Promising target

Telbivudine, an existing hepatitis drug, illuminates potential target in zebrafish study.

Hi, Laurie.
The linked article in EurekAlert quotes co-author Ted Allison as saying, "SOD1 is a protein that is known to misfold and misbehave in most cases of patients with ALS."
In your December 6th post about Biogen carrying on research started by Ionis Pharmaceuticals, it says, "...IONIS-SOD1 rx (BIIB067) for the treatment of SOD1 mutations, representing ~2% of total ALS cases."

I realize (and appreciate) that you are just passing along information of interest to PALS and CALS, but given your knowledge about ALS symptoms and treatment, I wondered if you had any insight into why one article associates SOD1 mutations with "most cases" of ALS, and the other one with "~2% of total ALS cases"?

Thanks!
Bill

People who have SOD1 FALS have a mutation in the gene. The issue seen in SALS is related to the protein encoded by the gene but the problem has a different underlying cause than the FALS type that is believed to result from the mutated gene. I don’t think the SALS causes are well understood yet

The Biogen antisense therapy targets the mutation which is why it is FALS specific

Would this drug, then, only be beneficial for FALS and not SALS? I've been confused and looking for proper clarification since the article came out.

Thanks.

The article cited in the first post is discussing a drug that acts on the protein which is where they see an issue in SALS so it is not a FALS specific possibility unlike biogen antisense. It is very early though and we have seen many promising candidates in animals and lab dishes not pan out. The more deep our understanding though the more likely we are to start getting answers

Thanks, Nikki, for the helpful clarifications! What caught my attention was the statement in the EurekAlert article that:

"The discovery of telbivudine as a potential treatment is particularly exciting because the drug is already in use for treating patients with hepatitis. 'It is already proven safe to use in patients, and it has very good potential for repurposing to use in a new clinical setting against ALS,' said Allison."

No one - self included - wants to get their hopes up about something that, as you put it, won't pan out, but at least telbivudine is an existing drug already approved for use in humans, although for a different condition.

Best regards,
Bill