SEATTLE, Wash. (Ivanhoe Newswire) — The CDC estimates 470,000 children in America live with epilepsy. For nearly half of those kids, multiple drugs and brain surgery have had little to no effect. Researchers in Seattle are testing a different kind of drug, and they’re excited about early results.
Nineteen-year-old Shanahan Dameral has had epileptic seizures for 14 years, despite taking five anti-seizure meds daily and having two brain surgeries.
Dameral explained, “You know that feeling like when somebody tips your chair back and you get that feeling? Just like that.”
Doctors Russ Saneto and Jason Hauptman theorized that targeting a protein pathway called mTOR could help. It’s overactive with epilepsy. Nab-rapamycin inhibits the pathway.
Jason Hauptman, MD, PhD, a Pediatric Neurosurgeon from Seattle Children’s Hospital said, “Our thought is that by changing the way this protein acts in these children with epilepsy, we can, in turn, change their epilepsy.”
Dameral was in the phase one trial-three infusions, once a week. His mom noticed changes.
“The seizures were definitely shorter. They were much different and much more manageable,” said Linley Allen, Dameral’s mom.
“He didn’t have any side effects except for one bloody nose. At the end of five weeks, he didn’t have any more seizures,” said Russ Saneto, DO, PhD, Professor of Neurology at Seattle Children’s Hospital.
Linley says he’s had one to three seizures a month since the trial, compared to four a week before.
Dameral said, “It lessened the seizures. It did that, which was a thrill, to be honest.”
The researchers at Seattle Children’s Hospital are encouraged and looking ahead to the next trial.
Dr. Hauptman said, “We’re going for improvement, and even that would be a win in my book, for these children. And we’re hoping for a cure.”
A slightly different version of nab-rapamycin is approved for other diseases, which could expedite getting it to the public after trials. Seattle Children’s is recruiting for another phase one trial, hoping to launch phase two in the next year or two.
You can get more information on the trial here: Renée Rivers at 206-987-1697 or firstname.lastname@example.org
Contributors to this news report include: Wendy Chioji, Field Producer; Bruce Maniscalco, Videographer; Cyndy McGrath, Supervising Producer; Roque Correa, Editor.
To receive a free weekly e-mail on Medical Breakthroughs from Ivanhoe, sign up at: http://www.ivanhoe.com/ftk
TOPIC: EPILEPSY DRUG TRIAL SLOWS SHANAHAN’S SEIZURES
REPORT: MB #4576
BACKGROUND: Epilepsy is a chronic disorder, the hallmark of which is recurrent, unprovoked seizures. A person is diagnosed with epilepsy if they have two unprovoked seizures (or one unprovoked seizure with the likelihood of more) that were not caused by some known and reversible medical condition like alcohol withdrawal or extremely low blood sugar. The seizures in epilepsy may be related to a brain injury or a family tendency, but often the cause is completely unknown. Many people with epilepsy have more than one type of seizure and may have other symptoms of neurological problems as well. Sometimes EEG (electroencephalogram) testing, clinical history, family history, and outlook are similar among a group of people with epilepsy. In these situations, their condition can be defined as a specific epilepsy syndrome.
TREATMENT: Most authors report that antiepileptic drugs provide complete control for more than half of all patients with epilepsy and reduce the number of seizures in another 20 to 30 percent. The remaining 20 percent do not respond to current medications and their condition is termed “intractable” or “refractory to treatment.” Some of these people may have other treatment options, such as surgery, or the ketogenic diet. Reports in the medical literature suggest between 75 and 80 percent of patients with idiopathic, generalized epilepsy have reliable, long term control of seizures on currently available drugs. Some patient surveys, however, show larger percentages of people continuing to have seizures, although they may describe their condition as being under control.
NEW RESEARCH: According to Seattle Children’s Hospital, Nab-rapamycin is designed to target mTOR, an overactive protein implicated in a number of childhood brain disorders. Given in conjunction with existing antiepileptic drugs, the hope is that Nab-rapamycin will decrease seizure severity by reducing the activity of mTOR. Another mTOR inhibitor similar to Nab-rapamycin has been used in the clinic to safely treat a seizure disorder called tuberous sclerosis complex (TSC). This drug improves seizures in children with TSC by targeting a known genetic mutation, which causes hyperactivity in the mTOR pathway. Up to 18 children and young adults between the ages of 3-26 will receive Nab-rapamycin as part of the first phase of this research. Researchers anticipate that results from this trial will inform a larger, multi-center study, reaching children beyond Seattle Children’s in the next two to three years.
FOR MORE INFORMATION ON THIS REPORT, PLEASE CONTACT:
If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at email@example.com