Research into Alzheimer’s disease took another tentative step forward as scientists said that an injection may help stall the damage wrought by the mind-robbing illness.
But much like last week, when U.S. regulators cleared the first new medicine in 18 years against the advice of agency advisers, the news was muddled. Only a subgroup of volunteers in the study of a therapy from Axon Neuroscience showed slowing of the most dreaded Alzheimer’s symptom: cognitive decline.
The researchers’ main goal was to determine whether the injection was safe -- and it was. Because it didn’t help cognitive or daily functioning in patients overall, the findings require caution and will need confirmation, the scientists said.
Axon, the closely-held biotech behind the research, targets tangles of a protein called tau in the brain, which the scientists view as related to the disease’s degenerative process.
The company’s Alzheimer’s vaccine was tested in almost 200 patients with mild disease over two years. However, lead researcher Petr Novak and colleagues believe that not all patients recruited for the trial had the disease, which can be hard to distinguish from other forms of dementia. Specifically, not all tested positive for the tau protein.
“Once we cut out these patients, then we see a significant difference on cognitive and neuro-degeneration markers,” Novak said in an interview.
Big Pharma Circling
In the subgroup of 109 patients whose brains contained tau tangles, volunteers who got the drug scored better than those given a placebo on a number of markers, including one that rates dementia by assessing variables such as problem solving, memory and orientation.
Another school of Alzheimer’s research focuses on the buildup of a protein called amyloid, which clumps into plaques in patients’ brains. Amyloid plaques and tau tangles are hallmarks of the disease, but much of the scientific effort has focused on attacking amyloid. That abnormal protein is the target of the Biogen Inc. medicine that won U.S. regulators’ endorsement a week ago, becoming the first new Alzheimer’s therapy in almost two decades.
With its focus on tau, Axon has received a surge in interest from pharma companies lately, according to Chief Executive Officer Michal Fresser. The Slovakian company is now in talks with more than five potential partners for the Alzheimer’s therapy, its lead product, he said.
The U.S. decision to approve Biogen’s drug “opened some additional phone calls,” Fresser said. “After the Biogen results, the Alzheimer’s field has a lot more attractivity.”
The Axon vaccine, called AADvac1, is a type of injected immunotherapy treatment that induces antibodies against a toxic form of the tau protein typical of Alzheimer’s.
The trial results were published Monday in the medical journal Nature Aging -- almost a year after the study ended. Axon is now looking for a partner to help fund another trial with as many as 400 patients with confirmed Alzheimer’s that would start late this year or early next year.
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