Desmoplastic small and round cell tumour (DSRCT) and synovial sarcoma (SySa) are rare, aggressive tumours of the soft tissue. Adolescents and young adults are disproportionately affected by the two types of sarcoma. The disease has been difficult to treat and relapses are common.
The PAMSARC clinical trial, which has now started at the NCT Heidelberg, is dedicated to finding better treatment options for the two types of sarcoma. It has emerged from the results of the DKFZ/NCT/DKTK MASTER programme and the HEROES-AYA consortium. The latter is a flagship project of the National Decade Against Cancer, led by the NCT Heidelberg and the Hopp Children's Cancer Center Heidelberg (KiTZ), which translates fundamental findings on the biology of specific sarcomas in adolescents and young adults into clinical action.
Richard Schlenk, head of the NCT Clinical Trial Center in Heidelberg, designed PAMSARC and is leading the study. He says: ‘When patients suffer a relapse, it is usually followed by intensive chemotherapy to reduce the tumour mass or at least stop its growth. In PAMSARC, we are investigating the extent to which the drug pasireotide can maintain this treatment success.’
Pasireotide inhibits growth by binding to special receptors, the somatostatin receptors. It is approved for the treatment of Cushing's disease and acromegaly, two disorders caused by tumours in the pituitary gland. The PAMSARC study is testing the drug outside of its originally intended and approved indications.
‘In developing PAMSARC, we worked very closely with the German Sarcoma Foundation as a research partner – it was a very valuable dialogue between equals,’ says Richard Schlenk.
Patients who are eligible to participate in PAMSARC are those in whom the somatostatin receptors are present on the tumour cell surfaces far more frequently than normal. The researchers use molecular biological methods, such as those used in the DKFZ/NCT/DKTK MASTER programme, to determine whether this is the case. Participants must be between 13 and 50 years old. A total of 28 people can be enrolled in the study.
‘The first four patients are currently being screened for PAMSARC. Through the connection to HEROES-AYA, we can recruit in a cohort of adolescents and adults,’ says Richard Schlenk. PAMSARC is the first study at NCT Heidelberg for adolescents and young adults and thus brings paediatric and adult oncology together.
In PAMSARC, all participants will receive a monthly injection of the active substance pasireotide. The study will monitor the extent to which the treatment affects the time to disease progression, overall survival and aspects of quality of life in patients.
Stefan Fröhling, Managing Director at the NCT Heidelberg and Head of Translational Medical Oncology at the DKFZ, says: ‘With the HEROES-AYA consortium, we are pursuing the goal of quickly translating research findings into clinical applications for sarcomas. The PAMSARC study, which was designed in a very short time, is an excellent example of this.’
PAMSARC is an NCT proof-of-concept study from Heidelberg that serves as a bridge study in the nationwide NCT. The NCT is a long-term collaboration between the DKFZ, excellent partners in university medicine and other outstanding research partners at various locations in Germany.
