Archivos de etiqueta: investigación

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 bodyXCHARXs 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 anunció recientemente resultados preliminares positivos de su ensayo de prueba de concepto 1b de fase de SYNT001 en pacientes con pénfigo vulgar y foliáceo. Es emocionante para IPPF compartir buenas noticias relacionadas con la investigación y los tratamientos. El comunicado de prensa completo de Syntimmune se puede encontrar .. El siguiente es un extracto:

Syntimmune, Inc., una compañía de biotecnología en etapa clínica que desarrolla terapias de anticuerpos dirigidas a FcRn, anunció hoy resultados preliminares positivos de su ensayo de prueba de concepto de fase 1b de SYNT001 en pacientes con pénfigo vulgar y pénfigo foliáceo. Los datos mostraron beneficios clínicamente significativos de SYNT001, con un perfil de seguridad y tolerabilidad favorable similar al observado en el estudio de fase 1a.

"Sigue habiendo una clara necesidad insatisfecha de un tratamiento seguro y de acción rápida para los pacientes con pénfigo, que enfrentan síntomas graves y complicaciones asociadas con su enfermedad", dijo Donna Culton, MD, Ph.D., profesora asistente de la Universidad de Escuela de Medicina de Carolina del Norte. Culton presentó los resultados preliminares del estudio Phase 1b en la conferencia International Investigative Dermatology que se celebrará en 16-19, 2018 en Orlando, Florida. "Estos datos preliminares demuestran seguridad, así como una reducción rápida en las puntuaciones de PDAI y la disminución de los niveles de IgG con el tratamiento de SYNT001, que respaldan nuevos estudios de este fármaco como una nueva opción terapéutica potencial", dijo Culton.

Lea el comunicado de prensa de Syntimmune, que incluye información adicional, aquí.