NF-2 is a genetically determined, very rare primary cancer that is extremely rare in children but is much more severe than in adults. The condition not only shortens lives, but also causes complex and severe disabilities. The disease results in the development of tumors of the central and peripheral nervous systems. This leads to paresis and paralysis due to the compression of tumors on various brain structures and peripheral nerves. One of the consequences is deafness with tinnitus. Balance disorder also occurs with facial and trigeminal nerve paresis as a complication, which dramatically accentuates symptoms and increases suffering for patients. In addition to paralysis, the development of peripheral tumors is accompanied by severe chronic pain that is difficult to treat.
Previous forms of therapy based on cancer surgery or pharmacotherapy have not worked. Radiation therapy even as technically advanced as proton therapy has also proved ineffective.
These experiences have led researchers to focus on so-called oncology targeted therapies. Preclinical trials and experimental studies testing the efficacy of small molecule drugs that are selective inhibitors of the ALK(RTK) tyrosine kinase receptor in the development of NF-2-specific tumors have proven promising.
A team of researchers from the Chair and Department of Pediatric Oncology, Hematology and Transplantology MUW, has developed a therapeutic protocol for the treatment with crizonitib (one of the TRK inhibitors) specifically for children with NF-2.
Since so far no company producing ALK kinase inhibitors has undertaken a clinical trial in this area, this gap will be filled by the researchers of the Medical University of Warsaw, whose project with the acronym KRONF2 has received funding in the competition of the Medical Research Agency.
This has provided an opportunity for effective treatment that if not eliminates the formation of tumors, should at least significantly reduce the disability and suffering of young patients and bring them back to society.