A new hormone treatment improved the cognitive function of six men with Down syndrome by 10-30 percent, scientists said Thursday, adding that the “promising” results may raise hope for improved quality of life of the patients.
However, the scientists stressed that the small study did not point toward a cure for cognitive disorders in people with Down syndrome and that much more research is needed.
“The experiment is very satisfying, even if we remain cautious,” said Nelly Pitteloud of the Lausanne University Hospital in Switzerland and co-author of a new study in the journal Science.
According to the World Health Organization, Down syndrome is the most common genetic form of intellectual disability, occurring in about one in 1,000 people.
However, previous research has not significantly improved cognition when applied to people with the condition, so the latest findings are “particularly important,” the study said.
Recent findings have suggested that how gonadotropin-releasing hormone (GnRH) is produced in the brain may affect cognitive functioning such as memory, language and learning.
GnRH hormones regulate the amount of testosterone and estrogen that are produced and the increased levels help stimulate puberty.
“We wondered if this hormone could play a role in establishing the symptoms of people with Down syndrome,” said Vincent Prevot, co-author of the study and head of neuroscience research at the French institute. INSERT.
Mice research
The team established for the first time that five microRNA strands that regulate GnRH production were dysfunctional in mice specifically engineered for Down syndrome research.
They then showed that cognitive impairments, as well as loss of smell, a common symptom of Down syndrome, were linked to dysfunctional GnRH secretion in the mice.
The team then gave the mice a GnRH drug used to treat low testosterone and delay puberty in humans, finding that it restored some cognitive function and the sense of smell.
A pilot study was carried out in Switzerland with seven men with Down’s syndrome between the ages of 20 and 50.
Each received the treatment through the arm every two hours over a six-month period, with the drug delivered in pulses to mimic the frequency of the hormone in people without Down syndrome.
During the treatment, cognition and smell tests were performed, as well as magnetic resonance scans.
Six of the seven men showed improvement in cognition without significant side effects, but none showed a change in their sense of smell.
“We saw a 10 to 30 percent improvement in cognitive functions, particularly with visuospatial function, three-dimensional representation, understanding instructions, and attention,” Pitteloud said.
Patients were asked to draw a simple 3D bed at various stages throughout therapy. Many struggled at first, but in the end the efforts were noticeably better.
Improve the quality of life
The authors acknowledged some limitations of the study, including its size and that the patients’ choice was “pushed by their parents.”
“The clinical trial only focused on seven male patients; we still have a lot of work to do to prove the effectiveness of GnRH treatment for Down syndrome,” Pitteloud said.
A larger study with a placebo and between 50 and 60 patients, a third of them women, is expected to begin in the coming months.
“We will not cure the cognitive disorders of people with Down syndrome, but the improvement observed in our results already seems fundamental enough to expect to improve their quality of life,” Pitteloud said.
Fabian Fernandez, an expert on cognition and Down syndrome at the University of Arizona who was not involved in the research, praised the “powerful study.”
He told AFP that while it was “difficult to imagine” how such an intensive treatment could be used for young people, it might be better suited to delay the Alzheimer’s-related dementia that many adults with Alzheimer’s syndrome suffer from. Down.
It was also difficult to predict how this improvement might affect the lives of people with the disease, he said.
“For some, however, it could be significant as it would allow them to be more independent with activities of daily living, such as maintaining and enjoying hobbies, finding belongings, using household appliances and traveling alone.”
© Agence France-Presse