DRUG DEVELOPMENT IS COSTLY AND TIME-CONSUMING, and that can be particularly true in neurology. Treatments that target the central nervous system have some of the lowest success rates of any therapies tested in clinical trials, so many companies, driven by the bottom line, have scaled back on development. Promising cures may be left on the table.

One way to counter this trend is to make it easier and cheaper to coordinate neurological drug trials. Six years ago, the National Institute of Neurological Disorders and Stroke (NINDS) partnered with researchers at more than two dozen hospitals and formed NeuroNEXT, a program that is making headway in finding new treatments for multiple sclerosis, Huntington’s disease, stroke and other conditions. Merit Cudkowicz, chief of the neurology service at Massachusetts General Hospital and the principal investigator for the Clinical Coordination Center of NeuroNEXT, discusses the pioneering effort.

So what is NeuroNEXT?
We are a network of 25 medical centers with expertise in neurology. We pool our resources to run better phase II clinical trials, which is the part of the approval process that assesses the safety, dosing and general effectiveness of new drugs. Massachusetts General Hospital serves as the clinical coordination center for these sites, while all the data analysis is conducted at the University of Iowa. Researchers who have a promising treatment—they might be from academia or industry—apply to NeuroNEXT, and we help them with their application for funding from the NINDS. If they get a grant, NeuroNEXT helps execute the study at our member sites.

Can you give an example?
For years, tissue plasminogen activator (tPA) has been the only approved treatment for strokes in the United States. It has to be delivered intravenously within three hours of stroke onset to help break down clots and restore blood flow to the brain. But for some patients, tPA has side effects, including internal bleeding and other complications.

A promising new therapy we are testing, called 3K3A-APC, is given right after tPA and is designed to help keep brain cells alive while reducing inflammation and bleeding risks related to tPA treatment. It was developed by researchers at a small company called ZZ Biotech, who were interested in using our network. We assign a principal investigator to these efforts—in this case, Patrick Lyden, chair of the department of neurology at Cedars-Sinai Medical Center.

What did NeuroNext do?
One of the first hurdles in a clinical trial is organizing a group of medical centers that can test the prospective drug in their patients. Traditionally, you have to reach out to each center with a separate contract, and each one reviews the protocol for safety and ethics. It’s a highly duplicative process, and it can sometimes take so long that the understanding of the biology of the disease changes to the extent that therapies are no longer highly relevant by the time testing begins.

Our network uses a centralized review board, and each member hospital has signed a contract so that it is ready to go for every study we do. Instead of waiting months or up to a year for full approval of a proposed trial, it takes only 45 days. This means that we can start testing therapies in people much, much sooner!

And when the centers approve the trial?
It’s sometimes hard to get enough people for a study, especially for rarer conditions. But our centers have access to big populations and a track record of great enrollment. And NeuroNEXT itself employs a staff fully dedicated to the recruitment and retention of participants for the clinical trials. We’re also assisted by a team of disease advocates—people who themselves have the illness or who have a close family member who has the illness. They can offer insights about study design and help get the word out about a trial. As a result, each one of our trials has enrolled on or ahead of schedule.

Has this changed who applies for NIH funding?
Historically, the only researchers who had a chance of getting funded for a multicenter clinical trial were those who were very experienced. That really barred any ideas coming from people who had never run a trial before. But our network offers mentorship that really lowers the barrier for young investigators and lab-based Ph.D. scientists who don’t have medical degrees.

Is this approach unique to neurology, or is it being applied in other disciplines?
Some aspects—our collaboration with private pharmaceutical companies, for instance—were borrowed from other fields, such as oncology. But our centralized review board, master contracts, mentorship in study design and conduct, collaboration among 25 experienced member hospitals and our efficient enrollment—that is all new and unique. Some other fields, however, are starting to adopt these tactics. For instance, similar networks were recently established for clinical trials related to trauma treatment and stroke.

What’s next for NeuroNEXT?
Our work has mostly focused on improving efficiencies once a trial is funded by the NIH. But it still takes a long time to get funding—the time from when you have a good idea and apply to the network to when you are actually funded is still a year and a half. So maybe there are ways we can work together to accelerate the grant application and review process.

To that end, we started a pipeline committee that provides free consultation to companies that have therapies ready for phase II testing. Because experts in our network have expertise in a broad range of diseases, we can provide some insight. We hope that the companies will then apply to our network, so that these drugs can come into trials more quickly. And it’s starting to work. The proposals are starting to come through.