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Stimulating Stem Cell Regeneration

Stimulating Stem Cell Regeneration

NeoTherapeutics, Inc. (157 Technology Dr., Irvine, CA 92618, Tel: 949/788-6700, Fax: 949/788-6706) has revealed that its lead compound, Neotrofin, stimulated the proliferation of brain stem cells in adult mice. Neotrofin is a purine analogue that induces expression of several neurotrophic factors. Brain stem cells are a self-renewing population of cells that can restock the supply of mature nerve cells and may be the foundation for self-healing in the brain after injury. Data were presented at the Ischaemic Injury: Novel Targets and Therapeutics conference in London, an event regularly attended by experts and opinion leaders in the field of neuroscience.

In two blinded, dose-response studies, mice were given a single administration of Neotrofin and, 24 hours later, the number of newly formed brain stem cells was counted. Analysis revealed that in animals treated with doses between 1 mg/kg and 10 mg/kg of Neotrofin, there was a significantly higher number of newly generated brain cells. Studies are underway to examine the effect of multiple doses of Neotrofin and to determine whether the newly formed brain stem cells will mature to become neurons.

“At this stage, we can say that there seems to be a rapid increase in the number of new brain stem cells in animals treated with Neotrofin. This is a very exciting finding, made even more so by the fact that it was reproduced in two separate experiments, and that three different investigators, all blinded to the treatment that the animals received, obtained the same results,” states Eve M. Taylor, the senior scientist at NeoTherapeutics who led the study.

“Our greatest hope is that these cells might one day prove to slow down or reverse a variety of neurodegenerative brain disorders, such as Alzheimer s disease and Parkinson’s disease,” says Alvin Glasky, CEO and chief scientific officer of NeoTherapeutics.

Neotrofin is in clinical trials for Alzheimer’s disease and has been shown to be safe and well tolerated in over 1,000 patients. In a Phase II clinical study conducted by Steven Potkin of the University of California, Irvine, patients treated with 500-mg and 1,000-mg doses of Neotrofin experienced statistically significant improvement in memory, attention, and judgment. These behavioral improvements were consistent with changes in brain metabolism seen using Positron Emission Tomography scanning and changes in brain waves measured by electroencephalography. A trial using higher doses is planned.

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