Summary of outcomes resulting from past summits and overview of the current state of alopecia areata research.

1. Summary of preceding
Alopecia Areata research summits
Natasha Atanaskova Mesinkovska MD PhD
Alopecia Areata Research Summit 2018

3. Alopecia areata - 3,500 years and Counting
Kevin McElwee, PhD

4. Alopecia areata - 3,500 years and Counting
• Causes:
• Allergy
• Immunity
• Infectious
• nerves and stress
Kevin McElwee, PhD

6. ALOPECIA AREATA
RESEARCH SUMMIT
Forging the Future
FINAL PROGRAM
December 4-5, 2018
Alfred Lerner Hall, Columbia University

7. “Forging the future”
• 2018
• Since 2008 - DECADE
• 7th meeting

8. The history of Alopecia areata summit
The idea:
• Getting together interested scientist, clinicians and patients
1. Find treatment - cost effective
2. Advance research
3. Engage patients in therapeutic development

9. History of Alopecia areata summit: 2008

10. • The NAAF Registry!!!
• Patient database
• Repository of serum and DNA
• NIH-funded whole genome scan

11. Foster collaboration

12. 2010

14. NAAF BOARD of DIRECTORS

15. The timeline of AA symposium
• 2008 in Bethesda- Genetics and Immunology
• 2009 in Denver - Immunology, animal models, drugs used in other
diseases.
• 2010 in NYC- demand of new treatments by AA patients.
• What tools were needed to foster clinical research – tools, biomarkers,
outcome measures.

16. The timeline
• 2012 in Bethesda –
• consolidating the recently
uncovered mechanisms in AA
• targeted by modern therapies used
in other diseases
• Development of core uniform
protocol.

17. The timeline
• 2014 in Bethesda
• Larger meeting, 90 participants
• New drugs showing promising results in AA
• New technologies and directions
• Clinical trials
• 2016 in NYC
• Advanced genetics and immunology
• New animal models
• Epidemiology
• QOL
• translational research
• First Summit: Industry partners!

18. Nine industry representatives!
Session 5 – Industry Roundtable
152016 AA Research Summit
Nine industry representatives! Stakeholder support of new products for alopecia areata. Engaging the
patient voice in therapeutic development.
Company Speaker Title
Aclaris Therapeutics, Inc. Dr. Kimberley Forbes-McKean Senior Vice President, Drug Development
BiologicsMD, Inc. Mr. J. David Owens President & CEO
Concert Pharmaceuticals, Inc. Dr. Roger Tung Co-Founder, President & CEO
Gilead Sciences, Inc. Dr. Thomas O'Riordan Senior Director, Clinical Research
Incyte Corporation Dr. Richard L. Leff Group VP, Drug Development
Legacy Healthcare Mr. Saad Harti President
LEO Pharma Inc. Dr. Michael Sierra Vice President, LEO Science & Tech Hub
Pfizer, Inc. Dr. Elena Peeva
Executive Director, Inflammation &
Immunology Clinical Research
RXi Pharmaceuticals Corp Dr. Geert Cauwenbergh President & CEO

19. Basic
Science
Clinical
Trials
Patient
Focused
Regulatory

20. Basic Science
Clinical Trials
Patient
Focused
Pharmaceutical

21. Ralf Paus: Revising the Immune Privilege
Hypothesis
• New physiological role of gamma delta T cells
• Scout for stressed hair follicle signals
• Langerhans cells, Mast cells, among others.
• a step toward a truly ‘curative’ therapy
• would involve restoration of peripheral tolerance

22. Marta Bertolini

23. Michael Rendl: Niche Control of HF Formation
and Regeneration
• hair regeneration wave profiling
• Hair-GEL.net
• resource to catalogue hair genes
• Searchable database for candidate AA genes/antigens
• 14 different cell types in adult HF

24. Emma Guttman: Cytokine Targeted Therapy:
Lessons from AD and Other Diseases
• Atopic dermatitis (AD)
• Highest comorbidity for AA (38%).
• IL13 SNPS and Th2 cytokines
• Dupilumab – Atopic dermatitis and AA.
• Apremilast ?
• Ustekinumab: ? higher dose in AD than psoriasis.

25. John Harris: Understanding Parallels between
Vitiligo and AA

26. Amos Gilhar: Non-conventional T-cells in the
Pathogenesis of AA
• NKG2D expressed also on ILCs
• humanized mouse AA model
• iNKT cells in AA
• New inexpensive treatments: Alpha-GalCer

27. Amos Gilhar: Non-conventional T-cells in the
Pathogenesis of AA
• NKG2D expressed also on ILCs
• humanized mouse AA model
• iNKT cells in AA
• New inexpensive treatments: Alpha-GalCer

28. So much more…
• Annemieke de Jong: Identifying Pathogenic TCRs in AA
• Natasha Botchkareva: Role of miRNAs in AA
• Dan Kaplan: T cells, DCs Autoimmunity and the Hair Follicle
• Alessandro Sette: Epitope Identification Screening in AA
• Anastasia Khvorova: RNA Chemistry toward Modulation of Gene
Expression in Skin
• Tiffany Scharschmidt: Commensal Microbes and HF Morphogenesis
Drive Treg Migration into Skin
• Michael Rosenblum: Tregs in Skin and HF Stem Cell Differentiation

29. Basic Science
Clinical Trials
Patient
Focused
Pharmaceutical

30. Madeleine Duvic: Update on AA Registry
• 11,180 AA patients
• All subtypes of AA: AU 40%; AT 20%; AAP 40%
• All ethnic/racial groups represented.
• DNA, serum samples
• 6000 patients -not yet sampled
• re-contacted for participation clinical trials.
• Lynn Petukhova: Comorbidities in the AA Registry

31. George Cotsarelis: Nutritional Factors Influencing
Alopecia
• Optimum conditions for hair shaft formation
• Micronutrients
• Vitamin D, Zinc, Iron.
• Is telogen effluvium a trigger for AA?

32. Angela Christiano: Update on Genetics and
Immunology
• GWAS studies
• At least 14 genes involved in AA
• Environmental factors: microbiome

33. Christiano
2016

34. “YAK YAK YAK about JAK JAK JAK”
• Cleveland, Columbia, Duke, Stanford, Yale
• Response rates: consistent across sites (range 50-75%):
• Heterogeneous groups of patients:
• duration of disease
• dosing regimens
• length of treatment

35. JAK inhibitors clinical trials
• Cleveland: tofacitinib 54%
• Columbia: tofacitinib 65%, ruxolitinib 75%
• Duke/Incyte: topical JAK ~50%
• Stanford: tofacitinib 66%
• Yale: tofacitinib 60% adults, 75% adolescents

36. JAK Take Home Lessons: from Combined
Studies
• Relapse after treatment in all studies
• sometimes worse than baseline, 4-8 weeks after stopping
• Flares while on treatment
• 12% in Yale
• Ruxolitinib patients regrowth visible earlier than tofacitinib
• Tofacitinib required dose escalation, longer treatment periods.
• Responses with long duration of disease
• ? suggested that <10 years better responses
• Regional differences in regrowth: Eyebrows, lashes and body and facial hair
• Combo : pulse prednisone Q4 weeks for 3 doses (Yale) , ILK ( NYU).
• No reported improvement in AGA .
• AE: generally mild.
• Acne (8% Yale ),trace hematuria (Columbia), eczema herpeticum (Stanford); LFT, lipid
abnormality (Cleveland), URIs.

37. Terrific talks
• Jerry Shapiro: Microneedling and PRP
• Ali Jabbari: Biomarkers for AA and Clinical Trials
• Antonella Tosti: Repurposing Drugs for AA – Vytorin
• Teresa DiLorenzo: Similarities Between Diabetis Type 1 and AA
• Leslie Castelo-Soccio: Using Computer Vision to Quantitate Pediatric AA
• Tito Mendoza: Update on the Use of the AA Symptom Impact Scale
• Elise Olsen: A new visual Aid for Assessing Hair Loss in AA

38. Basic Science
Clinical Trials
Patient
Focused
Pharmaceutical

39. Angela Rodgers: What Treatment Means to
Patients
• No safe, affordable, accessible treatment is a problem
• “a magnificent effect on psychological status”
• socialization from childhood to adulthood
• (Shahin, A. et al., 2014).
• Increased prevalence of mental health issues

40. Basic
Science
Clinical
Trials
Patient
Focused
Regulatory

41. Regulatory
• NIH
• NIAMS
• FDA
• PCORI
• Industry
• Medical economic
• Health Insurance

42. Thank You
natasha@naaf.org