Sceleroderma Research 2014-01 Update
Dr. Richard Neubig, the new chair of MSU’s Department of Pharmacology and Toxicology, appreciates the freedom he’s experienced in academia to pursue his research, especially in instances when findings take studies in directions far afield from their origins.
Neubig, a pharmacologist who has a robust research portfolio that he’s built since joining the University of Michigan faculty in 1983, has been especially aware of that freedom in one avenue of exploration that’s opened doors to possible future treatments for a number of serious diseases.
He was looking at a chemical compound that he initially hoped might show promise for treating melanoma and breast cancer when a colleague at another university published a paper showing that the compound might have applications for treating diseases where fibrosis is a major element.
Fibrosis is a process during which basic repair cells convert to cells called myofibroblasts and overproduce collagen. If you’ve had a cut that healed with a noticeable, bumpy scar you’ve seen fibrosis in action. It’s also a major component in a large number of conditions, from Crohn’s disease and liver cirrhosis to rare but serious pulmonary fibrosis to the entire family of scleroderma diseases.
Scleroderma is a condition that affects an estimated 300,000 Americans. It can take many forms, though it is often associated with systemic sclerosis, a condition that causes thickening of the skin and loss of flexibility in hands and feet, along with key organs like the lungs and kidneys.
“Oftentimes, what’s most dangerous about this disease is that it’s not just a skin disease. The fibrosis process also affects the kidneys and lungs and other organs,” Neubig says. “It used to be that most people with scleroderma would die of kidney failure. There are now approaches that will help blunt the kidney damage, but the lungs are a very important target organ now, and often, in people with systemic sclerosis, the lung disease is a huge problem that often leads to deaths.”
While other scleroderma treatments have looked at reducing the inflammation that contributes to scleroderma, Neubig’s work is focusing on stopping or reversing the fibrosis process at the cellular level. In his transition from the University of Michigan to MSU, he’s completed a mouse study that is showing very promising results that he hopes to publish this fall.
Though his findings are positive, there’s still a lot of work to do before a treatment is available for scleroderma or other fibrosis disease sufferers. The chemical structure and compound that would be used for human clinical studies need to be refined, clinical trials developed (likely in collaboration with a private sector pharmaceutical company) and any treatment would go through the FDA approval process.
“Certainly the experiments in vitro, in petri dishes and microscopes, look fantastic,” he says. “They look really, really good. We have also done experiments with human scleroderma cells where we can actually reverse the state in which they make lots of collagen and induce fibrosis.”
The work is being conducted by a research team comprised of Neubig and his former U of M colleagues Dr. Scott Larsen, director of the Vahlteich Medicinal Chemistry core; Dr. Dinesh Khanna, associate professor of internal medicine who heads Michigan’s scleroderma program; and Dr. David Fox, professor of internal medicine and chief of rheumatology. Because Neubig is now doing his research from East Lansing instead of Ann Arbor, the scleroderma work is now also a joint MSU/U of M project.
Fundraising and Gift Giving for Scleroderma
From its beginnings to its current cross-university team, the effort is also unique in the way it’s been supported. While Neubig and his collaborators are pursuing traditional grants and development avenues, they’re also raising funds to continue the research via an online crowdfunding site that was developed in collaboration with Jonathon and Lisa Rye, a southeastern Michigan couple whose family has been affected by scleroderma.
The Ryes have been strong advocates for and supporters of research to find a cure. They educated themselves about the disease, learned about the research community and made connections with Fox and Khanna, and later with Neubig.
The Ryes have not only made their own philanthropic contributions to scleroderma research, they also built a crowdfunding site to encourage others to support the work. The site offers donors the chance to support the research at either the University of Michigan or at Michigan State University.
“As we see it, this is two great institutions working to solve this disease,” Jonathon Rye says. “What we’re hoping is to raise as much money, either directly or through the web as we can so Rick and Scott can develop better compounds so a biotech company will take it on. The whole thing is coming together quite nicely.”
While sites like Kickstarter and GoFundMe are often used by nonprofits and entrepreneurs, the Scleroderma Cure Fund is only the fifth site of its kind that is connected with major research universities.
“We were the fifth in the country, so you get the advantage of that and you get the advantage of Rick (Neubig),” Jonathon Rye says. “We are optimistic that Rick and Scott (Larsen) have found the answer.”
The Scleroderma Cure Fund site offers a simple and quick option for donors who want to support research, but who might feel like universities are only interested in larger checks or who don’t want to deal with trying to send funds via the U.S. Mail.
“We’re up to 4,500 likes [on Facebook], Lisa Rye says. For smaller donors who might not think they are making a difference, if all they can give is $5, it does matter. We can get this going with smaller amounts and larger numbers of people giving.”
Neubig also sees value in adding the crowdfunding option to other resource-generating efforts.
“It provides a way to number one, move things more quickly and number two, it really connects us with the people who are interested in these cures and allows us to really connect with the end users who would potentially benefit from the research,” he notes. “I’ve met a few people who’ve been affected by scleroderma. Doing more of that will be very good for us in that it allows us to really see the direct impact as opposed to some esoteric study that we can’t easily envision what the direct impact is.”