ALS Alert Newsletter

The Packard Center is pleased to announce a grant to Gabsang Lee, PhD and Tom Lloyd, MD, PhD, both of Johns Hopkins University, to study whether data from an unbiased lipid screening can yield information about new therapeutics to reverse ALS symptoms in cell and animal models. The Lee and Lloyd labs will study two different types of motor neurons from ALS induced pluripotent stem cells (iPSCs).

One motor neuron subtype controls body movement (vulnerable in ALS patients); the other controls eye movement, which is mostly spared in ALS. By comparing these two types of motor neurons, Lloyd and Lee have identified a mis-regulated lipid pathway. Importantly, pharmacological modulation of the lipid pathway can rescue ALS phenotypes in three different model systems (human motor neurons in a dish, fly and mouse). This project will continue their work to uncover another lipid pathway as a novel therapeutic target.

“As a new investigator in the ALS field, it was extremely challenging to receive any research funding,” says Lee. “The Packard Center has generously supported our study from the beginning and empowered us to identify a new therapeutic target.”

This project was funded in part through a grant from Wings Over Wall Street™/Muscular Dystrophy Association.