Tag Archives: research

Aimee S. Payne, MD, PhD, and Nicola J. Mason, BVetMed, PhD

Aimee S. Payne, MD, PhD, and Nicola J. Mason, BVetMed, PhD

Transformative Research Award Will Support Further Pemphigus Research

Nicola J. Mason, BVetMed, PhD, associate professor of Medicine and Pathobiology at the School of Veterinary Medicine at the University of Pennsylvania, and Aimee S. Payne, MD, PhD, the Albert M. Kligman Associate Professor of Dermatology at the Perelman School of Medicine at the University of Pennsylvania, have received the prestigious NIH Director’s Transformative Research Award. The award is part of the NIH Common Fund’s High-Risk, High-Reward Research program, which was established to accelerate the pace of biomedical discoveries by supporting exceptionally creative scientists with highly innovative research.

Autoimmunity occurs when the body’s immune system mistakenly attacks normal tissues, thereby causing diseases such as rheumatoid arthritis, systemic lupus, and type 1 diabetes. According to the Autoimmune Disease Research Center at John Hopkins, at least ten million Americans suffer from the more than eighty illnesses caused by autoimmunity. Under the grant, Mason and Payne are looking to evaluate a genetically engineered cell-based therapy approach to treat pet dogs with naturally occurring autoimmune skin disease known as pemphigus. Dogs are one of the few other species to develop pemphigus naturally and the condition mirrors pemphigus in human patients. Evaluation of this approach to treat pet dogs with this debilitating disease may ultimately lead to breakthrough therapies for humans.

“The successful treatment of autoimmunity in the family dog using this unique approach would not only be a breakthrough in veterinary medicine,” said Mason, “but could also change the way autoimmune disease is treated in humans. We believe that this work may facilitate the translation of cellular immunotherapies for a broad range of canine and human diseases, including autoimmunity, transplant rejection, infectious disease and cancer.”

Mason and Payne will continue to focus on their novel gene-engineered chimeric autoantibody receptor T cell (CAART) immunotherapy and its potential to cause lasting remission of antibody-mediated disease.

“Our study of CAART immunotherapy in companion dogs with naturally occurring autoimmune disease will be synergistic with our efforts to develop similar human therapies,” said Payne. “By comparing how these complex cellular immunotherapies work in dogs versus humans, we will better understand how to engineer and deliver these therapies to potentially cure disease.”

Mason, who earned her BVetMed from the University of London, and her PhD from the University of Pennsylvania, is a board-certified veterinary internist and immunologist. For the past ten years, she has been actively involved in evaluating the immunological responses of immune-based therapies in client-owned dogs suffering from lymphoma, osteosarcoma, and hemangiosarcoma. Mason’s research laboratory is currently developing CAR-T cell therapies for dogs with B cell lymphoma, and she serves as the PI and lead investigator on the first clinical trial evaluating CAR-T cell therapies in dogs.

Read the full press release at PennVet. 

Syntimmune recently announced positive preliminary results from its phase 1b proof-of-concept trial of SYNT001 in pemphigus vulgaris and foliaceus patients. It’s exciting for the IPPF to share good news related to research and treatments. The full press release from Syntimmune can be found here. The following is an excerpt:

 

Syntimmune, Inc., a clinical-stage biotechnology company developing antibody therapeutics targeting FcRn, today announced positive preliminary results from its Phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus vulgaris and pemphigus foliaceus. The data showed clinically meaningful benefit of SYNT001, with a favorable safety and tolerability profile similar to that observed in the Phase 1a study.

“There remains a clear unmet need for a safe and fast-acting treatment for patients with pemphigus, who face serious symptoms and complications associated with their disease,” said Donna Culton, M.D., Ph.D., an assistant professor at the University of North Carolina School of Medicine. Culton presented preliminary results of the Phase 1b study at the International Investigative Dermatology conference being held on May 16-19, 2018 in Orlando, FL. “These preliminary data demonstrate safety as well as a rapid reduction in PDAI scores and lowering of IgG levels with treatment of SYNT001, which support further studies of this drug as a potential new therapeutic option,” Culton said.

Read Syntimmune’s press release, including additional information, here.