A novel injection therapy could restore cognitive function in people with Down syndrome

77 percent of those who have the syndrome have symptoms that resemble those of Alzheimer’s disease as they get older. Typical neurodegenerative illnesses and gradual loss of scent are also commonly seen from the prepubertal period, with males potentially experiencing sexual maturation problems.

Restoring physiological GnRH system function

Recent research has revealed that the neurons producing gonadotropin-releasing hormone, which is known for controlling reproduction through the hypothalamus, may also have an impact on other brain regions that may be involved in cognitive processes.

With this idea in mind, the research team studied the mechanism that regulates GnRH in mouse models of Down syndrome. The results have shown five strands of microRNA, which regulate the production of this hormone, are dysfunctional. This extra chromosome leads to abnormalities in the neurons that secrete GnRH.

The Inserm researchers, who indicated the direct correlation between abnormal GnRH secretion and the mice’s growing cognitive and olfactory deficiencies, were also able to show that cognitive and olfactory functions in trisomic mice can be recovered by restoring physiological GnRH system function.

Following a protocol identical to the one used in humans, the researchers tested the effectiveness of pulsatile GnRH treatment on cognitive and olfactory deficits in trisomic mice. The team showed that mice’s olfactory and cognitive skills had returned after 15 days.

The next step was a pilot clinical trial in patients to evaluate the efficacy of the treatment. Via a pump on the arm, seven men with Down syndrome between 20 and 50 years old got one subcutaneous GnRH dose every two hours for six months. Cognitive and olfactory tests, MRI scans, and other procedures were carried out before and after the treatment.

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