SynCav1 is an experimental gene therapy for the treatment of neurological disorders such as ALS and Alzheimer’s disease.

First developed at the University of California San Diego, SynCav1 has been licensed to CavoGene LifeSciences.

How SynCav1 works

In the brain, a protein called caveolin-1 is found in pockets that line the outside of nerve cells called caveolae. Research has indicated that caveolin-1 is involved in taking things into the cell (endocytosis) and releasing things from the cell (exocytosis). This process is especially important in nerve cells where cell-to-cell communication involves the release and reabsorption of neurotransmitters (cell signaling molecules) into the space between nerve cells.

Research has shown that SynCav1 increases the number of small bubbles containing neurotransmitters and nerve growth factors as well as the number of receptor proteins that respond to these molecules. This increase leads to healthier nerve cells and better communication between them.

ALS is caused by the progressive death of upper and lower motor neurons. Nerve cells with stronger connections and receiving more nerve growth factors might be healthier and less likely to die. By maintaining nerve cell health, SynCav1 could, therefore, delay the onset or prevent the progression of ALS.

SynCav1 research

SynCav1 is currently being tested in preclinical studies.

One study showed that nerve cells grown in the laboratory treated with SynCav1 produced more caveolin-1. The cells also produced more neurotransmitter and nerve growth factor receptors. Finally, they had a larger dendritic tree, which is the part of a nerve cell that accepts signals from other nerve cells.

Several mice studies with SynCav1 have shown improvements in brain function, in motor and memory function in animals with brain trauma, and in learning ability. These results suggest that SynCav1 could be a potential treatment for neurodegenerative disorders, such as ALS, that affect learning, memory, and motor function.

A recent study involving a mouse model of ALS and mice treated with SynCav1 showed that when the animals were crossbred, the hybrid offsprings had higher body weight, better motor function, and longer survival, suggesting that SynCav1 could potentially be used in the future as a treatment to delay ALS onset and even increase life expectancy.

It is important to note, however, that SynCav1 is still in the early stages of preclinical development, and more research is needed before these findings can be translated to the clinic.


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