A critical issue to consider for potential regenerative therapies for Alzheimer's is the ongoing and progressive burden of brain cell death caused by the disease.
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According to Roberta Brinton, Ph.D., of the University of Southern California, both aging and Alzheimer's disease are characterized by a decline in the ability of the body (including the brain) to self-renew and repair, but the capacity for regeneration is retained, albeit at a decreased level.
Allopregnanolone (also known as Allo) is a neurosteroid found in the brain and bloodstream.
In previous studies, it has shown promise as a potential regenerative therapy to promote brain cell creation and improve cognitive function in older animals and animal models of Alzheimer's disease.
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Allopregnanolone Regenerative Therapy to Begin Phase 1 Trials
At Alzheimer's Association International Conference 2013, Brinton reported the design of a Phase 1, multiple ascending dose, clinical trial of Allo in participants diagnosed with MCI due to Alzheimer's and mild Alzheimer's, with doses administered once-per-week for 12 weeks to establish a safe and tolerated dose. Because Allo is naturally expressed in the brain and reaches relatively high levels during the third trimester of pregnancy, the scientists were able to advance beyond the time limits of a typical first stage of safety testing.
Secondary goals of the trial include assessing potential short-term effects of Allo dosing on cognition and MRI indicators of AD and informing a subsequent Phase 2 proof of concept trial with MRI-based biomarkers of regenerative efficacy.
"Allopregnanolone is a well-characterized agent with a very promising track record of promoting neural stem cell generation and restoring cognitive function in animal models of Alzheimer's," said Brinton. "We consider Allopregnanolone a first in class regenerative therapeutic for MCI and Alzheimer's. Our hope is that, through further research, we will add Allo to the roster of Alzheimer's treatments.
"A critical issue to consider for potential regenerative therapies for Alzheimer's is the ongoing and progressive burden of brain cell death caused by the disease. It is not sufficient solely to generate new neurons and to promote their survival; it is necessary to reduce the ongoing burden of pathology for there to be long-term benefits for cognition and function," Brinton added. "We were very encouraged to discover that Allo reduced the burden of Alzheimer's pathology. Our latest findings are very exciting as they show that Allo increases the energy capacity of the brain. This is important because the generation of new neurons, new synaptic circuits and synaptic transmission all require substantial energy."Related Content
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