Parkinson’s disease overview: Parkinson’s disease (PD) is a long-term and progressive brain disorder that most commonly affects those over the age of 60. People with PD have difficulty IN controlling their body movements, and symptoms become worse as the condition progresses, leading to the impairment of the patient’s ability to function in daily life situations. The motor (movement-related) and non-motor symptoms result from a loss of NEURAL cells in the brain that affect movement control, as well as other areas, such as mood, sleep and thought. PD is one of the most common neurological disorders: it affected approximately 5.2 million people worldwide in 2004, with 4 to 20 new cases reported per 100,000 people per year.
Treatment: At present, there is no cure for PD, but once a clear diagnosis is made, symptoms can be treated quite effectively in most cases. The aim of current treatments is to control and relieve symptoms, so that people can improve their quality of life.
Strategy and mechanism of action proposed by nLife: nLife suggests a new treatment strategy that would reduce and delay the neurodegenerative process, and therefore act as a Disease-Modifying Agent as opposed to the current strategies. nLife proposes to stop the progression of the disease by silencing alpha-synuclein gene which is expressed at high levels in monoaminergic neurons of Parkinson’s patients and the scientific community considers to play a key role in this pathology. nLife’s therapy will modulate gene expression specifically in monoaminergic neurons (those which produce dopamine, serotonin or norepinephrine), where the increase of alpha-synuclein expression induces neuronal toxicity and neurodegeneration. Thus, nLife hopes to stop the disease progression, becoming the first company with this therapeutic approach.
Michael J Fox Foundation is supporting this program (see)