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Dr. Waanders is committed to developing more effective treatments for the many children diagnosed with brain tumors each year. Brain tumors are the leading cause of cancer-related death in children. Mutations in BRAF, an oncogene that can drive cancer growth, are prevalent in pediatric astrocytomas. By studying how mutated BRAF can be targeted by the newest classes of cancer drugs, she hopes to understand why and how these tumors develop in children and which treatments might work best.
Dr. Smanski is examining magnetic nanoparticles (MNPs), which possess unique physical properties that have led to several clinical applications in cancer diagnosis and therapy. Several species of bacteria have been found to naturally produce MNPs with exquisite control over size and shape that is unmatched by current chemical synthesis methods. He aims to understand how bacterial synthesis of MNPs relies on the coordinated expression of several dozen genes.
Diffuse intrinsic pontine glioma, or DIPG, is an incurable brain cancer that mostly strikes young children. The median survival rate is less than one year after diagnosis. To date, there are no chemotherapeutic or targeted agents that have proven to be beneficial for treatment of these cancers. Dr. Becher leads one of very few laboratories around the world that focus exclusively on this type of deadly brain cancer. He has identified EZH2-mediated epigenetic mechanisms that underlie the development of DIPG.
Mutations in the BRAF gene occur in 10-15% of colorectal cancers and predict poor outcome. Drugs that block the action of mutant BRAF are under active clinical development, and one drug that blocks BRAF was recently approved by the Food and Drug Administration (FDA) for treatment of metastatic melanoma. However, these BRAF inhibitor drugs alone have not been effective in BRAF mutant colorectal cancer patients, suggesting that improved approaches are needed. Dr.
The PI3K/AKT/mTOR signaling pathway normally conveys cues from the cell's environment into programs that promote cellular growth, division, and motility. Components of the PI3K signaling pathway are mutated in greater than 70% of all breast cancers and promote the persistent and exaggerated cell growth that is necessary for tumor formation and survival. This pathway is therefore a promising target for treating breast cancers; however, drugs designed to target the PI3K signaling pathway are initially effective but resistance rapidly develops. Dr.
The uncontrolled growth and metastasis of cancer cells is driven by changes in the genes expressed by these cells, relative to cells in healthy tissue. Understanding these gene expression changes provides key insights into the behaviors of cancer cells and guides the design of anti-cancer therapies.
Heritable factors are an important determinant of cancer risk. At present, only a small fraction of this genetic risk is explained by known cancer predisposition genes. Our preliminary data suggests that in pediatric cancers or cancers that occur in early adulthood, de novo or "new" genetic mutations may be identified that contribute to cancer causation. As such, the aim of our study is to study children with specific types of cancer (leukemia and neuroblastoma) without a family history of the disease to determine if we can identify the genetic cause of their cancer.
Bone marrow transplantation, or allogeneic hematopoietic stem cell transplant (HCT), is the only curative therapy for many patients with leukemia. Certain immune cells, called T cells, contained in the donor HCT graft can cause a "graft versus leukemia" (GVL) effect which eliminates leukemic cells. Unfortunately, there are also donor T cells in the HCT graft that can cause a condition called "graft versus host disease" (GVHD). GVHD is a life-threatening immune response that remains the major barrier to the success of transplantation. Dr.
Basal cell carcinoma (BCC) is the most common type of skin cancer. Mutations in the Hedgehog (HH) signaling pathway are frequently found in these cancers. Early-stage clinical studies of a HH pathway inhibitor drug have been successful, with 55% of patients reported to respond. However, most tumors change during the course of therapy and drug resistance eventually develops.
The goal of cancer immunotherapy is to adapt the natural components of the immune system to eradicate cancer. T cells are a type of immune cell highly evolved to detect and eradicate diseased cells. Dr. Davila is developing a novel treatment approach that involves genetic engineering of T cells as a safe and effective immunotherapy for blood cancers such as B cell lymphoma.