Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases

Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such as Rett syndrome, Alzheimer’s, Parkinson’s, and other disorders of the central nervous system. It was initially indicated for the treatment of frontotemporal dementia.

About Rett Syndrome

Rett syndrome is a severe, rare neurological disorder that primarily affects females. It was once thought to be a form of autism, but has since been distinguished as its own disorder. It is a genetic disorder, with a mutation occurring on the X chromosome. The exact location of this mutation and its effects are unknown, but researchers do know that it is typically a sporadic mutation.

Symptoms of this condition usually appear between the first 12-18 months of life. Effects include slowed brain growth, a small head, issues with muscle coordination, social anxiety, lack of language skills, seizures, uncoordinated breathing, and a tense or irritable disposition.

After these symptoms are noticed, doctors will conduct a clinical examination and rule out other conditions, such as autism. Genetic testing will be used to confirm, and it can also show the severity of a specific case. There is no cure for Rett syndrome, and treatment consists of symptom management. Doctors will suggest physical, speech, and occupational therapy. They may also prescribe anti-seizure medications.

About Alzheimer’s Disease

Alzheimer’s disease is a progressive disorder that causes the degeneration and death of brain cells. It does occur in stages, with severity increasing as time goes on. In the later stages of the disease, people will experience significant memory loss and be unable to carry out everyday tasks.

The major symptom of this condition is memory loss. Other issues that people experience include problems with thinking and reasoning, making judgments and decisions, planning and completing familiar tasks, and preserved skills. They will experience changes in personality and behavior. Other complications can also arise from Alzheimer’s, such as aspiration, pneumonia, infections, falls, fractures, bedsores, malnutrition, and dehydration.

Medical professionals believe that Alzheimer’s is caused by a combination of genetic, environmental, and lifestyle factors. In the case of the form of Alzheimer’s being studied by Roche, it is caused by genetic factors that are inherited in an autosomal dominant pattern. What healthcare professionals do know is that brain cells deteriorate and die. They also suspect that plaques and tangles, which are both proteins, play an important role.

There are risk factors that may heighten the chances of developing Alzheimer’s disease. Age is a major factor, as people usually develop this condition after 65. If one has a relative that is affected, they are also at a higher risk. Females are also affected at a higher rate than males. Those with past head trauma, poor sleeping patterns, poor exercise patterns, and have other unhealthy lifestyle habits are have a higher chance of Alzheimer’s disease.

Doctors will typically diagnose Alzheimer’s disease after a finding of characteristic symptoms. Tests will be used to confirm a diagnosis, such as a physical and neurological exam and brain imaging, like MRIs and CT scans. After it is confirmed that one has Alzheimer’s, treatment typically consists of cholinesterase inhibitors and memamtine.

About Parkinson’s Disease

Parkinson’s disease is a progressive disorder that affects the central nervous system (CNS). It is characterized by its effect on movement through five different stages. As the disease progresses, severity increases. Stage one is characterized by subtle tremors on one side of the body. In stage two symptoms are more noticeable, with tremors and rigidity on both sides of the body. Stage three brings loss of balance and slow movement, while stage four makes it impossible for one to live independently. Stage five is the most severe, as patients cannot stand or walk. Hallucinations and delusions are common symptoms of this stage.

Parkinson’s disease occurs due to the death of motor neurons, some of which produce dopamine. Dopamine is important in the transmittance of messages to the muscles from the brain, so the loss of dopamine results in the loss of motor functions. Abnormal brain activity occurs when these neurons are lost. Doctors do not know why these motor neurons die, but they do suspect a few factors that play a role, such as genetics, environmental factors like toxins, and Lewy bodies.

There are no FDA approved therapies for Parkinson’s, and treatment is symptomatic. Treatment options include dopamine substitutes, carbidopa-levodopa, MAO-B inhibitors, catechol-O-methyltransferase (COMT) inhibitors, anticholinergics, and amantadine.

ANAVEX®3-71

ANAVEX®3-71 is a small molecule that is intended to target the M1 and sigma-1 muscarinic receptors. It is administered orally. The FDA granted it the Orphan Drug designation for the treatment of FTD, but Anavex decided to expand its indications after the results of preclinical trials.

These studies evaluated the effects of ANAVEX®3-71 on mice and found that it resulted in disease modifying activity for Alzheimer’s disease. It was these results that led to the announcement of a phase 1 trial.

This trial will enroll a minimum of 36 healthy male and female participants. It will be double-blind, placebo-controlled, and randomized. Using escalating doses, researchers will evaluate the safety, efficacy, tolerability, and pharmacokinetics of ANAVEX®3-71. Soon after, patients with frontotemporal dementia will be included in the study.

Anavex is excited to announce the initiation of this trial, as its success would offer a viable treatment for those with neurodegenerative disorders.

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