Batten disease, also known as neuronal ceroid lipofuscinoses (NCLs) is a fatal, inherited disorder of the nervous system.
It involves a build up of lipopigments that accumulate and kill brain and central nervous system neurons. It is considered a lysosomal storage disorders and that can take it’s victim’s lives by their teens and young adulthood. There is no cure and treatments aim to alleviate symptoms only. To learn more about Batten disease, click here.
Right now, researchers assess clinical symptoms of patients with batten disease in order to observe the effectiveness of potential treatments. This gives long-term insight but does very little to inform the short-term. If scientists know more about how a treatment effects the short-term, they can better develop treatments that target the disease and not just the symptoms. Identifying specific biomarkers can help this cause, as biomarkers are telling of how far a disease has progressed.
A recent study in the Journal of Proteome Research may have accomplished just that, where they think they have identified potential biomarkers in the three main Batten disease categories!
And while researchers were limited in studying deceased patients, they are hopeful that they can use these biomarkers to assess disease progression, which in turn will lead to better treatments. To read more about the study, click here.