Narcolepsy is a chronic neurodegenerative condition that predominantly affects children and teens, disrupting their ability to control sleep and wakefulness. Currently, treatments for narcolepsy focus on managing symptoms to improve the quality of life, as no cure exists. The condition is characterized by the loss of a specific population of neurons known as hypocretin neurons, located in the hypothalamus of the brain.
What if we could restore these lost neurons, allowing patients to regain balance and lost functions, and reduce the need for ongoing palliative medication? Our ultimate goal is to repair the brains of narcoleptic children and young adults through a groundbreaking stem cell therapy. By recreating the lost hypocretin neurons from stem cells in a dish, we aim to restore their ability to regulate sleep and wakefulness naturally.
Impact
Narcolepsy is currently managed with medications like stimulants, antidepressants, and sodium oxybate, which primarily focus on alleviating symptoms without addressing the underlying neuronal loss. By restoring the lost hypocretin neurons, this therapy could significantly reduce or eliminate the need for these ongoing medications, thereby minimizing side effects and alleviating the burden of lifelong treatment. Moreover, effectively restoring the ability to regulate sleep and wakefulness could greatly enhance social, academic, and occupational functioning for children and teens, enabling them to participate more fully in life without the constant disruption of narcoleptic episodes.
In the long term, this approach could lower the overall costs associated with managing narcolepsy, including expenses for medications, healthcare visits, and productivity losses due to the condition.
From a scientific perspective, the ability to generate specific neurons like hypocretin neurons in a dish could provide valuable insights into the mechanisms of neuronal development, function, and repair. It might also help us understand why hypocretin neurons are particularly vulnerable and prone to degeneration, potentially leading to breakthroughs not only in the treatment of narcolepsy but also in understanding the disease itself.
reNEW research
Associate Professor Agnete Kirkeby and her group has successfully generated several types of neurons from stem cells, and a joint effort between her group, and industrial and academic partners has led to the generation of a stem cell therapy for Parkinson’s disease currently in phase I trial with success. Generating a good protocol for the production of specific cell types takes time, and the hunt for the best combination of factors that generate hypocretin neurons is well underway.
Image description
A human adult brain cell (neuron) in a dish. This brain cell has underwent a journey of 50 days originating from a human embryonic stem cell, and with the addition of specific factors, has diverged into an adult neuron. A neuron consists of several specialized structures including dendrites (top part), the branching extensions of the cells body (middle part) that receive signals from other neurons, and the axon, the long single projection that extends from the body which sends electrical impulses towards other neurons, muscles, or glands. The colors represents the potential different neurons that we can make in a dish.
Credits
Louise Piilgaard, Postdoc; Anika K Mueller, PhD fellow; Agnete Kirkeby Lab; reNEW Copenhagen.
