Targeting the cause of nerve cell damage
Annually, approximately 6 000 people worldwide are diagnosed with the rare disease Amyotrophic lateral sclerosis, commonly known as ALS. The disease is a progressive neuromuscular disease leading to a rapid degeneration of the central nervous system’s ability to control muscle activity throughout the body. The mean age of onset for ALS is around 60 but it may affect younger individuals as well. Due to the devastating effect and pace of progression of the disease, the life-span is for many individuals limited to just a few years after diagnosis. For this reason, the need for new and effective treatments remains large and urgent.
Current treatments include drugs that relieve severe symptoms. While there are some therapies approved to treat ALS, the disease generally progresses rapidly irrespective of treatment and there is accordingly a need to develop disease modifying drugs for ALS.
In ALS, the protein TDP-43 is misfolded, causing it to aggregate and accumulate in motor neurons in the central nervous system. This creates a toxic environment, causing the nerve cell to degenerate and die. ALS arises in motor neurons in the brain, the brain stem, and the spinal cord, which are areas that control the body’s movements. Misfolded and aggregated TDP-43 has also been shown in many patients with frontotemporal dementia and Alzheimer’s disease.
We develop drug candidates targeting soluble aggregates of TDP-43 – oligomers and protofibrils. These forms are thought to be the most harmful to nerve cells. In the project ND3014, we are aiming to develop selective antibody treatments targeting TDP-43. The antibodies make it easier for the immune system to detect and eliminate the toxic aggregates of TDP-43, leading, hopefully to a disease modifying effect with slowing of disease progression.
Project in ALS
BioArctic’s portfolio currently include the ALS project ND3014 which consists of selective antibodies against the protein TPD-43, which plays a key role in the development of the neurodegenerative disorder Amyotrophic lateral sclerosis (ALS).
ND3014 – other CNS disorders
The ND3014 drug project consists of selective antibodies against the protein TPD-43, which plays a key role in the development of the neurodegenerative disorder Amyotrophic lateral sclerosis, or ALS. The accumulation of TDP-43 aggregates is a common clinical finding in ALS as these observed in the brain tissue in the vast majority of the patients. Misfolded TDP-43 is also involved in the development of dementia in the frontotemporal lobe, and has been demonstrated in more than half of patients with Alzheimer’s disease.