Synthetic oil-based drug could slow ALS symptoms


A drug made from a kind of synthetic oil may help to delay the destruction of motor neurons in amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), a disease brought into the spotlight by last year’s popular ice bucket challenge, researchers in Australia said.

In a study published in the journal PLOS ONE, researchers at the University of Queensland found 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), after it had positive results in testing on mice.

“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 experimental 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,” according to a description on the company’s website.

Dr. Borges’ research team found that motor neuron loss was reduced by a third and loss of limb strength and body weight were delayed in the mice treated with the drug. 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 added 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.


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