Oncothon is a telethon spanning 24 hours, dedicated to gathering donations for childhood cancer research.

Prof. Gevorg Tamamyan is the Chief Editor of Oncodaily, head of Pediatric Cancer and Blood Disorders center of Armenia. Prof. Leo Kager is the outpatient depratment at Santana Children’s Hospital in Vienna.

Dr.Jaume Mora scientific director of the oncology and hematology area at Sant Joan de Deu Hospital in Barcelona. Dr. Khaled Ghanem is the medical director at BASMA Pediatric Oncology Unit in Damascus, Syria. These 3 speakers will discuss pediatric cancer care.

Speakers

Professor Leo Kager holds board certificates in Pediatrics and Pediatric Hematology/Oncology. He is the head of the Outpatient Department of Hematology and Oncology at the St. Anna Children’s Hospital the head of the Children’s Cancer Research Institute in Vienna, a Professor of Pediatrics at the Medical University Vienna, and an active member of the Austrian Mountain Rescue Service.

His research focuses on pediatric sarcomas, pediatric renal tumors, and rare hematological diseases. Moreover, he is an osteosarcoma expert. He has acted as a reviewer for the NEJM, Lancet Oncology, Annals of Oncology, Leukemia, etc., and published 100+ articles in Pubmed.

Jaume Mora serves as the scientific director of the Oncology and Hematology department at SJD Barcelona Children’s Hospital, concurrently leading the developmental tumors laboratory within the same institution.

He holds memberships in several prestigious national and international scientific organizations, notably the International Pediatric Oncology Society, where he was honored with the Schweisguth Prize.

Recognized for his contributions to oncology research, he received the Young Investigator Award (YIA) and the Career Development Award (CDA) from the American Society of Clinical Oncology (ASCO) in 2000. Additionally, he was the recipient of the BBVA Foundation Award in 2011 and the First Prize of the Spanish Association Against Cancer (AECC) in 2006 for his pioneering work in childhood cancer studies.

Mora’s research primarily delves into the origins of various childhood tumors, encompassing neuroblastoma, Ewing sarcoma, retinoblastoma, Wilms tumor, and DIPG.

Khaled Ghanem, M.D. is a consultant pediatric oncologist and the medical director of the BASMA Pediatric Oncology Unit in Damascus, Syria. Dr. Ghanem’s main area of interest is childhood cancer care in areas of conflict and crisis. He has been intensively involved in the capacity building of childhood cancer care in his own country since the end of the Syrian war in 2018. 

His contribution has led to a major improvement in levels of care and patient outcomes over the past 5 years. He is a regional pediatric oncology leader and received multiple international awards from ASH, EBMT and was recently selected for the first SIOP Young Rising Star Award during SIOP 2022 in Barcelona.

Moderator

Gevorg Tamamyan is the Editor-in-chief of OncoDaily, Head of the Pediatric Cancer and Blood Disorders Center of Armenia, Chairman and Professor of the Department of Hematology and Pediatric Oncology at Yerevan State Medical University, Dr. Tamamyan has also been elected as the President of SIOP Asia 2024 and the Pediatric Oncology East and Mediterranean (POEM) Group.

0:15 Introduction
3:15 Leo Kager
10:07 Jaume Mora
18:12 Discussion
23:27 Khaled Ghanem
25:53 Discussion

Follow the transcript below

Gevorg Tamamyan: We are continuing with our first Global OncoThon and it’s already nine hours we are live and we are today start trying to raise awareness, we are raising awareness about pediatric cancer and trying to get funds sponsorship for the pediatric cancer research and drug development specifically through the Alice Fund, we are trying to support OncoEuros biosciences in their great work to develop pediatric cancer medications and to run clinical trials in pediatric oncology field.

We have incredible guests from all over the world and without further Ado I’m going to introduce our next wonderful speakers and guests.  It’s a great honor for me to introduce Dr. Leo Kager, Dr. Jaume Mora, and Dr. Khaled Ghanem from three different countries.

Professor Kager is my mentor and I’m happy to say that, because I learned so much from him and I was fortunate to get a training under his mentorship he’s the head of outpatient department at the at one of the leading Children’s Hospital in Europe Santana Children’s Hospital and he has been done incredible research and work in the sarcoma field and he he is the Vice President of cooperative of to sarcoma study group and world known pediatric oncologist.

Dr. Jaume Mora the scientific director of another great pediatric Cancer Center  this time from Barcelona, a world known expert in the neuroblastoma field and they build a really wonderful program around different cancers, pediatric cancers at the Barcelona Center and now is it is one of the leading pediatric cancer centers  globally.

And my good friend Dr. Khaled Ghanem from Syria he is leading the BASMA Unit in Syria Damascus, when he returned back he started doing great work with very limited resources trying to trying to cure kids with cancer and he’s going to show also what amazing work he has been doing there.

So I would like to start with Professor Kager, my dear mentor,  how he was doing in this pediatric oncology field and what his his opinion about the development and his ideas about the Pediatric drug development and what he would like to share today with us on International Childhood Cancer Day.

Leo Kager:  Yeah hello everybody, hello Gevorg, it’s a great to seeing you. I’m not your Mentor you’re my mentor yeah to be honest just one publication that we had together on a review, you were on the paper and that was cited 160 times I’ve never had a review that was cited so-so high, so you you’re you’re doing really a fantastic job.

So I started at Saint Ana Children’s Hospital 30 years ago to work in the field of pediatric oncology in the last 10 years I also switched in a little bit to hematology so because it’s the full punch of our field and in Vienna we are lucky because we have the children’s cancer research institute which was founded 35 years ago.

And I have the privilege to be the head of this Research Institute and in the research institute we have about 30 people working there, young researchers from more than 35 countries so this is and I think this is very important that you bring together the people in the field of pediatric oncology we deal with extremely rare diseases and we have to learn from each other.

But Gevorg, we met first at the open Medical Institute in in Salzburg, the Salzburg seminars um and I think this is also an important part for having, bringing pediatric oncology forward we have every second year there these seminars in Salzburg where people from the chob, people from a Austria and from all over the world come together and share their their knowledge.

So we learn a lot uh at this conferences and and so on so but I do not want to talk too long because I think the other speakers want to talk as well or or do you want me to continue.

Gevorg Tamamyan: Please continue we have time and we’ll have speaker and then we’ll have some discussion 

Leo Kager: Okay. So, what I consider very important we have in in the field of pediatric oncology, we have diseases acute, for example, acute lymphoblastic leukemia. Every drug you try in this disease, obviously it works more or less and then we have diseases like osteosarcoma. Every drug we try in the last 30 years we had these drugs that we use now MAP is the standard.  They worked 30 years ago and we do not see any new, great signals from other tracks except for the DKIs.

So, and this is I think, very important, in the field of leukemia, we do a lot of harm with our chemotherapy. And I’m heading here also the late effects clinic. So to see, osteonecrosis, we get reviews, risk factors for osteosarcoma, but we have actually no treatment at all for osteonecrosis, right. And, so, and this is a real big burden for the patients. And here we have seen, of course, improvements with the, novel therapies, especially the immune therapies.

And I think here it’s very important that pediatric oncology comes closer to the adult oncologists, because they cannot give that intense, chemotherapy, so they have to treat other, treat with other modalities. And so we can learn from the adults, for example, AML M3, we have treated our patients very intensive. The adults have shown us that we just need two drugs to cure a patient with, APL.

And so, and on the other hand, in diseases like osteosarcoma, it’s very important that we have, like, for with your Aamos 1, the international trial that we have, such international collaborations. And for the first time, we have now in Europe, the so-called Foster Consortium, that is, fight osteosarcoma through European research. And we will, we got funding and we will open a trial with a TKI soon, and this will be spread throughout Europe.

So for the first time, we have a real, European, osteosarcoma study. And I think here this is very important. And, it’s my first thoughts.

Gevorg Tamamyan: Thank you very much, Dr. Kager. Thank you very much for sharing that.

Dr. Mora, Could you please share your ideas about pediatric drug development and your experience? You’ve been in this field for many years and have been leading final trials, so your insights are invaluable. Where do you see gaps in our current approach, and what steps do you think we should take to propel the field forward?

Jaume Mora: Well, good morning everybody, and thanks Gevorg and colleagues for sharing their thoughts. In this very particular and small world where we’re dealing with very rare diseases.

My first thoughts regarding your question are about how long it has taken our community to realize how important it is for us to carve our own path instead of following the tracks from the adult cancer field.

For a long time, we’ve been distracted because the majority of pharmacological development and investments were focused on adult cancer. We were lured by the new drugs and potentials developed for adult cancers, thinking we just needed to follow their paths to find answers for our patients. However, that approach has proven to be wrong and unsuccessful.

It’s now well-recognized that very few drugs have been approved for pediatric cancers in the last 20 years, highlighting our misguided approach. We are responsible for this failure. Unlike adult cancers, the reasons why children and adolescents develop cancer have nothing to do with aging-induced cancer. Carcinomas have nothing to do with children’s cancers.

Therefore, drugs developed for adult cancers, including targeted therapies like those for lung cancers, do not necessarily work the same way for pediatric cancers. For example, drugs targeting EGFR or BRAF mutations in lung cancer don’t have the same efficacy when dealing with mutations in neuroblastoma.

Realizing this, we’ve started to perform our own research, but it takes much longer due to the rarity of pediatric cancers and the lower amount of investment. It’s possible we are also less experienced compared to adult cancer field investigators. Bringing a new discovery into a patient’s life takes a considerable amount of time.

For instance, studies on anti-GD2 antibodies, which have shown promise in increasing the survival of children with high-risk neuroblastoma, took over 30 years to finally result in FDA approvals.

Moreover, we face numerous regulatory hurdles in getting our drugs approved. Therefore, advocating for specific research, funding, career support for investigators, and a real focus on understanding children’s tumors are crucial. We need to develop drugs specifically tailored to help pediatric patients rather than relying solely on developments in the adult cancer field, which have proven to be inadequate for our needs.

Gevorg Tamamyan: Thank you very much, Dr. Mora. Certainly, as we often emphasize in the pediatric oncology world, kids are not small adults. Therefore, we need to be more proactive in advocating for dedicated research in pediatric cancer, rather than it being seen as an appendix to adult studies. Funding should be specifically allocated for pediatric cancer research.

I’d like to give the floor to Dr. Ghanem, who is working in incredibly resource-limited areas but has achieved remarkable results despite the challenges. While we talk about boosting drug development from 85 to 100% in developed countries, the reality in other parts of the world is vastly different. For instance, even obtaining medications that are 50 years old can be challenging in some regions. It’s essential to consider this perspective.

Dr. Ghanem, could you please share your insights on this matter? Your experience working in resource-limited settings will provide valuable perspective. Additionally, when advocating for new research, we must ensure that low and middle-income countries are included and that the disparity in access to treatments does not widen.

Until he comes back, he lost connection; I think in Syria, the connection is a problem. Before he comes, one question to you: What do you think? This is the global OncoThon; we are trying to get people together to advocate to try to move the agenda forward. What else on the practical note, like tomorrow, what else we will be able to do that we move at least like one meter forward? Is there anything right now we can do in that regard? Who would like to be first? Professor Kager, I think you want to.

Leo Kager: So, I mentioned before, the example with adults, not to get the wrong opinion. I fully agree with Dr. Mora that in the field of solid tumors, etc., it’s not easy to learn from adults, but in certain diseases, it’s important also to have the view on the adults to make this point. We’ve been doing this for 35 years in the CCRI here, focusing solely on children and young adults, not adult oncology.

Our research is solely dedicated to understanding the mechanisms and identifying which drugs will work in certain pediatric patients. While we have made progress with drug screens for certain patients, the big platforms do not always yield the expected results. We wanted to see treatments that work in every patient, but that’s often an illusion.

However, employing a mechanistic model and improving drug screening platforms in pediatric oncology can lead to significant advancements in the next few years.

Gevorg Tamamyan: Thank you very much, Dr. Kager. CC is indeed an incredible organization, and I’m sure it has contributed significantly to many advancements in pediatric oncology.

Dr. Ghanem is back. Let’s give him the floor, and then Dr. Mora will answer the question. Khaled, can you hear us? I think we are experiencing some voice issues again. Let’s try to correct them. Maybe Dr. Mora will answer the question, and then we’ll come back to Dr. Ghanem.

Leo Kager: Um, well, there are so many different variables that we can entertain here to respond to your question, but I think it’s important to realize and highlight the fact that the most important prognostic factor nowadays to cure a child with cancer worldwide is, you know, the postal code—where the patient and the family reside. So, the inequalities in access to cancer care are huge. Well, I’m not discovering anything novel to anyone here, but I think we need to highlight such, right?

It is well-known that at the CC in Vienna, for instance, they can cure close to, you know, 80% of all the children with cancer, most likely even higher for the leukemias. In Latin America, for instance, where we’ve been working very actively, the median survival for children with ALL doesn’t reach 50%, for instance, and it’s much lower. In China, it’s been reported recently a 54% survival rate.

Khaled Ghanem: If you hear me, my message is very simple here: we all want to support children with cancer all over the world. However, children with cancer in crisis areas, especially in some countries in our region, need actually more support than others. In countries where there is no crisis or war, we all know that the burden of childhood cancer on families and children is known to change their lives physically, emotionally, and socially.

However, in crisis areas where there is economic crisis or war, this burden significantly intensifies to a level that sometimes the patients or families cannot tolerate. So, our message from areas under crisis, like Syria and other countries in the world, is that please support these children and make your effort to have initiatives very specific to children with cancer in crisis areas.

Gevorg Tamamyan: Thank you very much, Dr. Ghaem. Yes, especially in crisis areas, it’s often the case, and I can echo your sentiments because we have experienced living in these areas as well, and I know what it is.

Yes, we heard your voice message very well. Thank you very much for taking the time to connect. Dr. Mora, sorry we interrupted you. Please continue. I think you were talking about the same topic Dr. Ghanem was mentioning.

Leo Kager: What Dr. Ghanem was talking about is well-taken and very pertinent. Our colleague from Syria is highlighting the fact that children with cancer can be cured, but it mostly depends on the socio-economic environment. Crises or wars are the worst-case scenarios where life is threatened not only by cancer but by many other factors. All my admiration goes to colleagues worldwide who are struggling with families and patients in such terrible conditions.

Having said that, it’s essential as a community to realize that we have the knowledge, tools, and mainly very old and cheap drugs to cure many children with cancer. It’s all a matter of developing local expertise and providing the right drugs for everybody to access first-line therapy, at least for the most easily curable children’s tumors. That’s why OMS has launched the Global Pro program, where we aim to cure or at least achieve up to 90% cure rates worldwide for the six most common and treatable children’s cancers.

Regardless of where we are, we should be communicating, helping each other, and providing resources so that more and more families can have the opportunity for cure. We know how to effectively treat Wilms tumor, retinoblastoma, low-grade Lymphoma, and so on. There’s no excuse for us as a community to deny these families the real opportunity for a cure.

Gevorg Tamamyan: Thank you so much. Thank you very much, Dr. [MOA], Dr. Kager, and Dr. Gham, for taking the time in this very busy day for all of us to join our discussion and sharing your ideas about the problems we are facing. Thank you very much, and have a great day. We are moving to the next session. Thank you.