All-female cholangiocarcinoma advisory board forms alliances, shares patient stories
A cholangiocarcinoma advisory board composed entirely of women has given physicians who treat the rare malignancy, as well as patients and their advocates, an opportunity to collaborate, share best practices and forge alliances.
“Cholangiocarcinoma is a rare cancer type that forms in the bile ducts of the liver,” Susan Moran, MD, MSCE, chief medical officer of QED Therapeutics and one of the founders of the advisory board, said during an interview with Healio. “There is a huge unmet need for treatment, as there are very few effective treatments to date and it is associated with a dire prognosis.”
Moran spoke with Healio about what prompted the creation of the all-female advisory board and the support she received along the way in her career that has helped her achieve success.
Healio: What is unique about this population of patients with cholangiocarcinoma?
Moran: We are targeting a specific tumor with a specific FGFR mutation, known as FGFR2 fusion. This type of tumor tends to occur more commonly among women and specifically among younger women. The FDA is currently evaluating infigratinib (BridgeBio Pharma/QED Therapeutics) as a potential FGFR-targeted therapy for this patient subset, for which the current standard of care is chemotherapy, and outcomes are grim — median survival for patients with advanced or metastatic disease is less than 5 years. QED is focused on developing infigratinib for other FGFR-driven conditions, in addition to cholangiocarcinoma. We are running a phase 3 trial in urothelial cancer with FGFR3 mutations. In addition, we are studying infigratinib at a very low dose in children with achondroplasia, the most common form of disproportionate short stature, which is caused by an alteration in the FGFR3 gene.
Healio: What prompted the creation of the cholangiocarcinoma women’s advisory board?
Moran: My colleagues and I had noticed from participation in cholangiocarcinoma advocacy meetings, as well as in the data that we had, that patients with this specific FGFR-driven cholangiocarcinoma tended to be women. We also noticed that researchers and specialists treating these patients tended to women. We had an internal discussion to potentially gather all these women together to form an advisory board including physicians, patient advocates and patients themselves.
Healio: What does the advisory board entail?
Moran: The first Women in Cholangiocarcinoma Advisory Board meeting was held in October 2019 in parallel with the inaugural Cholangiocarcinoma Summit in Phoenix. The meeting was attended by 13 medical, surgical and radiation oncologists, as well as three patients and the founder of the Cholangiocarcinoma Foundation patient advocacy organization. Our second meeting was held virtually in October 2020, in conjunction with the virtual Cholangiocarcinoma Summit. We hope to continue to meet annually, and hopefully in person in 2021.
During these meetings, we review the current and emerging data on chemotherapy and targeted agents being investigated for this patient population, as well as best practices for biomarker testing to identify whether patients are candidates for targeted therapy. Approximately half of all cholangiocarcinoma tumors have a genetic marker that could make the patient eligible for a clinical trial of targeted therapy, such as infigratinib. However, testing to identify molecular targets of treatment often does not happen until late in the disease course, if at all.
We have also had patients join us and talk about their cancer journey. Additionally, we talk about how cholangiocarcinoma affects women, specifically younger women with young children who are still working, and how they manage some of the side effects of their treatments.
Healio: What support have you received along the way in your career that has helped you achieve success?
Moran: I have been fortunate enough to have support from people I work with throughout my career. In my early role in industry, I had a great mentor who established a formal training program for physicians who were making the transition from academia to industry. I also maintain a strong network of other women physicians, in which we support and encourage each other to take chances, including assuming roles of greater responsibility. A big risk that I took was becoming chief medical officer of QED Therapeutics, where I lead drug development for cholangiocarcinoma. I am glad that I took this chance, and I would encourage others to be bold in their pursuits.
For more information:
Susan Moran, MD, MSCE, can be reached at firstname.lastname@example.org.