UCLA RESEARCH ALERT
Researchers at UCLA have developed a molecular compound that improves balance and coordination in mice with early stage Parkinson’s disease. Further, the drug, called CLR01, reduced the amount of a toxic protein in the brain that is thought to be one of the prime culprits in the development of the disorder.
Parkinson’s disease is a nervous system disorder that affects movement. It’s estimated that as many as 1 million Americans live with Parkinson’s, and that roughly 60,000 are diagnosed with it each year. There is no cure. The disease is chronic and progressive, and over time can worsen from tremors in a person’s hands and slow movements, to impaired balance and coordination and, ultimately, overall rigidity of the body, including difficulty swallowing and speaking.
While the cause is not known, growing evidence points to the protein alpha-synuclein. The protein binds together in “clumps,” called aggregates, becoming toxic and killing brain neurons that produce dopamine, a neurotransmitter needed to send signals among neurons involved in controlling movements.
Earlier research by Gal Bitan, an associate professor of neurology at the David Geffen School of Medicine at UCLA, and colleagues led to the development of CLR01, which is known as a molecular tweezer — a complex compound capable of disrupting the formation of toxic protein clumps. Shaped like the letter “C,” CLR01 wraps around chains of lysine, a basic amino acid that is a constituent of most proteins. In the previous work in zebrafish, the scientists showed that the tweezer could decrease the clumping of alpha-synuclein and prevent its negative effects without detectable toxicity or side effects to normal, functioning cells in the brain.
Modified from the original press release by University of Pennsylvania
Breaking up amyloid fibrils in semen makes cells less likely to be infected
A paper, published in the journal eLife describes a small molecule that also disrupts amyloids in semen that promote HIV infection. This molecule, called CLR01, also attacks the virus itself.
Tweezers-shaped CLR01 can both disrupt fibril formation and disassemble fibrils that have already formed. CLR01 prevents HIV particles from interacting with fibrils by disrupting the membranes that surround the virus particles and can displace the virus particles that have already bound to the fibrils. In the presence of CLR01, human cells exposed to semen that contained HIV were at least 100-fold less likely to become infected with the virus.
CLR01 was also effective at directly disrupting other enveloped viruses, including hepatitis C virus, human cytomegalovirus, and herpes simplex virus, but was ineffective against the non-enveloped human adenovirus type 5. CLR01 may also be effective against many other enveloped viruses including flu and Ebola.
Amyloid fibrils formed by different proteins in the brain are associated with neurodegenerative disorders, such as Parkinson’s disease, and CLR01 could also be useful for these disorders, say the authors. The Breakthrough Treatment for Degenerative Diseases is a collaborative, international team pursuing CLR01 for these disorders.
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