(Vienna, 08.09.2021) Childhood cancer is different from cancer in adults. Thus, it needs research specifically tailored to pediatric tumors, which requires dedicated funding. The need for medicines suitable for children, as well as improvements in long-term follow-up care for cancer survivors have been discussed with EU Health Commissioner Stella Kyriakides, M.Ed., and Austrian Health Minister Wolfgang Mückstein, MD, at St. Anna Children's Cancer Research Institute and St. Anna Children's Hospital.
Due to intensive research throughout the last decades, survival rates for childhood cancer have improved considerably. "However, this comes at a high price," explains Prof. Ruth Ladenstein, MD, MBA, cPM, head of the Clinical Trials Unit S2IRP at St. Anna Children's Cancer Research Institute (St. Anna CCRI). "We owe a great many successes to long-used drugs that are not specifically approved for the application in children (i.e., off-label use). Of course, that carries risks."
Put children into the research focus
60 to 70 percent of all childhood cancer survivors struggle with late effects of the disease and its treatment. "Therefore, we urgently need new drugs that are specifically tailored to children and are not only more effective, but also better tolerated," says Ruth Ladenstein, who also works as an oncologist at St. Anna Children’s Hospital. In this regard, pediatric cancer research needs to focus on both, early drug development and subsequent clinical trials across Europe. Assoc.-Prof. Kaan Boztug, MD, Scientific Director of St. Anna CCRI: "At our Comprehensive Cancer Center, jointly comprising St. Anna CCRI and St. Anna Children's Hospital, we cover the entire spectrum from bench to bedside, i.e., from basic research to new applications in clinical trials."
The selection of suitable drugs for cancer in children and adolescents needs to be based mainly on a molecular understanding of the diseases in order to initiate a revolution in pediatric cancer therapy with higher chances of better long-term survival and minimization of late effects. "Even those tumors that do not respond to established therapies at the moment should be treated efficiently and successfully in the future. To this end, all modern technologies in the research field must be exploited and the understanding of carcinogenesis in childhood cancer entities must be expanded," continues Kaan Boztug, who is also a physician at St. Anna Children's Hospital. In addition, there is a need to clarify, which of the results from adult oncology can be translated into childhood cancer. For example, St. Anna CCRI recently showed that so-called ALK alterations in childhood nerve tumors reduce the chance of survival. Accordingly, ALK inhibitors used in adult cancers could also be helpful in children with nerve tumors.
EU is an essential funding source
St. Anna CCRI closely collaborates with other centers of expertise, both nationally and internationally. "We built up a network of strong partners and together we can achieve a lot. However, we often still lack adequate funding because drug development in the field of rare diseases – and pediatric cancer is one of them – is not driven economically, compared with common indications in the adult field," explains Ruth Ladenstein. "This means that children need special attention and also special funding opportunities, so that the many open questions are taken into account in future funding programs," says Ruth Ladenstein.
That this hope comes true seems to be a realistic scenario. The EU Commissioner Stella Kyriakides, M.Ed., responsible for Health and Food Safety, points out: "Cancer is the leading cause of death in children over 12 months of age in Europe. The recent pandemic has confronted us with additional challenges. Regular checkups and sometimes, even therapies have been postponed. We all know what that means – not only in terms of clinical outcomes, but also of psychological stress for the families. That is why we put a special focus on childhood cancer in Europe’s Beating Cancer Plan, which is budgeted at 4 billion euros. We have now launched the first calls in the EU4Health program, which includes setting up a network for young adult survivors of childhood cancer." According to the Austrian Health Minister Wolfgang Mückstein, MD, Austria will fully support not only Europe’s Beating Cancer Plan, but also the Mission Cancer. The EU Mission Cancer initiative, co-developed by Ruth Ladenstein as a member of the EU Mission Cancer Board, aims to ensure that the problems associated specifically with pediatric cancers are actively addressed.
Equal treatment across borders
Another topic of discussion have been inequalities of childhood cancer survival across Europe. "We must ensure that children in Europe have access to state-of-the-art therapies," emphasizes Stella Kyriakides. Prof. Wolfgang Holter, MD, Medical Director of St. Anna Children's Hospital and Head of St. Anna CCRI, points out: "Our Children's Cancer Center has a long tradition in Cross Border Healthcare. It is our strong desire that the best clinical expertise as well as the latest research findings benefit all children in Europe, regardless of where they live." One tool to achieve equal chances of survival for all children with cancer in Europe is the European Reference Network for Pediatric Oncology, ERN PaedCan – coordinated by Ruth Ladenstein." This network connects comprehensive pediatric cancer centers, accredited by the European Commission to create a clear governance structure for knowledge sharing and care coordination," says Ruth Ladenstein. Wolfgang Mückstein comments, "Such networks for the first time provide a structural collaboration in healthcare across Europe. This will continue to be very important in the future."
ERN PaedCan comprises numerous strategies to make specialized know-how and life-saving pediatric oncology treatments accessible throughout the EU. One of them is the so-called twinning: Centers with high potential in “widening countries” become affiliated partners. Through twinning programs, these centers receive support from an established center to achieve higher standards of education, research and care. "To overcome existing inequalities in Europe, we need more twinning projects, which must be funded through non-competitive grants," Ruth Ladenstein urges. Stella Kyriakides agrees: "Dedicated training programs to support the pediatric cancer community in building strong multidisciplinary workflows are essential. With Europe’s Beating Cancer Plan, we have set ourselves an ambitious program. What matters now is the implementation, and here I ask all relevant stakeholders, media professionals and patients to work together. Thank you once again for the commitment and research you are doing here. Together, we can support children much better."
Meet the needs of childhood cancer survivors
A key goal of Europe’s Beating Cancer Plan is to improve the quality of life for cancer patients and survivors. Late-effects of childhood and adolescent cancer can remain undetected, or follow-up care is insufficient or rather it comes too late. After the age of 18, pediatric care is no longer responsible for the necessary long-term follow-up care. However, most adult medical facilities do not meet the special needs of this patient group. This is due to a lack of complete quality-assured information transfer in a condensed form for follow-up care with targeted medical check-ups as well as treatment plans for follow-up care for the diagnosis and therapy of late effects after successful cancer therapy.
One tool designed to counteract this lack of information and to facilitate individual follow-up care is the Survivorship Passport (SUPA), which was developed in several EU-funded projects and has since been awaiting implementation throughout Europe. As a first step, the Austrian Cancer Plan included the implementation of SUPA as an operational goal back in 2014. According to this, every person suffering from childhood cancer in Austria should receive a SUPA, which summarizes oncological diagnoses and therapies and contains individual follow-up recommendations.
“Survivors Austria”, a patient organization that has been a strong supporter of the project from the beginning, is once again calling for SUPA to be implemented in the electronic health record ELGA. Board member Mag. Carina Schneider, who has become involved as a patient representative since her own cancer diagnosis in her adolescence, calls on politicians to wait no longer: "We have established the Survivorship Passport model in Austria so many years ago, all of Europe has invested a lot in its development. Now its integration into ELGA is the necessary next step, so that SUPA can finally support survivors. When will our healthcare system make it possible for us to make use of SUPA to better deal with late-effects?"
"You are right. The Survivorship Passport has been postponed because of the pandemic. Now we care about moving this important issue forward," announces Wolfgang Mückstein.
Katharina Reich, MD, Head of Department VII for Public Health and the Health Systems, adds, "It is of great concern to us that survivors of childhood cancer can have their detailed medical history available like a read thread, by means of using state-of-the-art structures. Intensive work is being done on the project."
Photo: (from left) Mag. Carina Schneider, Univ.-Prof. Dr. Wolfgang Holter, Dr. Eleni Tomazou, Stella Kyriakides, M.Ed., Univ.-Prof. Dr. Suzanne Rödler, Assoc.-Prof. Dr. Kaan Boztug, Dr. Sabine Taschner-Mandl, Dr. Wolfgang Mückstein, Mag. Jörg Bürger, Univ.-Prof. Dr. Ruth Ladenstein, MBA, cPM, Dr. Davide Seruggia, Dr. Erwin Senoner, Dr. Katherina Reich
Copyright: St. Anna Children´s Hospital/Kellner