Ana Pereira, MD

“We need a larger study to really establish better both efficacy and safety in this population, which is Alzheimer disease.”

A common approach in some trials of therapies for Alzheimer disease has been to attempt to repurpose already available agents for the neurodegenerative disease. Ana Pereira, MD, assistant professor of neurology and neuroscience, Mount Sinai Medical Center, recently led a pilot study that took this very approach with a drug familiar to those treating amyotrophic lateral sclerosis (ALS), riluzole.

This study, which included just under 50 patients, assessed the glutaminergic agent in patients with Alzheimer disease. Riluzole treatment met the primary end point—showing significantly less decline in areas of interest in the brain, as measured by fluorodeoxyglucose (FDG)-positron emission tomography (PET. As well, the second outcome measures showed a strong correlation between cognitive measures, such as the ADAS-cog, and FDG-PET brain metabolism with riluzole treatment. The findings were presented at the virtual Alzheimer’s Drug Discovery Foundation International Conference.

To find out more about why this repurposing effort was undertaken and what the clinical community should take away from these data, 

 spoke with Pereria in an interview. She explained how these data spark an interest for her to continue moving forward with its assessment and noted that a larger study will ultimately still be necessary to confirm the potential of the drug. Pereira also expressed her gratitude for the patients in the trial and their commitment to partnering in this attempt at discover

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The assistant professor of neurology and neuroscience at Mount Sinai Medical Center detailed the promise marked by the results of a recent pilot trial of riluzole in Alzheimer disease.

Ana Pereira, MD: Repurposing Riluzole for Alzheimer Disease

“Our FDG-PET measures also correlated very well with cognitive measures including the ADAS-cog…as well as the MMSE.”

Even with the excitement around the possible first approval in Alzheimer disease slated for early 2021 with aducanumab, those physicians working in this specialty, the pending action at the FDA is still lit with the backdrop of the hundreds of failed drugs that have left the treatment paradigm rather slim.

These failures have led some to look to repurpose already proven agents to explore their potential in making an impact on the disease’s progression. One such effort, led by Ana Pereira, MD, assistant professor of neurology and neuroscience, Mount Sinai Medical Center, was recently presented at the virtual Alzheimer’s Drug Discovery Foundation International Conference. This pilot clinical trial featured the therapy riluzole, which has long been used in the treatment of amyotrophic lateral sclerosis (ALS).

This study evaluated riluzole in patients with Alzheimer disease, and it met the primary end point, with fluorodeoxyglucose (FDG)-positron emission tomography (PET) measures showing significantly less decline in areas of interest in the brain. Additionally, the second outcome measure showed a strong correlation between cognitive measures and FDG-PET brain metabolism with riluzole treatment.

To find out more about the study and its findings, 

 spoke with Pereria in an interview. She offered some details on the overall findings of the study, which support a larger, phase 3 assessment, and gave her perspective on some of the more interesting aspects of the data.

The assistant professor of neurology and neuroscience at Mount Sinai Medical Center detailed the findings of a recent pilot trial of riluzole in Alzheimer disease she and colleagues conducted.

Ana Pereira, MD: Pilot Study of Riluzole in Alzheimer Disease

“The combination of the preclinical data, which was promising, made me as a clinician-scientist want to translate that into the clinic and test these hypotheses in a clinical trial.”

At the recent virtual Alzheimer’s Drug Discovery Foundation International Conference, Ana Pereira, MD, assistant professor of neurology and neuroscience, Mount Sinai Medical Center, presented data from a clinical pilot trial of riluzole in patients with Alzheimer disease. All told, the trial met its primary end point, with fluorodeoxyglucose (FDG)-positron emission tomography (PET) measures showing significantly less decline in areas of interest in the brain.

There were no signs of change in the N-acetylaspartate, as measured by magnetic resonance spectroscopy Although, the second outcome measure showed a strong correlation between cognitive measures and FDG-PET brain metabolism with riluzole treatment. The results are still not enough to recommend off-label use, however, Pereria and colleagues noted.

Pereira and colleagues concluded that the data support a larger and longer clinical trial to evaluate riluzole’s potential as a treatment in Alzheimer. This work, Pereria told 

, was influenced by a number of data indicating that blocking glutamatergic neurotransmission may offer a therapeutic approach to the disease. Pereira expanded on what these influences were and what ultimately prompted her to study the agent in this interview.

The assistant professor of neurology and neuroscience at Mount Sinai Medical Center discussed the literature that influenced a pilot trial of riluzole in Alzheimer disease.

Author | Ana Pereira, MD

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