Thursday, December 09, 2021 3:44:44 PM
The investigators were led by Kapil Bharti, Ph.D., who directs the NEI Ocular and Stem Cell Translational Research Section, and Kiyoharu (Josh) Miyagishima, Ph.D. and Ruchi Sharma, Ph.D., staff scientists in the section and leading authors of the study.
Next, using the patient-derived RPE model, they tested two potential treatment strategies: A gene therapy approach to encourage expression of normal CTRP5 in the RPE model, and the use of the diabetes drug metformin, which appears to modulate AMPK activity, re-sensitizing it to changes in cellular energy status. Both strategies prevented signs of L-ORD in RPE models.
“Importantly, we now have two potential strategies to disrupt the L-ORD disease process. While gene therapy may be years away, metformin is a drug that’s long been used to treat diabetes,” said Bharti, who with NEI collaborators is planning a clinical trial to test the drug in people with L-ORD.
https://www.nih.gov/news-events/news-releases/nih-study-traces-molecular-link-gene-late-onset-retinal-degeneration#.YbI3HelYrvs.linkedin
I think NEI stands for the National Eye Institute which is part of the NIH.
His point that "While gene therapy may be years away, metformin is a drug that’s long been used to treat diabetes" suggests to me that they do not think metformin therapy will take long to prove out in trials.
JMO
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