Update on Myozyme and Lumizyme drugs from Sanofi for Lafora Disease treatment

Chelsea’s Hope continues to champion the development of effective Lafora disease (LD) therapeutics in both the research community and with interested companies. One recurring question is whether currently used therapeutics for other glycogen storage diseases could be used in LD. As you know, this would greatly benefit our community, allowing rapid therapeutic adoption.

Because of the similarities between LD and Pompe disease, there has been considerable interest if Myozyme and Lumizyme (from Sanofi Pharmaceuticals) could be effective in LD. The diseases are similar in the accumulation of aberrant glycogen, but different in where the aberrant glycogen is located. In LD, Lafora Bodies (LBs) are in the cytoplasm of cells, whereas in Pompe, the aberrant glycogen is in lysosomes. Because of the strong connections between Chelsea’s Hope and the LD research community, several groups have explored this possibility. Unfortunately, they have found that Myozyme does not effectively target brain LBs in LD mouse models. Given the similarities between Myozyme and Lumizyme, the results also strongly suggest that Lumizyme would be ineffective for LD. These data will be published in a peer-reviewed journal in the coming months. As a service to the community, Chelsea’s Hope wanted to be sure these data were communicated as soon as possible.

While disappointing, these results help to focus our efforts on the multiple therapeutic strategies which have shown to be effective in pre-clinical LD models. Multiple therapeutics were presented at the recent LD meeting, including cytoplasmic targeted enzymes and substrate reduction strategies. We continue to advocate for these to be developed and tested with maximal speed.
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