Recipient: University Hospital Regensburg
Funding: GO-Bio Phase I (01.08.2014 bis 31.03.2019, 6.596.492 Euro)

Summary

Of all the neurodegenerative diseases, amyotrophic lateral sclerosis (ALS) is among the most severe. The condition particularly affects the motor components of the nervous system, meaning that patients suffer an inexorably progressive paralysis in the muscles of the body. In most cases, the prognosis of the disease is very poor. The molecular causes of ALS are manifold, and no effective causal therapy exists to date. The team led by Ulrich Bogdahn at the Uniklinik Regensburg is working to change this. The GO-Bio project also envisages the founding of the company ‘CampoNeuro Pharma’. The approach that is being pursued by the biomedical scientists in Regensburg is the application of innovative active ingredient to reactivate nerve cell regeneration. The key to this is an ‘antisense agent’ that effectuates a highly precise interruption of the TGFβ signal pathway in the brain. People with neurodegenerative conditions exhibit an increased concentration of the TGFβ molecule. However, excessive quantities of TGFβ will inhibit the ability of the central nervous system to regenerate and form new nerve cells. The work of the project team focuses on a reactivation of this natural compensatory mechanism, thus enabling patients to lead a life that is as normal as possible. The aim is to effectively increase the process of neurogenesis in the central nervous system through the targeted administration of the antisense agent in the cerebral ventricles of the brain. In turn, this would counteract the loss of nerve cells. In the first phase of GO-Bio, Ulrich Bogdahn and his team are planning the development of the drug candidates up to the start of clinical studies on patients. Alongside, they will develop biomarkers that will serve as indicators for patient safety and enable specific conclusions on the effects of the preparation. These are also expected to provide comprehensive insights into the disease itself, as well as its molecular, cellular and functional mechanisms. The second phase of GO-Bio will encompass clinical development and the founding of the biotechnology company ‘CampoNeuro Pharma’.