A new, possibly out of a range, study is underway that holds promise for improving cognitive abilities in people with Down syndrome, Science reports..

While still preliminary work, it may represent a step towards a goal that has eluded scientists for decades..

Down syndrome is the most common genetic cause of mental retardation, affecting about one in 600 children.. It occurs when a person is born with an extra copy of chromosome 21, which ultimately results in distinctive physical features, an increased risk of many health problems, and mild to moderate mental impairment..

Many people with Down syndrome do well, especially through access to therapy, education, and health care.. But researchers are still looking for drugs that could reduce differences in learning and communication skills and help more people with Down syndrome live independently..

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Previously, many drugs have shown promising results in mice, none in human trials has shown a significant improvement in cognition..

In a new study, researchers looked at a protein called gonadotropin-releasing hormone (GnRH). It has long been known as a major regulator of reproduction and has been widely used in the treatment of infertility..

This hormone has recently been found to play an important role in brain development.. If GnRH-secreting neurons do not develop properly, they can cause infertility, impair the sense of smell, and also affect the development of people with Down syndrome.. GnRH in particular affects the development of speech and other cognitive abilities in infants and toddlers and is critical for the formation of brain connections during adolescence..

Vincent Prevost, a neuroendocrinologist at the University of Lille, wondered if low GnRH levels during early development might play a similar role in Down syndrome..

To find out, he and his team conducted a series of experiments on mice that were genetically modified to have an extra chromosome similar to that found in people with Down syndrome.. They tested the memory and sense of smell of rodents and found that both deteriorate after puberty.. The group found that the mice also had abnormalities in GnRH-secreting neurons..

The team was able to restore GnRH production in cells using microRNAs—strands of RNA that act as switches for gene expression—and reverse smell and memory deficits in rodents.. When they gave the drug Lutrelef, commonly used to replace GnRH in humans, to mice with Down syndrome, their ability to remember different objects and distinguish smells was consistent with that of normal mice after 2 weeks of treatment..

“At this point, I was very excited and thought that we should try to move to people,” says Nelly Pittelow, a neuroendocrinologist at the University Hospital of Lausanne.. She and Prevost teamed up for a small pilot study of seven men with Down syndrome aged 20 to 50..

Participants and their legal guardians agreed to the study, which involved administering Luthrelef through a small needle and pump taped to their shoulders.. The pump delivered the drug every 2 hours, mimicking the body's natural release of the hormone.. After 6 months, the men showed a 10-30% improvement on the Montreal Cognitive Test, a standard measure of mental retardation.

However, discussing treatment can cause mixed feelings in the Down syndrome community, especially when it is presented as a disease that needs to be “cured,” says Kathleen Small, director of family services and medical care for the nonprofit Down Syndrome Connection and mother of a child with Down syndrome.. According to her, improving memory or communication skills is unlikely to change the personality of patients.. It will just improve their quality of life..

However, some researchers warn that high levels of certain hormones, such as GnRH, may also increase the risk of cancer.. In particular, Brian Scottko of Massachusetts General Hospital made such a warning, which can be especially dangerous for people with Down syndrome, who are already notoriously at higher risk of leukemia..

Pittelaud and Prevost are currently recruiting 32 men and women with Down syndrome for a placebo-controlled trial with Lutrelef. While the improvements in cognitive scores in their pre-test were small, Pittelaud says parents reported significant differences — for example, some found it easier to talk to their sons on the phone. Others have seen improvements in concentration and memory, which can help with everyday tasks like navigating the city, says Pitteludd.. According to him, the main goal of the study is to see an improvement in the quality of daily life of people with Down syndrome..

Important! This publication is based on the latest and current scientific research in the field of medicine and is for general information purposes only.. Publication cannot be the basis for establishing any diagnoses. If you are ill or need a diagnosis, see your doctor!