Researcher with FSHD Studies the Disorder to Help Others

Editor’s note, June 2017: Since we profiled Dr. Wuebbles in June 2016, he let us know that he is continuing to work on Galectin-1 and notes that he is working on a manuscript on the effects of Galectin-1 in a MDC1A model where it doubles life expectancy. His longtime mentor Dr. Peter Jones has since joined Dr. Wuebbles at University of Nevada in Reno, and Dr. Wuebbles reports that his lab is collaborating with Dr. Jones on defining the muscle physiology of his new FSHD model mouse and are testing some therapeutics in this new mouse model. In addition, in his role as Chief Scientific Officer at StrykaGen, he is close to beginning a clinical trial to determine if a drug that is FDA approved for cancer would be effective in treating Duchenne muscular dystrophy. Dr. Wuebbles is the co-first author of the manuscript describing this new therapeutic, which was recently published in Molecular Therapy. He also updated us on the effects of living with FSHD: “My health is slowly declining but I’m still getting around OK with help. My balance is the real problem, and I find myself looking for walls to lean on while I walk. I’m slowly but surely moving away from the lab into doing more writing and administration.”  

When Dr. Ryan Wuebbles stepped into a lab in 2002 to study muscular dystrophy, he wasn’t just a bright-eyed, eager graduate student.

He was also a young man who wanted to understand his own disease.

Dr. Ryan Wuebbles works under a hood in the lab.

Dr. Wuebbles, now a research assistant professor at the University of Nevada in Reno, was diagnosed with FSHD, or facioscapulohumeral muscular dystrophy, in his early 20s.

He first experienced symptoms at age 12, when he lost the ability to do a full sit-up. Later, though he could easily bench press 225 pounds, his muscles didn’t work quite like his fellow gym rats. Instead of tiring slowly, his muscles would abruptly stop working. Without a spotter, he routinely dropped weights on himself when his muscles failed without warning.

“Like any diagnosis like that, you’re worried about the future and what the future’s going to hold,” he says. “How fast are you going to lose mobility and your activity and what’s it going to look like when you’re confined to a wheelchair? How are you going to live?”

Most of all, Dr. Wuebbles wanted to understand what was happening to him and why. He had been studying electrical engineering but decided to make a switch.

“Especially when I was being diagnosed, I became very interested in biology,” he says. “’Why were my muscles giving out on me?’ It didn’t seem very fair. I was lifting weights. I was strong and yet my muscles started to fail me. I wanted to understand exactly why this was happening. So I switched to biology and I’m glad I did.”

Soon after his diagnosis, the first-year grad student arrived at the University of Illinois lab of MDA grant recipient Dr. Peter Jones. He asked to work with him on FSHD, a disorder Dr. Jones had never heard of. When asked why, Dr. Wuebbles shared his story. Inspired, and recognizing that working on FSHD fit into the scope of the lab’s research, Dr. Jones brought him on board and the two men started working together.

Soon enough, Dr. Wuebbles earned his PhD in that lab and moved on. But, 14 years later, using what he first learned in Dr. Jones’ lab, the researcher made a promising discovery that could help patients with Duchenne muscular dystrophy, or DMD.

It is called Galectin-1, a therapy that stabilizes and protects the interaction between muscle fibers and the glue that holds those fibers together in the body. The medicine lessens deterioration of those fibers when the muscles are used, extending muscle life for those patients. The pre-clinical data have been positive and the next stop will be human trials.

“Duchenne children have it worse off than I do by a long shot,” Dr. Wuebbles says. “I feel pretty blessed that I have the abilities still that I do, because I’ve met several Duchenne patients in the field we’re in and I really want to get them something that’s going to alleviate the burden.”

Dr. Wuebbles credits the MDA with helping him discover this therapeutic. He received a career development grant in 2012 and says the link is simple but extremely important — the MDA’s grant gave him the time he needed to actually do the research.

“Without that funding, therapeutics like Galectin-1 would have never come about,” he says.

Dr. Ryan Wuebbles with a special robot, called Janus, that he and his colleagues use to develop new drugs.

Dr. Wuebbles received his doctorate in 2009 and got married the same year. He now has two kids, ages 2 and 5. His 5-year-old loves riding on the scooter and helps his dad a lot. The boy will pick things up off the ground that his father can’t reach and relishes weeding the yard, which Dr. Wuebbles appreciates.

“A big part of my life now is definitely my kids,” he says, explaining that he and his wife went through a special procedure to help ensure that their kids would be born healthy. “That’s another way way I’ve lived without constraint.”

Getting around the lab isn’t as easy as it used to be, but he adapts. Instead of running up five flights of stairs to his work space, he uses the elevator. His lab mates have become a second family. They lift things that he cannot and perform other tasks to help him.

Dr. Wuebbles continues to do what he loves to do, and live his life to the fullest, despite the challenges.

“I’m a curious person and I like understanding how things work,” he says, explaining why he continues doing research, despite the complexity of his disease and a tough funding environment. “You love the science, you love figuring things out, and it’s interesting. It keeps you interested in what you’re doing. It’s fun to come to work every day.”

Alongside from working in his new role as co-founder of a biopharmaceutical company dedicated to developing medicines for muscle disease, the researcher has come full circle. He will be collaborating once again with Dr. Jones on FSHD.

Using a highly accurate model of the disorder newly developed by Dr. Jones, the scientists will be able to test treatments to see if they can help those, like Dr. Wuebbles, who live with FSHD every day.

“The more dedicated researchers that any of these diseases have, the faster a treatment is going to develop,” he says. “The more we understand, the better our chance are of finding a cure.”

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