Stem cell therapies - a special case of therapy and research

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A S P E C I AL C AS E O F T H E R AP Y & R E S E AR C H
STEM CELL THERAPIES

CONTENT
• I. POTENTIAL
• II. CLASSIFICATION
• III. CLINICAL RESEARCH
• IV. PATIENT DEMAND
• V. REGULATORY AND LEGAL FRAMEW...

I. STEM CELLS POTENTIAL
BASIC RESEARCH – DRUG TESTING - THERAPIES

• Basic research
• Cell division and differentiation
• Genetic and molecular controls
• Testing new drugs
• Safety testing...

©2008 Terese Winslow

Source: NIH
• Proliferate extensively and generate sufficient quantities of
cells for making tissue.
• Differentiate into ...

II. CLASSIFICATION
DIFFERENTIATION POTENTIAL & ORIGIN

• TOTIPOTENT
• Embryonic, first 4 days
• Most versatile, can form the
whole body
• PLURIPOTENT
• Embryonic, after 4 days
•...

Morula
Blastocyst
Inner Cell Mass (ESCs)
Epiblasts (ESCs)
ENDODERM MESODERM ECTODERM
TotipotentPluripotentMultipotentUnipo...

ENDODERM MESODERM ECTODERM
MultipotentUnipotent
ENDODERM
LUNG PANCREAS LIVER GIT UGT
Pulmonary
Epithelial SCs
Pancreatic
S...

ENDODERM
MultipotentUnipotent
ENDODERM
KIDNEY BONE & BONE MARROW
BLOOD
VESSELS
HEART
Kidney Hematopoietic
SCs
Leukocytes
E...

Bonfield, TL: Adult Mesenchymal Stem Cells: An Innovative Therapeutic for Lung Diseases (Discovery Medicine, 2010)

ENDODERM
MultipotentUnipotent
ENDODERM
NERVOUS SYSTEM SKIN
Ocular SCs Neural SCs
Neurons
Astrocytes
Oligodendrocytes
Skin ...

EMBRYONIC STEM CELLS
M O U S E E M B R Y O N I C S T E M C E L L S F O R M I N G S T E M C E L L C O L O N I E S
O N A F I...

ADULT STEM CELLS
A D U L T S T E M C E L L S C A N B E E X T R A C T E D F R O M M A N Y A R E A S O F T H E
B O D Y , I N...

III. CLINICAL RESEARCH
I N D I A , C H I N A , M E X I C O , U S A , E U
S U B S T A N T I A T E D C L A I M S O F T E N A...

CLINICALTRIALS.GOV
ALL STEM CELL TRIALS
Total: 5,101 studies
Europe: 1,157 studies
Poland: 328
Germany: 287
Italy: 239
UK:...

CLINICALTRIALS.GOV
ALL STEM CELL TRIALS
Approved for marketing: 1
Available: 12
Recruiting: 1,702
Enrolling by invitation:...

CLINICALTRIALS.GOV
ALL STEM CELL TRIALS
2627
750
494
456
320 88
Other
Other + NIH
Industry
Other + Industry
NIH
Combinatio...

CLINICALTRIALS.GOV
ALL STEM CELL TRIALS
Bacterial and Fungal Diseases
Behaviors and Mental Disorders
Blood and Lymph Condi...

CLINICALTRIALS.GOV
37 EMBRYONIC STEM CELL TRIALS

CLINICALTRIALS.GOV
285 UMBILICAL STEM CELL TRIALS

CLINICALTRIALS.GOV
529 MESENCHYMAL STEM CELL TRIALS
Middle East: 47
Iran (30)
Europe: 123
Spain (49)

CLINICALTRIALS.GOV
4,968 ADULT STEM CELL TRIALS
Poland: 322
France: 276
Italy: 235
Spain: 195
UK: 190
Israel: 110
Iran: 51...

WHO REGISTRY ICTRP
5,401 RECORDS FOR 4,804 TRIALS

STEM CELLS IN PUBMED
233,458 RESULTS, OF WHICH 4,412 ARE CLINICAL TRIALS

IV. PATIENT DEMAND
COMMUNICATION OF BENEFITS AND RISKS

CHALLENGES
IN RESEARCH AND THERAPY INVOLVING STEM CELLS
The best ways to regulate stem cell therapies remain unclear. Stem...

KNOWNS AND UNKNOWNS
NAP: Stem cell therapies (Nov 2014)
• Objective outcome measures would be useful for assessing the
ef...

• Patients
• Difficult to estimate true numbers of
patients seeking stem cell
treatments in the U.S. and overseas
• Main d...

I N N O V A T I V E S U R G E R Y I S G O V E R N E D B Y T H E C O M M O N R U L E W H I C H
I M P O S E S R E S T R I C ...

IV. STEM CELLS AS REGUATED
THERAPEUTICS

APPROVED STEM CELL TREATMENTS
ARE FEW AND FAR BETWEEN

APPROVED STEM CELL TREATMENTS
ARE FEW AND FAR BETWEEN

REGULATORY AND LEGAL FRAMEWORKS
Stem cell therapies are regulated as biologics and are subject to premarket approval
under...

1995 2000 2005 2010 2015
U.S. POLICY
REGARDING STEM CELL RESEARCH
1996 The Dickey-
Wicker Amendment
2000 NIH guidelines fo...

U.S. POLICY
REGARDING STEM CELL RESEARCH
Executive Order 13505
Removing Barriers to Responsible
Scientific Research Involv...

UNITED STATES
REGULATORY AND LEGAL FRAMEWORKS
1995 2000 2005 2010 2015
1997
Proposed
Approach to
Regulation of
Cellular an...

21 CFR 1271: CGTP
GENERAL PROVISIONS
PURPOSE and SCOPE:
• Unified registration and listing system
• Manufacturing establis...

21 CFR 1271: CGTP
GENERAL PROVISIONS
HCT/P’s
Articles containing or consisting of
human cells or tissues that are
intended...

HCT/P is regulated solely under the Section 361 of the PHS Act if:
(1) The HCT/P is minimally manipulated;
(2) The HCT/P i...

A
21 CFR 1271: CGTP
GENERAL PROVISIONS
EXCEPTIONS for REGISTRATION:
HCT/P’s solely
for nonclinical
scientific or
education...

REGISTRATION:
• Register establishment
• Submit a list of every HCT/P that the
establishment manufactures
• Submit any cha...

B
PUBLIC RECORDS
Filled 3356 Forms will be available
for public inspection
• A list of all HCT/P's
• A list of all HCT/P's...

C
Donor-eligibility determination required:
• Manufacturer must screen and test donors for eligibility
• Concern: relevant...

DONOR SCREENING
All donors
• Risk factors and clinical evidence of
HIV, hepatitis B and C, CJD, syphilis
• Xenotransplanta...

C
21 CFR 1271: CGTP
DONOR ELIGIBILITY
INELIGIBLE DONORS’ HCT/PS CAN BE USED:
• For a close blood relative
• Reproductive c...

C
ESTABLISH AND MAINTAIN PROCEDURES
Design, Define, Document, Implement,
Follow, Review and Revise
• All steps in testing,...

Regulations more specifically applicable will supersede the more general.
21 CFR 1271: CGTP
CURRENT GOOD TISSUE PRACTICE
S...

CGTP governs the methods used in, and the
facilities and controls used for, the
manufacture of HCT/Ps including all steps ...

21 CFR 1271: CGTP
CURRENT GOOD
TISSUE PRACTICE
PERSONNEL:
Manufacturer must have sufficient, competent
and appropriately t...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ENVIRONMENTFACILITIES
EQUIPMENT
SUPPLIES
REAGENTS
PROCESSRECOVERY
LABELLING STORAGE
DONORSDISTRIBUTION
21 CFR 1271: CGTP
C...

ADVERSE REACTIONS
• Non-reproductive only
• Manufacturers must investigate and
follow-up any adverse reaction involving a
...

BIOLOGICAL PRODUCT DEVIATION REPORT
Manufacturers must report HCT/P deviations relating to the core CGTP
requirements with...

INTERNATIONAL LAW
“Practices which are contrary to human dignity, such as reproductive cloning of
human beings, shall not ...

EUROPE
RESEARCH ON EMBRYOS
2000 2005 2010 2015
2000: Opinion on
the Ethical Aspects
of Human Stem Cell
Research and Use
20...

EUROPE
RESEARCH ON EMBRYOS
The Biopatent
Directive (98/44/EC)
Human Tissues and Cells
Directive (2004/23/EC)
2013 The Euro...

EUROPE
RESEARCH ON EMBRYOS
• 63% fall into the first two categories
• Legal sources of human embryonic stem cells (hESCs) ...

EUROPE
RESEARCH ON EMBRYOS
• Human embryos excluded from patentability for industrial or
commercial purposes
• “Embryo” no...

EUROPE
RESEARCH ON EMBRYOS
VERY
PERMISSIVE
PERMISSIVE WITH
RESTRICTIONS
RESTRICTIVE BY
DEFAULT
VERY
RESTRICTIVE
UNLEGISLAT...

EUROPE
RESEARCH ON EMBRYOS
UK
Sweden
Belgium
Romania
Turkey
Lithuania
Germany
Italy
Slovakia
Croatia
Austria
Ireland
Polan...

STEM CELL RESEARCH IN EUROPE
ADVANCED-THERAPY MEDICINAL PRODUCTS
Advanced-therapy medicinal products (ATMPs) are medicines...

HOLOCLAR
APPROVED AS ATMP IN EUROPE
FIRST STEM-CELL THERAPY RECOMMENDED FOR
APPROVAL IN EU
The applicant is Chiesi Farmace...

STEM CELL RESEARCH IN EUROPE
ADVANCED-THERAPY MEDICINAL PRODUCTS

REGULATORY AND LEGAL
FRAMEWORKS IN CHINA
NEW REGULATIONS:
• Ethical guidelines and regulations for human embryonic stem ce...

IV. PROFESSIONAL SOCIETIES
THE INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH (ISSCR)
• the largest professional organizatio...

IV. PROFESSIONAL SOCIETIES
THE INTERNATIONAL SOCIETY FOR CELLULAR THERAPY (ISCT)
• The general scientific consensus is tha...

THANK YOU

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