Survival guide to stem cell research and therapies

Survival guide to stem cell research and therapies
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1334 E Chandler Blvd 5A-19
Phoenix 85048, AZ, USA
T: +1-480-409-0778
COURSE SYLLABUS
Course Title: Survival guide to stem cell research and therapies
Instructor: Arete-Zoe
Delivery method: Indirect (Internet)
Length: 1.5 hours of video content / 15 lectures
Estimated study time: 2-4 days
CPE credits: n/a
Catalog Description:
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.

BROAD COMPETENCIES AND TERMINAL OBJECTIVES:
 Find and interpret information from publicly available sources and make better treatment
decisions.
 Appreciate the unique characteristics of different types of stem cells and their typical use in
research and therapy.
 Understand the difference between research and therapy
 Find and interpret relevant information on stem cell research in two main clinical research
registries - ClinicalTrials.gov and ICTRP
 Export/download and collate data from trial registries, and interpret the findings in context of
other sources such as scientific libraries and regulatory data.
 Locate and assess relevant information in other sources, such as the NIH registry and the FDA
registry.
 Find relevant published literature in PubMed and interpret the findings.
 Relate to patient motivations to seek unproven stem cell therapies off shore.
 Understand the main guiding principles of stem cells research and therapies in the U.S. and in
Europe.
 Cite the most relevant regulations and guidelines governing stem cell research and therapies in
the U.S. and in Europe, and explain the nature and history of controversies and restrictions
relating to embryonic stem cells.
 Understand the nature of changes of regulatory environment in China.
 Understand the role of professional societies in the regulation and self-regulation of stem cell
research and therapies.
Target audience:
 Patients and their families and friends who need to find more information about stem cells, who
wish to learn how to find relevant information online, and how to work with publicly available
resources such as databases and registries, to be able to make informed decisions for
themselves.
 The course can also be useful as a primer for those who need to learn the essentials about stem
cells as part of their professional training.
 The course is not intended for medical professionals
Course Material List:
Required: Students have to be able to follow moderately complex instruction and
scientific and legal texts with possibly unfamiliar terminology.
Assigned reading as listed
Recommended: Suggested readings expand on the topic and provide context and depth of
understanding of the topic. These materials are not essential for completion of
the course.
Required software: Access to a computer with office software, such as MS Excel or its equivalent.

COURSE BREAKDOWN
Contents
COURSE SYLLABUS................................................................................................................................... 1
BROAD COMPETENCIES AND TERMINAL OBJECTIVES:.............................................................................. 2
COURSE BREAKDOWN ............................................................................................................................. 3
SECTION 1: INTRODUCTION ..................................................................................................................... 4
Lecture 1 - Introduction ....................................................................................................................... 4
Lecture 2 - The Potential of Stem Cell Treatments................................................................................ 4
SECTION 2: CLASSIFICATION..................................................................................................................... 4
Lecture 3 - Classification of stem cells .................................................................................................. 4
SECTION 3: CLINICAL TRIALS..................................................................................................................... 5
Lecture 4 - Stem cell clinical trials involving humans around the world................................................. 5
Lecture 5 - Analysis of stem cell trials available in the Clinical Trials.gov registry .................................. 6
Lecture 6 - Stem cell research available in the ICTRP platform and published literature........................ 6
SECTION 4: PATIENT DEMAND FOR STEM CELL THERAPIES....................................................................... 7
Lecture 7 - Patient demand.................................................................................................................. 7
SECTION 5: REGULATORY AND LEGAL FRAMEWORK ................................................................................ 7
Lecture 8 - Stem cells regulations overview.......................................................................................... 7
Lecture 9 - U.S. policy........................................................................................................................... 8
Lecture 10 - U.S. regulatory and legal framework governing stem cells ................................................ 8
Lecture 11 - International law .............................................................................................................. 9
Lecture 12 - European stem cell regulations and policy ........................................................................ 9
Lecture 13 - Stem cell regulation in China............................................................................................. 9
SECTION 6: PROFESSIONAL SOCIETIES.................................................................................................... 10
Lecture 14 - Professional societies...................................................................................................... 10

SECTION 1: INTRODUCTION
Learning objective: Understand the ethical standards that govern the use of unproven treatments in
clinical practice, and appreciate the difference between preclinical and clinical research and therapeutic
intervention.
Introduction
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.
Resources:
 Declaration of Helsinki
The Potential of Stem Cell Treatments
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.
Resources:
 NIH: Stem cells basics
 Survey
SECTION 2: CLASSIFICATION
Learning objective: Learn essential terminology in stem cell research and classification of stem cells by
their origin and ability to differentiate.
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 course materials.

Resources:
 Adult mesenchymal stem cells
 Visual Stem cell glossary at all things stem cell
 Boston children's hospital: About stem cells
Quiz
SECTION 3: CLINICAL TRIALS
Learning objective: Find and interpret relevant information on stem cell research from two main clinical
research registries - ClinicalTrials.gov and ICTRP, and complement the datasets with published literature.
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 sources of funding.
Information from clinical trial registries shall be complemented by whatever has been published on the
topic in scientific literature.
Stem cell clinical trials involving humans around the world
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.
Resources:
 NIH database ClinicalTrials.gov
 WHO platform ICTRP
 PubMed
 NIH grants and funding - human embryonic stem cells registry

Analysis of stem cell trials available in the Clinical Trials.gov registry
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.
Stem cell research available in the ICTRP platform and published literature
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.
Clinical trials: project
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?

Resources:
 Pubmed search
 Pubmed advanced search
 ClinicalTrials by condition
 FDA approvals
 Biologics products and establishments
SECTION 4: PATIENT DEMAND FOR STEM CELL THERAPIES
Learning objectives: Relate to patient motivations to seek unproven stem cell therapies off shore, and
understand the challenges relating to marketing claims, objective outcomes, and patient autonomy.
Patient demand
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.
Resources:
 Stem cell therapies
 Stem cell fraud: 60 minutes investigation
SECTION 5: REGULATORY AND LEGAL FRAMEWORK
Learning objective: Understand the main guiding principles of stem cells research and therapies in the
U.S. and in Europe, and cite the most relevant regulations and guidelines governing stem cell research
and therapies.
Stem cells regulations overview
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.
Resources:
 Prochymal
 Radiation and nuclear countermeasures program
 Osiris
 Mesenchymal stem cell therapy for acute radiation syndrome
Quiz
U.S. policy
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.
Resources:
 2001 policy
 Bush memorandum
U.S. regulatory and legal framework governing stem cells
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.
Resources:
 Sherley vs. Sebelius, appeal 2012
 Sherley vs. Sebelius, 2011
 Sherley vs. Sebelius, petition for certiorari denied 2013
 S.5 - Stem Cell Research Enhancement Act of 2007
 H.R.810 - Stem Cell Research Enhancement Act of 2005
 H.R.2880 - Balanced Budget Downpayment Act, I
 EO 13435
 National Institutes of Health Guidelines on Human Stem Cell Research
 Proposed approach, 1997

 Part 1270 - Current Good Tissue Practice
 EO 13505
 Human cell and tissue establishment registration
 Title 21 CFR Chapter 9 - §355 New drugs
International law
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.
Resources:
 Universal Declaration on the Human Genome and Human Rights
 Selected National and International Laws and Regulations
 InfoCuria: Oliver Brüstle v Greenpeace eV.
European stem cell regulations and policy
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.
Resources:
 EuroStemCell
 The European Patent Convention
 The EU Parliament adopted a Resolution on voluntary and unpaid donation of tissues and cells (2012)
 European Science Foundation
 Stem cell research and patenting
 EuroStemCell: Stem cell patents and the European Court of Justice: news coverage
Stem cell regulation in China
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.
Resources:
 China announces stem-cell rules
 Health authority announces step to rein in 'wild' stem cell treatment
SECTION 6: PROFESSIONAL SOCIETIES
Learning objective: Understand the role of professional societies in the establishment of professional
standards and communication with patients.
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.
Resources:
 International Society for Cellular Therapy
 International Society for stem cell research

Survival guide to stem cell research and therapies

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