Ulrich Bogdahn: In pursuit of a treatment for ALS

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The neurologist Ulrich Bogdahn is working to boost the activity of endogenous stem cells in ALS patients and thereby to regenerate damaged nervous systems. Source: Prof. Dr. med Ulrich Bogdahn

18.09.2014  - 

Ulrich Bogdahn is a physician and scientist to his very core. His professional vision: to make a contribution to an effective treatment for neurodegenerative diseases. At this time, the medical director of the Department of Neurology at the University of Regensburg is conducting research into an effective therapy for a previously incurable nerve disease – amyotrophic lateral sclerosis (ALS) – which crashed into the media spotlight this summer as a result of the ‘Ice Bucket Challenge’. Bogdahn’s approach is to regrow the nerve cells in the affected patients. The 63-year-old doctor is now working to carry this research idea into clinical practice. As one of seven winners of this year’s ‘GO-Bio’ competition, his ambitious project will be funded to the tune of tens of millions of euros over the coming years by the Federal Ministry of Education and Research (BMBF).

While celebrities around the world are taking part in the ‘Ice Bucket Challenge’ to drum up donations for the fight against the disease ALS, Ulrich Bogdahn is deliberately shunning the limelight: “We just want to fulfil our responsibilities. It is still too early to rejoice,” says the clinician. Despite initial successes, the neurologist emphasises a more modest approach. The path towards a treatment for the neurological illness ALS remains a long one. However, endurance seems to be in the blood of the 63-year-old scientist from Regensburg. As a former competitive athlete in rowing and passionate racing cyclist, he knows how to allocate his energy in order to successfully arrive at the destination. Bogdahn studied medicine in Marburg and Freiburg, specialising in neurology. His doctorate was on the subject of immunology. During his assistant professorship in Würzburg, Bogdahn was occupied with experimental brain tumour therapies, a field in which he has been specialised for some time in his work as a clinician. Since 1997, he is the founder and medical director of the Department of Neurology at the University of Regensburg. Although he sees himself first and foremost as a doctor, he has strong connections with clinical research over many decades.

Throwing light on damage by ALS

Since 2005, Bogdahn has been occupied above all with the subjects of neurodegeneration and oncology, with a particular emphasis on ALS. In amyotrophic lateral sclerosis, the motor nervous system is permanently damaged. Slowly but surely, parts of the body become paralysed. ALS is generally fatal. To date, it is not possible to causally treat or cure the disease. More recently, however, Bogdahn and his team at the University Clinic in Regensburg, which is also participating in the German Network for Motor Neuron Diseases, have taken the field a significant step forward. A first breakthrough was the discovery of the crucial role played by the molecule TGF-beta in the generation of new nerve cells from stem cells of the central nervous system. “This molecule is formed in abundance during inflammation and degenerative processes. When present in large quantities, the formation of new nerve cells is slowed down to a greater extent than is already caused by ageing,” explains the researcher. In the next step, Bogdahn and his team developed a concept to render visible the damage to the brain caused by ALS. Here, the researchers employed imaging techniques, neurophysiological methods and stem cell markers. Combined, this enables an ongoing visual evaluation of the progress of the treatment. “We have indeed used these biomarkers to track over 20 ALS patients as a part of their treatment.”

Boosting neurogenesis with the body’s own stem cells

For Bogdahn’s team, the perseverance has paid off. In April this year, their results convinced the jury of GO-Bio, a competition for start-up teams hoping to gain BMBF seed funding. The project from the ALS researchers will now be supported with millions of euros over the coming years. “We want to help the body’s own stem cells to repair the neurodegenerative damage, or at least to compensate better,” says Bogdahn. The corresponding active substance has already been developed. This concerns a so-called antisense agent, which is inserted in the brain ventricles via a mini-gene probe and where it reduces the activity of the TGF-beta molecule.  Through continuous administration of this drug, the researchers hope to give the ALS-damaged central nervous system the ability to regenerate and form new nerve cells.

GO-Bio funding to form a company

In the first phase of the GO-Bio project, Ulrich Bogdahn’s research group is planning to develop their drug for clinical testing on patients, which are hoped to begin as soon as possible. “I am assuming that we will have the drug available for clinical use in two and a half years at the latest, maybe even sooner.” Moreover, the biomarker will be refined to enable conclusions to be drawn on the effect of the preparation on individual patients. In the second phase, the drug will be prepared for clinical development and for the founding of a biotech company, Campo Euro Pharma, which will market the ALS treatment. Bogdahn already has “very promising” links with relevant partners in industry. The next stage appears to be within reach: As Bogdahn explains, this is “to bring the disease to a standstill at the point of diagnosis, at least in the majority of patients.” Throughout, the doctor is keen to play down expectations. ALS is a highly complex disease with an extremely complex genetic background. “From the beginning, we should maintain a cautious optimism until we see the first clean clinical data,” says Bogdahn.

Author: Beatrix Boldt