Trials and Tribulations

One of the biggest challenges confronting researchers and start-up firms seeking to develop new neurotech therapies is designing, funding, and completing successful clinical trials. Even when adequate funding is available from grants, investors, or corporate partners, clinical trials often present obstacles such as recruiting the appropriate participants and centers, defining the right endpoints and control arms, and submitting trial data for regulatory approval.

For this reason, we were encouraged to hear about a new program from the National Institute of Neurological Disorders and Stroke called NeuroNEXT, which stands for National Network for Excellence in Neuro­science Clinical Trials. The idea behind the seven-year, $84 million program is to expedite the development of new neurological therapies from lab to clinic by offering participating investigators common resources. Currently, the program has 25 clinical centers across the U.S., plus a coordinating clinical center at Massachusetts General Hospital and a data coordinating center at the University of Iowa. The staff at these two central sites will take responsibility for clinical implementation of the trial and data analysis. The clinical coordinating center will also house a central institutional review board, which will review protocols for all the participating sites.

While NeuroNEXT is currently oriented more toward pharmaceutical therapies and diagnostics, the potential for expanding this program to the neurotech device industry is great, in our view. Indeed, since many new implantable neurotech device therapies are targeted at individuals who have failed to respond to specific drug therapies, device trials could readily take advantage of participants who did not benefit from drug trials for a particular disorder.

The first disorder targeted by the NeuroNEXT program is spinal muscular atrophy at the phase II stage of development. SMA is a rare, inherited disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves. The muscles most frequently affected are those of the neck and trunk that control posture, those of the legs and arms that control movement, and those in the area of the ribs that help breathing.

One of the sites participating in this program is UT Southwestern Medical Center, which will receive $1.4 million from NINDS. “We want to bring the fruits of discovery in the laboratory as quickly as we can to the patients who need them,” said Mark Goldberg, chairman of neurology and neurotherapeutics at UT Southwestern and a co-principal investigator for the project.

We welcome the advent of the NeuroNEXT program and hope that it will be expanded to more disorders and therapies in the years ahead.

James Cavuoto
Editor and Publisher