The document discusses achievements and future trends in vitiligo research by the Vitiligo Research Foundation. It summarizes the Foundation's approach of understanding repigmentation through collecting clinical data and biosamples, open-source data sharing, and disseminating knowledge. It outlines current vitiligo treatments including phototherapy and experimental options. It also discusses the Foundation's work developing an electronic health record, vitiligo biobank, and data analysis to help accelerate drug development for vitiligo. Emerging trends highlighted include increased patient self-treatment options using home phototherapy and nutraceuticals.
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Vitiligo Research Foundation Achievements and Future Trends
1. Vitiligo Research Foundation:Vitiligo Research Foundation:
Achievements,Achievements,
Present, Future TrendsPresent, Future Trends
Yan Valle
CEO VR Foundation
2. I. Our approach
II. Vitiligo treatments
III. Vitiligo drug development
IV. Electronic health record
V. Vitiligo biobank
VI. Data analysis
VII. Future trends
What’s ahead
4. Mission
“to accelerate the end of suffering
for millions of people with vitiligo”
We support, we educate, we care.
Open source research concept.
Vitiligo Research Foundation
501(c)3 Non-profit organization
Consultative member of the UN ECOSOC
6. VRF Approach:
Seeking for novel treatments
Identify key
molecular targets in
pathogenesis
Identify key
molecular targets in
pathogenesis
Identify key
molecular targets of
existing treatments
Identify key
molecular targets of
existing treatments
Existing drugs
for repurposing
Existing drugs
for repurposing
Search for
existing
solutions
Search for
existing
solutions
Evaluate safety
& feasibility
Evaluate safety
& feasibility
Drugs under
development
for other
indications
Drugs under
development
for other
indicationsOriginal ideas
for
development
Original ideas
for
development
Identify vitiligo-
related side-effects of
other drugs
Identify vitiligo-
related side-effects of
other drugs
7. VRF Approach:
Collecting clinical data
Vitiligo CloudBank:
First Vitiligo-specific
Electronic Patient Record
& Registry of Biosamples
10. In 2011-2016: Vitiligo Master Classes were held in
Barcelona, St. Petersburg, Kitzbuhel, Sao Paolo, Shenyang,
Almaty, Amritsar, Istanbul, Split, Tehran, Shymkent, Sofia,
Batumi, Tel-Aviv, New York, Hyderabad, Moscow, Manaus
VRF Approach:
Disseminating knowledge
12. Debate: Vitiligo or Vitiligos?
Classification of vitiligo: a challenging endeavour.
Hercogová J, Schwartz RA, Lotti TM.
Dermatol Ther. 2012 Nov;25 Suppl 1:S10-6. doi:
10.1111/dth.12010.
13. “Vitiligo is a spectrum of under-investigated
diseases with different clinical presentations,
unknown etiology, fragmented genetic data
and pathogenetic hypothesis.”
The phenotypic manifestation
is NOT a disease!
Vitiligo defined:
14. > No vitiligo-specific drugs (US/EU)
12 pharma companies launched internal R&D, but
nothing in the pipeline in late phase of development
> New generics can stop progression
Not available everywhere
> Average remission period is 4-7 years
No relapse prevention (?)
> Phototherapy remains ‘a golden standard’
Home-based option emerge as equal to clinic-based
> Experimental treatments emerge
Aspirin; Low-dose cytokines; Plant extracts;
Needling to induce wound-healing response
> Cosmeceuticals and Nutraceuticals fill the gap
> Camouflage preserves high Quality of Life
Vitiligo in 2016
15. 1. Stabilise
2. Repigment
3. Prevent recurrencies
4. Provide ongoing support
5. Educate community & public
Vitiligo: a correct approach
16. 1. Vitiligo Stabilization
Treatment Efficacy Comments
PUVA 40% Extended treatment
Side-effects
NB-UVB 70% Extended treatment
Steroids (OMP) 85%-90% Side-effects
Gingko biloba 80%-100% For slowly progressing
Minocycline 76%-90% Antibiotic
Levamisole 83%-94% Serious side effects
Leflunomide 90% Serious side effects
Aspirin 100% Moderate term (12 weeks)
Risk of side-effects
Neovir 73.3%
96.4%
Short term (9-19 days)
NO side-effects
Immunomax 72% Short term (9-19 days)
NO side-effects
17. Stabilization: experimental systemic treatments
Gingko Biloba
Anti-inflammatory, immunomodulatory, antioxidative
mode of action.
80% (N=25) vs placebo 36% (N=22)
40 mg 3x/day per os, 12 weeks
100% (N=11)
40 mg 2x/day per os, 12 weeks
Aspirin
Pro-melagenic, reducing level of anti-inflammatory
cytokines in blood.
100% (N=16) vs placebo
300 mg 1x/day per os, 12 weeks
18. Stabilization: experimental systemic treatments
Minocycline
Anti-infammatory, immunomodulatory, antioxidative
mode of action. Melanocyte protection from oxidative
stress.
90% (N=32)
100 mg 1x/day per os, 4 weeks
76% (N=25) vs 88% (N=25, dexametasone OMP)
100 mg 1x/day per os, 6 months (PMID: 24448120)
Low-dose oral cytokines
Low-dose IL-4, IL-10, bFGF and β-endorphin represent
a novel therapeutic option for vitiligo.
19. Stabilization: experimental systemic treatments
Neovir (available only in Russia, Ukraine and Kazakhstan)
Immunomodulator and corrector of immunophenotype of
peripheral blood
73.3% (N=60); 96.4% (N=57)
250 mg 1x/day every 2nd
day, 10 intramuscular injections
20. Afamelanotide
Analog for a-MSH
Clinical trials completed
Can fasten re-
pigmentation (+NB-UVB)
Expensive
Tretinoin
Vitamin A
Contradictory data on
melanogenic effect
Reduces atrophy during
steroid therapy
Prostoglandin E2
Melanogenic factor
Immonumodulating activity
Effective on focal and
segmental vitiligo, side effects
reported
Experimental local treatments
21. Steroids vs Calcineurine Inhibitors
• Tacrolimus is more effective when applied 2x/day (13% -> 40%)
• The combination of NB-UVB and tacrolimus is no more effective
than NB-UVB alone -- but Tacrolimus plus Excimer laser is more
effective than Excimer laser alone
• Occlusion potentiates Tacrolimus effect (40% -> 81% face & neck,
0% -> 80% limbs)
• Tacrolimus is more effective with stable vitiligo
• Tacrolimus and steroids demonstrate similar efficacy
• Tacrolimus (0.1%) is more potent than pimecrolimus (1%)
• Pimecrolimus is less effective than mometazone (42% vs 65%),
equally effective with clobetasol
22. Whole-body Focused
NB-UVB 311 nm
Requires 9-18 months, 2-3 times per week.
Achieves avg. 80% stabilization with 43% - 72% repigmentation efficacy.
Poor efficacy on acral areas and segmental vitiligo.
Home-based phototherapy is nearly equal to clinic-based in effectiveness.
2. Vitiligo Re-pigmentation
Local
23. Excimer laser & light 308 nm
•Requires 2 sessions per week.
•Efficacy (re-pigmentation >50%) 50% - 86%
•Acral areas respond in 76% cases, but with low
re-pigmentation.
•Best for segmental vitiligo: 44% patients
achieve >50% re-pigmentation.
•Sligthly more efficient vs NB-UVB at a higher
price.
Vitiligo Re-pigmentation
24. Photocil ® (USA)
Vitiligo Re-pigmentation
Selective phototherapy
using lotion and sunlight
- Passes approximately
280 mJ/cm2 NB- UVB
(peak 311nm) in 90
minutes sunlight exposure
- Filters 95% of harmful
UVA
- Filters 92% of harmful
UVB
- FDA registered, soon will
be available in the EU
25. Basic Fibroblast Growth Factor (bFGF)
• Derived from keratinocytes.
• bFGF is a polypeptide which has 146 amino acid residues in
the complete primary structure.
• The active fragment is decapeptide containing 10 amino
acids.
Capable of:
• producing wide variety of cells including melanocytes,
keratinocytes, fibroblasts
• formation of new blood vessels
• multiplying melanocytes from hair follicles surrounding
the affected skin and
• also acts as a chemotactic agent to direct the new
melanocytes to the vitiligo patch.
Vitiligo Re-pigmentation
27. Skin trauma as monotherapy?
•Dermabrasion
Microdermabrasion potentiates
effect of Pimecrolimus (42% -> 65%)
•Laser ablasion (СO2- и Erbium: YAG-
lasers)
•Needling (Dermaroller, ACS Pen etc.)
28. A new era of surgical management of vitiligo
by Prof. Davinder Parsad
29. Under-investigated area, promising results:
• Low-dose UVB phototherapy (including home
treatment lamps )
• The Dead Sea heliotherapy
• Nutraceuticlas and food supplements
• Neovir ® as prophylactics
3. Prevent Recurrence
30. Polipodium Leukotomas
Vitiligo Supplements
• Immunomodulating and photoprotective
properties of plant extract
• Traditionally used in Central and South
America
• Positive use experience in Brazil
• Effective in combination with NB-UVB:
44% vs 27% (face and neck)
31. Camouflage, among may options:
ZANDERM (USA)
•DHA-based
•12 shades
•Stays for 3-4 days
•Non-marking, mess-free
•Convenient
4. Provide Ongoing Support
32. • Monthly newsletters
• Vitiligo Q&A (7 languages)
• Ebooks:
- 10 Vitiligo Treatment Protocols
- No-Nonsense Guide To Vitiligo
(publication in December 2016)
Send your request for free copy via email
5. Educate Community & Public
35. Drug Development
Expensive:
> 1,000+ Bio-samples
> 10,000+ Patients
> 10+ years
> and between
$100,000,000 and
$10,000,000,000
Why there is no drug for vitiligo?
Poor understanding of:
> Disease pathogenesis
> Disease models
> Immune and other
cells involved
> Target delivery
systems
46. What is a Biobank?What is a Biobank?
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
48. Biospecimens in Vitiligo BioBankBiospecimens in Vitiligo BioBank
Serum
A variety of parameters can be
extracted (from cytokines/GF,
metabolites to microRNA)
Common clinical use
Routine collection procedure
Time-course or drug response
analysis opportunity
Usually collected along with
other blood tests, with high
patient tolerance
Blood
Can be used in genetic
research, present and future
No special intervention
required if collected along with
serum
Hair bulbs
Limited use in genetic research
No special requirements for
collection and storage
49. Why do we need aWhy do we need a
Vitiligo Biobank?Vitiligo Biobank?
To empower vitiligo researchers with biospecimens,
collected and annotated by standardized procedures
To enable investigation of unseen correlations
between known parameters
To identify factors previously not linked to the
disease
To efficiently enroll patients into clinical studies
(There are already 350+ biobanks in the US and EU)
50. Emerging Trend: Virtual BiobanksEmerging Trend: Virtual Biobanks
A targeted collection of high-value samples along
with a disease-specific dataset.
Both case and control samples are readily
available in statistically meaningful quantity.
Some available biological information (typically,
DNA) is extracted a-priori from specimens.
Epidemiological and demographic data is
embedded with the dataset.
52. Virtual Biobanking Tips:
1. Create a continually updated inventory of
biospecimens that can be used as your research tool and
marketing materials for the academia and
pharmaceutical industry.
2. Create statistically meaningful case-control sample
sets (i.e. patient/control or responders/non-responders).
3. Consider pre-processing of specimens so that the time
to results may be shortened, specifically with regard to
DNA extraction.
55. Work in this direction will help use large amount of existing data. However, we
need to resolve the noise-signal aspect in these studies.
Social networks: unstructured data
57. Pilot research project on
1,000+ patient records data set
from Vitiligo CloudBank conducted
by Dr. Lipi Thurkal and colleagues at the
Institute of Genomics and Integrative Biology
in New Delhi (India)
58.
59.
60. Vitiligo vulgaris (63%) was the
most common type of vitiligo
among the patients
Occurrence and Progression
The disease progressed
slowly in 86% of the
patients
61.
62.
63.
64.
65.
66. Distribution of affected area
The most affected area is lower
extremities, followed by upper
extremities
Location
Percentage
occurrence
(Female)
Percentage
occurrence
(Male)
Pearson
Chi-
Square (p
value)
Scalp 18.2% 6.7% .088
Face &
Neck
49.1% 44.4% .643
Chest &
Abdomen
60.0% 37.8% .027
Buttock 45.5% 20.0% .018
Upper
extremities
65.5% 48.9% .095
Lower
extremities
83.6% 71.1% .133
Genitals 12.7% 15.6% .685
Location v/s sex
Patch Distribution
69. • Patient self-education, self-prescription and self-treatment.
Always ask patient what other substances they use to avoid
cross-effects (such as vitamins / antibiotics).
• Home phototherapy treatment
Works as good as clinic-based, for 1/3 cost and more
convenient. Requires supervision by dermatologist.
• Nutraceuticals and food supplements
Probably, Polipodium Leukotomas and Ginko Biloba work --
but beware of fake products. Nano-products may be an
effective option for 10X price.
• Camouflage
Vitiligo Trends for 2017
Networking is a must for vitiligo – multifactorial, poor ways of patient stratification, lengthy treatments to assess efficiency
In both the sexes, lower part of the body was the most common affected area.
Among females, chest& abdomen and buttock shows a higher incidence than males
The overall picture is that the non-profit VR Foundation today possesses resources, great competence and a deep commitment to biobank-based research; and that it is these assets are the base for future joint efforts in expedited vitiligo therapy development.