Name: Dr. John Paul Shen
Focus area: Appendiceal cancer
Grant: 2022 Career Development Award The Problem: Traditionally, oncologists have targeted appendix cancer with the same chemotherapy regimens used for colon cancer. Yet appendix tumors differ from colon tumors and require more nuanced treatment. We believed—and wanted to prove—it would be more effective to design and implement chemo specifically for patients with appendix cancer.
The Outcome: We found that appendix tumors respond to chemotherapy quite differently than colon tumors. But we also found that appendix tumors can greatly differ from one another, depending on their grade and type. In fact, we found that patients with low-grade tumors don’t benefit from certain types of chemotherapy. They may, however, benefit from specific combination therapies. We also found that measuring serum tumor markers can help oncologists more accurately predict a patient’s unique prognosis.
The Implications: The key takeaway from our work is that appendix cancer shouldn’t be grouped with colorectal cancer. Instead, it needs to be treated as its own disease. Based on these findings and similar projects, the National Comprehensive Cancer Network is revising treatment guidelines for appendix cancer. Moreover, the Society of Surgical Oncology recently published new guidelines for targeting these cancers.
What’s Next? There are still no U.S. Food and Drug Administration-approved drugs for appendix cancer. Based on new data, including results from our Conquer Cancer-funded research, we now have strong evidence to support using therapies specifically for appendix cancer. Now, we’re in the process of finding industry partners to sponsor future clinical trials 
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 Name: Dr. Abhishek Mangaonkar
Focus area: Chronic myelomonocytic leukemia
Grant: 2018 Endowed Young Investigator Award
The Problem: Patients with chronic myelomonocytic leukemia (CMML) present with significant differences in disease based on genetics and other factors. My research mentor encouraged me to examine the relationship between CMML-causing mutations and epigenetic changes. Using Conquer Cancer funding, we studied epigenetic alterations in patients with CMML. We hoped to find biological pathways to help us to better understand the diverse nature of this disease.
The Outcome: We found that patients’ immune tolerance (and other factors like T-cell immunity) could play vital roles in how the disease presents on a genetic level. These factors may also explain how CMML progresses to acute myeloid leukemia (AML). I have since focused on how dendritic cells cause immune tolerance for patients with this type of cancer.
The Implications: A critical aspect of research for any type of cancer is to find biologic pathways that affect disease progression. My funding let me focus on immune tolerance and dysregulation. This has potential implications for treating patients with other types of leukemias.
What’s Next? The next step in my research is to find a suitable therapeutic target to disrupt immune tolerance and prevent disease progression in patients with CMML.
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 Name: Dr. Abdulazeez Salawu
Focus area: Soft tissue sarcoma
Grant: 2022 Young Investigator Award
The Problem: Soft tissue sarcoma is a rare cancer that often recurs after surgery. Nearly half of all patients experience metastasis despite aggressive treatment. Making things harder, sarcomas are genetically diverse. Because of that, there hasn’t been a reliable, one-size-fits-all indicator to monitor patients for recurrence. This gap in our ability to detect disease early is what drove me to explore circulating tumor DNA (ctDNA). This approach could yield a personalized method of catching recurrence before it shows up on scans.
The Outcome: So far, we’ve seen promising results. In our study, ctDNA was detectable in about 80% of patients at diagnosis. The levels dropped to zero after surgery in most patients. Importantly, for all but one patient, ctDNA reappeared weeks before we detected residual disease through imaging. This suggests ctDNA could be a highly sensitive way to track disease status. It could also help doctors intervene earlier.
The Implications: If ctDNA can reliably signal recurrence in sarcoma, it could be a game-changer for patients. This approach could help us tailor treatments more effectively. It could also allow us to spare patients from unnecessary treatment or to recommend more aggressive follow-up if needed. Ultimately, we need to catch recurrence early on. If we can, it will help to improve survival outcomes by identifying patients who need additional treatment to reach remission.
What’s Next? The next step is to expand this research with other centers to further confirm these findings. We also plan to look more closely at how monitoring ctDNA can guide the selection of patients who may benefit from adjuvant therapies.
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 Name: Dr. Anna Spreafico
Focus area: Pancreatic neuroendocrine tumors
Grant: 2014 Young Investigator Award
The Problem: Pancreatic cancer is particularly aggressive. Only 12% of people survive five years after diagnosis. With my Conquer Cancer-funded research, I wanted to define the molecular causes of disease progression in pancreatic neuroendocrine tumors (NETs)—a rare type of cancer. Additionally, limited research funding exists for pancreatic NETs compared to other types of cancer.
The Outcome: We defined the characteristics of an aggressive subtype of pancreatic NETs and mapped out the mechanisms that cause it to multiply. This initial work paved the way for future studies that can help doctors assess the risks for individual patients. It can also help them build more personalized care plans and work toward promising treatments.
The Implications: This was an important translational project that contributed meaningful results to the oncology community and paved the path for subsequent research into early drug development for pancreatic NETs.
What’s Next? This project gave me the skills to mentor the next generation of early-career researchers, including five Conquer Cancer grant recipients. I now guide junior investigators and continue to pursue translational research in early drug development.
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 Name: Dr. Pavlos Msaouel
Focus area: Renal medullary carcinoma
Grant: 2017 Young Investigator Award
The Problem: Patients with rare and aggressive kidney cancers like renal medullary carcinoma (RMC) have limited options for treatment. They also face poor survival rates. Average survival is only around 13 months. If left untreated, the disease can be fatal within just three months of development in the body. Nearly 50% of patients with RMC pass away within one year of diagnosis. RMC also predominantly affects younger populations of African descent. Better understanding the biology of RMC can help us to optimize treatment for this rare disease and fill a major gap in cancer care.
The Outcome: Conquer Cancer funding allowed us to launch the first funded research project on RMC. Before our study, RMC was relatively invisible, with very little awareness in the oncology community. Using this funding, we analyzed RMC tissues to deepen our understanding of RMC on a genetic and molecular level. This project laid the foundation for exploring innovative treatment strategies and translating ideas to clinical settings.
The Implications: The advances we make in rare cancer research are directly applicable to improving care for countless other types of cancer. In less than a decade, we’ve moved from patients with RMC having less than one year to live to patients surviving much longer. Close to 10% of patients become completely disease-free, including many who might have been considered incurable several years ago. These milestones reflect the immense value of addressing unmet needs in rare kidney cancer research. They also prove that we can make a substantive impact for patients with rare diseases—and every other type of cancer.
What’s Next? The data we acquired helped us launch three ongoing clinical trials dedicated to further investigating RMC. We’re in the process of uncovering new and promising treatments for a subset of patients with a type of bladder cancer far more common than RMC. Conquer Cancer funding is the reason we were able to do this. Without it, our discoveries may have been delayed by nearly a decade.
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 Name: Dr. Bahar Laderian
Focus area: Rare tumor biology
Grant: 2019 Young Investigator Award
The Problem: Pheochromocytomas and paragangliomas are rare. Because research in this field remains underfunded, they also remain understudied. There’s a significant knowledge gap in rare neuroendocrine tumors and adrenal gland cancers. Patients often face uncertainty and find it hard to find oncologists with expertise in these rare conditions. I help to bridge this gap by studying the biology of these cancers and exploring innovative treatments to improve patient outcomes.
The Outcome: My team and I used single-cell analysis in normal adrenal tissue. This method allows us to isolate and examine normal cells within the adrenal gland. This technique has enabled us to investigate why certain mutations cause cancer in the medulla but not in the cortex of the adrenal gland—and rarely in any other organ.
The Implications: By identifying why SDH germline mutations cause cancer in medullary cells but not in cortical cells of the adrenal gland, we’re closer to developing strategies to delay cancer onset in these cells.
What’s Next? After completing my current research, I plan to use the findings to develop targeted treatments for patients with germline SDH mutations. The goal is to prevent the development of pheochromocytomas and paragangliomas. |
