Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).
This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.
In September 2008, MDA gave Repligen approximately $1 million to develop this molecule for FA. (See MDA Awards $1 Million to Repligen.)
MDA has a strong commitment to research, annually spending more than $40 million on research projects to find treatments and ultimately cures for neuromuscular diseases.
About Friedreich's ataxia
Friedreich's ataxia is caused by mutated genes for the frataxin protein, resulting in a frataxin deficiency and malfunction of the nervous system and heart.
The disease is characterized by incoordination (ataxia), severe weakness and cardiac muscle degeneration.
Current treatments are largely supportive. Restoring frataxin production is a goal of treatment development.
About the new compound
The compound Repligen is developing is a histone deacetylase (HDAC) inhibitor, a molecule that keeps "read me" signals called acetyl groups on genes. Cells interpret these signals to mean that a gene is available to be "read" and used for protein production.
Repligen's molecule specifically inhibits HDAC3 (see Experimental Compound for FA Hits Its Molecular Target), which its developers hope will restore these "read me" signals to frataxin genes and thereby increase production of the frataxin protein.
Meaning for people with FA
The new MDA grant will support completion of preclinical (laboratory) safety testing for Repligen's HDAC3 inhibitor, a prerequisite for clinical (human) trials. If the drug passes these lab-based safety tests, human trials could begin within a few years.