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  Biotechnologie / Life S...  
Leukonet: Evidenz für neue Therapien bei seltenen ZNS-Erkrankungen | 22.11.2010 | Wissenschaft
Males are more beneficial than previously assumed | 19.11.2010 | Science
  Biotechnologie / Life S...  
Unter einer solchen Therapie ließe sich laut Klör deutlich früher eine Verringerung des Blutflusses mit funktioneller Bildgebung erfassen, als dass eine Größenänderung des Tumors an sich im Schnittbild messbar werde. Tatsächlich nutzen speziell größere Kliniken diese neue „parametrische“ Bildanalyse bereits in der Differentialdiagnose von ZNS-Erkrankungen sowie in der Onkologie oder Rheumatologie.
The parameter imaging method is particularly suitable for the development of new therapeutics, medical devices and diagnostic methods. “Clinical data, including the imaging data, that achieve what they set out to achieve, highlight the potential of new therapies and point the way towards further targeted development,” Zinggrebe said. “The crux with newer drug substances used for treating cancer is that they have an excellent effect in some patient subgroups, but hardly any in others. This is mainly due to the drugs’ specific mechanism of action. Functional imaging provides information on whether a specific treatment scheme is effective or not at a relatively early stage. “This helps save valuable time, especially in cases where one drug proves ineffective and needs to be replaced by a different one,” Klör added. For example, a relatively new class of substances, known as angiogenesis inhibitors, does not directly kill the tumour cells. These drugs prevent the growth of new blood vessels, which tumours need in order to grow larger. Klör explains that functional imaging has the potential to detect the success or failure of cancer therapies involving angiogenesis inhibitors much earlier in the treatment process than slice images, which simply provide information about changes in tumour size. In fact, many hospitals, especially larger ones, are already using the innovative parameter imaging method for the differential diagnosis of CNS diseases, in the fields of oncology and rheumatology. “However, the application of such methods is not yet routine,” Zinggrebe added.
  Biotechnologie / Life S...  
Diese Stoffwechselerkrankungen beruhen in der Regel auf einem genetisch bedingten Enzymdefekt und führen oft zu schwersten Schäden im Bereich des zentralen Nervensystems (ZNS). „Dass die Transplantation von Blutstammzellen eines gesunden Spenders den Ausbruch eines neurologischen Krankheitsbildes verhindern kann, ist auf den ersten Blick sehr überraschend", berichtet PD Dr.med.
These metabolic disorders are often the result of a genetic enzyme defect and they lead to severe damage to the central nervous system (CNS). “When you first look at it, the fact that the transplantation of haematopoietic stem cells of a healthy donor can prevent the development of a neurological disease seems very surprising,” said Dr. Ingo Müller, chief physician in the University Children's Hospital in Tübingen. However, it appears that these haematopoietic stem cells are able to cross the blood-brain barrier and differentiate in the brain into supporting cells that are able to produce the missing enzyme. This enables doctors to correct the defective cell metabolism – at least locally. “This mechanism is referred to as cross correction,” said Müller who had already focussed on this phenomenon during a several-year research stay at the St. Jude Children’s Hospital in Memphis (USA). “It is not yet known in detail what happens,” said the scientist, but the results achieved so far are extremely promising.
  Biotechnologie / Life S...  
Zu verschieden sind ihre Funktionen: Das bis in die feinsten Kapillaren verzweigte Gefäßnetz sorgt dafür, dass alle Gewebe ausreichend mit Blut versorgt sind; die Nerven verzweigen sich vom ZNS (Gehirn und Rückenmark) aus, um elektrische Signale an die peripheren Organe zu vermitteln.
At first sight, the nervous and vascular systems have little in common. Their functions are far too different: the vascular network, an interweaving network of capillaries, supplies all tissues with sufficient amounts of blood; the nerves originate in the CNS (brain and spinal marrow) and transmit electrical signals to the peripheral organs. Despite all these differences, the nerves and vessels of vertebrates, including humans, are closely linked anatomically, something Vesalius presented most impressively in his marvellous woodcut illustrations back in the 16th century. Nowadays, the term “neurovascular bundle” is applied to body nerves, veins, lymph vessels and arteries that travel together in the body, suggesting biological links between these organ systems.
  Biotechnologie / Life S...  
Ein Forschungsschwerpunkt beschäftigt sich mit der bislang unheilbaren amyotrophen Lateralsklerose (ALS). An dieser chronischen Erkrankung des ZNS, an der drei bis acht Menschen von 100.000, meistens ab dem 50. Lebensjahr erkranken, forscht der 50-jährige Ulmer Neurologe Albert Ludolph seit 25 Jahren.
One major area of research focuses on amyotrophic lateral sclerosis (ALS), a condition for which there is currently no cure. Three to eight in 100,000 people suffer from this disorder, mainly found in people over 50. 50-year-old neurologist Albert Ludolph has been investigating ALS for 25 years. According to a French biotechnology company, approximately 80,000 people in the EU, USA and Japan suffer from ALS which rapidly leads to death of the patient. Only recently, Dr. Ludolph and his coworkers identified the third ALS gene. In people suffering from ALS, the motor nerve cells in the brain and spinal marrow are damaged. This disrupts the control of the skeletal muscles, leads to amyotrophy and death. For the majority of ALS patients the cause of the disease is not yet known. It is possible to influence the progression of the disease by a therapy using drugs which influence the neurotransmission of glutamate. Using transgenic mice, the Ulm neurologists are analysing the disease mechanisms of hereditary ALS which will eventually allow the development and testing of drugs.
  Biotechnologie / Life S...  
Bei dieser seltenen neurometabolischen Erkrankung kommt es aufgrund eines mutierten Gens zum Fehlen eines bestimmten Enzyms im Fettsäurestoffwechsel, der sogenannten Arylsulfatase A. Dadurch können Abbauprodukte der Fettsäuren nicht mehr verarbeitet werden. Es kommt zu Ablagerungen, die vor allem das ZNS schädigen.
When he returned to Tübingen, Müller, who has a degree in medicine and biochemistry, decided to establish the transplantation of stem cells for children suffering from metachromatic leukodystrophy (MLD), a disease that until then had been regarded as incurable. This rare neurometabolic disease is characterised by a deficiency of the enzyme arylsulfatase A as a result of mutations. The enzyme is involved in the fatty acid metabolism; without this enzyme, degradation products of the fatty acids cannot be processed and they build up in the body. The CNS is the area that is the most affected of all body tissues; the myelin sheaths protect nerve fibres. Without this fatty covering, the nerve fibres are eventually destroyed and cease to function. Affected children often develop severe neurological symptoms in the first years of life; they begin to have difficulty walking, further progression of the disease leads to paralysis, the loss of the ability to talk and blindness. The earlier the onset of the symptoms, the worse the prognosis.
  Biotechnologie / Life S...  
Funktionelle medizinische Bildgebungsverfahren haben einen hohen Stellenwert in der modernen medizinischen Diagnostik. Sie messen wichtige Größen wie Stoffwechselaktivität oder Blutfluss und helfen somit in der Diagnostik von vielen Erkrankungen, vor allem denen des zentralen Nervensystems (ZNS).
Functional medical imaging modalities are of great significance in modern medical diagnostics. By delivering information on changes in metabolism and blood flow, they make an important contribution to diagnosing diseases, in particular those of the central nervous system (CNS) such as acute stroke, brain tumours and neurodegenerative diseases such as multiple sclerosis (MS) and Alzheimer’s. Existing ways of diagnosing diseases are now being expanded with the arrival of the innovative “parameter imaging method”. syneed imaging, a division of the Konstanz-based contract research organisation syneed medidata GmbH focused on medical imaging, uses proprietary, highly sensitive software for the analysis of dynamic image data. This software complements standard methods such as computed tomography and magnetic resonance imaging and increases the significance of image data in clinical trials and therapy decisions.
  Biotechnologie / Life S...  
Zur weiteren Optimierung wird jetzt erforscht, ob sich die Nervenwurzel der einen Körperseite besser eignet als die der anderen oder ob man mit einer Verpflanzung auf beiden Seiten die besten Ergebnisse erzielt. Wenn zwei periphere Nerven erfolgreich miteinander verbunden werden können, ist es nicht weit zur Idee, das ZNS zur Überbrückung zu nutzen.
The method works well – at least in principle. The scientists are now working on the optimisation of the method. They are investigating whether the nerve root of one half of the body is better suited than the other or whether it is necessary to transplant on both sides for best results. Should the scientists succeed in successfully connecting two peripheral nerves, then it is not a big step to using the CNS as a kind of bridge. As soon as Tatagiba and his team have enough money, they will look into the fascinating option of nerve regeneration in greater depth. Despite his optimism, Tatagiba also understands the limits of this approach and does not want to raise false hopes: “The spinal marrow function is highly complex. That is the reason why complete regeneration is highly improbable.” However, the regeneration already foreseen will undoubtedly lead to an improvement in body function and hence quality of life.
  Biotechnologie / Life S...  
Nach der Akquisition der Amnestix wird SYGNIS einen besonderen Fokus auf die Zusammenarbeit mit TGen legen, um damit diese Kooperation zu intensivieren und die Einlizenzierung weiterer ZNS-Projekte zu ermöglichen.
Following the acquisition of Amnestix, SYGNIS will now be placing a special focus on the working relationship with TGen to intensify this collaboration and to make the in-licensing of further CNS projects possible. In addition, SYGNIS will continue to strengthen and extend the patent position of AX200 and move ahead with pre-clinical projects relating to KIBRA and the neuronal stem cell programme. The company’s goal is to set up collaborations for these projects with pharmaceutical partners in the coming months. The effective execution of the multinational phase II efficacy study is also a key focus.
  Biotechnologie / Life S...  
Auch nach einer Verletzung im zentralen Nervensystem (ZNS) versuchen die Nervenfasern nachzuwachsen. Es bilden sich winzige Fortsätze, die allerdings nicht einmal Millimeterlänge erreichen. Das liegt daran, dass das Myelin des ZNS im Gegensatz zum Myelin des peripheren Nervensystems (PNS) Faktoren enthält, die das Nervenwachstum hemmen.
After central nervous system (CNS) damage, the nerve fibres also try to grow back. They form small protuberances that nevertheless grow no more than a few millimetres. The reason for this is that, in contrast to myelin of the peripheral nervous system (PNS), myelin of the CNS contains factors that inhibit nerve growth. Prof. Martin Schwab from the ETH Zurich already found in the 1980s that the myelin sheath of the CNS has a negative effect on regeneration. Nowadays several inhibiting proteins (e.g., NOGO-A) are known.
  Biotechnologie / Life S...  
Für solche Analysen sind Methoden der Enzymbiochemie nötig, um die Aktivität von Neurotransmittern im synaptischen Spalt oder die dynamische Aktivität von Rezeptoren an den Nervenzellmembranen zu messen. Das ZNS war von vornherein einer der Schwerpunkte von IBAM.
The company continues to carry out much the same, and even more, as before. The company was founded by Dr. Peter-Andreas Löschmann from the Department of Human Medicine, Prof. Dr. Thomas Feuerstein from the Department of Neuropharmacology, Dr. Norbert Limberger from the Department of Pharmacy and Dr. Rainer Knörle from the Department of Physical Biochemistry. The company is also contracted by big companies, such as Novartis and Bayer. “For example, our clients might have a compound that exerts its effect on the nervous system where it reduces the sensation of pain,” said Knörle. “The companies usually contract us to identify the underlying molecular mechanism and to find ways to reduce or eliminate the compound’s potential side effects.” The analyses carried out by Knörle and the IBAM team involve methods that are used in the field of enzyme biochemistry in order to measure the activity of neurotransmitters in the synaptic cleft or the dynamic activity of receptors at nerve cell membranes. The CNS has been the researchers’ major research priority ever since the company’s establishment.
  Biotechnologie / Life S...  
Die Ulmer Neurologen setzen aus guten Gründen auf diesen Nischenmarkt. Längst spiele die klinische Forschung in Deutschland an Krankheiten des Zentralen Nervensystems (ZNS) international keine Rolle mehr.
The Ulm neurologists have good reasons to rely on this niche market. According to Ludolph, America's stranglehold on the development of profitable 'blockbusters' for common diseases such as Alzheimer’s and Parkinson’s seems impossible to break. As a result, German scientists stand a better chance of competing by developing orphan drugs for the niche markets. Promising clinical research is being carried out on central nervous system (CNS) disorders - a market, as yet, unexploited. Currently, CNS research in the German pharmaceutical industry is, according to Ludolph, restricted to a research group in a company in the south of Germany.
  Biotechnologie / Life S...  
Auch nach einer Verletzung im zentralen Nervensystem (ZNS) versuchen die Nervenfasern nachzuwachsen. Es bilden sich winzige Fortsätze, die allerdings nicht einmal Millimeterlänge erreichen. Das liegt daran, dass das Myelin des ZNS im Gegensatz zum Myelin des peripheren Nervensystems (PNS) Faktoren enthält, die das Nervenwachstum hemmen.
After central nervous system (CNS) damage, the nerve fibres also try to grow back. They form small protuberances that nevertheless grow no more than a few millimetres. The reason for this is that, in contrast to myelin of the peripheral nervous system (PNS), myelin of the CNS contains factors that inhibit nerve growth. Prof. Martin Schwab from the ETH Zurich already found in the 1980s that the myelin sheath of the CNS has a negative effect on regeneration. Nowadays several inhibiting proteins (e.g., NOGO-A) are known.
  Biotechnologie / Life S...  
Auch nach einer Verletzung im zentralen Nervensystem (ZNS) versuchen die Nervenfasern nachzuwachsen. Es bilden sich winzige Fortsätze, die allerdings nicht einmal Millimeterlänge erreichen. Das liegt daran, dass das Myelin des ZNS im Gegensatz zum Myelin des peripheren Nervensystems (PNS) Faktoren enthält, die das Nervenwachstum hemmen.
After central nervous system (CNS) damage, the nerve fibres also try to grow back. They form small protuberances that nevertheless grow no more than a few millimetres. The reason for this is that, in contrast to myelin of the peripheral nervous system (PNS), myelin of the CNS contains factors that inhibit nerve growth. Prof. Martin Schwab from the ETH Zurich already found in the 1980s that the myelin sheath of the CNS has a negative effect on regeneration. Nowadays several inhibiting proteins (e.g., NOGO-A) are known.
  Biotechnologie / Life S...  
SYGNIS hat auf dem Weg in Richtung eines produktorientierten pharmazeutischen Unternehmens mit der Spezialisierung auf die Behandlung von Erkrankungen des Zentralen Nervensystems (ZNS) erneut einen Schritt nach vorne gemacht.
SYGNIS Pharma AG announced the results of the 2008/2009 fiscal year, which ended on 31 March 2009 and presented the milestones of the reporting period. On the way to becoming a product-based pharmaceutical company specialising in the treatment of disorders of the central nervous system (CNS), SYGNIS has taken further steps forward. One of the key milestones of the reporting period is the completion of the preparations for the multinational phase II efficacy study for AX200 for the treatment of acute stroke, which was initiated in spring in the first centres.
  Biotechnologie / Life S...  
Das Tor zu diesen Fragen stieß das 1994 entdeckte Leptin auf. Dieses von den Fettzellen ausgeschüttete Hormon reguliert das Energiegleichgewicht und meldet dem ZNS die aktuelle Energiemenge. Es reguliert die Insulinsensitivität und ist nach Wabitschs Einschätzung auch an der Entstehung des Diabetes beteiligt.
These issues all arose from the discovery of leptin in 1994. Leptin is a hormone that is released by fat cells; it regulates the energy equilibrium and informs the CNS about the current energy stocks. It regulates insulin sensitivity and Wabitsch assumes that it is also involved in the development of diabetes.
  Biotechnologie / Life S...  
Die Ulmer Neurologen setzen aus guten Gründen auf diesen Nischenmarkt. Längst spiele die klinische Forschung in Deutschland an Krankheiten des Zentralen Nervensystems (ZNS) international keine Rolle mehr.
The Ulm neurologists have good reasons to rely on this niche market. According to Ludolph, America's stranglehold on the development of profitable 'blockbusters' for common diseases such as Alzheimer’s and Parkinson’s seems impossible to break. As a result, German scientists stand a better chance of competing by developing orphan drugs for the niche markets. Promising clinical research is being carried out on central nervous system (CNS) disorders - a market, as yet, unexploited. Currently, CNS research in the German pharmaceutical industry is, according to Ludolph, restricted to a research group in a company in the south of Germany.
  Biotechnologie / Life S...  
Bei der Aufnahme und Auswertung entsprechender Parameter-Bilddaten besteht noch großer Standardisierungsbedarf. Im Rahmen einer Studie möchte syneed imaging erstmalig eine Standardisierung der Perfusionsbildgebung am Beispiel von ZNS-Erkrankungen erzielen.
One of the great challenges in MRI perfusion imaging is the inconsistency in the acquisition of data and the complexity of existing post-processing methods. There is therefore great need for standardisation. syneed imaging has initiated a study aimed at standardising perfusion imaging using CNS diseases as the example. This study pools the results from other studies in which MRI data of patients with acute stroke, brain tumours and Alzheimer’s were collected.
  Biotechnologie / Life S...  
Die Pflanzen aus der Gattung Sideritis, die in verschiedenen Mittelmeerländern als „Griechischer Bergtee“ serviert werden, haben heilende Wirkungen auf das ZNS. (© IBAM GbR)
Sideritis plants, which are served in numerous Mediterranean countries as “Greek mountain tea” have a remedial effect on the CNS. (© IBAM GbR)
  Biotechnologie / Life S...  
Yorkville Advisors, LLC, welche ein Portfolio mit Fonds von New Jersey aus verwaltet, verantwortlich für die Investition in die SYGNIS Pharma AG, sagte: “Yorkville bietet innovative Finanzierungsinstrumente für börsengelistete Technologieunternehmen. Der vielversprechende neuartige Ansatz der SYGNIS bei der Entwicklung von ZNSTherapien fällt in einen Bereich, für den es einen großen medizinischen Bedarf sowie ein entsprechendes Marktpotenzial gibt, aber bislang nur unzureichende Therapiemöglichkeiten zur Verfügung stehen. Wir sind stolz, in dieses führende innovative deutsche ZNS-Unternehmen zu investieren.”
Yorkville Advisors, LLC, the investment manager to a family of Funds based in New Jersey, led the investment into SYGNIS Pharma AG. Dr. Nowak, stated: “Yorkville provides innovative financing tools for public technology companies. Given the large unmet medical need, the market potential and promising novel approach of SYGNIS to CNS therapeutics, we’re especially delighted to be investing in this leading German CNS player.”
  Biotechnologie / Life S...  
ZNS
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  Biotechnologie / Life S...  
Leukodystrophien wie die MLD sind durch einen Myelinabbau im ZNS charakterisiert. Die MRT-Aufnahmen links zeigen die krankhaften Veränderungen im Marklager (siehe Pfeile) – die Aufnahmen rechts stammen von einer gesunden Kontrollperson.
The LEUKONET network was established in Germany in 2002 to systematically investigate leukodystrophies characterised by the progressive degeneration of myelin. Although funding from the German Federal Ministry of Education and Research (BMBF) will come to an end in 2011, "we will continue our work under the LEUKOTREAT project, a new EU-funded project," said Prof. Dr. med. Ingeborg Krägeloh-Mann.
  Biotechnologie / Life S...  
Ascendis Pharma A/S ist ein junges, auf neue Drug-Delivery-Versionen bereits erprobter Arzneimittel in den Bereichen der Endokrinologie, ZNS-Erkrankungen und Infektionskrankheiten spezialisiertes Pharmaunternehmen.
Ascendis Pharma A/S is an emerging pharmaceutical company that specializes in new drug delivery versions of marketed drugs in the fields of endocrinology, central nervous system and infectious diseases. The parent company is headquartered in Copenhagen, Denmark. Ascendis Pharma maintains a research site (Ascendis Pharma GmbH) with 25 employees in the Heidelberg Technology Park under the management of Dr. Dirk Vetter. The company opened an office in Palo Alto, California (USA) in 2010, from where Jan Møller Mikkelsen, President and CEO of Ascendis Pharma, coordinates the company’s business and strategic development. “We chose to set up an office in the Silicon Valley in order to improve our situation vis-à-vis American venture capital investors,” said Vetter in an interview with BioRN in Heidelberg.