New Uses for Old Drugs

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  • It typically takes ten to fifteen years to get a new drug to market not to mention the billions of dollars invested. This herculean effort is how the COVID vaccines were developed thanks in part to the years of research done on RNAs before we needed it.

    But not all drugs are brand new. Oftentimes we repurpose a drug for a different disease and the benefits are enormous. It's cheaper and the timeline is cut since it's already been approved.

    How then do drug companies decide which drugs might work on other diseases? Just read the fine print for a drug's side effects because those are the places in the body that the drug affects and are good targets of study.

    Recently, two drugs gemfibrozil and retinoic acid were combined to treat Alzheimer's. Gemfibrozil is a precursor to statins for lowering cholesterol. Retinoic acid is widely used to treat cancer. New studies show combining them impacts brain cells called astrocytes, which could be contributing to amyloid plaque build-up in Alzheimer's patients.

    In studies, the two drugs reduce this plaque build-up and improve cognitive function. Mouse studies show retinoic acid stimulates astrocytes to take up and destroy amyloid beta. And this activity is stimulated by the second drug, gemfibrozil. Both drugs appear to also unleash the PPAR-alpha protein which also eliminates amyloid beta.

    If approved for Alzheimer's, these drugs will cost patients just pennies on the dollar.

More Information

Has a treatment for Alzheimer's been sitting on pharmacy shelves for decades? Scientists have two possible candidates
Two drugs approved decades ago not only counteract brain damage caused by Alzheimer's disease in animal models, the same therapeutic combination may also improve cognition...

Activation of PPAR? enhances astroglial uptake and degradation of ?-amyloid
Astrocytes are a type of glial cell that are activated in the brain tissue of patients with Alzheimer's disease to induce the accumulation of amyloid (A?). We previously found that a combination of low-dose gemfibrozil (GFB; a drug approved to treat high cholesterol) and retinoic acid (RA; a vitamin A derivative) induces lysosomal bio-genesis through peroxisome proliferator-activated receptor ? (PPAR?)-mediated transcription of the gene encoding transcription factor EB (TFEB), a master regulator of lysosomal biogenesis and autophagy. Here, we found that the same combination (GFB-RA) enhanced the uptake of A? from the extracellular space and its subsequent degradation in astrocytes through a PPAR?-dependent pathway...