The Icahn School of Medicine at Mount Sinai Health System is spearheading a collaborative effort to investigate dupilumab as a treatment for children with alopecia areata, a disease that causes extensive hair loss.
Emma Guttman-Yassky, MD, PhD, the Waldman Professor and Chair of Dermatology at Icahn Mount Sinai and the Mount Sinai Health System, has been awarded a new $6.6 million, five-year National Institutes of Health (NIH) U01 grant to support this work.
Alopecia areata is a common, hair-specific skin disease that affects more than 6.6 million people in the United States and is more prevalent in children than adults. The cause is unknown, and there is currently no treatment for it.
This grant, a part of NIH's continuous commitment to fostering innovation and excellence in scientific inquiry, will fund a collaborative effort to investigate the safety, tolerability, efficacy, and durability of response of subcutaneous administration of dupilumab in pediatric patients with extensive alopecia areata, defined as affecting at least 30 percent of the scalp.
"Alopecia areata causes tremendous psychosocial distress and greatly affects the patient's quality of life as well as negatively impacting the entire family. This is particularly impactful in children, as they do not yet have the coping mechanisms adults may have," said Dr. Guttman-Yassky. "Current treatment options for alopecia areata harbor negative side effects for long-term use. In addition, the moment you stop using the treatment, the hair typically sheds a few months later. This funding will enable us to embark on a transformative journey, pushing the boundaries of alopecia research and paving the way for innovative solutions to this burdensome disease."
Alopecia areata is recognizable by patchy hair loss, most often in the scalp and beard. About 14-25 percent of patients progress to total scalp or even total body hair loss. In 40 percent of patients, the disease begins before they are 20 years old, often increasing their risk of major depression and anxiety.
"There is evidence of a strong relationship between alopecia areata and atopic diseases like eczema, allergic rhinitis, asthma, and food allergy in the general population, and an even stronger association in children," said Dr. Guttman. "Among children with alopecia areata, 33 percent have atopic dermatitis, 21 percent have asthma, 20 percent have hay fever, and 14 percent have another type of allergy."
Knowing this association, Dr. Guttman research will explore the use of dupilumab, a monoclonal antibody that has shown strong efficacy and safety and is approved for a number of diseases, including atopic dermatitis (sometimes known as eczema) and asthma. A recent clinical trial of adult alopecia patients with severe hair loss showed that dupilumab induced significant hair regrowth, encouraging Dr. Guttman to explore dupilumab as a treatment for alopecia areata in pediatric patients with a history of atopic diseases.
To conduct this work, Dr. Guttman will lead a controlled trial of 76 children (6-17 years old) with alopecia affecting at least 50 percent of the scalp, who will be randomized 2:1 (dupilumab to placebo) for 48 weeks, followed by 48 weeks open-label dupilumab to all participants so everyone can benefit, and lastly 16 weeks follow-up, for a total of 112 weeks.
"This exciting NIH-funded clinical trial aims to expand our mechanistic understanding of AA, and shed light on the regulatory immune circuits in the pathogenesis of AA, particularly in children and adolescents," said. Dr. Guttman. "If successful, this trial holds promise to change the treatment of AA, enabling the use and further development of targeted therapeutics, which can be safe for long-term use."
