Parkinson's Disease

Disease Background

Parkinson's disease (PD) is characterized by chronic neurodegeneration, in the midbrain region of the brain, affecting mainly dopaminergic neurons.

Greater than 500,000 Americans suffer from this progressive, debilitating disease. Current therapies affect some of the symptoms, but there are no therapies that reverse or halt the motor dysfunction. Unfortunately, many Parkinson’s patients also suffer from cognitive disabilities as the disease progresses. Mixed results have been found with transplantation of fetal neural stem cells into the brains of PD patients. Originally introduced in the late ‘80s, serious side effects were noted, including dyskinesia following implantation of stem cells. Other invasive, growth factor therapeutic approaches have been attempted, but also with little success.

Overview of Neuronascent’s Lead Parkinson’s Disease Candidates

Neuronascent has discovered non-invasive (without brain injection) therapeutic candidates that are neuron regenerative. In other words, the therapies aim to promote the proliferation, differentiation and ensure the survival of nascent neurons in the midbrain region, an area where neurons are selectively lost in Parkinson’s disease.

Our lead candidate for Alzheimer’s disease, NNI-362 that is effective in promoting new neurons in a number of disease models, was shown to halt all further dopaminergic connection loss in a pilot model of chronic, progressive Parkinson’s disease. Though the pilot study used a very short administration period, NNI-362 treatment already demonstrated a trend toward regeneration of neuron connections in the caudate putamen (see graph) and at the same time increased the number of new neuronal progenitors, suggesting neuron regeneration, not just neuroprotection. This suggests that NNI-362 should be tested in future human efficacy trials for Parkinson’s disease as well as Alzheimer’s disease, once first-in-human safety testing is complete. Neuronascent’s back-up therapeutic candidate, NNI-370 was optimized from a completely separate and novel small molecule family. It represents an exciting new class of potential therapies, that in vitro promotes new neuron growth from ventral midbrain stem cells and that inhibits neuron loss due to key initiators of dopaminergic neuron death. This patented therapeutic is now in the preclinical testing phase.


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