- Overview of Current Project
We have two on-going project that focus on Neuronal Calcium Sensor 1 (NCS1) as the target. NCS1 is a multi-functional calcium-binding protein that interacts with many proteins and modulates cell signaling, motility, and survival. Cells with diminished NCS1 levels have decreased calcium signaling, motility and survival. Several diseases occur when NCS1 levels are out of balance. We have identified compounds that target NCS1 and ameliorate symptoms in those diseases. In the first example, NCS1 is a novel receptor for paclitaxel, a chemotherapeutic drug. This interaction greatly amplifies signaling which initiates a cascade of protein degradation, subsequently leading to chemotherapy induced peripheral neuropathy (CIPN) and cognitive impairment (chemobrain). We found a drug that will prevent these negative side effects of chemotherapy, without altering the efficacy of the cancer therapy. We formed Osmol Therapeutics and we are currently seeking funding for a clinical trial. In the second example, we formed Center Pharm to focus on Wolfram Syndrome, a rare and deadly genetically defined neuroendocrine disorder. Currently no available disease-modifying therapies exist for Wolfram Syndrome patients. The standard-of-care is limited to palliative approaches to control the neuroendocrine symptoms. The syndrome is caused by loss-of-function mutations in Wolframin (encoded by WFS1) and is diagnosed in patients with childhood-onset diabetes mellitus, a gradual loss of vision caused by optic atrophy, and deafness. WFS1 and NCS1 are functionally intertwined and WFS1 inactivation leads to downregulation of NCS1 expression and function. We identified a new drug that rescues cell function in a cellular model of Wolfram Syndrome and we are currently seeking funding for testing this drug in mouse models of Wolfram Syndrome. Once validated we will begin a clinical trial in collaboration with Dr. Fumihiko Urano, a world-renowned expert in Wolfram Syndrome and the person who maintains the US Wolfram Syndrome patient registry.
- Favorite off-time activity
Spending time in the gym, baking, knitting (see the mittens I made with my calcium design)
- Best advice for young innovators
When possible, implement the advice of investors Persistence is critical.