Scleroderma Research

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Neubig Lab MSU Scleroderma update – February 2015

We greatly appreciate the interest that you have all shown in our studies of new scleroderma treatments. We have gotten many e-mails from around the country and even the world about progress on our studies in part based on the publicity from the TV4 interview. The ultimate goal of our work is to develop new treatments for individuals with scleroderma and other diseases of fibrosis. The path from a new therapeutic concept to an actual drug is, unfortunately, long. We are making progress but it seems very slow – to us and I’m sure to you all. We are still years away from any clinical trials. Here is an update on our work and that of our collaborators.

New publication showing reversal of fibrosis

Our concept of treating scleroderma and other diseases of fibrosis by blocking a key genetic switch has received another boost. In a collaboration with Drs. Sisson and Horowitz in the Pulmonary Medicine division at the University of Michigan we have shown that our compound CCG-203971 can reverse fibrosis in the lungs of mice. The paper is online at the American Journal of Pathology. The journal considered this important enough to write a commentary regarding the significance of our result showing reversal of established lung fibrosis.

Presentation at Scleroderma Foundation (Michigan Chapter)

Dr. Neubig will be presenting an update at the Scleroderma Foundation’s (Michigan Chapter) gathering Let's Talk Scleroderma 2015. It is on April 25, 2015 (DoubleTree by Hilton Detroit-Novi). Learn more about this patient-oriented conference.

New compounds

With the Larsen Lab at the University of Michigan, we have identified new compounds that reduce pro-fibrotic gene expression and myofibroblast activation in human skin fibroblasts in culture. They have significant improvements in both potency (so that smaller doses can be used) and their ability to stay in the body long enough to have a beneficial effect. Animal studies to test their ability to reverse skin fibrosis are underway. We hope to have these results soon.

Collaborators

Modern drug discovery and development requires a substantial team effort. We have an outstanding group of trainees and co-investigators. Andrew Haak, a PhD student who just finished in my lab, and Dr. Eliza Tsou, a research fellow at the University of Michigan, have been key players. The work is also a close collaboration between my lab at MSU and faculty at the University of Michigan. Dr. Scott Larsen, director of the Vahlteich Medicinal Chemistry core, a former Pharmacia and Pfizer researcher, has provided key insights into the process of drug development as well as driving the chemical optimization of our compounds. Dr. Dinesh Khanna, associate professor of internal medicine and head of Michigan’s scleroderma program and Dr. David Fox, professor of internal medicine and chief of rheumatology supervise the collection of human scleroderma skin samples for compound studies as well as the in vivo testing of compounds. The recent publication was from Drs. Sisson and Horowitz in Pulmonary Medicine at UM.

Funding

Support for this work has come from Jonathan and Lisa Rye and from many of you. We greatly appreciate your assistance in this important project. We also now have a substantial grant from the National Institutes of Health (Arthritis and Musculoskeletal and Skin Diseases).

We greatly appreciate your interest and support. Know that we are completely dedicated to making a difference in scleroderma and systemic sclerosis with this work.

Rick Neubig
Professor and Chair
Pharmacology & Toxicology
Michigan State University