2 Drugs Identified: They Could Halt the Progression of All Neurodegenerative Diseases

Several years ago, a team of scientists identified a pathway that caused brain cell death in mice. Today, the same team has discovered two other drugs that can block this pathway and avert neurodegeneration.

These drugs caused minor side effects in mice and one of the meds is already licensed for human use and is ready for clinical trials. Misfolded proteins are known to build up in the brain in several neurodegenerative illnesses. 

They’re also a major contributing factor to dementias like Parkinson’s and Alzheimer’s, as well as prion diseases. 

What Did the Team Discover During Their Research?

Previously, the team discovered that the accumulated misfolded proteins in mice with prion disease tend to make the natural defense mechanism overactive by switching off the essential production of new proteins in the brain cells.

What’s more, they discovered that restoring protein production using an experimental drug helped cease the process of neurodegeneration. But, the problem was that the tested drug was toxic to the pancreas and as such, it wasn’t suitable for human testing.

In the new study published in the journal Brain, the team tested 1,040 compounds from the National Institute for Neurological Disorders and Stroke, first in worms which have a functioning nervous system and are a suitable experimental model for drug screening for use on the nervous system, and then in mammalian cells. 

This unveiled different candidate compounds that the scientists could test in mice models of prion disease and a type of familial tauopathy (frontotemporal dementia-FTD), both of which have been protected by the experimental yet toxic compounds in the previous studies conducted by this team.

2 Drugs That Could Play a Role in the Prevention of Neurodegenerative Decline

The team identified two drugs that successfully restored protein production in mice. One is trazodone hydrochloride, a licensed antidepressant, and the other is dibenzoylmethane, a compound being trialed as a drug for the treatment of cancer.

Both of these drugs were successful at preventing the occurrence of the signs of brain cell damage in most of the prion-ill mice and also restored memory in the FTD mice. In both mice models, the drugs lowered brain shrinkage that’s associated with neurodegenerative disease. 

According to the head of the team, Professor Giovanna Malluci, they know that trazodone is safe for use in humans, so a clinical trial would be possible to test if the drug’s protective effects on brain cells in mice with neurodegeneration will also apply to humans in early stages of dementias. 

Malluci notes that in two to three years, they could find out if this approach helps slow down the progression of the disease. This may be the exciting first step toward the treatment of these disorders. 

She goes on to add that trazodone has been used for the treatment of symptoms of patients in later stages of dementia so they know it’s safe for this group. The goal is to find out if giving this drug to patients at early stages may help slow down the disease.