ENRare tumours have few large trials and no ready-made regimen for every case, so the choice of drug increasingly rests not on the organ but on the changes found in the tumour cells. Targeted drugs, medicines aimed at a specific molecular target in the tumour, are one way of doing this.
Targeted therapy in Germany for a rare tumour starts with the search for such a target. Below: how targets are found, the options for tumours of the small intestine and the thymus (the gland behind the breastbone), where this is done and how to arrange it. Surgery and other methods are covered under small intestine cancer treatment, while “thymus therapy” with animal thymus extracts is something else entirely and has nothing to do with treating thymus cancer. General principles are explained on the targeted therapy page, and thymoma treatment and cost on a separate page.
Why molecular testing decides in rare tumours
Molecular testing decides because the choice of targeted drug increasingly rests on a target found in the tumour itself, and without testing nobody knows whether there is one. The usual method is next-generation sequencing (NGS): testing tens or hundreds of genes in tumour tissue at once. A paraffin block, tissue embedded in wax after an earlier biopsy or operation, is usually enough. Sometimes the blood is tested for fragments of tumour DNA.
The results go to a molecular tumour board (Molekulares Tumorboard) of oncologists, pathologists, geneticists and bioinformaticians. It decides whether there is a target with a proven drug, whether a clinical trial makes sense, or whether there is no target. Not every patient has a target, and then surgery and chemotherapy remain the basis.
Targeted therapy for small intestine cancer
Targeted therapy for small intestine cancer depends on the tumour type, because the name covers different diseases: adenocarcinoma (cancer of the bowel lining), gastrointestinal stromal tumour or GIST (from the bowel wall), neuroendocrine tumours or NET (from hormone-producing cells) and lymphomas (from immune cells). Each is treated differently.
GIST is the classic example of targeted treatment: with KIT or PDGFRA mutations, tyrosine kinase inhibitors are used, drugs that block the cell’s growth signal. The mutation type affects the choice of drug and the dose. For NET, doctors use somatostatin analogues, which resemble one of the body’s hormones and hold back tumour growth. If the tumour keeps growing, the tumour board decides on targeted drugs or radioligand therapy, in which a radioactive substance reaches tumour cells via their receptors.
In small bowel adenocarcinoma, doctors test HER2 (a growth receptor on the cell surface), the BRAF gene, microsatellite instability (a fault in DNA error repair) and other targets; with instability, immunotherapy is discussed. For chemotherapy, see chemotherapy in Germany. NET treatment is set out in the guideline on gastrointestinal neuroendocrine tumours of the German Society for Gastroenterology (DGVS), in German; it was issued in 2018 and is currently being updated.
Targeted therapy for thymus cancer
For thymus tumours, targeted therapy is not the first step: treatment rests on surgery, with radiotherapy and chemotherapy where needed. Thymoma and thymic carcinoma are tumours of the thymus, a small gland behind the breastbone. Thymomas often come with autoimmune diseases, above all myasthenia gravis: muscle weakness from faulty signal transmission between nerve and muscle. For treatment and billing, see thymoma treatment and cost.
Targeted therapy is discussed when the tumour has spread or returned after chemotherapy: multikinase inhibitors, which block several growth signals at once, and drugs acting on the mTOR pathway, which controls cell growth. A rare KIT mutation is a reason to discuss a drug aimed at that gene. Some prescriptions are off-label, outside the drug’s licensed uses, and require agreement with the tumour board and the insurer.
Immunotherapy, which releases the brakes on the immune system, calls for particular caution in thymoma: it can trigger severe autoimmune reactions against the body’s own organs. So an experienced centre makes the decision.
Where molecular testing and treatment are offered in Germany
Molecular testing and treatment selection are offered by personalised medicine centres at university hospitals. At the LMU Comprehensive Cancer Center in Munich, Dr. Benedikt Westphalen heads precision oncology, which matches treatment to the tumour’s molecular profile, and the Centre for Personalised Medicine. At Freiburg University Hospital, Prof. Justus Duyster is Medical Director of haematology and oncology and speaker of the Freiburg Centre for Personalised Medicine.
University Hospital Würzburg has a neuroendocrine tumour centre certified by the European Neuroendocrine Tumor Society (ENETS) and a Centre for Personalised Medicine; Heidelberg University Hospital also has one. These centres belong to the German Network for Personalised Medicine (DNPM) of university hospitals, so tissue can be sent for testing before travel.
Frequently asked questions
Can targeted therapy be chosen without travelling to Germany?
Partly: the molecular analysis can be done on tissue sent from home, but treatment is prescribed after an in-person consultation. Paraffin blocks and the histology report go to a personalised medicine centre for sequencing, and the molecular tumour board reports whether there is a target and which drug to discuss. Before prescribing, the doctor examines the patient and reviews recent tests.
What if no target is found in the tumour?
Then treatment follows the usual rules: surgery and chemotherapy remain the basis, with radiotherapy for some tumours. The tumour board may suggest a clinical trial if the patient meets its criteria. A missing target does not mean nothing can be done; it means only that no drug with proven benefit against this tumour’s changes exists yet.
Can targeted therapy continue at home?
Yes, if the drug comes as tablets and is available in the patient’s country. Once treatment has started in Germany, the plan goes to the local oncologist: the drug, follow-up tests and scans, and warning signs that need a doctor at once. Follow-up results are best sent to the German centre so that treatment can be adjusted.
How AlenMed arranges treatment
AlenMed starts with the documents: histology report, paraffin tissue blocks, discharge letters and scans. We translate them into German and send them to a personalised medicine centre, which replies whether there is a target and which drug is under discussion.
Molecular testing and drugs are paid according to the hospital’s estimate (Kostenvoranschlag). AlenMed prepares the visa invitation, arranges an interpreter and accommodation, and hands the plan to the local oncologist if tablet treatment continues at home. AlenMed’s office is in Munich; partner hospitals are all over Germany.
This material is for information only and is not an offer. The treatment plan is set by the doctor at an in-person consultation.
News on this topic
Charité treats first beta-thalassemia patient with CRISPR
At Berlin Charité, a 19-year-old beta-thalassemia patient became the first in Germany to receive the newly approved CRISPR gene therapy Exagamglogene Autotemcel.
Real-World Breast Cancer Study Confirms High Five-Year Survival Rates in Germany
A landmark study of over 43,000 German patients reveals a five-year invasive disease-free survival rate of 82.9 percent in early hormone receptor-positive breast cancer.
FDA Approves Jülich Diagnostic Radiotracer FET for Brain Tumor Imaging
The FDA approves the diagnostic radiotracer FET (Pixclara®) for evaluating brain gliomas, highlighting decades of neuro-oncology research at Forschungszentrum Jülich.
