Thanks to a $250,000 grant from the National Multiple Sclerosis Society, Endece Neural is forging ahead with preclinical research of a drug compound that it believes could revolutionize treatment options for people living with multiple sclerosis (MS).
The disease, which impacts more than 2.1 million people across the globe, occurs as the immune system attacks the central nervous system, which consists of the brain, spinal cord, and optic nerves. Those who suffer from MS face varying levels of symptoms, such as bladder and bowel dysfunction, vision problems, pain and numbness, and issues with walking and balance.
Current treatment options slow the progression of the disease and its effects, but with the lead compound, NDC-1308, Endece Neural aims to help patients improve their functioning and live without such severe symptoms.
Endece Neural, a subsidiary company of Mequon-based drug discovery company Endece LLC, is using grant dollars from the National Multiple Sclerosis Society, along with funding from four internal private investors, to conduct a study analyzing the efficacy of NDC-1308.
The investigational compound, and the company’s study with it, addresses the myelin, a fatty substance that protects nerves against damage.
In MS patients, the body attacks the myelin. Nerves, then left exposed, are defenseless against damage. The damage, once done, can cause distorted nerve impulses between the brain and spinal cord, which prompts the onset of the disease’s disabling symptoms.
Unlike other treatments, NDC-1308 aims to repair the myelin of damaged axons, or nerve fibers, and consequently improve individuals’ symptoms.
“What we’re excited about is that the MS patients could feasibly continue to take the current drugs to slow the progression and then by having our drug to put the myelin back on, that could be a balance that could be very beneficial for them,” said Dr. James Yarger, chief executive officer and co-founder of Endece Neural.
The company’s related study has been an ongoing one since July and will run through February 2014.
Mice that do not have a myelin sheath around some of their nerve fibers are at the center of the study as researchers try to induce remyelination within them using NDC-1308.
While researchers have chemically removed the myelin sheath within some mice they are studying, in other mice they have prompted the immune system to remove the myelin sheath, much like what MS patients experience.
Within the last decade, mouse models have been developed to mimic human patients with MS, according to Yarger.
While Endece Neural is responsible for the drug discovery and the design of the study as well as the monitoring and analysis of collected data, the study is being conducted through Cleveland, Ohio-based Renovo Neural Inc.
Following Endece Neural’s study on mice, NDC-1308 will be applied to safety studies using larger animals to ensure that the investigational compound is, in fact, safe to use in humans. Such studies typically last six to eight months, according to Yarger.
Before Endece Neural then proceeds forward with clinical trials in humans, the U.S. Food and Drug Administration (FDA) will need to give its consent through an Investigational New Drug Application.
The document details information about drug studies’ safety data, clinical strategy, and clinical program.
“With that, (the) FDA reviews our data thoroughly, mostly our safety data, to make sure this drug is safe to go into humans,” Yarger said.
He is optimistic that clinical trials using the compound in humans will take flight sometime around the end of 2014 or early 2015.
“All of our data is very positive,” Yarger said. “So at this point, I am highly, highly positive that this will go into clinic.”
So far, the safety profile is favorable, he said.
“We’re done a lot of studies in mice, and we’ve seen no health issues in mice.”
Earlier this month, Endece Neural scientists took findings from the preclinical study to Copenhagen, Denmark, where they presented data at the 29th Congress of the European Committee for Treatment and Research in Multiple Sclerosis.
The conference drew 8,000 of the world’s leading MS researchers and gave Endece Neural a chance to put its progress on the world stage.
While NDC-1308 won’t cure MS, it could likely enable people struggling with the disease to regain a higher quality of life.
“By putting the myelin back on, it could give people their lives back so they could essentially live with the disease and not have the major symptoms they have now,” Yarger said.
