMC - PubMed

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Evidence generation and reproducibility in cell and gene therapy research: A call to action

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Evidence generation and reproducibility in cell and gene therapy research: A call to action

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