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Posts tagged ‘neonatal skin’

A Letter from Our Director: Dear Members, PeDRA Gets It.

Amidst a growing sea of scientific work focused on skin disorders in adults, there is only a limited amount focused on children. In 2012, a group of pediatric dermatologists founded PeDRA, the Pediatric Dermatology Research Alliance, in hopes to better treat and cure dermatologic diseases in infants, children and adolescents. Their mission is to foster collaborative clinical, translational, and basic science research.photo(1) This past weekend, Executive Director Jean Pickford, attended the very first annual PeDRA (Pediatric Dermatology Research Alliance) conference, and is happy to report that there is “no stopping” this dedicated team of doctors, when it comes to finding a cure for ichthyosis. Today, she’d like to share her experience with those whom this remarkable new alliance will mostly affect; our members.

October 23, 2013

Some days in life are predictable. And then, there are the days that greet you with such utter amazement, no matter how you describe it, you will never do it justice. In fact, I happened to have just that type of day last weekend in Chicago at the PeDRA (Pediatric Dermatology Research Alliance) conference. So please allow me to do my best job at sharing the highlights from that meeting. As you read the words, I only hope you can feel even a twinge of the powerful energy I experienced.

Despite missing my early morning flight to Chicago due to another commuter’s rush-hour accident on the highway, I managed to still arrive at the 1st Annual PeDRA Conference, albeit late. The conference was attended by 80 pediatric dermatologists and six patient advocacy groups. The goal of this meeting was to gather the top leaders in pediatric dermatology research in one room to share, develop, and discuss new and innovative research to treat skin disorders. At the top of their list is, gratefully, ichthyosis.

Because of ichthyosis’ rarity, I have become accustomed to attending these types of meetings, and having other more prevalent skin disorders in the forefront. I am usually in the “back of the house” as ichthyosis is typically not a hot topic on the agenda. However, at this meeting, I happily climbed over the rows, and took my place in the front row, as ichthyosis was in fact, in the limelight. Investigators from around the country presented innovative ideas, current research projects, proposed grants, and novel approaches throughout the three-day conference. After each presentation, one thing became very clear – I knew that FIRST was part of something remarkable.

One of the biggest highlights throughout all these discussions was actually feeling the passion of these physician/scientists, as they presented and discussed their work. They are fueled by curiosity; relentless in their research efforts; and simply put – they get it. They understand how important it is to find better treatments and cures —and they really care. They also understand what an important role FIRST plays in their research. Because we have access to patients and a pulse on the most pressing areas of concern, we complement their work in an immeasurable way. It was clearly evident at this meeting that FIRST and the other patient advocacy groups were held in the highest regard.

So what was accomplished? Four major areas of focus were determined, with multiple physician/scientists signing on to various leadership roles. Those projects included:

Neonatal Premature Skin – One group was organized to address the area of neonatal premature skin. This neonatal skin group will work together with neonatologists to develop and test scientifically based barrier formulations aimed both at improving barrier function and promoting its maturation.
Observational Study - A second project was launched to begin a multicenter cooperative prospective observational study, enrolling infants who will receive a genetic diagnosis and be followed on a monthly basis. The goal is to help physicians to better understand who is at risk for complications and allow the development of protocols to better anticipate and manage these complications.
NIH Rare Disease Clinical Research Consortia – A third project focused on a grant that will be submitted to the NIH Rare Diseases Clinical Research Consortia this year. The grant focuses on a multicenter collaborative network that will enroll patients with rare ichthyoses and perform coordinated longitudinal (over the course of time) and pilot (brand new) studies. It will also focus on defining clinical relevant biomarkers (changes found in skin or blood, such as in levels of a specific protein, that mark disease activity) and creating a basis for clinical trials leading to cures.
Inflammatory Ichthyosis – The final project addresses inflammatory ichthyosis and its correlation to atopic dermatitis (eczema) and shared biomarkers that drive inflammation and itching.

PeDRA plans to meet as a full group each year, and I for one, will not miss a moment (and yes, I’m already planning to leave an extra hour early for the airport!). I am very impressed and excited to see how the work develops from this dedicated group of game-changers for ichthyosis. I am proud and privileged to be a part of their efforts.

You can look forward to hearing updates and information from FIRST regarding the progress of PeDRA and of these projects in the future.

Let’s continue to educate, inspire, and connect,

Jean