What do scientists mean when they refer to “cancer models”? Models can take many forms in cancer research, from single cells in a petri dish to large animals developing cancer similar to people, but all are designed to copy key features found in human cancer. They help scientists learn about how cancer forms and test ideas about curing cancer that would be too hard, or take too long, in people. Cancer models are important tools for developing and testing potential treatments. Before a drug ever reaches the clinic, it has been tested and shown promise in many different cancer models.

One of the research goals of the MyPART Network is to develop models of rare tumors. One of the challenges of rare tumor research is the lack of available tumor tissue to study. The lack of tumor tissue can make it difficult to create models to study rare tumors. MyPART scientists use cancer tissue when patients have surgery at the NIH Clinical Center to make cancer cell lines grown in petri dishes; “organoid” models, which are groups of cancer and supporting cells grown together in dishes; and “patient-derived xenograft” (PDX) models, where human cancer cells grow in a special mouse that lacks an immune system but feeds the tumor with its own blood system. Not all rare tumors will grow into a cell culture or organoid or PDX, and often scientists need to try to grow many tumors before they find one that can be used as a model.

Another way MyPART scientists develop rare tumor models is to find out what mutations are common in tumors and use these mutations to try to get normal cells or animals to form the same kind of tumor. Through many years of studying the rare stomach and intestine tumors from pediatric and “wild-type” Gastrointestinal Stromal Tumor (GIST) patients at an NIH Clinical Center rare tumor clinic, scientists learned that genes for SDH are often mutated. Dr. Ardori is a MyPART scientist currently working to develop a mouse model of SDH-deficient Gastrointestinal Stromal Tumor (GIST). Dr. Ardori explains, “Mouse models can be a powerful tool for studying many aspects of tumors and for developing new cancer therapies.” He has used genetic “scissors” called CRIPSR to cut and paste mouse DNA so the mouse gets the same mutation that is found in SDH-deficient GIST. The CRISPR scissors have revolutionized how fast scientists can make new models based on mutations found in cancer and this year the Nobel prize in Chemistry was given to Emmanuelle Charpentier and Jennifer Doudna, who discovered it!

Models of human cancer can be created using a variety of different laboratory methods, but they all start with patients who are willing to participate in research. Without these patients, scientists would not have the surgical samples to try to make new cell, organoid, or PDX models. Without patients willing to have DNA and RNA from their tumor sequenced, scientists would not know which genes to try mutating in mice (or flies, or fish, or rats, or pigs!) to form tumors in animal models. Cancer models can take time to develop but are crucial to eventually developing the best treatments for cancer. MyPART is always grateful to the rare tumor patients who participate in our studies, potentially benefitting future generations of rare tumor patients.  

To learn more about MyPART research efforts, visit our website here.

Dr. Ardori is a scientist for MyPART. He works in the lab to increase our understanding of the biology of rare cancers by designing approaches for creating new mouse models to aid in the study of rare cancers. Dr. Ardori was born in Italy and obtained his master’s degree in Biomolecular Sciences at the University of Urbino “Carlo Bo” while studying glioblastoma multiforme, a type of rare brain tumor. He then earned his PhD from the Polytechnic University of Marche in Ancona, Italy where he studied molecular pathology.

In 2010, he moved to the US and joined the lab of Dr. Lino Tessarollo as a post-doctoral fellow, and later a research fellow, at the NCI Frederick campus. Since Dr. Tessarollo is Director of the Mouse Cancer Genetics Program (MCGP) and the Gene Targeting Facility, Dr. Ardori was able to gain valuable experience creating genetically engineered mouse models. He had the opportunity to collaborate with several different laboratories seeking expertise in genetic engineering mouse models, which allowed him to further develop his skills. Dr. Ardori says, “this [collaboration] contributed exponentially to increase my experience in gene editing in a way that would not have been achievable by working solely on projects from a single laboratory.” He brings this expertise to MyPART, where he is currently working on a mouse model for SDH-deficient GIST, a rare gastrointestinal tumor.

When asked about the best part about MyPART, Dr. Ardori explains, “MyPART has the wonderful ability of connecting people with so many different [areas of] expertise and converging [this expertise into] one purpose only: working on improving the condition of rare tumor patients in any possible way.”

TargetCancer Foundation supports research on rare cancers. The organization was founded in 2009 by Paul Poth after he was diagnosed with cholangiocarcinoma, a rare tumor that forms in the bile duct, and learned his tumor was so rare that there were no available treatments. Since the organization was founded, it has supported a variety of rare cancer research efforts. TargetCancer Foundation recently launched a clinical study called TCF-001 TRACK. TCF-001 TRACK aims to enroll 400 rare cancer patients who will receive comprehensive genomic profiling of tissue and blood, and receive treatment recommendations from a virtual molecular tumor board. TRACK will both inform treatment options for patients, and help researchers learn more about rare cancers.

In addition to research support, TargetCancer Foundation provides resources for patients, including treatment and clinical trial guidance, sharing of personal stories from patients and updates on scientific progress. TargetCancer Foundation aims to connect patients, advocates, researchers, and doctors to accelerate rare cancer research. MyPART is pleased to partner with the TargetCancer Foundation to make an impact in the lives of patients with rare tumors.