LONDON: New findings may help explain why Alzheimer’s disease progresses more quickly in women and could lead to new avenues for future research and treatment, researchers say.
Alzheimer’s disease is characterized by abnormal levels of tau protein in the brain, which disrupts communication between brain cells and leads to cognitive decline.
Some patients also have abnormal clumps of a protein called alpha-synuclein that is associated with Parkinson’s disease.
In patients with Alzheimer’s who had higher levels of both proteins in their brains, women’s brains changed 20 times faster than men’s, suggesting that alpha-synuclein may contribute to faster progression of dementia in women, Mayo Clinic researchers report in JAMA Network Open.
“We cannot continue to treat Alzheimer’s disease as if it behaves exactly the same in everyone when we see disease-related changes unfolding at wildly different rates,” Dr. Kejal Kantarsi, the study’s lead author, said in a statement.
Researchers studied 415 volunteers with Alzheimer’s disease who agreed to track changes in their brains over time by testing their cerebrospinal fluid to detect abnormal alpha-synuclein and imaging changes in tau accumulation.
About 17% of participants showed evidence of abnormal alpha-synuclein.
“Identifying these sex-specific differences could help us design more targeted clinical trials and, ultimately, more personalized treatment strategies,” Kantarci said.
“This opens up a whole new direction in understanding why women bear a disproportionate burden of dementia,” study leader Dr. Elijah Mack said in a statement.
“If we can reveal the mechanism behind this vulnerability, we may uncover targets we didn’t think of before.”
Improved treatment for rare childhood epilepsy shows promising results
An experimental disease-modifying drug developed by Stoke Therapeutics and Biogen is helping children and teens with Dravet syndrome, a rare but devastating genetic form of epilepsy, to reduce seizures and improve quality of life in early- and mid-stage trials.
Researchers reported in the New England Journal of Medicine that children who took zurvonersen regularly for up to three years had a 91% reduction in seizures, from 17 to 1.5 to 7 seizures per month, depending on the drug regimen tested.
Eighty-one patients aged 2 to 18 years received 10 mg to 70 mg of zorevunersen injected directly into the cerebrospinal fluid over six months, either as a single dose or as a booster dose after two or three months.
75 of 81 people participated in the Phase 2 trial and continue to receive the drug every four months.
Researchers found Zorefunersen was generally well tolerated, with most side effects being mild to moderate.
Dravet syndrome is notoriously difficult to treat, affecting approximately 1 in 15,000 children.
In Dravet’s case, the patient’s copy of the SCN1A gene fails to produce enough protein to maintain normal nerve cell function, leading to developmental delays, coordination and eating problems, severe seizures, and premature death.
Zorefunersen works by increasing protein production of a patient’s healthy genes.
Larger trials of the drug are currently underway.
“I often see patients with hereditary epilepsy, which is difficult to treat and whose effects go beyond seizures and where treatment options are very limited,” study leader Helen Cross of University College London said in a statement.
The mother of Freddie, an 8-year-old boy involved in the trial, said in a statement that it had “completely changed our lives”.
“We are now living a life we never imagined possible and most importantly, it is a life that Freddy can enjoy.”
(Reporting by Nancy Lapid; Additional reporting by Shovana Ellen Morris; Editing by Bill Burkrot)
