Overcoming Barriers to Facilitate the Regulation of Multi-centre Regenerative Medicine Clinical Trials

Overview

Journal Stem Cell Res Ther

Publisher Biomed Central

Date 2018 Nov 10

PMID 30409192

Citations 10

Authors

Erika Kleiderman

Audrey Boily

Craig Hasilo

Bartha Maria Knoppers

Affiliations

Soon will be listed here.

Abstract

In the context of regenerative medicine and cellular therapies, the treatment under study often targets a less common disease or condition for which recruitment of a large number of research participants at any given site is challenging, if not impossible. One way to overcome this challenge is with a multi-centre clinical trial. This manuscript first aims to briefly outline the existing ethical, legal and social implications as well as the regulatory frameworks associated with multi-centre regenerative medicine clinical trials. Second, it considers the regulatory limitations and barriers surrounding the initiation of such trials in Canada, the USA and Europe. Third, it concludes with a set of recommendations for facilitating multi-centre clinical trials, at both national and international levels.

Citing Articles

Assessing the generalisability of a multicentre qualitative dementia research: the experience and challenges faced by the MinD project in Europe.

Lim J, Niedderer K, Tournier I, Almeida R, Harrison D, Holthoff-Detto V Open Res Eur. 2023; 1:64.

PMID: 37645110 PMC: 10446055. DOI: 10.12688/openreseurope.13700.3.

Recent advances on 3D-printed PCL-based composite scaffolds for bone tissue engineering.

Gharibshahian M, Salehi M, Beheshtizadeh N, Kamalabadi-Farahani M, Atashi A, Nourbakhsh M Front Bioeng Biotechnol. 2023; 11:1168504.

PMID: 37469447 PMC: 10353441. DOI: 10.3389/fbioe.2023.1168504.

Early-Phase Clinical Trials of Bio-Artificial Organ Technology: A Systematic Review of Ethical Issues.

de Jongh D, Massey E, Cronin A, Schermer M, Bunnik E Transpl Int. 2022; 35:10751.

PMID: 36388425 PMC: 9659568. DOI: 10.3389/ti.2022.10751.

The Direct to Consumer Stem Cell Market and the Role of Primary Care Providers in Correcting Misinformation.

Arthurs J, Martin Lillie C, Master Z, Shapiro S J Prim Care Community Health. 2022; 13:21501319221121460.

PMID: 36112830 PMC: 9476238. DOI: 10.1177/21501319221121460.

The Ethical Implications of Tissue Engineering for Regenerative Purposes: A Systematic Review.

de Kanter A, Jongsma K, Verhaar M, Bredenoord A Tissue Eng Part B Rev. 2022; 29(2):167-187.

PMID: 36112697 PMC: 10122262. DOI: 10.1089/ten.TEB.2022.0033.

References

Dunbar C, High K, Joung J, Kohn D, Ozawa K, Sadelain M . Gene therapy comes of age. Science. 2018; 359(6372). DOI: 10.1126/science.aan4672. View

Daley G, Hyun I, Apperley J, Barker R, Benvenisty N, Bredenoord A . Setting Global Standards for Stem Cell Research and Clinical Translation: The 2016 ISSCR Guidelines. Stem Cell Reports. 2016; 6(6):787-797. PMC: 4912385. DOI: 10.1016/j.stemcr.2016.05.001. View

King N, Perrin J . Ethical issues in stem cell research and therapy. Stem Cell Res Ther. 2014; 5(4):85. PMC: 4097842. DOI: 10.1186/scrt474. View

Hauskeller C . Can harmonized regulation overcome intra-European differences? Insights from a European Phase III stem cell trial. Regen Med. 2017; 12(6):599-609. DOI: 10.2217/rme-2017-0064. View

Boran T, Menezes-Ferreira M, Reischl I, Celis P, Ferry N, Gansbacher B . Clinical Development and Commercialization of Advanced Therapy Medicinal Products in the European Union: How Are the Product Pipeline and Regulatory Framework Evolving?. Hum Gene Ther Clin Dev. 2017; 28(3):126-135. DOI: 10.1089/humc.2016.193. View

Illes J, Sipp D, Kleiderman E, Benjaminy S, Isasi R, Lomax G . A blueprint for the next generation of ELSI research, training, and outreach in regenerative medicine. NPJ Regen Med. 2018; 2:21. PMC: 5677945. DOI: 10.1038/s41536-017-0026-z. View

Marks P, Gottlieb S . Balancing Safety and Innovation for Cell-Based Regenerative Medicine. N Engl J Med. 2018; 378(10):954-959. DOI: 10.1056/NEJMsr1715626. View

Martell K, Trounson A, Baum E . Stem cell therapies in clinical trials: workshop on best practices and the need for harmonization. Cell Stem Cell. 2010; 7(4):451-4. DOI: 10.1016/j.stem.2010.09.004. View

Berger I, Ahmad A, Bansal A, Kapoor T, Sipp D, Rasko J . Global Distribution of Businesses Marketing Stem Cell-Based Interventions. Cell Stem Cell. 2016; 19(2):158-162. DOI: 10.1016/j.stem.2016.07.015. View

10.

Caulfield T, Rachul C, Zarzeczny A . The evolution of policy issues in stem cell research: an international survey. Stem Cell Rev Rep. 2012; 8(4):1037-42. DOI: 10.1007/s12015-012-9404-5. View

11.

McCormick J, Huso H . Stem cells and ethics: current issues. J Cardiovasc Transl Res. 2010; 3(2):122-7. PMC: 3933367. DOI: 10.1007/s12265-009-9155-0. View

12.

Lysaght T, Kerridge I, Sipp D, Porter G, Capps B . Ethical and Regulatory Challenges with Autologous Adult Stem Cells: A Comparative Review of International Regulations. J Bioeth Inq. 2017; 14(2):261-273. DOI: 10.1007/s11673-017-9776-y. View

13.

Neofytou E, OBrien C, Couture L, Wu J . Hurdles to clinical translation of human induced pluripotent stem cells. J Clin Invest. 2015; 125(7):2551-7. PMC: 4563685. DOI: 10.1172/JCI80575. View

14.

Hartmann J, Schussler-Lenz M, Bondanza A, Buchholz C . Clinical development of CAR T cells-challenges and opportunities in translating innovative treatment concepts. EMBO Mol Med. 2017; 9(9):1183-1197. PMC: 5582407. DOI: 10.15252/emmm.201607485. View

15.

Sleeboom-Faulkner M, Chekar C, Faulkner A, Heitmeyer C, Marouda M, Rosemann A . Comparing national home-keeping and the regulation of translational stem cell applications: An international perspective. Soc Sci Med. 2016; 153:240-9. DOI: 10.1016/j.socscimed.2016.01.047. View

16.

Mason C, McCall M, Culme-Seymour E, Suthasan S, Edwards-Parton S, Bonfiglio G . The global cell therapy industry continues to rise during the second and third quarters of 2012. Cell Stem Cell. 2012; 11(6):735-9. DOI: 10.1016/j.stem.2012.11.013. View

17.

Nagpal A, Juttner C, Hamilton-Bruce M, Rolan P, Koblar S . Stem cell therapy clinical research: A regulatory conundrum for academia. Adv Drug Deliv Rev. 2016; 122:105-114. DOI: 10.1016/j.addr.2016.10.001. View

18.

Buskard N . Bone marrow transplantation in Canada. Can Med Assoc J. 1980; 123(1):19-22. PMC: 1704559. View

19.

Rosemann A . Why regenerative stem cell medicine progresses slower than expected. J Cell Biochem. 2014; 115(12):2073-6. DOI: 10.1002/jcb.24894. View

20.

Benjaminy S, Lo C, Illes J . Social Responsibility in Stem Cell Research - Is the News All Bad?. Stem Cell Rev Rep. 2016; 12(3):269-75. DOI: 10.1007/s12015-016-9646-8. View