Rare Disease Day: development candidate SOMscan® receives orphan drug status
Eckert & Ziegler is also investing in a drug for rare diseases through equity investment OctreoPharm Sciences GmbH: a peptide (SOMscan®) for detecting neuroendocrine tumours, which the European Medicines Agency EMA recently awarded the coveted and financially rewarding orphan drug status.
With only two to five cases per 100,000 people, neuroendocrine tumors (NETs) belong to the category of rare diseases. NETs primarily occur in the gastrointestinal tract and the pancreas. They are called neuroendocrine because their cells are similar to the cells of endocrinal glands, such as insulin-producing cells in the pancreas. NETs grow slowly but can metastasize very early. Most of the patients diagnosed already have metastases, usually of the liver, and can therefore often no longer be cured by surgery.
"With the new drug we hope to make a method of diagnosis available to patients soon which allows for detection of neuroendocrine tumors at an early stage," said Karolin Riehle, press spokeswoman for Eckert & Ziegler AG. Depending on the radionuclide, the peptide can be used for diagnosis (Gallium-68) as well as for treatment (Yttrium-90 labeled) of the tumor. "The preliminary clinical results are so encouraging that we can soon start the necessary European clinical trials," said Dr. Hakim Bouterfa, Managing Director of OctreoPharm Sciences GmbH.
"There is huge potential in diagnosing rare diseases earlier and treating them better, thereby increasing the quality of life. The success of diagnostics and therapy depends on the active agent's ability to attach itself to the tumor. SOMscan® looks like a promising development candidate", says Professor Dr. med. Richard P. Baum, chief physician at the Central Clinic Bad Berka. The clinic (ENETS Center of Excellence) is one of leading facilities in the diagnosis and treatment of neuroendocrine tumours - both in Europe and worldwide. Radioreceptor therapy is a therapy based on nuclear medicine which can selectively fight the tumor, as the beta radiation only affects the diseased cells without damaging healthy tissue. These radiopeptides have shown to be particularly effective in patients where the tumor has a particular kind of receptor (magnet-like adhesion sites on the surface of tumor cells). The radio-labeled peptides are injected into the blood stream and attach themselves to the tumor cells and destroy them with beta radiation.
Heike H., a young patient from the Lake Constance region, benefited from this treatment. She developed a neuroendocrine tumor in 2001 and underwent a series of operations in the following years. In 2008 and 2009, she received her first and then second course of peptide receptor radionuclide therapy at the Central Clinic Bad Berka, which stopped the cancer from developing for a number of years. Her therapy has been so successful that Heike H. was able to conceive and gave birth to a healthy child.
About Octreopharm Sciences
OctreoPharm Sciences GmbH is a nuclear medicine specialist focusing on the clinical development of radiopharmaceuticals for the diagnosis and therapeutic control of tumors. Octreopharm Sciences GmbH is held by Eckert & Ziegler AG, VC Funds Technology managed by IBB Berlin GmbH, KfW Mittelstandsbank, Eckert Life Science Accelerator GmbH, OctreoPharm GmbH and Chinese investor Shaanxi Xinyida Investment Co. Ltd.
About neuroendocrine tumors
Neuroendocrine tumors (NETs) represent a group of rare tumors. In Germany there are an estimated one to two cases per 100,000 people. NETs generally occur in the gastrointestinal tract and the pancreas. They are called neuroendocrine because their cells are similar to those of endocrine glands such as the pancreas or thyroid gland. NETs grow slowly but spread very early. Most of the patients diagnosed already have metastases, usually of the liver, and can therefore often no longer be cured by surgery.
About rare diseases
Diseases are defined as rare diseases when less than five in 10,000 are afflicted. For medical and economic reasons, these fields are not sufficiently researched. These patients often lack medical care. In order to improve this situation, medical authorities are encouraging research of diagnosis and therapies for rare diseases and simplifying the approval procedure for new drugs.
Eckert & Ziegler Strahlen- und Medizintechnik AG
Eckert & Ziegler Strahlen- und Medizintechnik AG (ISIN DE0005659700) is one of the world's largest providers of isotope technology for radiation therapy and nuclear medicine and has almost 700 employees worldwide.
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