M1 Antibody

SPAST, the gene that is mutated in SPG4-HSP, produces two spastin proteins, called M1 and M87.  M87 is easy to detect and study with antibody tools that are available, while M1 is very hard to detect or study with available antibodies.  This has been a significant obstacle for researchers because M1 is the protein that becomes toxic when mutated, resulting in corticospinal degeneration for SPG4 patients.

 

The Drexel University team, led by Dr. Peter Baas, is in the process of developing a new monoclonal antibody that specifically detects M1.  Initial results are encouraging.  When the final purified antibody is ready, it will enable researchers to detect and study M1 in ways never before possible.  This may lead to M1 in body fluids serving as a biomarker for disease progression, a therapeutic to block the toxicity of M1 in patients, and benchmark in evaluating the success of ongoing gene therapy efforts.