The new treatment uses “peptide brush” polymers, which act as a shield to prevent proteins from binding to each other. It is the first polymer-based treatment for Huntington’s disease, an incurable and debilitating disease that causes the breakdown of nerve cells in the brain.
Patients with Huntington’s disease have a genetic mutation that triggers the misfolding and clumping of proteins in the brain. These clumps interfere with cellular function and eventually lead to neuron death. As the disease progresses, patients lose the ability to speak, walk, swallow and concentrate.
Most patients die within 10 to 20 years after the first symptoms appear.
Peptide brush polymers
The new treatment uses peptide brush polymers, which act as a shield to prevent proteins from binding to each other.
The studycarried out at this stage in the Huntington’s mouse model, provides a first demonstration of
- the effectiveness of this new approach in saving neurons and reversing symptoms;
- no significant side effects were noted, suggesting that the therapy is non-toxic and well tolerated.
Future clinical trials will still need to be carried out to validate the effectiveness and safety of the treatment in humans, as this treatment can be administered
in the form of a simple weekly injection,
and for prevention, in patients carrying the genetic mutation.
One of the lead authors, Nathan Gianneschi of Northwestern who led this development, recalls that indeed “If you have this genetic mutation, you will have Huntington’s disease. It is inevitable; there is no way out. There is no real treatment to stop or reverse the disease, and there is no cure. These patients really need help. This new way of treating the disease by preventing misfolded proteins from clumping together represents immense hope.”
Other possible applications? The polymer will be further optimized, with plans to explore its use in other neurodegenerative diseases.
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