Therapy to improve cognitive function in Down syndrome patients

In a study, the researchers sought to determine whether GnRH injection therapy was effective in enhancing the cognitive abilities of a small group of Down syndrome patients. In an animal model of Down syndrome, they discovered GnRH neuron malfunction and its effects on the condition's impairment of cognitive performance.

Then a pilot study later published in Science testing GnRH pulsatile injection therapy was conducted in seven patients. The results were promising : the therapy led to improved cognitive function and brain connectivity.

The scientists were able to demonstrate that restoring physiological GnRH system function restores cognitive and olfactory functions in trisomic mice. The next stage for the scientists and doctors involved a pilot clinical trial in patients to evaluate the effects of this treatment. Seven men with Down syndrome, between 20 and 50 years of age, received one subcutaneous dose of GnRH every two hours for 6 months via a pump placed on the arm. Cognition and olfactory tests as well as MRI exams were performed before and after the treatment.

From the clinical viewpoint, cognitive performance increased in 6 of the 7 patients with better three-dimensional representation, a better understanding of instructions, improved reasoning, attention, and episodic memory. These measures to improve cognitive functions were confirmed by brain imaging which revealed a significant increase in functional connectivity.

Ref:

Science, September 1, 2022, https://doi.org/10.1126/science.abq4515