Potential Gaucher Treatment Lucerastat Well Tolerated in Early Clinical Trials

Potential Gaucher Treatment Lucerastat Well Tolerated in Early Clinical Trials

The drug compound lucerastat, a potential treatment for conditions such as Gaucher disease, is well-tolerated overall and does not seem to cause any serious adverse side effects, according to a study conducted by scientists at Actelion Pharmaceuticals.

They also found that increasing the dose of the drug doesn’t seem to increase the occurrence of adverse side effects.

The report, “Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects,” was published in the Orphanet Journal of Rare Diseases.

“These results warrant further investigation of substrate reduction therapy with lucerastat in patients with glycolipid storage disorders,” Dr. Nicholas Guérard and co-authors wrote.

Substrate reduction therapy is an approach where the production of the fatty substances that build up in Gaucher disease is blocked by certain chemicals. Lucerastat is one such chemical and works by blocking the activity of the enzyme producing these fatty substances, therefore restoring the balance of these compounds in the body.

The researchers evaluated the safety, tolerability, and pharmacokinetics (or movement inside the body) of lucerastat in healthy volunteers in two separate randomized and double-blind placebo-controlled clinical trials.

The first trial (NCT02944487) used a single ascending dose (SAD) of the compound, while the second (NCT02944474) used multiple ascending doses (MAD).

For the SAD study, 31 participants received either a placebo or a single dose of 100, 300, 500, or 1000 mg of lucerastat. Eight more people received two doses of 1000 mg of lucerastat or a placebo separated by 12 hours.

For the MAD study, 37 participants received either a placebo or 200, 500, or 1000 mg of lucerastat twice a day for seven consecutive days.

The researchers then collected blood and urine samples from the participants to determine the concentration of lucerastat. They found that the maximum observed levels of lucerastat in the blood were comparable between studies. Similarly, the time necessary for the maximum level of the drug in blood to be reached was within the same range across dose groups in both studies.

In the SAD study, the researchers recorded 15 adverse side effects in 10 people, and in the MAD study, they recorded 18 adverse side effects in 15 people. These included fatigue, constipation, back pain, cough, hot flushes, headache, and somnolence, but none were severe or serious. The team did not observe any clinically relevant abnormalities in the vital signs of the participants.

The authors concluded that lucerastat is well tolerated overall and that the incidence of drug-related adverse side effects does not increase with dose. The next step is to test the drug compound in people with lipid storage disorders such as Gaucher disease.

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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

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