A failed fragile X syndrome drug can reduce cocaine use, clinical trial finds

A team of pharmaceutical researchers at Novartis Biomedical Research, working with an international team of associates, has found that the drug mavoglurant can reduce use in people with cocaine use disorder. In their study, published in the journal Science Translational Medicine, the group conducted a Phase II clinical trial with 68 people with a diagnosed cocaine use disorder.

Mavoglurant was first developed by a team at Novartis as a treatment for fragile X syndrome, which is a genetic neurodevelopmental condition that usually leads to some degree of intellectual disability. Trials for the drug were disappointing, prompting Novartis to explore other possible uses.

Testing explored its use as a treatment for Levodopa-induced dyskinesia (a type of dyskinesia associated with the prescription of L-Dopa for Parkinson’s disease), reducing smoking withdrawal symptoms or even OCD. Eventually, the team hit on the idea of using it to reduce craving symptoms in people with alcohol and cocaine use disorders. Initial testing showed some success, leading to a successful Phase I clinical trial.

In their Phase II trial, the research team recruited 68 people previously diagnosed with cocaine use disorders to take either a placebo or a single dose (one pill) of mavoglurant twice a day for 98 days. Use of cocaine by the participants was self-reported and tested via urine and hair throughout the trial.

The research team found that use of the drug led to “significant reductions” in cocaine use compared to the placebo in most of the participants. The researchers also found that the medication led to a decrease in alcohol use by most of the participants, although this reduction in alcohol use was not statistically significant. Side effects included dizziness, nausea and headache, though the participants who experienced them described them as manageable. The researchers describe the testing outcome thus far as effective, safe and tolerable.

If the drug passes all its trials and at some point is approved by the FDA and other medical agencies, it would represent the first drug to be marketed as a therapy for treating cocaine abuse. Current treatments involve psychosocial sessions such as cognitive-behavioral therapy and counseling, which, to date, have proven to be far less effective than desired.

© 2025 Science X Network

A team of pharmaceutical researchers at Novartis Biomedical Research, working with an international team of associates, has found that the drug mavoglurant can reduce use in people with cocaine use disorder. In their study, published in the journal Science Translational Medicine, the group conducted a Phase II clinical trial with 68 people with a diagnosed cocaine use disorder.

Mavoglurant was first developed by a team at Novartis as a treatment for fragile X syndrome, which is a genetic neurodevelopmental condition that usually leads to some degree of intellectual disability. Trials for the drug were disappointing, prompting Novartis to explore other possible uses.

Testing explored its use as a treatment for Levodopa-induced dyskinesia (a type of dyskinesia associated with the prescription of L-Dopa for Parkinson’s disease), reducing smoking withdrawal symptoms or even OCD. Eventually, the team hit on the idea of using it to reduce craving symptoms in people with alcohol and cocaine use disorders. Initial testing showed some success, leading to a successful Phase I clinical trial.

In their Phase II trial, the research team recruited 68 people previously diagnosed with cocaine use disorders to take either a placebo or a single dose (one pill) of mavoglurant twice a day for 98 days. Use of cocaine by the participants was self-reported and tested via urine and hair throughout the trial.

The research team found that use of the drug led to “significant reductions” in cocaine use compared to the placebo in most of the participants. The researchers also found that the medication led to a decrease in alcohol use by most of the participants, although this reduction in alcohol use was not statistically significant. Side effects included dizziness, nausea and headache, though the participants who experienced them described them as manageable. The researchers describe the testing outcome thus far as effective, safe and tolerable.

If the drug passes all its trials and at some point is approved by the FDA and other medical agencies, it would represent the first drug to be marketed as a therapy for treating cocaine abuse. Current treatments involve psychosocial sessions such as cognitive-behavioral therapy and counseling, which, to date, have proven to be far less effective than desired.

© 2025 Science X Network