C3 Awards Grant to Dr. Melissa Spencer to Screen for Calpain 3 Targets

Coalition to Cure Calpain 3 (C3) is excited to announce that we are funding a new research project with Dr. Melissa Spencer at the University of California Los Angeles. The project is titled “Identification of calpain 3 substrates through use of 2D DIGE and mass spectrometry, and testing muscle-building compounds in an LGMD2A model.”

Identification of calpain 3 substrates through use of 2D DIGE and mass spectrometry
Calpain 3 is a type of protein called a protease, which means that it can cleave other proteins. Its role in muscle health has not been fully established. The first goal of Dr. Spencer’s project is to identify the targets of calpain 3’s cleavage activity using a technique called 2D-DIGE to measure and compare relative amounts of proteins in normal muscle and muscle lacking calpain 3. The identification of targets will contribute to our knowledge of calpain 3’s normal role in muscle health and will provide insight into why loss of calpain 3 causes muscular dystrophy.

Testing muscle-building compounds in an LGMD2A model
The second goal of Dr. Spencer’s project is to test several compounds that are already in the FDA pipeline to determine if they will also be effective in LGMD2A. These compounds promote muscle growth and are currently in development for other forms of muscular dystrophy. If Dr. Spencer’s research results show that these compounds result in improved muscle size, improved function, and lack of toxicity in LGMD2A models, then this will suggest that these compounds may be beneficial for LGMD2A patients.

Dr. Spencer states, “We are hopeful that this funding from C3 will allow us to make fundamental insights about the normal function of calpain 3, and from these insights, we hope to identify new drug targets. We are also excited about the possibility of testing compounds that are closer to the clinic and may be beneficial to patients in the short term.”

WHY THIS MATTERS TO PATIENTS
According to Dr. Jennifer Levy, C3 Scientific Director, “Dr. Spencer’s project is exciting for several reasons. It will:
– contribute to our knowledge about calpain 3’s biological role,
– help us understand why dysfunctional calpain 3 causes LGMD2A,
– test a class of compounds for safety and efficacy in a disease model.”