Stories about: rapamycin

Another use for mTOR inhibitors: Preserving vanishing bones in Gorham-Stout syndrome

Gorham Stout rapamycin sirolimus
In Gorham-Stout, lymphatic vessels gone amok eat away at bone. Sirolimus appears to reverse this process.

The mTOR pathway is fundamental to nearly every cell in the body. It drives processes related to cell growth, protein production and metabolism, influencing everything from neurocognition to tumor growth. Because of this broad role, indications for drugs targeting the mTOR pathway are also remarkably broad.

Alexander Malloy, 14, is one of the first patients to benefit from a new use: curbing rapid bone loss in patients with a rare “vanishing bone disease,” or Gorham-Stout syndrome. It was discovered when Alex, who had mild scoliosis, started getting worse. To his parents’ shock, an MRI scan showed he was missing bones in his spine.

Gorham-Stout is actually the result of a rare vascular anomaly.

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Preventing autism after infant seizures

Early seizures may disrupt circuit formation in babies' brains, leading to autism. But new research suggests that an existing drug can reverse this.

This is the third post in a series about new approaches for seizures and epilepsy. Read the first and second posts.

We already know that there’s some kind of connection between epilepsy and autism: Children who have seizures as newborns not uncommonly develop autism, and studies indicate that about 40 percent of patients with autism also have epilepsy. New research at Boston Children’s Hospital finds a reason for the link, and suggests a way to break it — using an existing drug that’s already been given safely to children.

In the online journal PLoS ONE, Frances Jensen, MD, in the Department of Neurology and the F.M. Kirby Neurobiology Center at Boston Children’s, and lab members Delia Talos, PhD, Hongyu Sun, MD, PhD, and Xiangping Zhou, MD, PhD, showed in a rat model that early-life seizures not only lead to epilepsy later in life, but also produce autistic-like behaviors.

Drilling deeper, they showed that early seizures hyper-activate a group of signaling molecules collectively known as the mTOR pathway.

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Is rapamycin the new aspirin?

rapamycin
Easter Island, home of rapamycin (Ndecam/Flickr)

I’ve heard it said that if aspirin had to go through today’s FDA approval process, it would never be approved for over-the-counter use because it just does so many things. Lately, it’s been hard to cover biomedical research at Children’s without stumbling on another drug that’s also FDA-approved and also seems to have multiple uses: rapamycin.

It’s a drug that targets a pathway fundamental to nearly every cell in the body, yet is seemingly good for nearly everything. But how can one drug touch on so many cells and tissues and organs and still be both effective and safe?

First found in the 1960s in soil bacteria collected on Easter Island (the drug’s name comes from the island’s native name, Rapa Nui), rapamycin is a naturally derived antibiotic, antifungal and immunosuppressant.

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