Post on 07-May-2015
description
Golden Standard in Biobanking for Vitiligo
and Rare Diseases
Igor V. Korobko, VR Founda0on Chief Scien0fic Director
What is a biobank for a specific disease? “... biobanks are defined as collections of samples of human substances (e.g. cells, blood, tissue or DNA) that are or can be associated with personal data and information on their donors. Biobanks have a twofold character, as collections of both samples and data”. European Medicines Agency. Draft concept paper on the development of a guideline on biobanks issues relevant to pharmacogenetics
Why there is a need for biobanks for rare and neglected diseases?
• Samples for lab research
• Clinical data analysis “… to establish new patient stratification principles and for revealing unknown disease correlations”
Jensen et al., Nature Review Genetics (2012) 13: 395-405
• Irrelevant factors affecting the disease
Why there is a need for biobanks for rare and neglected diseases?
• Lengthy biosample collection
• Lack of standards in data and sample collection
• Networking problems
What biobank is needed for? ü to fuel research with well-described biosample
collection
ü to enable finding overseen correlations between clinical parameters
ü to identify factors (inducers, treatments) previously not linked to the disease
ü to efficiently enroll patients into clinical studies
and more ...
Good rare/neglected disease biobank ingredients: a recipe
ü Standard protocol for data collection ü Standard protocol for biosample
collection ü Networking ü Common database (make available for
researchers, attach search engines and analytical tools)
ü Ethical concerns ü Motivation for physicians ü Funding
Vitiligo Biobank: implementation of the concept for a neglected disease
Vitiligo Biobank is an initiative launched, supported and operated by the VR Foundation
Expecting impact of Vitiligo biobank on vitiligo management
• Vitiligo is a complex multifactorial disease with diverse triggers and mechanisms of pathogenesis.
• Despite being one of the most common skin diseases, owing to its non-life threatening nature, vitiligo is truly neglected disease.
• Existing treatments have limited efficiency.
The need for research of vitiligo to understand mechanisms of pathogenesis, to develop novel treatment modalities, to offer personalized treatment options to patients.
Expecting impact of Vitiligo biobank on vitiligo management
Biobank would promote and speed up reaching these goals through:
ü expediting experimental (both lab & clinical) research
ü revealing overseen correlations between clinical parameters
ü repurposing existing treatments & identifying unconventional ways of disease management
Expecting impact of Vitiligo biobank on vitiligo management
... But not only through enabling access to data and biosamples
Truly collaborative project between researchers, clinicians and patients, in pursuit for vitiligo therapy development
Vitiligo biobank: data collection Standard protocols for data collection
ü takes into account all possibly informative parameters yet excluded those which are not important to save time at data collection
ü is compiled based on numerous physician's opinions and analytical analysis of revealed or possible correlation
ü is built based on tabulated data, an important issue for subsequent analysis
Vitiligo biobank today: data collection Patient Biobank Record forms enveloping:
ü maximally tabulated data collection; ü range of parameters carefully defined for all essential events to be
recorded; and designed for:
ü primary visits; ü follow-up visits; ü specific supplementary forms for transplantation procedures.
Guideline for data collection ü to ensure quality and uniformity of the collected data, dataset
labeling & ethical concern compliance.
Vitiligo biobank: sample collection Standard protocols and guidelines for biosample
collection depending on the sample type
Types of collected samples Criteria for sample type selection:
ü patient's compliance, ü usability in research for understanding of
pathomechanism, ü usability as biosamples for routine clinical tests, ü suitability for ready-to-go sample collection, ü cost of collection, ü accessibility to proper storage conditions.
Vitiligo biobank today: sample collection Serum
ü reading of versatile parameters (from cytokines/GF, metabolites to microRNA)
ü easy to admit for clinical use ü routine collection procedure ü time-course/response analysis
opportunity ü collected along with other blood
tests thus having high patient tolerance
Blood ü genetic research, present and
future ü no special intervention if
collected along with serum
Hair bulbs
ü limited genetic research capability
ü no special requirements for collection and storage
Vitiligo biobank: ethical concerns Vitiligo bioblank implicates policy of strict adherence to all applicable ethical concerns
and requirements
Vitiligo biobank: networking Multiple sites for biosample and data collection
enables robust growth of database
Implemented concept:
ü local storage of biosamples, centralized storage of
data
ü biosamples are available for research on demand
ü availability is either physical or virtual
Vitiligo biobank: networking Benefits of the implemented concept:
ü no need for sample transportation so expenses are cut
ü no conflict with local legal issues
ü increase in biobank analytical power
ü broadening geography of vitiligo research
ü promoting local vitiligo research projects through the use of local biobank opportunities
Vitiligo biobank today: networking Six locations worldwide operating now, several to
join shortly
Vitiligo biobank: data recording, storage and management
Integrated solution for data recording, collection and storage
ü data entry and analysis
ü datasets selection based on pre-set parameters (virtual patient enrollment)
ü biosample availability information
ü ensuring high compliance of the physician
Database and more...
Cloudy: a system providing capabilities for…
ü data storage in searchable and analyzable format ü dynamic database field updates ü entering and storage of non-tabulated data such as
text and images ü network access (with later local versions
synchronized with database to be released) ... and more for physician & patient!
Vitiligo biobank: data recording, storage and management
Cloudy system:
merging data collection (research area) with capabilities for
easy and robust tracing of patient's disease conditions and treatment response
Biobank: attracting physicians
Biobank: attracting physicians
Features:
ü Electronic medical record tuned up for vitiligo
ü Visit scheduling
ü One-click frequently used treatment protocol addition
ü Visualization of selected parameters in a time scale
Vitiligo biobank today: overview Six locations worldwide operating now, several to join shortly
More than 650 datasets were acquired since the beginning of operation in July 2012
Cloudy smart interface and data collection electronic system to be released soon
Welcome to join Vitiligo biobank :
in networking, research projects, funding
Thank you for your attention !
www.vrfounda/on.org
Web: vi/ligobiobank.org Email: biobank@vrfounda/on.org