FORT LEE, N.J.–Neurologix, Inc. has licensed the humanin gene from Keio University in Tokyo, an agreement providing Neuro-logix exclusive worldwide rights (excluding Japan) to develop and commercialize therapeutics to treat brain and other central nervous system (CNS) disorders (excluding amyotrophic lateral sclerosis) using the technology.
The humanin gene will be used in combination with Neurologix's proprietary gene transfer technology and is expected to be the company's lead compound for a novel and promising approach to Alzheimer's disease. Asked the advantage for his company to the licensing agreement, Neurologix Chief Executive Officer Michael Sorell replied: "It fits with our overall theme of treating neurological diseases with naturally occurring peptides that can packaged into an AAB construct and used as peptide factories within the brain. It is, in effect, a drug-delivery device. Our focus is on naturally occurring peptides that are involved in physiological roles, neurotransmitters and so on.
"Our scientists have identified that small peptides can be packaged readily into an AAV (adeno-associated virus) construct and injected directly into the brain. Drs. Michael Kaplitt and Matthew During, our scientific founders, pioneered this area in the early 1990s and were authors of many of the key papers in the field.
"In the case of humanin, which was below the radar screen of many groups, we believe it may be given simply and directly into spinal fluid by a single injection and taken up into the proper cells lining the spinal fluid and the gene product released over the cerebral cortex, where we hope it will play a role in protecting larger areas of the brain from the plaque that occurs in Alzheimer's disease. This would avoid the necessity of neurosurgery in these patients and allow market penetration to be rapid and extensive."
According to data compiled by the Alzheimer's Association (AA), an estimated 4.5 million Americans have Alzheimer's disease, more than double the number in 1980. National direct and indirect annual costs of caring for individuals with Alzheimer's disease are at least $100 billion, according to estimates used by the AA and the National Institute on Aging.
"You really do not have to do the arithmetic for a long time to see how valuable it [the humanin gene] is for Neurologix," Sorell said. "This is totally complimentary to everything we are doing. Our efforts in Parkinson's involves using GABA, a naturally occurring neurotransmitter, to dampen the activity in the subthalamic nucleus. In epilepsy, we are using the peptide Neuropeptide Y, which is a naturally occurring transmitter to dampen the excitability of the epileptogenic focus in the temporal lobe."
According to a press release, humanin, a 24-amino acid peptide, was identified by Dr. Ikuo Nishimoto and colleagues at Keio University by screening for novel genes that prevent the amyloid toxicity associated with Alzheimer's disease, as reported in the Proceedings of the National Academy of Sciences in 2001. The peptide is secreted, binds to the receptor on the cell membrane and then acts potently through parallel mechanisms to inhibit cell death pathways active in Alzheimer's disease and other neurodegenerative disorders. Humanin appears to be one of the body's internal anti-Alzheimer's defenses and is specifically found in brain regions that are naturally resistant to Alzheimer's disease. Further independent characterization of the mechanism of action for humanin was reported in follow-up studies by Dr. J.C. Reed and colleagues of the Burnham Institute in La Jolla, Calif., as published in Nature in 2003.
