The idea for the Center for Neurodegenerative Diseases and Repair (CNDR) was first hatched when Christopher Gomez, the head of the U of C’s department of neurology, realized that his lab was not ideally equipped to test potential cures for the class of nervous system disorders in which he specializes: spinocerebellar ataxia. In these diseases, degeneration of the cerebellum or spinal cord causes problems with movement coordination and control. Researchers under Gomez are working furiously to examine potential cures for the several dozen types of the disorder.
When potential cures were discovered, the researchers in Gomez’s lab attempted trials on lab mice, but because of shortcomings with equipment and resources, valuable research was being foiled.
To combat this, Gomez teamed up with Xiaoxi Zhuang—an assistant professor in neurobiology whose work with Parkinson’s Disease has afforded him extensive experience with testing on mice—as well as with a handful of other researchers to establish a mouse motor facility in order to track the progress of certain diseases and therapies.
They pooled their resources and bought a treadmill and a spinning bar known as a “rota-rod” to test motor coordination in mice. Now, Gomez says, researchers from across the U of C are bringing their mice to this facility.
This type of interdisciplinary and interdepartmental cooperation is exactly what Gomez wants to see more of as his department works to establish the new center.
“One of the CNDR’s main goals is to promote that [cooperation], to make it a very productive place for us to work for us and to attract new recruits,” Gomez said.
Life of the brain
The University has researchers working on diseases as diverse as Alzheimer’s, amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s Disease), and Creutzfeldt-Jakob Disease (CJD, closely related to mad cow disease). But scientists claim that several concepts are fundamental to many neurodegenerative problems.
Raymond Roos, a professor in the department of neurology, works with ALS, a disease that causes motor neurons to die. As the disease progresses, the patient loses control over muscles throughout the body. The muscles atrophy and the patient eventually dies. Many researchers suspect that the disease occurs when a key protein folds incorrectly, either as the result of a mutated gene or for unknown reasons, somehow killing the neurons.
Incorrectly folded proteins are also suspected of playing a role in other neurodegenerative diseases. For example, Alzheimer’s dementia symptoms are caused in part by plaques of incorrectly folded amyloid beta protein. Infectious misfolded proteins known as prions cause CJD and mad cow disease.
James Mastrianni, an associate professor of neurology who studies prion diseases, says the CNDR could make research involving these fundamental concepts more efficient.
“It’s very possible that one treatment may be useful to a variety of diseases. So if you have all these people working on different diseases with different proteins but the same process of misfolding, having several neuroscientists working together…will make the research much quicker,” Mastrianni said.
“Researchers have a chance to synergize in a very powerful way: to share insights, research, and techniques,” Roos added.
From the College through his residency, Gomez spent 14 years at the U of C. He returned recently, in part to establish the CNDR. The foundation was already they: “The idea is we already had strength in Alzheimer’s, a strong Parkinson’s effort, an ALS guy, me with ataxia, and many other strengths,” Gomez said. But there are some holes, and Gomez hopes to use the CNDR to recruit several experts to strengthen the U of C’s work.
For instance, he wants a stem cell researcher who could develop novel therapies to treat a range of neurodegenerative diseases.
Some scientists are looking into the use of viruses to deliver genetic therapies directly into degenerating cells, but the U of C also lacks a viral vector researcher.
Many neurodegenerative diseases are caused by broken molecules inside neurons. An expert in small-molecule drug discovery could help to sift through thousands of candidates to find the right therapy to stop those molecules from breaking.
Finally, Gomez wants someone who works with biomarkers, substances in the body whose measurements can be used to monitor the progress of diseases and treatments. “When a disease takes a turn for the worse, like multiple sclerosis sometimes does unexpectedly, it would be nice to have a marker showing when it’s time to bring out the big guns,” he said.
But so far the CNDR hasn’t landed any new recruits.
Still a vision
The Center is not yet a reality. Asked when the CNDR would be fully up and running, Gomez leaned back in his chair and let out a low whistle. “That’s an interesting question.”
“It depends on your definition of center,” he said. Many future CNDR members already participate in a weekly seminar series where researchers collaborate and bounce ideas off each other. This is the main aim of the CNDR, but the Center still has no recruits or independent financing under its belt.
“I think if we got one recruit it would have a major effect. Or maybe when we get a donation it will be established. Sometimes it’s just a matter of a sign,” Gomez said.
Sangram Sisodia, a professor in the department of neurobiology, hopes it’s sooner rather than later. “We’re working hard to create the Center… half a dozen other institutions have done it. The time to do it is now. It’s a very important aspect of this University’s development,” Sisodia said.
Gomez also hopes to establish the Center as soon as possible. By bringing diverse researchers together under one named roof, scientists hope to put the University’s intellectual talent and material resources to work for as many labs as possible. It could help attract new recruits and make the U of C preeminent in the neurodegenerative disease field.
University neurologists are excited about all these possibilities. But in the end, the CNDR is about one thing.
“The real goal is to cure disease,” Gomez said. “To make an environment where we can, as quickly as possible, move along the pipeline from a misbehaving cell or disabled person to fixing the broken molecule and mending the broken bodies.”