(This is an excerpt of the Health Rounds newsletter, where we present latest medical studies on Tuesdays and Thursdays.)
March 6 (Reuters) – New findings could help explain why Alzheimer’s dementia often progresses faster in women and may lead to fresh avenues of research and future treatments, researchers said.
Alzheimer’s disease is marked by abnormal amounts of tau protein in the brain that disrupt communication between brain cells and contribute to cognitive decline.
Some patients also have abnormal clumping of a protein associated with Parkinson’s disease called alpha-synuclein.
Among patients with Alzheimer’s disease and elevated brain levels of both proteins, brain changes occurred up to 20 times faster in women than in men, suggesting that alpha-synuclein may drive faster dementia progression in women, Mayo Clinic researchers reported in JAMA Network Open.
“When we see disease-related changes unfolding at dramatically different rates, we cannot keep approaching Alzheimer’s as though it behaves exactly the same way in everyone,” study senior author Dr. Kejal Kantarci said in a statement.
The researchers studied 415 volunteers with Alzheimer’s disease who agreed to have their brain changes tracked over time with cerebrospinal fluid testing to detect abnormal alpha-synuclein and imaging to measure changes in tau accumulation.
About 17% of participants showed evidence of abnormal alpha-synuclein.
“Recognizing these sex-specific differences could help us design more targeted clinical trials and ultimately more personalized treatment strategies,” Kantarci said.
“This opens an entirely new direction for understanding why women bear a disproportionate burden of dementia,” study leader Dr. Elijah Mak said in a statement.
“If we can unravel the mechanisms behind this vulnerability, we may uncover targets we haven’t considered before.”
DISEASE-MODIFYING TREATMENT FOR RARE CHILDHOOD SEIZURE DISORDER SHOWS PROMISE
An experimental disease-modifying drug being developed by Stoke Therapeutics and Biogen helped reduce seizures and improve quality of life for children and teenagers with Dravet syndrome, a rare but devastating genetic form of epilepsy, in early and mid-stage trials.
Children who regularly took zorevunersen for up to three years experienced up to 91% fewer seizures, with monthly episode counts falling from 17 to between 1.5 and 7, depending on the drug regimen tested, researchers reported in The New England Journal of Medicine.
Eighty-one patients ages 2 to 18 received between 10 milligrams and 70 mg of zorevunersen injected directly into the cerebrospinal fluid, either as a single dose or with additional doses two or three months later, over the course of six months.
Seventy-five of the 81 took part in second-stage trials and continued to receive the drug every four months.
Zorevunersen was generally well tolerated, and most side effects were mild to moderate, researchers found.
Dravet syndrome is notoriously hard to treat and affects about 1 in 15,000 children.
With Dravet, one copy of the patient’s two SCN1A genes fails to produce enough protein for nerve cells to function properly, resulting in developmental delays, coordination and eating problems, severe seizures, and early death.
Zorevunersen works by increasing the protein production of the patient’s healthy copy of the gene.
Larger trials of the drug are underway.
“I regularly see patients with hard-to-treat genetic epilepsies with impacts that go beyond seizures and it’s heartbreaking when treatment options are limited,” study leader Helen Cross from University College London said in a statement.
The mother of an 8-year-old named Freddie who participated in the trial said in a statement that it “has completely changed our lives.”
“We now have a life we didn’t ever think was possible and most importantly it’s a life that Freddie can enjoy.”
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(Reporting by Nancy Lapid; additional reporting by Shawana Alleyne-Morris; Editing by Bill Berkrot)
