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Dr. Rudloff's Research at the National Cancer Institute at the National Institutes of Health:
 

Donations for Running for Rachel have supported progress towards finding new and better treatments for duodenal and pancreas cancer. Donations from previous years have enabled several new discoveries, the opening of a new clinical trial, produced at least two new leads for novel, first-in-class anti-cancer drugs, and – since 2016 – contributed towards training of young scientists through our just recently created ‘Rachel Guss and ‘Bob’ Pomerenk Pancreas Cancer Research Fellowship. Finding better ways and drugs to fight cancer has been our very goal of the research we are supporting – research to hopefully help patients like Rachel in the future. A brief outline of the accomplishments supported with Running for Rachel donations includes:
We have opened a clinical study, initially for pancreas, but planned to be expanded for duodenal cancer patients [NCT03040986, NCI-2017-00118, Selumetinib Sulfate in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer With KRAS G12R Mutations], where we select patients with a certain genetic make-up of their tumor for treatment with a new cancer drug (MEK inhibitor). The rationale for this ‘precision medicine’ concept was developed in Dr. Rudloff’s laboratory with support through RfR donations. A summary of the study design (Re-staging = patients undergo repeat imaging in the form of X=rays, CT scans to see if their tumor has shrunk) is shown below.

 

Running for Rachel donations have supported work on a first-in-time, first-in-class (PNC disassembler) new small molecule cancer drug which targets the ability of cancer cells to spread (named metarrestin). Spread to other organs, or metastasis, throughout the body of cancer patients is the most common cause of death from cancer. The development of this drug has been awarded with one of the BrIDGs (Bridging the Interventional Development Gap) Awards of the National Center of Advancing Translational Sciences (NCATS) which will support the necessary safety and toxicity studies for FDA to grant permission to give the drug, for the first time, to patients in the future. That includes work on a formulation and to determine the first human dose wherefore studies are currently conducted in several different animal species. The figure below shows that the target of the drug, the peri-nucleolar complex or PNC, is only present in the evolved, metastatic cancer stage, as well as the insertion of a catheter into pigs to conduct above safety and dose-range finding studies. Importantly, this drug targets metastasis from different cancers, including duodenal cancer and other GI cancers. We therefore hope that we are able to provide patients like Rachel with an effective treatment option in the future. 

 

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. Like metarrestin this anti-cancer drug is a new development of laboratory efforts supported by Running for Rachel donations.
One of our proudest accomplishments however is the very recent establishment of a dedicated ‘Rachel Guss and ‘Bob’ Pomerenk Pancreatic and Duodenal Cancer Research Fellowship’ at NIH through donations from Running for Rachel. Funds from Running for Rachel support since 2016 a young scientist with a special interest in these malignancies, working 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.