Other clinical trials and new advanced therapies in children

In addition to pharmaceutical and device research, clinical medical research in children and adolescents is very diverse and covers all specializations in pediatrics, including surgery and orthopedics, as well as pediatric and adolescent psychiatry. The total number of new recognizable diseases is constantly increasing with studies of children’s genomes (genes), metabolism and pathogens, among other things. In medicine, new methods are constantly being sought to identify and treat various types of untreated childhood diseases.

Researchers and companies in children’s diseases also collaborate extensively with other disciplines (e.g. engineers and biochemists) using the latest technology and the latest researched knowledge. Among other things, the excellent treatment results of children’s blood and cancer diseases are the result of long-term and high-quality research work and international cooperation between researchers.

The latest treatments for children’s diseases have been found with the help of Advanced Therapy Medicinal Products (ATMPs), i.e. so-called gene and cell therapies and tissue modifications. These studies of ATMPs are part of drug trials and target very serious and rare diseases for which other treatment options are not sufficient. In these very early (stage 1 and 2) studies, the number of subjects is very small at once and research requires specialized researchers and research centers. The latest ATMP treatments have so far been developed for children to treat some rare diseases and blood and cancer diseases (e.g. gene preparation for spinal muscle atrophy, SMA and CAR-T cell therapy for B-cell lymphoblastic leukemia, B-ALL). ATMPs may also include a medical device (e.g., a device as part of a structure), in which case they are so called combination products (medicine + medical device).