Cell Therapy Clinical Trials Part I
The five articles or white papers/research papers that provide recommendations on how to design and conduct a clinical trial for cell therapy include the "Guidelines for Phase I clinical trials," "The first decade of advanced cell therapy clinical trials using perinatal cells (2005–2015)," "Stem cell therapy clinical research: A regulatory conundrum for academia," "Bioethics of Clinical Applications of Stem Cells," and the "FDA Stem Cell Therapy Warning."
Cell Therapy Clinical Trials
1. Guidelines for Phase I clinical trials, 2018 edition
- This white paper published by The Association of the British Pharmaceutical Industry (ABPI) in 2018 gives specific guidelines on how to design and conduct different clinical trials, including cell therapy trials.
- ABPI has been publishing guidelines into conducting clinical trials since 1970s, and the 2018 edition reflects "the current EU legislation for the performance of Phase I clinical research as set down in the EU Clinical Trials Directive."
- The guidelines white paper includes specific directions for conducting trials in the European Union in accordance with the Clinical Trials Directive, and it provides the regulatory guidelines for the process.
- The main recommendations include answering initial questions such as population, age groups, etc. before the trial clinicians even apply for regulatory permits. Additionally, different types of trials require different regulatory permits, such as first-in-human trial (phase I exploratory trial), and subsequent parts/studies (clinical pharmacology trials).
2. The first decade of advanced cell therapy clinical trials using perinatal cells (2005–2015)
- This review of advanced cell therapy trials with perinatal cells was published in November 2017 in the magazine called Regenerative Medicine, and was written by Pedro S. Couto, Alexey Bersenev, and Frances Verter.
- This research provides the first review of advanced cell therapy trials with perinatal cells, and the review presented spans across 10 years (2005 – 2015).
- In the review, 281 clinical trials are summed up that line out the best practices for advanced cell therapy clinical trials that involve perinatal cells. The results include the most common cell source in the trials being "cord blood, but the cell type that provides the mechanism of action in the majority of trials is mesenchymal stem/stromal cells."
- The main recommendations include giving special attention to the most common cell sources, which include cord blood, as those have been proven to give the best results in clinical trials with perinatal cells.
3. Stem cell therapy clinical research: A regulatory conundrum for academia
- The research titled "Stem cell therapy clinical research: A regulatory conundrum for academia" was published in December 2017 by Anjali Nagpala, Chris Juttnera, Monica Anne Hamilton-Bruce, PaulRolan, and Simon A. Koblara.
- This review gives a summary of the most recent developments in regulatory frameworks in the countries that are at the forefront of stem cell clinical research.
- It discusses the "implications of these developments for researchers aiming to make the challenging transition from laboratory to clinical development of these therapies and considers possible pragmatic solutions that could accelerate this process that is essential to maintain research credibility and ensure patient safety."
- The main recommendations include the researchers applying for an industry-based clinical trial grant as those studies have been proven to be expedited.
4. Bioethics of Clinical Applications of Stem Cells
- "Bioethics of Clinical Applications of Stem Cells" was first published in April 2017 and revised and republished in December 2017. It was written by Carlo Petrini in the International Journal of Molecular Sciences.
- The academic review of clinical applications of stem cell clinical trials includes an overview of a multitude of problems, which include "safety, efficacy, information and consent, the right to unproven treatments, the right to try, costs, access, sustainability, scientific scrupulousness, patents, and regulatory aspects."
- Additionally, the main recommendations of the review include a comparison of the consequentialist and deontological approaches, and describes the top–down and bottom–up models of trials. Moreover, the review gives a suggestion about the practical frameworks for addressing the ethical issues, specifically when it comes to the "medical indications, patient preferences, quality of life, and contextual features."
5. FDA's Stem Cell Therapy Warning
- In its white paper published in 2019, the U.S. Food Drug Administration warns patients about the danger of unauthorized cell therapy clinical trials.
- In the review, the FDA notes it is increasing its "oversight and enforcement to protect people from dishonest and unscrupulous stem cell clinics, while continuing to encourage innovation so that the medical industry can properly harness the potential of stem cell products."
- It outlines the FDA approval process as well as safety concerns of clinical trials for cell therapy.
- The main recommendations include the fact that each study should be reviewed by the FDA before being submitted to be put into action by the hospitals. The FDA recommends that the agency will start taking harder action on clinical trials in the cell therapy field, which haven't been properly reviewed by the FDA.