Seattle Children’s Hospital Targets Protein Involved in Epilepsy

Photo by Marcelo Leal on Unsplash

An experienced finance executive, Warren Lammert is the CEO of Granite Point Capital in Boston, a hedge fund which manages more than $250 million in assets. His daughter was diagnosed with Dravet syndrome as a baby, which inspired him to cofound Epilepsy.com and the Epilepsy Therapy Project in the early 2000s. The Epilepsy Therapy Project subsequently merged with the Epilepsy Foundation, of which Warren Lammert is a former chairman.

In a new development in epilepsy treatment, a team based at Seattle Children’s Hospital announced the potentially life-changing effects of their experimental work targeting mTOR, a protein pathway that becomes overactive in people with the condition.

The researchers focused on altering the protein's behavior in the bodies of young people diagnosed with refractory, or treatment-resistant, epilepsy. Early results reported in 2019 showed that patients who received an initial form of this treatment displayed a noticeable improvement in symptoms.

Those who participated in a phase 1 trial received three weekly infusions of Nab-rapamycin (ABI-009), which physicians believed would block the mTOR pathway and lead to a decrease in symptoms. Nab-rapamycin is a drug developed by Aadi Bioscience.