Potential drug for mitochondrial disease gets FDA fast track status

[Anomie]

This drug could have some therapeutic potential for us. These are exciting times!


http://mitochondrialdiseasenews.com/2016/01/15/potential-drug-primary-mitochondrial-myopathy-granted-fda-fast-track-status/

 

[Remy]

This sounds like treating the root cause to me!

It can potentially modify the disease by preserving mitochondrial energy and restoring healthy physiology, while decreasing oxidative stress.

 

[IrisRV]

This sounds like treating the root cause to me!

Yeah, but what caused the mito dysfunction in the first place? Will we ever find the beginning of this?

This is the song that never ends, It just goes on and on my friends...

 

[Remy]

Yeah, but what caused the mito dysfunction in the first place? Will we ever find the beginning of this?

This is the song that never ends, It just goes on and on my friends...

My guess? Toxicants. In our food, in the air, in our water...and lack of meaningful nutrition in the face of excessive calories. It all adds up and makes us vulnerable.

But fixing that root cause won't happen unless extinction and reboot.

 

[IrisRV]

My guess? Toxicants. In our food, in the air, in our water...and lack of meaningful nutrition in the face of excessive calories. It all adds up and makes us vulnerable.

But fixing that root cause won't happen unless extinction and reboot.

I see your point, but then why only us? The rest of the world is living under those conditions, not just us. Why are we the only ones with this damned disease?

 

[Remy]

I see your point, but then why only us? The rest of the world is living under those conditions, not just us. Why are we the only ones with this damned disease?

I don't think we are!

 

[IrisRV]

I don't think we are!

Okay, why do we have it worse than they do? Genetics?

 

[Cort]

The drug seems to have a broad spectrum appeal

Developing drugs that target mitochondria is challenging because it is difficult to penetrate cell and outer mitochondrial membranes. In multiple nonclinical studies, Bendavia has been shown to reach the inner mitochondrial membrane and target cardiolipin, a lipid critical to maintaining mitochondrial function and cellular energy supply. In these studies, Bendavia has been shown to promote energy production and reduce the production of excess ROS.

They believe this drug may be helpful in many disorders not just mito genetic disorders....

• Heart failure2
• Kidney disease3
• Cardiovascular and metabolic diseases4
• Neurodegeneration5
• Certain skeletal muscle disorders6

The trial is in mitochondrial myopathy - the symptoms to which are similar to ME/CFS....

Mitochondrial myopathies are caused by mutations, or changes, in genes. They are inheritable, although they can occur with no family history, and they often affect members of the same family in different ways. Muscle weakness, exercise intolerance, trouble with balance, and impaired coordination are common features of mitochondrial myopathy (MM) given that muscle cells have particularly high energy needs.6

Interesting!

 

[Remy]

Okay, why do we have it worse than they do? Genetics?

Partly, for sure. I think we all have some threshold, that is probably mostly genetically determined, for how much stress we can take before things start to break down. And I mean all sorts of stress, not just emotional stress, obviously. In fact, probably the greatest stressors right now are environmental in nature.

So if you have a low threshold and a high exposure, then you get sick. If you have a high threshold and low exposure, you are gloriously healthy. Everyone else falls somewhere in the middle.

That's my theory anyway, subject to revision.

 

[Remy]

Developing drugs to target mitochondrial membranes may be difficult, but fish oil has been proven to do this in studies...it reorganizes the lipid bilayer and improves metabolism in high enough doses. I really think fish oil/omega 3s should be a cornerstone of nutritional supplementation in MECFS.

 

[IrisRV]

Developing drugs to target mitochondrial membranes may be difficult, but fish oil has been proven to do this in studies...it reorganizes the lipid bilayer and improves metabolism in high enough doses.

And high enough doses are how much, do you know? I like this idea because fish oil is readily available and relatively cheap. I'd take more in a flash if I had reason to think it would help. Do you know if there are upper limits for safety?

 

[Anomie]

Good news, this drug which is now called elamipretide has passed phase two of the clinical trials!

http://www.prnewswire.com/news-releases/stealth-biotherapeutics-reports-positive-results-for-primary-mitochondrial-myopathy-trial-300279791.html

 

[Anomie]

Update: They have started the second part of phase 2. Some interesting stats on the improvements in this article:

http://www.raredr.com/news/stealth-mitochondrial-diseases

It would be a once daily injection.

 

[Eset Isadore]

Is there any news regarding what happened with this one?

 

[Remy]

Is there any news regarding what happened with this one?

Looks like it is still in trials, but has been granted orphan drug status.
https://mitochondrialdiseasenews.com/tag/bendavia/