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Ali Nili, MD

Nili Ali_PFDS24_2
Postdoctoral Fellowship in Drug Discovery, 2024 Dana Farber Cancer Institute

A Targeted Approach for Treating a Rare Autoimmune Disease

Summary

Pemphigus is a rare autoimmune disease that causes painful blisters and erosions on the skin and the mucous membranes inside the mouth, nose, throat, and genitals. Pemphigus is most prevalent among people living in Israel, Iran, and the United States, particularly in the Ashkenazi population. Though uncommon, it can be devastating for those affected. In pemphigus, the body’s immune system mistakenly attacks itself, with immune cells (called B cells) producing antibodies that attack the adhesive structures in the mucous membrane and skin. Current medications, such as corticosteroids, shut down the faulty immune cells as well as the entire immune system, increasing the patient’s infection risk and causing severe side effects. Using protein engineering methods, I developed a novel targeted therapy that specifically clears out the harmful B cells without affecting the entire immune system. This therapy has demonstrated high efficacy and safety in several mouse models. My project also aims to develop more challenging models to better mimic real patient conditions before translating this treatment to the clinic. The next step would be to apply this treatment to other autoimmune diseases.

Watch a Q&A with Ali

I am grateful for the support of the PhRMA Foundation. To me, this fellowship signifies recognition of my research in autoimmune skin diseases and offers a precious chance to further my experience in basic science that helps me to advance my career as a physician-scientist. The two years of support have granted me the opportunity to delve deeper and explore with more sophisticated insight into developing novel targeted therapies, setting the stage for clinical translation of the approach, to the benefit of many patients.

Ali Nili