Progressive Supranuclear Palsy Headed Toward Cure

Progressive supranuclear palsy (PSP) is a brain disorder which impacts vision, cognition, movement, and speech. This disease results in weakness by affecting certain parts of the brain above the cell clusters, known as nuclei (supranuclear), which are involved in controlling eye movements. It usually manifests in mid-to late adulthood, and the survival rate of people suffering from PSP is five to nine years after the disease first appears. It is also referred to as Steele–Richardson–Olszewski syndrome.

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Signs and Symptoms

The signs and symptoms of PSP vary on an individual basis, and the first symptom is usually the loss of balance while walking. Patients may experience unexplained falls or stiffness in gait. With disease progression, problems in controlling eye movements and blurring of vision become the common observations. Slowing of the eye movements is the definitive clue for a proper PSP diagnosis. Alterations of mood and behavior, such as apathy and depression, are also exhibited by patients. Other symptoms of PSP are a mask-like facial expression, slowed movements, and monotone speech.

Causes

The exact cause of PSP is not yet established; the symptoms are caused by the gradual deterioration of brain cells in certain areas, specifically the brain stem. Abnormal tau protein deposits in the nerve cells are the hallmark of the disease. This protein is associated with microtubules, which help the axons transmit information through the nerves. Abnormal protein aggregates in the surrounding cells to form protein clumps, thereby spreading throughout the nervous system. Another possible theory is that of cellular damage by free radicals, which are continuously generated by cells during normal metabolism; these damage other molecules under certain circumstances.

Diagnostic Tests

There are no specific laboratory or imaging tests currently available for a definitive PSP diagnosis. It is often misdiagnosed as the symptoms are comparatively rare and similar to other movement disorders, such as Parkinson’s disease. Brain shrinkage may show on magnetic resonance imaging (MRI) scans, and a positron emission tomography (PET) scan may be used to detect early signs that may not appear on the MRI.

Treatment

No medication or surgical procedure is available for PSP currently, and the treatment is done based on the appearance of symptoms. Antiparkinsonian agents such as levodopa help in managing stiffness, slowness, and balance, but the effect is minimal and doesn’t last long. Anti-depressants are also prescribed to control excessive drooling and for pain relief, whereas botulinum injections are used for treating excessive eye closing.

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PSP therapeutics Pipeline

The PSP therapeutics pipeline is witnessing growth due to the receipt of the orphan drug status by several pharmaceutical companies for their under-development drugs. A provision of the U.S. Food and Drug Administration, the Orphan Drug Act grants a special status to a biological product or drug for the treatment of a rare condition upon a sponsor’s request. Certain incentives, such as tax credits, federal grants and a waiver of the filing fees, which is mentioned in the Prescription Drug User Fee Act, are provided under this designation.

One of the drugs in the PSP therapeutics pipeline is BIIB092. Being developed by Biogen Inc., it was under the Phase II development stage in November 2017. Also known as BMS-986168, it is a humanized monoclonal antibody, which works by inhibiting the tau protein. It is being developed to be administered through the intravenous route. Bristol-Myers Squibb Company gave the drug licensing rights to Biogen Inc.

Therefore, with the entry of more companies, the pipeline would advance, hopefully finding a definitive treatment for PSP.

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This release was published on openPR.