NeuroNEXT Accelerates Research in SMA, Other Neurological Diseases

The National Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT ) is accelerating the clinical trial process for neurological diseases like spinal muscular atrophy (SMA) with the expectation of bringing medications to market in less time, with fewer costs and less risks.

Clinical studies for neurological diseases are typically long and arduous, taking many years and a lot of money to complete. As such, developing medications to treat these conditions also is long and expensive.

With funding from the National Institutes of Health (NIH), and leadership from the University of Iowa, NeuroNEXT is a consortium that brings together 25 academic healthcare research institutions nationwide to facilitate and diminish costs associated with clinical trials for neurological diseases.

The consortium helps the process of investigating novel drugs and therapies by dividing the risk through a series of institutions, funding agencies and pharmaceutical companies, said Chris Coffey, director of the College of Public Health’s Clinical Trials and Statistical Data Management Center (CTSDMC), the data-coordinating center for the initiative.

“A single institution is no longer spending the resources to conduct a clinical trial,” Coffey said in a story on the University of Iowa website. “Now, the risk is shared among many institutions across the country.”

The consortium is designed to facilitate patient recruitment (admittedly difficult in rare diseases due to the scarcity of patients), to help control quality and methodological standardization across studies and to assist data-gathering in the most efficient and cost-effective way possible, which already facilitates cross-referencing and comparability of results.

“We act as a bridge to make sure all of the trials are conducted in the same way,” Coffey explained.

The consortium was funded in 2011, but results of its first trial weren’t published until December 2017, in Annals of Neurology, which is a large time gap that illustrates just how much time it can take to study neurological diseases.

The two-year study, “Natural history of infantile-onset spinal muscular atrophy,” led by researchers at the Ohio State University observed biomarkers that indicate infantile-onset SMA. The trial involved researchers at 15 NeuroNEXT sites and the data collected were used to inform the U.S. Food and Drug Administration (FDA) application for the approval of Spinraza (nusinersen).

Looking at the natural history of 26 SMA infants and 27 controls, the data delineated meaningful change in clinical trials in infantile-onset SMA, demonstrating the power and utility of NeuroNEXT to provide “real-world,” prospective natural history data sets and accelerate public and private drug development programs for rare diseases.

“Our study showed how SMA occurred and what the progression looks like in newborns,” Coffey said. “Now that we have properly described and know how to find those benchmarks, researchers are able to match up drugs that can effectively be used against it in this study.”

The gene therapy AVXS-101, being developed by AveXis for SMA, is currently in clinical trials and soon may be available, too.

Ultimately the goal is to encourage more clinical trials in neurological disorders, see those trials through to completion, and ascertain if there is enough promise to go forward in drug development.

NeuroNEXT focuses exclusively on Phase 2 studies, which means researchers are not looking for a definitive answer to whether a treatment is effective, but are instead seeking to learn more about a potential treatment to decide whether there is enough evidence to justify the cost and complexity of a larger, more definitive Phase 3 trial.