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NIH Grant Funds New Spinal Cord Injury Treatment

Spine

(Michael Dorausch, Flickr)

28 June 2018. A biotechnology company developing regenerative therapies for neurological disorders is receiving a Small Business Innovation Research grant to advance an experimental treatment for spinal cord injury. The $1.1 million award from National Institute of Neurological Disorders and Stroke, a part of National Institutes of Health, was made to BioAxone BioSciences Inc. in Cambridge, Massachusetts and RXi Pharmaceuticals Corp. in Marlborough, Mass.

Spinal cord injuries are often caused by a sudden, traumatic blow to the spine that bruises or tears into spinal cord tissue, resulting in fractures or compression to vertebrae, or in some cases severing the spinal cord. Depending on severity, people with spinal cord injuries often suffer loss of feeling or motor function in the limbs, and in some cases complete paralysis. According to the National Spinal Cord Injury Statistical Center, spinal cord injuries occur in 54 out of 1 million people in the U.S., adding some 17,500 new cases each year.

The grant supports BioAxone’s work with the phosphatase and tensin homolog or PTEN protein that appears to play a key role in regenerating axons, the fibers extending from the bodies of nerve cells that conduct electrochemical signals. The presence of PTEN is associated with limiting the growth and development of axons in nerve cells. As a result, blocking the actions of PTEN is believed to help encourage nerve cell regeneration in people with spinal cord injuries.

BioAxone’s technology, code-named BA-434, aims to silence PTEN expression at the site of spinal cord injuries. In preclinical tests, blocking PTEN effects is shown in lab rodents to encourage axon growth and rewire nerve cell circuits. To stop PTEN activity, BioAxone is using RNA-interference, genetic instructions and a natural process that regulates the over-expression of proteins from a gene causing a disorder. RXI Pharmaceuticals, a developer of RNA-interference treatments for cancer, is partnering with BioAxone on the project.

The grant from NINDS aims to advance BioAxone’s work with blocking PTEN’s effects to the point of an investigational new drug application, which asks FDA to begin clinical trials. The program calls for developing stable forms of BA-434 for drug delivery, and to determine optimum doses. The company also plans to conduct safety and efficacy tests with pigs induced with spinal cord injuries, since the spinal cords of pigs are nearly the same size as humans.

The project is a continuation of a Small Business Innovation Research or SBIR grant from NINDS that initially awarded nearly $736,000 to begin the work, with the remaining $1.06 million for completing the proposed tasks. RXi is receiving nearly $248,000 of the total award. SBIR awards given as grants and contracts, are research and development funds reserved for small businesses by major government research agencies, including NIH.

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