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