Tag Archives: pemphigus

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. 

Pemphigus and pemphigoid (P/P) patients may agree that in the early months of their condition it is a very painful and difficult time. However, the following suggestions may be helpful like learning how to care for our skin in terms of taking and applying topical medications, cleansers, moisturizers, dressings, and staying comfortable.

FDA logo

On Thursday, June 7th, the FDA approved Rituxan for the treatment of adults with moderate to severe pemphigus vulgaris (PV). Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of PV in more than 60 years.

It can be difficult to accept the news of a lifelong chronic disease. It may be easier to understand the diagnosis of appendicitis when a procedure is needed, or medication to treat a cold, or the need to use crutches for a couple of months because of a broken bone. But accepting a disease like asthma, diabetes, lupus, pemphigus, or pemphigoid may be more difficult. Managing your life first begins with a strategy to live with your medical condition.

Even if you don’t have a medical condition, it’s always wise to have your medical information accessible ICE (In Case of Emergency). It’s a good idea to wear a medical alert bracelet or carry a medical ID with you, but since many people carry a cellphone at all times, it’s a great idea to use or install a health app.

Now that you know what you have been diagnosed with, it is important that all of the doctors who treat you are aware of pemphigus and pemphigoid (P/P), too.

This includes pemphigus vulgaris, bullous pemphigoid, mucous membrane pemphigoid (aka ocular cicatricial pemphigoid), pemphigus foliaceus, pemphigus vegetans, IgA pemphigus, paraneoplastic pemphigus, or gestational pemphigoid.  

All P/P rare autoimmune diseases are skin-blistering conditions. Steroid medication is often recommended in conjunction with immunosuppressant therapy in order to lower steroid dosages and bring the patient to remission (or at the very least, on a low-dosage maintenance program).

Since this is a condition that affects the skin, any invasive surgery will require an adjustment to medications prior to and after the procedure. This includes dental work (i.e. tooth extraction). Definitely for knee replacements, hip replacements, and any other invasive procedure.

Therefore, it is very important to advise your primary care physician, dentist, internist, rheumatologist, ob-gyn, and any other medical professional you are seeing on a regular basis. Each doctor needs to know about your new diagnosis and the medications you are taking, including any adjustments throughout your treatments, so they can keep track as well.

Your entire medical team is working together to keep you healthy, and keeping all of them in the loop is to your advantage.

Photo of Rebecca Oling and Dr. Cataldo Leone

It wasn’t easy getting to this meeting. After a four-hour train ride, I decided to walk to my hotel, nearly two miles away. It was rush hour, days after a major snowstorm. Boston was cold, and the streets were crowded with traffic barely visible beyond mounds of blackened snow. The walk was neither refreshing nor picturesque; it ended with a gauntlet of addicts outside the methadone clinic near my hotel. No sooner did I arrive in my hotel room when the phone rang. He was early, and I was spent. Everyone has a story.

The International Pemphigus and Pemphigoid Foundation (IPPF) is pleased to announce the availability of a seed grant program to encourage and support patient-oriented research of pemphigus and pemphigoid. The intent of these grants is to support well-defined research projects that have a specified benefit for improving the treatment, health, disease management, or diagnosis of persons with pemphigus and pemphigoid. Consideration will also be given to studies that contribute to the body of medical knowledge of pemphigus and pemphigoid.