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I. Caroline Le Poole, Biologics for Vitiligo

Caroline LePoole

Vitiligo is an autoimmune disease affecting approximately 50 million people worldwide. It presents itself as progressive white lesions on the skin, especially noticeable in people of color. The condition may lead to complete depigmentation.

Comorbidities include amongst others alopecia, a condition that causes hair to fall out in small patches, and hypothyroid disease. While vitiligo manifests itself in the skin, it clearly has an all-encompassing impact on patient life, often leading to discrimination.

Caroline Le Poole, PhD, a professor of dermatology, microbiology and immunology at Northwestern’s Feinberg School of Medicine, has developed a DNA-based treatment using the modified heat shock protein HSP70i to reverse disease progression. When applied as a series of needle-free DNA-plasmid injections, the treatment was shown over a six-month study to reverse the disease in large animals.

Her research, published in journals including Science Translational Medicine, shows efficacy in both animal models. In 2019, Le Poole co-founded the startup company Temprian Therapeutics to take the treatment to clinical trials with CEO Kettil Cedercreutz. The company is currently in the process of raising $3 million for preclinical and Phase I/IIa clinical trials.

“It all started by our research team convincing the dermatology world to see [vitiligo] as an autoimmune disease,” she said. “A detailed understanding of the etiology was important for the development of an effective treatment.”

Le Poole’s doctoral studies at the University of Amsterdam focused on vitiligo etiology. Her dissertation and subsequent research helped her refine approaches to treat both vitiligo and melanoma, where immune responses to affected cells are in fact desirable. By the late 1990s, the Le Poole lab had shown that vitiligo results from T cells attacking and killing the body’s own pigment cells. The process tends to be triggered by severe stress brought on by external factors such as sunburn, other physical trauma, or emotional distress.

“We made a single amino acid substitution that changes HPS70i from activating the immune system, to becoming immunosuppressive, which is what we needed to reverse vitiligo,” she said. With this slight modification of the original molecule, Le Poole gained the ability to alter its function to benefit the patient.  Their findings received international attention as a finalist in The Nature Merck 2020 Spinoff Competition.

Le Poole believes that her line of research also could have implications for the fight against melanoma. Besides studying HSP70i, the Le Poole Lab is developing a regulatory T cell-based treatment for vitiligo to target patients with more advanced disease.

Le Poole notes that reserved attitudes towards DNA-based applications have faded in recent years, which has helped spur interest in this type of treatment and underscored the promise that Temprian Therapeutics holds. “When we tell investors about the severity of the disease, the lack of effective solutions, and what our development plans are, investors tend to listen,” she said.