ND3014 – other CNS disorders

Research to develop new antibodies for treating neurodegenerative disorders is under way at BioArctic. the ND3014 project aims to develop selective antibody treatments targeting TDP-43, a protein that is believed to play a crucial role in the development of the rare neurodegenerative disease ALS (amyotrophic lateral sclerosis).

ALS is a devastating neurodegenerative disease that affects the body’s ability to control muscle activity. TDP-43 (TAR DNA-binding protein 43) is a key protein that impacts the disease development. When this protein is misfolded, it creates toxic aggregates, causing a stepwise degeneration of motor neurons in the brain, the brain stem, and the spinal cord. Aggregates of TDP-43 in the central nervous system is a common clinical finding in ALS, found in most cases of patients in studies. Misfolded TDP-43 has also been shown in many patients with frontotemporal dementia and Alzheimer’s disease.

The aim of BioArctic’s development of selective antibody treatments toward TDP-43 is to decrease the levels of the toxic TDP-43 aggregates in nerve cells, and thus achieve a disease-modifying effect.

6,000 people are diagnosed with ALS annually world-wide. The disease often makes its debut around the age of 60, but younger people are also affected. The disease has a very aggressive progression, causing increased muscle fatigue and a high risk of morbidity within a few years after diagnosis. Current treatments include drugs that slow down the disease progression, or relive symptoms, aimed at improving the patient’s quality of life and the need for new treatments is large.