Epigenetics Could Be the Key to Progress on Lou Gehrig’s Disease


On July 4, 1939, one of the greatest baseball players of all time, Lou Gehrig, stood before a crowd at Yankee Stadium and uttered this famous line: “Today I consider myself the luckiest man on the face of the earth.”

The tragically ironic declaration was part of Gehrig’s farewell speech. The beloved slugger was retiring from baseball after being diagnosed with a disease that killed him less than two years later.

Amyotrophic lateral sclerosis (ALS), which came to be known as Lou Gehrig’s disease, was first recognized 150 years ago. But it remains incurable. It’s a neurodegenerative disease that slowly destroys the motor neurons, gradually taking away a patient’s ability to eat, speak, move, and breathe.

In a recent review article, scientists emphasized the importance of a field within ALS research, one that could someday lead to new treatment options: epigenetics. Growing evidence shows that epigenetics — changes to gene expression that don’t modify the DNA code — play a role in the disease. Because epigenetic markers can be targeted with pharmaceuticals, this research could eventually lead to new ALS therapeutics.

The article, authored by Professor Mariana Torrente (Brooklyn College, The Graduate Center), Ph.D. students Seth Bennett and Samantha Cobos, and research specialist Royena Tanaz, appears in the journal Translational Research.

Epigenetic markers are like an extra layer of genetic instructions on top of DNA. One example is histone modification: changes to the protein “spools” around which DNA winds to form a compact package.

“Epigenetic mechanisms are reversible,” Torrente said. “If there is an epigenetic problem, you can use a small molecule or peptide drug to counteract the effect and bring it back to normal.” This has already been done in research for cancer treatments, Torrente noted. The two currently approved ALS drugs can extend a patient’s lifetime but do not stop the disease’s progress or reverse its symptoms.

Research on epigenetics in the context of ALS is fairly new — only four or five years old, Torrente said. This young field is the focus of her lab, where they are now investigating histone modifications in ALS, both in yeast models and human cell lines.

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Epigenetics in amyotrophic lateral sclerosis
Translational Research, 2019

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Mariana Torrente (Assistant Professor, Chemistry, Biology) | Profile 1 | Profile 2