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Promising Alzheimer’s Drug Nearing Clinical Trials

J147, the molecular targeting drug, has been shown to treat Alzheimer’s disease and reverse aging in mice is nearing readiness for the clinical trials in humans. Scientists have been able to determine what exactly J147 does,reporting that the drug binds to a protein found within the mitochondria which is the energy generating powerhouse of the cells, showing that it makes the again cells, mice and flies appear more youthful as published in the journal Aging Cell.

 

Finding the target of J147 was key in being able to move forward to clinical trials. J147 was developed in 2011 after screening for compounds from plants that may have the ability to reverse the molecular and cellular signs of aging in the brain, it is a modified version of a molecule found within the curry spice curcumin, it has been shown to reverse memory defects, potentiates production of new brain cells, and to slow the progression of Alzheimer’s in mice.

 

A mitochondrial protein called ATP synthase has been identified as the molecular target of J147 that helps to generate ATP which is the cell’s energy currency within the mitochondria. It was shown that by manipulating its activity protected the neuronal cells from multiple toxicities that are associated with the aging brain. ATP synthase has previously been shown to have the ability to control aging in flies and C. elegans worms.

 

Additional studies conducted showed that modulating the activity of ATP synthase within J147 changes the levels of a number of other molecules including levels of ATP itself leading to a more stable and healthier mitochondria throughout disease and aging. The results are promising for not only moving forward with a treatment for Alzheimer’s but also as possible treatments in other age related diseases as well as it may be that targeting aging may also treat or slow down pathological conditions that are age related.

 

J147 has passed FDA required toxicology testing in animals, funding is now being sought after to begin phase 1 of human clinical trials.

 

 

Materials provided by Salk Institute for Biological Studies.

Note: Content may be edited for style and length.

Journal Reference:

Joshua Goldberg et al. The mitochondrial ATP synthase is a shared drug target for aging and dementia. Aging Cell, 2018 DOI: 10.1111/acel.12715

 

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