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Neuralstem secures FDA approval for Phase Ib MDD trial
ROCKVILLE, Md.—Neuralstem, Inc., a Maryland-based company that targets neurological disorders and diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted approval for Neuralstem to advance to Phase Ib in its ongoing clinical trial to test its neuroregenerative compound, NSI-189, as a treatment for major depressive disorder (MDD). Neuralstem's technology allows them to create neural stem cells of the human brain and spinal cord, and it allows for control of the differentiation of the cell into mature, physiologically relevant human neurons and glia.
The Phase Ib trial will seek to determine the safety and tolerability of NSI-189 in depressed patients. The trial is a randomized, double-blind, placebo- controlled, multiple-dose escalating trial. Phase Ia tested escalating doses of a single administration of the compound in healthy patients, and Phase Ib, which is expected to take approximately six months, will test the safety of escalating doses for 28 daily administrations in 24 depressed patients.
"We are pleased to be approved to begin testing NSI-189 in patients who suffer from depression," Karl Johe, Ph.D., Chief Scientific Officer and Chairman of Neuralstem's Board of Directors, said in a press release. "Loss of hippocampal volume is a known characteristic in depressed patients. NSI-189 stimulates neurogenesis and increases hippocampal volume in healthy adult mice, at the same time reversing behavioral symptoms in mouse depression models, so it could address depression at the source."
NSI-189 is a proprietary compound discovered by Neuralstem that stimulates new neuron growth in the hippocampus, the area of the brain that plays a role in short- and long-term memory as well as spatial navigation. Damage to the hippocampus can lead to cases of anterograde amnesia, in which a person cannot form or retain new memories, and this part of the brain is believed to be involved in MDD and other conditions, including Alzheimer's disease and post-traumatic stress disorder. The hippocampus is the first part of the brain that Alzheimer's attacks, and as Neuralstem notes on its website, "researchers now know that depressed patients have reduced hippocampal volume."
In healthy adult mice, NSI-189 stimulated neurogenesis in the hippocampus and significantly increased its volume. In mouse models of depression, the compound was shown to significantly improve behavioral responses associated with depression, and it is hoped that in humans, it will serve to reverse the hippocampal atrophy seen in MDD and its symptoms. Both the National Institutes of Health and the Defense Advanced Research Projects Agency have supported the program.
"It is exciting to see a new class of drugs that potentially offers a novel and different approach to this disease moving into patients," Maurizio Fava, M.D., Slater Family Professor of Psychiatry at Harvard Medical School and Executive Vice Chair of the Department of Psychiatry at Massachusetts General Hospital, said in a press release. Fava is one of the world's leading researchers in MDD, and helped with the design of Neuralstem's trial.
In addition to MDD, Neuralstem is also developing a cell therapy based on its technology for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, in which the death of motor neurons leads to paralysis. Neuralstem has received orphan status designation from the FDA for this trial. The company's cell therapy consists of transplantation of human spinal cord stem cells into the gray matter of a patient's spinal cord, and in preclinical animal tests, the cells made synaptic contact with the host motor neurons and also expressed neurotrophic growth factors, which are protective of cells. The company is also targeting spinal cord injury, Huntington's disease, chronic stroke and ischemic spastic paraplegia with its cell therapy platform.