KL1333 Mitochondria medicin

[elvira]

The medicin KL1333 has been approved a phase 2 study. It’s for mitochondrial diseases that one is born with - but do you think it could benefit some people with ME/CFS too?


https://www.mediconvillage.se/sv/ab...x-WUq-KXWergHgRFgmPp4Pz4tFEKcb_r4v-3ohlvBmF5Q

 

[elvira]

About KL1333

KL1333 is being developed towards a treatment for a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. The KL1333 compound is a potent modulator of the cellular levels of NAD+ and NADH, central co-enzymes in the cell’s energy metabolism. In a cohort of PMD patients in a Phase 1a/b study, the patients who received KL1333 showed both improvements in symptoms of fatigue as well as functional improvements. KL1333 has received orphan drug designation in both the USA and Europe.

 

[Martin aka paused||M.E.]

About KL1333

KL1333 is being developed towards a treatment for a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. The KL1333 compound is a potent modulator of the cellular levels of NAD+ and NADH, central co-enzymes in the cell’s energy metabolism. In a cohort of PMD patients in a Phase 1a/b study, the patients who received KL1333 showed both improvements in symptoms of fatigue as well as functional improvements. KL1333 has received orphan drug designation in both the USA and Europe.

The question is how it modulates NAD+/H. I think it increases it (I would like to know how?). If it does, there would be more electrons for the ATP synthesis. But the thing is: We don't actually know if that's the problem. Studies (some) are showing normal ATP levels.
Questionable

 

[godlovesatrier]

Depends if mitochondrial disease is truly at the heart of the illness. I guess the bigger question is which subset of patients may this improve? Although it would be great if it made 60% of patients better purely because they share a similar symptomatic overlap.

 

[elvira]

I was studying biotechnology when I got ill and wanted to be researcher within medicine. And now i’m sitting here waiting for research to be done as my only hope. Oh the irony

 

[godlovesatrier]

Crazy isn't it. I always think I'd have enjoyed being a research doctor or the like. Then again I really liked the idea of being a mountaineer too I guess it really suits my personality type.

 

[Wishful]

I agree that there's a chance that it will help some PWME. Whether that's 60% or .0000006% is the question. My guess would be on the lower end.