Synthetic Oil Delayed ALS Symptoms In Mice: Study

A potential treatment may help delay motor neuron loss in amyotrophic lateral sclerosis, a disease brought into the spotlight by the ice bucket challenge.

AsianScientist (Sep. 26, 2016) – New research in mice brings hope that triheptanoin, a synthetic triglyceride oil, might help to delay motor neuron loss and symptoms in the inevitably fatal motor neuron disease (MND) or amyotrophic lateral sclerosis (ALS). Their findings are published in PLOS ONE.

“MND is characterized by degeneration of motor neurons in the brain and spinal cord which leads to muscle weakness, paralysis and death, usually due to respiratory failure,” said Dr. Karin Borges of The University of Queensland. “There is evidence showing the disease interrupts the process providing the primary cellular energy source necessary for cell function and survival.”

Borges’ team tested if giving oral triheptanoin could prevent cell death and symptom onset in a mouse model of MND. Triheptanoin is an investigational medicine being developed by Ultragenyx Pharmaceuticals for patients with fatty-acid metabolism disorders and glucose transporter type 1 deficiency syndrome, which leads to epileptic seizures in young children.

Her team found that motor neuron loss was reduced by a third and loss of limb strength and body weight were delayed in the animals. Their data also showed an improvement in disease symptoms when treatment was initiated prior to their onset.

“One of the interesting properties of triheptanoin is that it helps the body to extract more energy from normal food, as well as providing additional fuel. This could be very important for patients with this rapidly progressive disease,” said Borges.

Borges said that the study raises hope that triheptanoin may be able to preserve motor neuron and muscle function when treatment is started at an early stage of the disease, but she also noted that more research is needed to determine whether this treatment is effective for patients with obvious muscle or neuron impairment.


The article can be found at: Tefera et al. (2016) Triheptanoin Protects Motor Neurons and Delays the Onset of Motor Symptoms in a Mouse Model of Amyotrophic Lateral Sclerosis.

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Source: University of Queensland.
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