Welcome to the sarcoma world with the third episode of ‘5 Min Sarcoma Talk’ on OncoDaily. In this episode, host Dr. Hovsepyan and esteemed sarcoma expert Dr. Weiss discuss the latest advancements and challenges in desmoid tumors, discover the promising agents under investigation, and more. Join us for an informative and inspiring journey into the sarcoma field.

About Aaron Weiss

Dr. Aaron Weiss is a pediatric oncologist/hematologist and division director of pediatric hematology-oncology at MaineHealth. He is an associate professor at Tufts University School of Medicine. He holds the Sam L Cohen medical director endowed chair at Maine Children’s Cancer Program. Within the Children’s Oncology Group, he is a Vice-Chair of the Soft Tissue Sarcoma Committee and associate Vice-Chair of the AYA Oncology Discipline Committee the Clinical Trials. Moreover, he chairs the Medical Advisory Board of the Desmoid Tumor Research Foundation.

About Shushan Hovsepyan

Dr. Shushan Hovsepyan is a pediatric oncologist and adjunct assistant professor at the Yerevan State Medical University. She earned her medical degree (M.D.) from the same university’s General Medicine faculty, where she was awarded the Nominal Student Scholarship. Currently, she is a clinical research coordinator at the Yeolyan Hematology and Oncology Center. She completed a clinical fellowship lasting over a year at the pediatric oncology unit of the National Institute of Cancer in Milan, Italy, with a primary focus on pediatric sarcomas and rare tumors. Additionally, she was a clinical observer at the pediatric oncology unit of St. Anna Children’s Research Hospital in Austria for six months. Furthermore, she held a research fellowship position at the European Organisation for Research and Treatment of Cancer in Brussels, Belgium. Dr. Hovsepyan also completed the Postgraduate Harvard Medical School’s Effective Writing for Healthcare program, focusing on writing and reviewing medical-scientific manuscripts. Dr. Hovsepyan is a member of the POEM Training Committee and a graduate of the College of the European School of Oncology. She is a former SIOP Global Health Network Education and Training Working Group co-chair.

About OncoDaily

OncoDaily.com was founded in 2023. It is a US-based oncology media platform, which features the latest news, insights, and patient stories from the world of oncology. Within a short period, it became one of the leading oncology media platforms globally. OncoDaily gathers content from various sources, including social media posts from renowned oncologists from all over the world, news from oncology societies and cancer centers, patient and survivor stories, and career-related information for professionals. The mission of OncoDaily is to empower patients, survivors, and professionals with the knowledge and inspiration they need to fight cancer. The motto of OncoDaily is “Cancer doesn’t take a day off – neither do we.”

Follow the transcript below

Shushan Hovsepian (S.H.): Greetings to all our listeners wherever you may be joining us from. Welcome back to our third episode of “Five Minutes Sarcoma Talk” on UNC Daily. I’m Shushan Hovsepyan, pediatric oncologist from Armenia. If you are interested in sarcomas, I would recommend our listeners to grab a cup of coffee as over the next few minutes we will explore the unique challenges of desmoid tumors. And today our distinguished guest is Dr. Aaron Weiss, who is the division chief of pediatric Hematology Oncology at MaineHealth and an Associate Professor at Tufts University School of Medicine and Sam Cohan medical director in the oncology program. He’s also the vice chair of soft tissue Committee in children’s oncology group and the vice chair of the clinical trials Aya oncology discipline committee. So welcome, Dr. Weiss.

Aaron Weiss (A.W.): Oh, thank you so much for having me. 

S.H. :Thanks a lot for accepting our invitation, and let’s, without further ado, let’s start. So as I said, we are going to discuss desmoid tumors. Could you explain what the desmoid tumors are and how they differ from other soft tissue sarcomas? What are the typical symptoms and signs of desmoid tumors?

A.W. : Desmoid tumors are extremely rare, more common in the young female age range of when they’re most fertile. It’s typically a slow-growing type of tumor that can appear almost anywhere in the body. The most common symptom is pain or just a visible lump if it’s closer to the surface. But many people will have these desmoid tumors for long periods of time before they’re discovered. Sometimes they’re discovered by accident, and sometimes they’re discovered when someone works somebody up for some type of particular discomfort.

S.H. :Thank you for mentioning that and building on that you said that it’s typically very rare tumor but we always encounter challenges for rare tumors as  there are no standard of care treatment so what are the currently primary treatment modalities available now both in systematic and also local therapies?

A.W. : Yeah, desmoid tumors are tricky because, for a long time, they were treated as a surgical disease, and surgeons were often just taking them out. But we were finding that many times they would come back. And actually, due to a lot of work that has been done, typically over in Europe, we’ve discovered that these tumors can sometimes spontaneously regress or get smaller on their own, or sometimes they can stabilize. And actually, even if we remove them, many times they come back. So we’ve looked to other types of modalities or treatments that we could offer patients. It’s also a little different how we think about treatment in children versus adults because of the size of the patients and potentially the toxicities or side effects, but right now the standard approach is typically actually observation and active which another in term of active surveillance where we image patients and watch them and actually not treat them at all that’s become kind of the Frontline standard for most patients but when it starts to grow or cause symptoms then we typically intervene and a lot of the decisions are based on the location and the age of the patient, so often times we use systemic therapy we have IV chemotherapy type medicines as well as oral medications that are a little bit more targeted with a little bit less side effects that often times I think now in the adult world at least most people standardly use some of the oral agents that have become available, and in children will sometimes use the oral agents or sometimes the IV chemotherapy medicines depending on the age of the patient and how safe we feel it is for them to be on it but if those if they’re not a candidate for systemic therapy or the tumor continues to grow or sometimes depending on location a local modality like you mentioned is often used and now more and more we’re using some of these local Interventional approaches with certain radiation techniques that have a little bit less side effects and have shown to be effective for many of our patient populations but we have worked together with a international committee to help develop kind of a standard approach for how these patients are managed because it’s so rare I think it’s important collectively that we come together to help decide the best way to treat these patients appropriately.

S.H. : That’s very encouraging to see that there is a network working on this kind of rare tumors and also you mentioned the role of surgery. I know that it’s changing and now a lot of patients are referred directly to oncologists but at least inLMICs it’s a common problem that surgeon move out the tumor and then patient comes to an oncologist so that’s for the conventional therapies and what about the experimental therapies are there any clinical trials both for children and adults of course we know that the data is coming from adults but at least  right now what is ongoing ?

A.W. : There are, in fact, there’s a clinical trial that recently finished in the adult population. You looking at a specific target targeted medicine called a gamma-secretase inhibitor. It was shown to be very effective when they gave it to patients that were randomized between getting the drug and getting the placebo or fake drug, and it was shown to be very effective. So while that was happening in parallel, we ran a study for children. So patients under 18 years of age with the same drug through the Children’s Oncology Group, which is the cancer Consortium for children in North America. That study actually closed after we accrued the appropriate number of patients, and we’ll be able to actually analyze the data starting in December of 2024. I think at that point we’ll have a sense of the safety of the medication as well as how effective it is to help determine where in the priority of medications we would put that particular medicine. So right now, that is the only clinical trial at least for children that’s recently closed. There is another trial that’s looking at another targeted medicine that affects the beta-catenin gene, which is part of the pathway if we think for tumor growth in desmoid tumors. That study being used not only for patients with desmoid but other cancers that have beta-catenin involved in their growth as well. That study’s also being run through the Children’s Oncology Group, and so we’ll have some data on the study was mainly for safety and finding the dose, but we’ll also have some ability to be able to see whether it has some effect as well. And so there is another study looking at a different gamma-secretase inhibitor that is accruing patients as well. But those are the current studies that at least are happening from a systemic therapy standpoint and from a clinical trial basis.

S.H. : That’s very helpful. And I guess next year we will have a lot of new results for children. And so, another thing moving to another aspect, you mentioned that one of the ways to find the solution is the network. What are the other ways? What you recommend dealing with these challenges associated with desmoid tumors, particularly considering their rarity?

A.W. : I think the most important thing is that we have as many people thinking about this as possible and getting all different types of providers in the room. We need Physicians who take care of children, we need Physicians that take care of adults, we need the scientists who are doing the basic science and translational research to help determine how desmoid tumors form. We need the people who are delivering certain types of interventions like interventional radiologic procedures, radiation surgeons. Getting all these people in a room together is the most important thing, and so we’ve been doing that. Through organizations like the Desmoid Tumor Research Foundation, they have an annual meeting. And as part of that meeting, they have a research day in which all these different people get together, both in person as well as a virtual component, to be able to talk about their research and also to help develop priorities of what we feel as desmoid tumor experts would be the best way to move the field forward and develop ways to cure and help with treatment of this disease. So I think the most important thing is getting like-minded people together to talk about these issues because if you work individually in separate silos, I think it’d be very difficult to make significant strides for this particular disease.

S.H. : Exactly. And also, funding is very important, even if the people are coming together and deciding or writing a protocol, if they don’t have the funding, it’s very tricky. And especially for Rarities, there are very few companies that are interested to invest.

A.W. : You’re absolutely right. I think that’s probably one of the hardest things that we suffer from is that although we can raise money through different means, it’s very difficult to get people behind something that’s just not as common and affects as many people.

S.H. : That’s true. And moving to your personal journey, how you decided to be personalized in pediatric oncology and especially in sarcomas? And how would you describe the role of mentors on this way?

A.W. : For many of us, it’s not always a path that we thought we’d be doing. I was going to be a general pediatrician. And I think through some of my experiences of patient encounters during my training, as well as mentors that you mentioned, helped shape what I enjoy doing. And I think what drew me most to the field was the ability to be able to take care of patients that are well, take care of patients that are not as well, take care of very sick patients and well patients in the same day, and deal with Cutting Edge research and be able to participate in that research in a meaningful way. It was kind of a field that encompassed everything, as well as long-term and longitudinal relationships with patients and families. And so, that was really what drew me most into that. And through sarcoma, my main interest in desmoid tumors is largely based upon those that have mentored me through my training. I’ve always been interested in clinical research and getting involved in clinical research. It just so happened that those people that I respected during my training and really took me under their wings are the ones working in that particular area of research. They allowed me opportunities to get involved. Now, in my role, where I not only take care of patients but also help develop clinical trials and strategically how to move things forward, to be able to see young investigators that are interested to help get them involved in projects either their own projects or project ideas that we have that we just don’t have enough people to help. Seeing them get involved and mentoring them and helping them to self-discover and get involved is really one of the most rewarding things about my job now that I enjoy most.

S.H. : That’s very motivational and inspiring also for Young oncologists and Dr. Weiss thanks a lot for sharing your expertise on desmoid tumors and also on your personal journey. I think it was a wonderful discussion. And to our listeners, thank you for joining us today, and we look forward to exploring more topics on sarcoma research and care in future episodes. Thanks a lot.

A.W. : Oh, thank you so much. And thank you for having this type of platform for us to talk about something that I think more people need to know about. So, thank you so much.