How many times have we heard: “This field has been thoroughly studied, and we know all there is to know; there is no reason to continue investigating, because there is nothing more to find out on the subject”? This has been the attitude of some researchers with respect to the importance of mitochondria and diseases. For more than 50 years, we have known that mitochondria, which are the “batteries” of the cell, are critical for energy production. But many believed they did not have other major roles in the health of the cell, so when it came to developing drugs against diseases, mitochondria were overlooked.
That idea has now been turned on its head.
Led by Discovery Eye Foundation (DEF) Research Director Dr. Cristina Kenney, the Mitochondria Research Group believed that, to really discover something new, you have to look in novel areas. This group has done just that. Using the transmitochondrial cybrid model, which are cell lines with identical nuclei, but with each line containing mitochondria from a different person, they have shown that the mitochondria have major regulation powers over cell behavior and expression of disease-related pathways. This is significant, because the mitochondria then become a target for therapies to combat diseases.
Kenney’s group is investigating various drugs and substances that will keep the mitochondria healthy and, ultimately, improve the health of the retinal cells in age-related macular degeneration (AMD). But it does not stop there. This same approach to developing mitochondria-targeting drugs is being pursued for drugs to treat Parkinson’s disease, Alzheimer’s disease, leukemia, various cancers, heart failure, thrombosis, stroke, diabetic retinopathy, Leber hereditary optic neuropathy, and even liver toxicity from acetaminophen.
The continued support from DEF, especially in the early stages of the mitochondria research, has fostered a new area to be opened up, specifically for AMD and diabetic retinopathy. In turn, this has allowed new collaborations among Kenney and researchers from the eye field, and laboratories studying the brain and neurodegeneration, cardiology, cancer therapies and methods to reduce side effects from cancer drugs. Kenney’s discoveries using cybrids have revolutionized the field of mitochondrial research, showing that mitochondria have wide-ranging biological effects never imagined and opening up the field of mitochondrial therapy to careful investigation.
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Lauren Hauptman
Lauren Hauptman Ink