In the hope of one day finding a cure for Huntington's Disease, medical research is working on future treatments. Although these treatments are still in the experimental stage, there are many promising avenues that offer hope to patients and their families: selective sirtuin 1 inhibitors for neuron protection, bone marrow transplantation to improve motor skills, gene silencing, or phosphodiesterase inhibition.
Huntington's Disease currently lacks a cure, but medical research is tirelessly working on its development. Today, numerous experimental treatments are emerging, providing hope for patients and their families.
This experimental treatment was discovered by Italian researchers from Siena Biotech in January 2010. They developed a selective sirtuin 1 inhibitor that could have modifying effects on Huntington's Disease and protect neurons.
Their hypothesis is that since Huntington's Disease is caused by a mutation in a protein called huntingtin, this sirtuin 1 inhibitor could reduce the activity of sirtuin 1, promoting the elimination of the defective huntingtin by cells. This inhibitor would act as a neuroprotective agent. Currently in testing, this new treatment has already received "orphan drug" status in Europe and the USA.
This new approach to treating Huntington's Disease is based on potential connections between the immune system and the development of the disease. Recent discoveries suggest that these connections are real and very complex.
The immune system can influence the brain, and vice versa, as phenomena occurring in the brain can have various effects on the immune system.
As evidence, the blood of mice with Huntington's Disease contains damaged immune system molecules.
The researchers' hypothesis is that since the immune system can affect the brain of a patient with Huntington's Disease, a healthy bone marrow transplant could provide important insights into the role of the immune system in the disease.
Currently in testing on mice, this new avenue shows promising results. The transplant has improved motor skills in mice with Huntington's Disease.
Gene silencing is a brand-new research avenue that seems particularly promising. This new drug, currently in development, would be capable of reducing the production of the mutated protein responsible for Huntington's Disease.
The principle is to develop a drug that can bind to the messenger molecule whose production is induced by the Huntington's Disease gene, blocking this production. Neurons in affected individuals could then regenerate.
Various drugs have been tested on animals with highly satisfactory results. The next step will be testing on human patients.
This research avenue focuses on the functioning of phosphodiesterase (PDE) enzymes and their role in synaptic function.
Researchers have observed that synaptic function is severely impaired in individuals with Huntington's Disease. They hypothesize that restoring proper synaptic function could reduce cell degeneration in affected individuals.
Medications, including phosphodiesterase inhibitors, are currently in development.
All of these promising avenues will need to be further tested and validated before we know whether they are truly effective in treating Huntington's Disease.
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