NEW HAVEN >> There is now no way to regenerate severed nerves in the central nervous system, but Dr. Stephen M. Strittmatter is confident hes found a way to repair them.

Hes even founded a company, ReNetX Bio Inc., to shepherd his new therapy through the maze of regulations, clinical trials and manufacturing processes, ultimately hoping to cure patients with devastating injuries.

We have this amazing, complex neural network that manages all our functions, stemming from the brain and spinal cord, said Strittmatter, professor of neurology and neuroscience in the Yale School of Medicine.

When a nerve fiber, or axon, in the central nervous system is damaged, such as in a paralyzing spinal cord injury, it doesnt grow back. Even though the nerve cell is still healthy in the adult brain or spinal cord, it cant grow and therefore function doesnt come back, he said.

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(The central nervous system, which manages all our functions, is separate from the peripheral nervous system, which performs other tasks, such as carrying stimuli from our senses, and which can regenerate.)

The axon, which starts at the cell body, or soma, can extend up to a meter in length, Strittmatter said. If the cell were the size of a baseball, the extension, the nerve fiber, would be the width of a pencil and be a quarter of a mile long, he said.

Strittmatter said he has investigated why nerve fibers cant grow in adults, and that led us to the idea that there are inhibitors that are present in the adult brain and spinal cord. They stop the axons from growing back to where theyre supposed to be.

In fact, there are three such inhibitors, called Nogo, MAG and OMgp, which exist in the myelin that coats the nerve fibers. They stick to the axon and tell it not to grow, Strittmatter said.

He and the researchers in his lab studied ways to stop the inhibitors from attaching to the axon. So we developed this protein, which we call Nogo Trap Its sort of like a double negative; it blocks the inhibitors [and] those new connections allow function to be recovered, he said.

So far, the therapy looks promising. Weve done experiments here that have shown that that works after rats and mice have spinal cord injuries, he said.

Now, ReNetX Bio, a new name for a company founded in 2010 as Axerion Therapeutics, faces the long process of turning an experimental therapy into a marketable drug, which they hope also will be effective for stroke and glaucoma.

Thats what the company is about, bringing it out of the lab and into the clinic, Strittmatter said.

The next step is getting Food and Drug Administration approval of Nogo Trap, also known as Axer-204, as an investigative new drug, which allows phase one clinical trials. That initial phase is only concerned with the drugs safety. The second and third phases test whether or not the drug is effective.

Erika R. Smith, named CEO of ReNetX in July, said there is a long list of other tasks to be addressed, including toxicology testing, scaling up manufacture of the drug and lots of paperwork. A lot of boxes get checked to make sure its OK to try in a clinical setting, she said.

Both Smith and Strittmatter said there are advantages to forming their own company.

I guess I feel like being involved I can help make sure that the right clinical trials are done, Strittmatter said.

Smith added, Theres a lot of challenges in early research that a lot of pharma companies arent willing to take the risk themselves. A lot of times companies wont come in really early.

Along the way, theres all kinds of roadblocks, things we cant expect, Strittmatter said. Drugs might get degraded faster in one species than another or there could be secondary complications like infections.

Were very excited that the experiments that have happened in the lab have gone very well, he said. However, there is a risk. Experimental animal studies can look great but maybe only 20 percent of the time can that be turned into a drug that can be used in people, he said.

But Smith noted the substantial funding that has come into the company to this point were estimating $15 million that has gotten the program to where it is. Much of that support has come from the National Institutes of Health, she said.

The company has a staff of five and is seeking to hire a chief medical officer, Smith said.

Yale University holds intellectual property rights and is a part owner of ReNetX. The company has licensed those patents from Yale so they can go on to do sales and clinical development, Strittmatter said. Yale would receive royalties if it were eventually sold as a drug.

The end goal that it gets to people and it makes a difference in their lives, Strittmatter said.

Smith said, The big picture of this is its a whole new paradigm change for any kind of injury in the central nervous system.

Call Ed Stannard at 203-680-9382.

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Connecticut doctor works to repair central nervous system injuries - Torrington Register Citizen