Donations for Running for Rachel will continue to be used for finding new and better treatments for duodenal and pancreas cancer. Donations from previous years have laid the foundation to better understand and identify the ‘achilles heel(s)’ of these tumors, and we are excited to report that two of these novel avenues are getting close to being ready to be tested in patients within a first-in-human clinical trial. Finding better ways and drugs to fight cancer has been our very ultimate goal of the research we are supporting – research to hopefully help patients like Rachel in the future.
A better understanding of the process ‘stemness’ and spread of these cancers, which are intimately linked, led to the identification of a small structure in the cancer cells termed ‘perinucleolar complex’ which appears to be essential for cancer cells to spread, an event which in the majority of patients is an ominous sign. We identified a drug which is able to disrupt this perinucleolar complex and have observed dramatic improvements in preclinical cancer models including small animals. The drug is safe to administer.
We are equally excited about a novel small peptide which reprograms the immune system fighting these cancers in a way that in combination with the standard chemotherapy agent gemcitabine not only makes cancers stop growing but leads to complete disappearance of these cancers in some animal models. We are currently working out the exact mechanism of action, the cellular target of this exciting new agent appears to be a certain subset of tumor-associated macrophages.
Our ‘final’ hurdle with both discoveries is the completion of the very important safety and toxicity studies with both agents mandated by the FDA prior to use in patients. We hope to raise over the next two to three years enough funds to successfully complete them.
In line with the greater maturity of the supported research over the last few years and an ever increasing generosity, we are planning to establish a ‘Rachel Guss Pancreatic and Duodenal Cancer Research Fellowship’ at NIH. Funds from Running for Rachel would support a young scientist, either PhD candidate or one of the clinical fellows of the Center for Cancer Research with a special interest in these malignancies, to work for one year in Dr. Rudloff’s laboratory (Thoracic & GI Oncology Branch, CCR/NIH) on drug development in pancreas and duodenal cancer. The progress briefly described above, and the need for better treatments make us confident that the Rachel Guss fellowship will attract some of the most devoted and brightest young scientist to help with this great mission.