Antibodies for Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is an adult-onset neurodegenerative disease affecting primarily motor neurons of the motor cortex, brainstem and spinal cord. Pathological hallmarks of ALS are protein inclusion bodies found in the diseased motor neurons. Disease starts as a mild muscle weakness which progresses to complete paralysis and eventual death, usually within 2-5 years from diagnosis. Diagnosis is based solely on clinical manifestations and is usually a process of elimination, which can take many months, even years. It is estimated that by the time a definitive diagnosis of ALS is given, up to 60% of motor neurons have already degenerated. Currently there are no effective treatments for ALS thus, an early diagnosis is critical to increase the efficacy of therapeutic interventions.
In a novel breakthrough in Amyotrophic Lateral Sclerosis (ALS) research, Dr. Robertson at the University of Toronto has identified components of inclusion bodies in diseased motor neurons that are alternatively spliced variants of two neuronal intermediate filament proteins. Using synthetic peptides corresponding to these unique epitopes, Dr. Robertson has generated splice variant specific polyclonal antibodies that label inclusion bodies in disease tissue. By using pathological and control samples of human spinal cord tissue, she has demonstrated that splice variants in these proteins show specificity for ALS. She has also initiated investigations of the peptides and their corresponding antibodies in a biomarker assay for ALS using human cerebral spinal fluid (CSF), blood and urine.
Dr. Robertson is now in the process of developing splice specific monoclonal antibodies and validating the sensitivity and specificity of the technology by increasing the sample size of human blood serum, tissue and CSF.
This discovery provides an extremely useful tool for ALS research and has the potential to be developed into a molecular biomarker profile used for clinical diagnosis, early stage detection, and the development of more specific treatments for ALS.
Provisional patent applications have been filed.
We are interested in licensing this technology to an experienced industrial partner, who will help drive and support further proof-of-principle investigations, clinical trials and regulatory processes. We are also soliciting for sponsored research collaboration agreements to further explore additional applications and improvements of this technology.
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