In a press release from late January 2021, biopharmaceutical company Seelos Therapeutics, Inc. (“Seelos”) announced the issuance of an Australian patent for Trehalose (SLS-005). The patent (#2019204513) centers around the use of Trehalose for a variety of neurodegenerative disorders characterized by abnormal protein aggregation. For example, the drug can be used for Pick’s disease, corticobasal degeneration, and oculopharyngeal muscular dystrophy, among others. Additional conditions will be explained below.
The patent covers the intravenously-administered treatment of a pharmaceutical formulation using trehalose as the only active ingredient. It is designed to assuage symptoms related to these neurodegenerative conditions.
Trehalose is described as:
a low molecular weight disaccharide (0.342 kDa).
It is able to cross the blood-brain barrier, help clear protein accumulation within cells, and stabilizes protein throughout the body. In animal studies, trehalose lowered abnormal protein aggregation. It is also able to help clear cells by activating Transcription Factor EB (TFEB), which many researchers believe holds the keys to treating a variety of rare diseases.
Trehalose – Treated Conditions
Progressive Supranuclear Palsy (PSP)
Doctors are not sure of the exact cause of progressive supranuclear palsy (PSP), a neurological disorder that impacts movement. However, doctors do know that the condition results from gradual nerve cell damage in and around the brain stem. Patients with PSP often have abnormal tau protein buildup. An estimated 3-6 in every 100,000 people have PSP. The condition typically affects those between 45-75 and often affects males more than females. Symptoms include:
- Progressively worsening balance and walking
- Slowed movement
- Insomnia or other sleep issues
- Slurred speech
- Hand tremors
- Blurred vision
- Muscle stiffness
- Difficulty talking, eating, and swallowing
- Changes in mood, behavior, personality, and critical thinking
Spinocerebellar Ataxia (SCA)
A variety of genetic mutations cause spinocerebellar ataxia (SCA), hereditary conditions resulting from nervous system damage. As the nervous system becomes damaged, areas of the brain and spinal cord that control movement begin to degenerate. Each form of SCA is delineated by its specific gene mutation. Symptoms include:
- Loss of coordination, especially when walking
- Irregular gait
- Processing and learning difficulties
- Memory problems
- Abnormal speech
- Involuntary eye movements
Friedreich Ataxia (FRDA)
FXN gene mutations cause Friedreich ataxia (FRDA), a rare genetic disease which causes neurological and movement problems. Normally, FXN produces frataxin, which plays a role in mitochondrial function and energy. Without enough frataxin, the body has difficulty performing normal functions. An estimated 1 in every 40,000 Americans has FRDA. In most cases, symptoms appear between ages 5-15. However, late-onset FRDA appears after 25, with very late-onset appearing after 40. While disease progression slows in late- and very late-onset cases, many patients with FRDA require a wheelchair. Symptoms include:
- Chest pain
- Shortness of breath and/or difficulty breathing
- Poor coordination
- Slurred speech
- Changes in vision and hearing
- Scoliosis (curving of the spine)