TDP-43 - Breakthrough? - Macquarie University

[powerpadman]

Anybody see this news last week? There's some online chatter about this being the biggest ALS breakthrough we've seen in years.

After uncovering, for the first time, the mechanism by which pathological TDP-43 accumulates in MND and FTD, researchers at Macquarie University, Sydney, created a genetic therapy to clear the blockages and prevent them from re-forming.

The researchers found that, in mice, a single dose of CTx1000 targeted only the ‘bad’ TDP-43, leaving the healthy version of it alone. Not only was it safe, it was effective even when symptoms were present at the time of treatment.

It’s taken Macquarie University researchers 15 years to reach this point.

 

[Nikki J]

While the concept is exciting it is still very early. Historically things successful in mice has often not been so in humans. Even it does what we hope it still needs a lot of work to get to humans so we are talking years for toxicity studies and drug development into human trials

Still targeting tdp43 is a very hopeful approach and the one time dosing would be a plus. This is the same technology for delivery that Dr Brown used in his sod1 2 person proof of concept trial that is going to a full phase 1 very soon. I suspect like Dr Brown’s if it works at all it will most successful in early and non aggressive disease