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Survival guide to stem cell research and therapies provides comprehensive guidance to publicly available resource materials, libraries and registries for people who are interested in understanding currently available treatment options involving human stem cells.
POTENTIAL: The first section explains how stem cells are currently used in research, drug testing, and therapy, and how they have to be manipulated before transfer to make any treatments possible.
CLASSIFICATION: Origin and ability of stem cells to differentiate into different cell types determines how different types of stem cells are typically used.
CLINICAL RESEARCH: In this section, we will introduce two most important registries of clinical trials: NIH registry ClinicalTrials gov and WHO International Clinical Trials Registry Platform. A project is part of this section to give students the opportunity to get hands on experience with collecting and collating relevant information from registries and libraries, and interpretation of the findings. Real time interactive sessions are included to allow students to ask questions and offer additional guidance.
PATIENT DEMAND: In this section, we briefly introduce challenges relating to marketing claims, objective outcome measures, advertising strategies and patient autonomy.
REGULATORY AND LEGAL FRAMEWORK: Stem cell therapies are regulated differently in various countries around the world. In this section, we will focus on regulations that govern stem cell research and therapies in the U.S. and in the European Union. Policies on stem cell research are driven by ethical concerns relating to research that utilizes human embryos. China recently announced new ethical guidelines and new rules for their stem cell clinics, regulating both trials and treatments.
PROFESSIONAL SOCIETIES: The last section explains the role of professional societies in stem cell research and therapies.
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|Section 1: The Potential of Stem Cell Treatments|
In the first section you will learn how stem cells are currently used in research, drug testing, and therapy, and how stem cells have to be manipulated before transfer to make any treatments possible.
The first lecture introduces the Declaration of Helsinki as the most relevant and appropriate ethical standard that governs the use of unproven treatments in clinical practice.
Details and sequence of the course are presented to guide students through the course content.
Audio and slide deck (pdf) are available for download.
The lecture explains the potential uses of human stem cells and the obstacles that must be overcome before this potential will be realized. Stem cells are used in basic (non-clinical) research, in testing new drugs, and for cell-based therapies.
To realize the promise of novel cell-based therapies, scientists must be able to manipulate stem cells so that they possess the necessary characteristics for successful differentiation, transplantation, and engraftment.
Please take a short survey after you have completed this section. The survey is not graded but will be used to address any concerns you may have regarding Q&A sessions and live interactions.
|Section 2: Classification of stem cells|
Second section is dedicated to classification of stem cells by their origin and their ability to differentiate into different cell types. Here you will see the fundamental difference between embryonic and adult stem cells and their typical use. The terminology is essential for understanding of the sections that follow.
This lecture provides students with essential terminology necessary to understand how stem cells function and how they are used.
There is a quiz in the end of this section. Students are advised to
allow enough time to find the correct answer with the help of provided
|Quiz 1||15 questions|
|Section 3: Clinical Research involving stem cells|
In Section III, Clinical Research, students will have the opportunity to explore registries of clinical studies and learn how to find relevant information about ongoing and completed clinical trials.
Students will have the opportunity to explore registries of clinical
studies and learn how to find relevant information about ongoing and
completed clinical trials. Students will see what kind of
trials are conducted around the world, with what type of stem cells, what kind of conditions are being studied the most, which studies have posted results available
to the public and how to find them, which trials are currently
recruiting and which were terminated or suspended, and what are the
Information from clinical trial registries shall be complemented by whatever has been published on the topic in scientific literature.
Lecture 4 introduces two most important registries of clinical trials: ClinicalTrials (NIH) and ICTRP (WHO). All currently conducted stem cell trials are placed on a map to illustrate the most popular destinations for stem cell research. The total volume of early research is then compared to available approved treatments.
A project is part of this section to give you the opportunity to get hands on experience with collecting and collating relevant information from registries and libraries, its visualization in the form of graphs and charts, and interpretation of the data. Detailed guidance how to download data from the registries is in the course material section. You are welcome to use real time interactive sessions to ask questions and share your ideas and concerns.
Clinical research in human subjects is conducted in 5 phases – 0, I, II, III and IV. Lecture 5 explains objectives of these research phases and also clarifies the difference between interventional and observational studies and expanded access to therapy.
Analysis of data downloaded from the NIH registry shows how many studies have published results available to the public, and allows comparison of research utilizing different stem cell types. Presented data from the NIH registry also provide useful information on sources of funding and studied conditions.
All stem cell trials available in the registry are placed on a map to illustrate popular destinations for stem cell research, and to compare research conducted on adult, umbilical, mesenchymal and embryonic stem cells.
WHO platform ICTRP contains information on trials from other parts of the world. Students will learn how to complement data from registries with information available in scientific literature.
Basic research in a laboratory and on animals is compared to research involving humans.
|Lecture 7||24 pages|
1) Download data for all stem cell trials from the NIH Clinical trial registry and the WHO ICTRP registry platform and import the datasets in Excel as shown in the project guide.
2) Pick a disease/condition you want to study.
3) Search PubMed for relevant queries.
4) Analyze the datasets from registries using the Excel template as a guide.
How many studies were identified for this particular condition (all - in vitro, in vivo, and on humans)?
Create a pie graph which would show how many studies were conducted in vitro, on laboratory animals, and how many involved humans.
How many people were enrolled in these studies in total?
Create a pie graph which would show number of participants by study phase.
In which countries were these studies conducted?
Create a column graph which would show number of human studies by phase.
Create a graph which would show number of publications in PubMed by year for animal studies
Create a graph which would show number of publications in PubMed by year for human studies
Create a graph which would show number of publications in PubMed by year for in vitro studies
Create a graph which would show how many human studies involved human embryonic stem cells, adult stem cells, and umbilical stem cells.
Create a graph which would show how many animal and in vitro studies involved human embryonic stem cells, adult stem cells, and umbilical stem cells.
How many of the identified studies are currently recruiting patients?
|Section 4: Patient demand for stem cell therapies|
Lecture 7 addresses main challenges in stem cell treatments such as regulatory uncertainty, advertising strategies, generating information on safety and efficacy of stem cell treatments, and the need to maintain patients’ autonomy.
Countries have taken different approaches to the regulation of stem cell research and therapies, and enforcement of existing regulations differs. As a result, patients who are willing and able to travel from one country to another can access treatments that are not available in their home countries.
The lecture contains links to external resources useful for understanding of the complex environment of stem cells research and therapy.
|Section 5: Regulatory and legal framework|
Stem cell therapies are regulated differently in various countries around the world, with some countries offering stem cell therapies that are not available elsewhere. In this section, we will discuss in detail regulations that govern stem cell research and therapies in the U.S. and in the European Union.
Policies on stem cell research are driven by ethical concerns relating to research that utilizes human embryos. Lot of attention is paid to research on embryos because of all the controversies involved. The total number of clinical trials involving embryonic stem cells is very low, compared to adult stem cell trials. Lot of hESC research is conducted in laboratories and on animals in non-clinical and pre-clinical studies that do not involve humans.
China recently announced new ethical guidelines and new rules for their stem cell clinics, regulating both trials and treatments.
A quiz in the end of this section shall be best approached with all the relevant regulations at hand. The intent is to make sure you can pick the right regulation or document and find the relevant piece of information. Allow yourself plenty of time to find the correct answer.
Lecture 9 discusses the history and development of U.S. policy from the Dickey-Wicker Amendment of 1996 to this day, covering the 2000 NIH guidelines for research using human pluripotent stem cells; the 2001 Presidential statement, which prohibited federal funding of most human embryonic stem cell research; two attempts to pass the "Stem Cell Research Enhancement Act" (2005 and 2007); Executive Order 13435 (2007); Executive Order 13505 "Removing Barriers to Responsible Scientific Research Involving Human Stem Cells" (2009), and Sherley v. Sebelius.
Lecture 10 covers legal development and the current regulatory landscape in stem cell research and therapies in the U.S. Final rule, codified as 21 CFR 1271, Title 21 of the Code of Federal Regulations, Part 1271, known as the Current Good Tissue Practices (CGTPs), is discussed in detail sufficient for patients to understand, which areas of stem cell research are regulated, and how.
Human cells, with the exception of those deemed minimally manipulated and used for autologous treatments, would be subject to FDA premarket approval as biologics.
Lecture 11 introduces Article 11 of the Universal Declaration on the Human Genome and Human Rights (UNESCO) and report The Use of Embryonic Stem Cells in Therapeutic Research by the International Bioethics Committee (IBC) of UNESCO.
Lecture 12 is dedicated to regulatory and legal framework and policy governing stem cell research in the European Union. Positions and recent rulings of the European Patent Office and the European Court of Justice are briefly discussed. The timeline presented here shows fierce debate and lack of consensus on the research on embryos in Europe. Positions of individual EU member states and implementation of EU rules vary widely from one country to another.
Directive 2004/23/EC sets standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells. Research and innovation in regenerative medicine are supported by the Biopatent Directive.
The lecture discusses authorization of advanced-therapy medicinal products (ATMPs), that is medicines for human use that are based on gene therapy, somatic-cell therapy or tissue engineering, and also presents the first stem-cell therapy recommended for approval in EU.
China is a popular destination for patients seeking stem cell treatments. New regulations and ethical guidelines have recently been published. Chinese state media claim that China is determined to rein in rogue use of stem cells in clinics while allowing research. The measures were announced on 21 August by China’s National Health and Family Planning Commission.
This section includes a quiz. Use all materials available for download in this section to find the right answer before you submit the quiz for grading.
|Quiz 2||13 questions|
|Section 6: Professional societies|
Potential contributions of professional societies and other organizations include self-regulation through accreditation and certification, development of standards, and creation of a platform for collaboration among stakeholders. Key goals are to develop an appropriate terminology, define the levels of scientific evidence needed to justify routine use or commercialization of a cellular therapy, address questions of “experimental” and “innovative” use, and understand the global regulatory landscape in order to identify gaps and contradictions.
Thank you for participating in this course and congratulations on its completion!
Veronika Valdova is a professional with wide range of experience within pharmaceutical and applied veterinary and medical research, pharmaceutical safety, and risk management in related disciplines. As a co-owner of consultancy Arete-Zoe, her current endeavors include optimizing organizational effectiveness to make business agile, competitive and profitable, while intrinsically safe, secure, and resilient.