Oncothon: Challenges And Successes In Pediatric Hematology and Oncology

Oncothon is a telethon spanning 24 hours, dedicated to gathering donations for childhood cancer research. Professor Schlegel is the Chief of Pediatric Hematology/Oncology/Stem Cell Transplantation departments at the University Children’s Hospital of Würzburg, Germany. During this presentation, he will represent successes and challenges of pediatric cancer care and also will talk about CAR-T cell therapy.

Speakers

Paul-Gerhardt Schlegel is Chief of the Pediatric Hematology, Oncology, and Pediatric Stem Cell Transplant Program of the University Children’s Hospital. He has been a member of The International Society of Pediatric Oncology (SIOP) since 1995 and The European Group for Blood and Marrow Transplantation (EBMT) since 1997.

Professor is a Consultant Member of the Munich State Certification Board for Pediatric Hematology and Oncology, State Chamber of Physicians, Member of the Board of the Comprehensive Cancer Center Mainfranken CCCMF, Wuerzburg and Pediatric Board Member, Secretary DAG-KBT, German Working Group for Blood and Bone Marrow Transplantation.

He has been awarded several highly regarded international prizes, which include the Nycomed Prize, the Young Investigator Travel Award of the Kind-Philipp Foundation, and the Young Investigator Translational Research Award.

Moderator

Ricardo García is the Co-Founder, Chief Executive Officer, and a Member of the Board of Directors at Oncoheroes Biosciences. Ricardo has a diverse background in computer engineering, management, and business administration.

His journey into entrepreneurship began early, leading to the successful sale of his first company when he was just 22 years old. This led Ricardo to start and grow multiple tech-focused companies, all driven by a passion for innovation.

In 2011, his son Richi was diagnosed with a brain tumor. This challenging experience prompted Ricardo to uproot his life and relocate to Boston to support his son’s treatment journey. During this period Ricardo discovered significant gaps and challenges in the support system for childhood cancer patients and their families.

Motivated by his journey, Ricardo founded the Richi Childhood Cancer Foundation, a non-profit organization with a global outreach.

The foundation aims to address the unmet needs in childhood cancer care and support, leveraging Ricardo’s passion for making a meaningful difference in the lives of children and families affected by cancer.

The Transcript of Oncothon: Challenges And Successes In Pediatric Hematology and Oncology

Ricardo Garcia: We have another video. This time we’re talking about an eight-minute video from another speaker that could not join us today. At this time, his name is Paul Schlegel from Germany.

He’s the chief of the pediatric hematology oncology and stem cell transplant program of the University Children’s Hospital of Würzburg. I hope I’ll say that correctly from Germany. Yeah. Let’s go ahead with the video.

Paul-Gerhardt Schlegel: Dear colleagues, a very warm welcome to Childhood Cancer Day 2024. My name is Paul Schlegel from Würzburg, Germany, and I’m extending our warmest regards from the pediatric hematology oncology team in Würzburg, Germany for Childhood Cancer Day.

Last year, as you well remember, we have had a most remarkable event of SIOP Asia meeting in Armenia.

In Yerevan, we have experienced extremely nice hospitality, but most importantly, we have had excellent talks and many, many discussions which hopefully will help to improve knowledge in these rare diseases and which will also hopefully help to improve treatment for those children in many countries around the world.

 Let me come to Childhood Cancer Day. What are the successes of our journey that we have reached so far? And these successes are really remarkable. Over the years, we have seen a tremendous improvement in the survival rates of many pediatric childhood cancers. 

When you look back at the 1950s and 60s, survival rates for most of these cancers were down as low as 0 to 10%. And over time these have improved. 

For example, shown here in red for acute lymphoblastic leukemia, which now has a prognosis of about 80 to 90%. And this tremendous improvement has been able to be reached for many other disease entities. 

When it comes to childhood cancer, let us remember that every child and every family really matters. There is one moment in the life of a family that changes a whole life.

When diagnosis of for example, of leukemia or of a brain tumor or solid tumor is offered to the family. And when they fear for the life of their children. These diseases are rare, and the children in many instances are critically ill.

Now, let us remember what has contributed to the success, to the tremendous success that we have seen over the last decades. And today, I’ve brought you three major ingredients, which I find have been essential to this remarkable development. 

Number one, we work in multidisciplinary multi-professional teams right from the beginning. Every child needs specific care in a pediatric oncology center. We have learned that only by connecting and by working closely together in clinical studies and registries the success rate of treatment has remarkably and tremendously improved.

And we have learned that it is very, very important to really consolidate our tools for diagnosis. And therefore, in many countries, probably most of the countries we now find a structure of reference laboratories.

These means that every diagnosed Gnostic step that is taken locally is also referenced in a national central lab.

Therefore providing a second network of security not only to the treating pediatric oncologists but also the families and most notably, to the children. This has been a tremendous effort to establish those national reference centers in many countries they exist.

In some countries they are right now in the process of being established. And this, in my opinion, has been one of the major hallmarks ingredients to really be able to achieve this success.

 Now let’s come back to our graphic. What do we see? We do not only see success, but we also see challenge. And let me show you this yellow mark. It shows that about 20% to 30% of children in some diseases cannot be cured by current means.

So this represents the challenge for the future years to come. And how do we address this challenge? Let’s go back to our cyclists on the mountains.

What are our challenges on the journey? And as one example, how one can address this, these challenges? I’ve brought you one example from a situation in refractory relapsed ALL, a situation where ALL has come back many times and has become unresponsive to chemotherapy.

And one of the major developments here was, as an example, the development of a so-called bispecific T cell engager.

This bispecific T cell engager is an artificial product, and it has two components. One component, shown here in red, is taken from an Anti-CD3 antibody, and it attaches to the CD3 receptor on a human T cell. The green one here comes from a CD19 antibody.

It’s linked to the red one by a protein spacer, and the green one attaches to the CD19 on the tumor cell lymphoma or leukemic cell. And by directing the T cell to the leukemic cell, this T cell gets activated and then, as shown with a red arrow, this T cell releases, for example, granzyme B and Perforin and other cytokines and they produce lysis of the subsequent tumor cell.

And with this principle, by a 28-day infusion, one is able to really cure an otherwise resistant leukemia. This is one of the most remarkable examples that we have seen in pediatric hematology oncology these last years.

Now, what do we achieve with such a bispecific engager? Here are the results of the Rialto Expanded Access Study, where you see in a population of pediatric patients who were refractory to all conventional treatment just by the addition of the antibody, plus or minus following stem cell transplantation, you can achieve long-term cure in quite a number of patients and thus saving many, many lives.

Here is shown the same results. Just split it by MRD responders and non-responders. It’s very obvious that those having a good MRD response are the ones who have a better outcome.

This is one example how to address this challenge and there are many other, many other challenges on the road and many other treatment options that are currently being evaluated and developed.

 Childhood Cancer Day we remember every single family that has been treated in our institution. We embrace their courage and their resilience in enduring all the treatments that have been necessary.

We embrace their courage to go forward and to help their children achieve life and achieve long term remission. This is a team effort of many, many players and many more to come.

On the right, you see a cartoon of one other development that is currently being developed at many places so-called Car-T cells. Chimeric antigen receptor T cells.

That will be another milestone in the treatment for our children. But for today, I want to close with the following remark. It is our goal. It is our impetus. It is our motivation to one day be able to say, no child should die of cancer. Thank you very much for your attention.

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