Epilepsy

Current treatment options for epilepsy only work for 2 out of 3 people. Furthermore, these treatments only help manage the symptoms and often do not address the underlying cause of seizures. This highlights the importance of identifying alternative approaches.

Stem cell research for epilepsy is still in its early stages, but there are two main ways in which stem cells could be used for the treatment and/or study of epilepsy:

1. Transplantation of interneurons (These are a population of nerve cells that enable communication between various cells in the nervous system .) Some of the neurons in our CNS are “excitatory” while others are “inhibitory.” The balanced function of each of these cell types is critical for proper functioning. During a seizure, this balance gets thrown off. Stem cell research has the potential to reduce seizures by restoring this balance. In animal studies, this approach was found to increase inhibition and reduced seizures. These results are promising, but more research is needed before this approach can be tested on people with epilepsy.
2. Induced pluripotent stem cells (iPSCs)
   1. Some epilepsies are caused because of a genetic mutation or change in neurons, as in Dravet Syndrome, a devastating form of childhood epilepsy caused by a mutation in a specific sodium channel.
   2. Stem cell researchers have found a way to take skin or blood or other adult cells from people with Dravet Syndrome and program them to be made into neurons that can be used to study the effects of specific drugs. This approach essentially generates a “disease in a dish” that will enable scientists to better understand the mechanisms involved in the development of epilepsy.

• Dr. Cory Nicholas and his team at Neurona Therapeutics are working with CIRM funding on the development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy, a type of drug-resistant chronic temporal lobe epilepsy. The product candidate is aimed at reducing and even eliminating seizures for people with epilepsy by secreting the inhibitory neurotransmitter GABA in order to rebalance neural electrical activity in the brain.
• Dr. Arturo Alvarez-Buylla and his team at UCSF have developed a cell-based therapy to modulate abnormal brain activity in the treatment of epilepsy. The transplanted cells have been shown to differentiate into inhibitory neurons in animal models and hold great promise. Further research is needed to bring this therapy to humans.
  • This research takes advantage of an established multi-lab collaboration between basic scientists and clinicians, along with the advice of neurosurgeons, epilepsy neurologists and laboratory experts on animal behavior.