Pediatric Research Center
The Pediatric Research Center currently represents almost 30 research groups carrying out clinical, translational and basic research in pediatrics in the University of Helsinki, Finland. Our mission is to promote research, to communicate the latest research results to physicians, patients and the general public, and to serve as a reference guide for researchers and companies seeking academic collaboration. We warmly invite students and young physician-scientists to join our growing team!
Cell therapy has a long track record in medicine dating back to the transfusion of whole blood to wounded soldiers during the Second World War. A couple of decades of cell culture and animal research were required before the medical community felt ready for hematopoietic stem cell transplantation – the next key step in cell therapy. In the late 60’s, this opened a whole new field of therapeutic options for patients with inborn defects of immunity, diseases of hematopoiesis or hematological malignancies.
Thereafter and during the past four decades, hematopoietic stem cell transplantation has established its role in the treatment of a multitude of diseases of the bone marrow or bone marrow-derived cells with more than 500 000 transplantations performed in the industrialized countries, and more than 40 000 annually in Europe, both for pediatric and adult patients.
Yet, these steps have been taken by using unmodified or only mildly manipulated cells either from the bone marrow or peripheral blood, and relapses have remained a key concern for those with aggressive, hematological malignancies.
Development in cell manipulation techniques during the past ten years has provided us with all new tools to tackle these problems. T-cells with chimeric antigen receptors (CAR-T) have emerged as a viable, novel option particularly for patients with aggressive, hematological malignancies, who have either relapsed after an allogeneic stem cell transplantation or have been resistant to conventional primary therapy modalities. The basic idea is to genetically engineer patient’s own defender T-cells so that they can attack the malignant cells when transfused back to the patient. The first of these products (tisagenlecleucel) has just recently been approved by the FDA for clinical use as a 2nd line therapy among patients with lymphoid malignancies, and several others are in the pipeline.
This technology will open a whole new era of targeted cellular therapy, not only in the treatment of hematological malignancies, but also in that of many solid tumors (e.g. neuroblastoma, sarcomas etc.) and perhaps certain forms of hemophilia A.
To establish a role for the Academic Medical Center Helsinki and the Helsinki University Hospital in this rapidly evolving field, the University of Helsinki announced an opening for a full professorship in Cell Therapy and Transfusion Medicine through a donation by the Finnish Red Cross Blood Service. The undersigned was appointed to this post from September the 1st. In addition to the development in clinical cell therapy outlined above, a wealth of novel research questions have emerged with this development and will be tackled through several PhD thesis projects to be launched during 2018, under the auspices of the Pediatric Research Center and in close collaboration with the research department of the Finnish Red Cross Blood Service and the key research groups in the Academic Medical Center Helsinki.
Pediatric surgical research tackles with unique challenges. Pediatric surgery encompasses a few common childhood diseases and a surplus of very rare complex congenital anomalies. Practically all of them fulfil the definition of rare disease and most carry significant long-term sequela. In addition to surgery, which sometimes starts before the child is born and may continue in adulthood, successful treatment of developmental defects requires continuous input from a multitude of other specialties not only during childhood but also after transition to adult care. In most patients conclusive assessment of surgical outcomes is not possible before adulthood. (Read more)
”How is it that my sister has been asked to participate in your study but I still have not received an invitation?”, asked a relative who had heard about our research project. Recruitment to clinical studies can be as easy as this – patients volunteer before we even contact them! We researchers sometimes hesitate to approach our patients and recruit them to our study, fearing that it will burden them or that they will feel offended by our invitation. But during the past years, I have time and again been surprised by children’s and adults’ positive attitude and enthusiasm towards research. They do want to be part of our research, they want to contribute to scientific development, and therefore they should have the opportunity to participate. (Read more)
A few days ago I had the opportunity to meet the members of a newly elected board of parents at the Children’s Hospital. The members of this board have an important client perspective to the daily activities and services provided by the Hospital, and I was therefore quite curious to know if research is visible in our Hospital and what the general attitudes toward it are. It was perhaps not a great surprise to learn that our reputation is extremely good, and the parents are confident that their children always get the best evidence-based care. (Read more)
Certain moments in life lead us to new paths. In autumn 2015 I was finally finishing my residency in pediatrics. I love my clinical work and had really enjoyed the journey. Meanwhile my husband had specialized in anesthesia, the family had grown, we had built a house, struggled with weekly on-calls - the usual business many of us junior physician couples go through. (Read more)
I have just entered a 2.5-month research period in the midst of my pediatric residency. During this time, excluding a few night shifts on call, I will be able to concentrate 100% on research. As my current research involves cell culture experiments, I would not be able to do it without these lengthy breaks from clinical work. (Read more)