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New ALS Drug Formula Shown Active, Safe

Neurons

(commonfund.nih.gov)

29 May 2018. A new formulation of a current drug to treat amyotrophic lateral sclerosis, or ALS, shows in its first clinical trial the drug produces more active ingredients against ALS than the current drug and causes no safety concerns. Results of the study, conducted among healthy volunteers, were reported by Treeway BV, a biotechnology company in Rotterdam, The Netherlands.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, the muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure. There are currently no cures for ALS, and few effective treatments for slowing progression of the disease.

One of the currently approved treatments for slowing the advance of ALS is edaravone, marketed under the brand name Radicava by Mitsubishi Tanabe Pharma America. Edaravone is an antioxidant that reduces oxidative stress that contributes to the damage to neurons in people with ALS. Radicava is given as an infusion each day for 14 days, followed by periodic daily infusions for 10 days at a time, and requiring a clinician to administer the drug. Radicava was first approved for sale in Japan, then approved by the Food and Drug Administration in May 2017 for marketing in the U.S.

Treeway developed an oral version of edaravone, code-named TW001, designed as an extended-release drug given in a single dose for faster and easier administration. The clinical trial enrolled 18 healthy volunteers, not ALS patients, to test the drug for adverse effects, but also to measure the concentrations of edaravone in the participants’ bodies. The company says TW001 in doses of 140 milligrams was well tolerated and participants experienced no safety concerns. In tests of Radicava, ALS patients most often reported bruising and gait disturbances, but also reactions such as hives, swelling, and shortness of breath that required medical attention.

The volunteers also tested 1 hour infusions of edaravone, with the study team measuring the availability of active ingredients of edaravone in the bodies of participants for both forms of the drug. Treeway says TW001 was available in higher proportions among the participants than the infused form of edaravone. The company says the results of the trial will help it plan a late-stage clinical study testing TW001 among ALS patients.

Treeway is a 6 year-old enterprise founded by two ALS patients, Bernard Muller and Robbert Jan Stuit. The company is also developing a gene therapy as a neuroprotective treatment for ALS still in preclinical stages. Muller and Stuit are also founders of Project MinE that seeks to better understand genetic causes of ALS. Project MinE, in which Treeway takes part, aims to analyze the genomes of 15,000 people with ALS and 7,500 people without the disease to find the genes most responsible for the disorder.

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