When a computer falls from a delivery truck, repair technicians simply replace the broken parts to keep the system working.
The solution is not quite that easy for humans.
If disease or injury attacks brain cells, humans don't have the option of replacement parts. They might live the rest of their lives disabled. But a small biotechnology company based in Lisle, Ill., hopes its efforts will improve that outcome.
NewNeural LLC, which was founded in August 2002, is working to develop products to help the brain replace damaged or destroyed cells. That damage can occur because of stroke or head injury, among other causes.
"We get lots of emails and phone calls. There are people out there desperate for solutions," said Robert Gonzalez, president, CEO and co-founder of NewNeural. The company's products "are for treating diseases that today don't have a cure."
The company's work has piqued the interest of the National Institute of Aging, a division of Bethesda, Md.-based National Institutes of Health, which just granted NewNeural a $136,000 Phase I Small Business Innovation Research grant to develop NN-818. The compound has demonstrated its ability to increase neural stem cells in aged animals. Neural stem cells are thought to be a part of the brain's internal healing mechanism.
NewNeural's technology is based on the discoveries of Kiminobu Sugaya, a NewNeural co-founder, its chairman and chief scientific officer. While at the University of Illinois at Chicago, Sugaya demonstrated the ability to transform human cells from marrow into cells that migrate and differentiate into cells with the morphology, markers and orientation of human neurons. Sugaya received a $1.4 million NIH grant for his work. The university filed for patents on the technology, which NewNeural has exclusively licensed.
The company has two "big bets" being studied at the preclinical stage, Gonzalez said. Those are ACT-N, the company's cell therapy approach, and the small molecule NN-818.
The company's autologous cell therapy-neurological (ACT-N) product is a treatment that begins with a sample of a patient's marrow. The marrow would be packaged and shipped to a facility for producing ACT-N, which then would be injected into the ventricle of the patient's brain. The transformed cells then would migrate through the brain and differentiate into new neurons and supporting neural cells. The hope is that the therapy would alleviate or reverse debilitating neurological conditions.
"There would be a significant improvement in motor or cognitive behaviors or functioning," Gonzalez told BioWorld Today.
The company and its handful of employees believe ACT-N also might be developed for other neurological indications, such as Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis, as well as spinal cord injuries.
NN-818 is designed to increase the number of a patient's neural stem cells, improving the brain's internal repair mechanism. Neither of the company's products use embryonic or fetal tissue or require immune suppression, Gonzalez said. NewNeural intends to initially target stroke and retinitis pigmentosa with its products.
Since its inception, NewNeural has raised a total of $1.1 million through grants and investments. The University of Illinois purchased $200,000 of the company's stock, in lieu of a cash payment for the license, and the company has sold about $720,000 worth of shares to investors. On top of that, Sugaya has received his own grant funding for research.
NewNeural is working to raise its initial $2 million seed round and hopes to build relationships with venture capital and strategic partners to support a $10 million Series A round that could bring the company's products into the clinic.
"If we had the funding that we'd like to have, we believe we could be in humans within two years," Gonzalez said.
NewNeural expects to partner its products in the clinic, while building a discovery engine to improve cell therapy and small molecules at the early research stage.
Neurological diseases and injuries affect more than 11 million people in the U.S., causing a loss of quality of life and costing more than $170 billion each year.
In addition to Gonzalez and Sugaya, NewNeural also was co-founded by Fady Charbel, its chief medical officer.