Comets Giving Days 2021

Solitary fibrous tumors, also known as hemangiopericytoma, is a very rare type of cancer for which there is no known cure, and no known chemo that consistently works well to combat it.  Patients usually use surgery and radiation to remove tumors as they appear, but the effectiveness is limited.  Because it is such a rare type of cancer, there has been relatively little research done on it, and most oncologists in the U.S. have little or no experience with it.  This makes it very distressing for the patients who have been diagnosed with it.  

Solitary fibrous tumors (SFTs) are suspected to be caused by a fusion of two genes – NAB2 and STAT6.  Dr. Hayenga and her colleagues have been working the past 7 months to genetically engineer cells that have the same genetic mutations that are present in SFTs.  They have recently had success.  Using the cancer cells, their next steps are: 

Baseline tests
- Characterize the growth behavior of these genetically engineered SFT/HPC cancer cells (as compared to the control cells without the modification)

Genetic Cure
- Develop a gene editor (CRISPR-Cas9) to reverse the cancer mutation
- Package this gene editor into an inert virus (adenovirus) and test its ability to correct the cancer cells in a petri-dish (in-vitro)
- Insert the tumor cells into mice and then test the virus packed gene editors ability to target and slow/stop the growth of the tumor in-vivo
- If it works, get the virus-gene editor system approved by the FDA (IND or compassion case) to try in patients who qualify

Pharmaceutical Cure
- Do genetic sequencing (RNA-Seq) to see if there is a novel protein created by the fused genes that can be targeted
- Test the ability of potentially promising pharmaceuticals on the market to kills/slow the growth of the cancer cells in-vitro
- Run a panel of 1,200 small molecules (proteins) with random functionalities to see if any target a signaling pathway of these cells and slow/stop their growth (i.e. try to find a novel pharmaceutical) 
- If any work, team up with a pharmaceutical company to try to make a drug, test in our animal model, then get FDA approval to use in patients.