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Genetics, Immunology, and
Therapeutic Targets
Breakout Group Report
What has already been done and how is it related?
• Identification of new targets…but there must be more
• Are we looking in the right places? Hair follicle..? ECM..?
• Are we looking for the right signals…? Other cytokines…?
• Are we looking at the right cells…? NK? Other regulatory cells?
• Efforts toward antigen discovery progress being made and needs
to keep going
• What can we learn from patients who don’t respond to JAK
inhibitors?
• How can we better define those who will respond to JAK
inhibitors?
What questions still need to be answered?
• Need to understand how to stratify/select patients
• What defines a JAK responder vs a JAK non-responder?
• Are all non-responders not responding for same reason(s)? Different
reason(s)?
• Are there confounding diagnoses in JAK treated patients (AGA, other
alopecias?)
• Can we define a set of markers to select/stratify JAK responders, and
group the non-responders for further studies?
• What is the mechanism of longstanding disease? Is there burnout?
• Correlation of clinical status with research efforts – new onset patients,
longstanding disease, pediatric populations, etc.
What resources are needed?
• Additional funding for immunological studies
• Exploration to other immune mechanisms – NK cells, gammadelta
T cells, Tregs, B cells, DCs, mast cells, monocytes, others
• Further genetic studies – only 14 confirmed genes so far…there
are likely many more. Defining complete architecture to find
more pathways, potentially the ‘minor’ ones
• Biology of the end organ – hair follicle, cycle, pigmentation- and
its role in disease
• Consideration of environmental factors that interact with genetics
• Biomarkers and tools to monitor response to treatments
What can NAAF do to help?
• Liaise with other autoimmune disease groups to leverage research
findings, funding schemes, research priorities, shared disease
mechanisms (T1D, RA, thyroiditis, vitiligo, etc)
• Continued funding of exploratory ideas, to advance new concepts
and young investigators
• Continue to drive drug development pathway
• Continued emphasis on developing new outcome measures
• Continue with Research Summit to help define priorities and set
the agenda for the coming years!

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Genetics, Immunology and Therapeutic Targets Breakout Report

  • 1. Genetics, Immunology, and Therapeutic Targets Breakout Group Report
  • 2. What has already been done and how is it related? • Identification of new targets…but there must be more • Are we looking in the right places? Hair follicle..? ECM..? • Are we looking for the right signals…? Other cytokines…? • Are we looking at the right cells…? NK? Other regulatory cells? • Efforts toward antigen discovery progress being made and needs to keep going • What can we learn from patients who don’t respond to JAK inhibitors? • How can we better define those who will respond to JAK inhibitors?
  • 3. What questions still need to be answered? • Need to understand how to stratify/select patients • What defines a JAK responder vs a JAK non-responder? • Are all non-responders not responding for same reason(s)? Different reason(s)? • Are there confounding diagnoses in JAK treated patients (AGA, other alopecias?) • Can we define a set of markers to select/stratify JAK responders, and group the non-responders for further studies? • What is the mechanism of longstanding disease? Is there burnout? • Correlation of clinical status with research efforts – new onset patients, longstanding disease, pediatric populations, etc.
  • 4. What resources are needed? • Additional funding for immunological studies • Exploration to other immune mechanisms – NK cells, gammadelta T cells, Tregs, B cells, DCs, mast cells, monocytes, others • Further genetic studies – only 14 confirmed genes so far…there are likely many more. Defining complete architecture to find more pathways, potentially the ‘minor’ ones • Biology of the end organ – hair follicle, cycle, pigmentation- and its role in disease • Consideration of environmental factors that interact with genetics • Biomarkers and tools to monitor response to treatments
  • 5. What can NAAF do to help? • Liaise with other autoimmune disease groups to leverage research findings, funding schemes, research priorities, shared disease mechanisms (T1D, RA, thyroiditis, vitiligo, etc) • Continued funding of exploratory ideas, to advance new concepts and young investigators • Continue to drive drug development pathway • Continued emphasis on developing new outcome measures • Continue with Research Summit to help define priorities and set the agenda for the coming years!