Forscherdatenbank
Prof. Dr. Stefan Pfister
Abteilung Pädiatrische Neuroonkologie
Im Neuenheimer Feld 580
69120 Heidelberg
Programm
Molecular Diagnostics, Early Detection, and Biomarker Development (MDEB)
Übersicht
INdividualized Therapy FOr Relapsed Malignancies in Childhood
The INFORM-Register Study (INdividualized Therapy for Relapsed Malignancies in Childhood or Individualized Therapy for Regression of Malignant Tumors in Children) is a DKTK cross-site initiative in the field of pediatric oncology, which is expected to develop from a registry trial into an umbrella trial. With INFORM, the possibilities of personalized medicine open up a second chance for cancer-prone children who suffer a relapse. The registry study started in January 2015 and has sequenced approximately 260 tumor samples from patients by the end of 2016.
The plan is to perform the genomic analysis of this INFORM-2 trial within the MDEB program (starting 2017).
Pediatric patients with disease relapse will be prospectively treated, according to obtained molecular information, with matching combinatorial targeted drugs. In particular, data from the analysis of ctDNA in blood plasma will be used to monitor patients during diagnosis and treatment. Tumor samples will be subjected to a comprehensive multi-platform molecular analysis that allows for the detection of several classes of genomic alterations.
A homepage, http://www.dkfz.de/de/inform provides information about the register. INFORM is coordinated by Prof. Stefan Pfister, Prof. Peter Lichter, Prof. Olaf Witt, Heidelberg, and Prof. Angelika Eggert, Berlin.
At the end of 2016 the register was extended to include Swedish and Austrian patients. The participation of other countries is being prepared.
The INFORM registry study is the basis for a subsequent clinical trial series INFORM2. The study participants are to be assigned according to their sequencing results into one of several parallel subgroups with combinations of targeted medications.
DKTK Junior Group Leader for Cancer Systems Biology
Single-cell approaches have not only revealed a wide variety of cell states, characterized by cells exhibiting striking differences in their transcriptional profile, but have also illuminated the mechanisms underlying state transitions in health and disease. Cellular plasticity and adaptive state changes have recently emerged as a basis for therapeutic resistance in cancer, and a better understanding of how cell state transitions are regulated is critical to develop therapeutic approaches that can overcome therapy resistance.
Our research focuses on understanding the mechanisms driving non-genetic cellular heterogeneity and therapy resistance in malignancy. Using novel single-cell sequencing approaches, we seek to develop new experimental and computational strategies to define altered cell states in both, cancer and immune cells. Our aim is to leverage a data driven strategy combined with single cell genomics and systems biology to address the challenges posed by heterogeneity in cancer, and to develop new strategies to overcome it, with the aim of translating laboratory-based findings into the clinic.