Elucidating Mechanisms Driving Renal Medullary Carcinomas to Deliver a Rational Approach to Cancer Directed Therapy
Through the natural history of patients with sickle cell trait or disease, we have learned that they are at risk of a rare cancer, renal medullary carcinoma. With a very poor prognosis, it is critical that we understand the molecular underpinnings of this disease in order to devise improved therapeutic options. Unfortunately, there are no models available to study this cancer in the laboratory.
Using new techniques and approaches, we have developed the first renal medullary carcinoma cell line from a patient with sickle cell trait. This novel model captures the known biology and will now allow us to explore additional biology. Furthermore, using high throughput technologies developed in our laboratory and at the Broad Institute of Harvard and MIT, we can probe this rare cancer to understand mechanisms behind tumorigenesis and look for vulnerabilities. This platform of technologies when combined has two purposes:
1) to identify biology of rare cancers such as renal medullary carcinomas and
2) to look to repurpose known and tested compounds with the hopes of turning them into effective therapeutic interventions.