About This Project

Ask the Scientists

What is the context of this research?

In 2011, a previously unknown mechanism was discovered to control the disease characteristics of the rare genetic disorder Niemann Pick Type C (NPC). NPC is also nicknamed "Childhood Alzheimer's", because the neurological symptoms are remarkably similar to Alzheimer's disease (AD). By re-activating the mechanism, disease characteristics in NPC patient cells were successfully reversed to look like normal cells. As a proof-of-concept, normal cells developed similar characteristics to those seen in NPC patient cells when the mechanism was inhibited. This suggests that this mechanism controls the underlying cause of the disease. The goal of this research is to identify a therapeutic agent that is mechanism specific for the treatment of NPC and AD.

What is the significance of this project?

Alzheimer’s disease (AD) affects more than 10 million people worldwide and that number is expected to grow to 22 million by 2025. In 2012, AD cost the nation more than $213 billion dollars (alz.org). Moreover, AD takes an incredible financial and emotional toll on the families that care for these individuals. Currently, there are only 5 FDA approved drugs that only temporarily treat the symptoms of AD, but none of them treat the underlying cause of AD.

Niemann Pick Type C (NPC) is a rare and deadly genetic disorder that effects children. Symptoms include aggressive neurodegeneration, fatty liver, and ultimately liver failure occurs within 10-15 years of age. Currently, there is no treatment for NPC and symptoms are only managed.

The discovery of this new mechanism allows for the development of a new class of therapeutics. A number of additional diseases share this mechanism and would be future targets for this new therapeutic agent. These diseases include Parkinson's disease, Cataracts, Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig's Disease), Cystic Fibrosis, and Lysosomal Storage Disorders.

What are the goals of the project?

The funds will be used towards laboratory space at a state-of-the-art biotechnology incubator that provides wet lab space ($300/month), potential drug compounds, and consumables (reagents, tubes, flasks).

The cell lines, my personal time, consultants time, and much of the equipment needed for this work has been donated or will be supplied free of charge.

We are happy to announce our partnership with the North Woodward Community Foundation, a 501(c)(3) organization, which will allow all of your donations to be TAX DEDUCTIBLE. Following the successful completion of our Microryza campaign, you will receive your donation letter from North Woodward Community Foundation to keep for your records. We want to thank North Woodward Community Foundation for lending their support to this very important project.