No telling how many years away from availability this is, but it seems a mitochondrial disease treatment called Elamipretide may eventually be a treatment for Mitochondrial Myopathy. As old as I am now I can’t imagine this would be much help to me. Natural aging on top of inefficient mitochondria have taken their toll on my body. I am old, and I feel old. But it’s exciting to think that the day may come in my lifetime there will be help those younger folks who struggle with this rare disease.
Read all about Elamipretide research and progress on Mitochondrial Disease News.
Phase 3 Trial is a Big Deal
Scientists test possible drug treatments on animals before getting to human trials. Human drug trials generally go through three stages. Phase 1 trials mainly want to be sure the new treatment being tested won’t harm healthy people. They track side effects compared to dosages. People volunteer and are often paid to be test subjects for Phase 1 trials. Phase 2 involves testing actual patients to see if it works or not. And of course they are on the lookout for bad side effects, even if the drug does what it in intended to do. That means any time a treatment protocol makes it through Phase 3, there is reason for optimism. Elamipretide is currently nearing the end of its Phase 3 Clinical trial.
And it seems to be actually helping the mitochondria work better. Its like “glue” that makes better connections during the chemical reaction chain that creates energy in the mitochondria. The heart muscle is illustrated in this drawing, but my problem mitochondria are in my skeletal muscles.
Clinical Trial Process is Expensive
If you read more about the whole clinical trial process, it’s not hard to see why drug research is so very expensive. Yes, we all want medicines to be cheap and easily affordable for everyone. But pharmaceutical companies will never get rich developing treatments for what are called “Rare Diseases”. It is not cost effective to do the R&D for a drug that only a few thousand people will ever need. So they have to charge more for the every day variety of meds, like for diabetes. That’s just being financially realistic. The pharmaceutical company normally applies for Orphan Drug classification for any rare diseaae research they undertake. The government provides tax incentives and a longer time as a copyrighted drug than normal medicines. And that’s meant to stimulate more research for rare disease treatments.
Ethical Drug Pricing
The problem comes when Big Pharma gets just plain greedy. They charge for the more common drugs at extravagantly elevated prices. These prices are far out of proportion to what they need to make their share holders profits. We’ve seen this happen with Insulin.
So those of us who live with one of the rare mitochondrial diseases hope for the day when a clinical trial results in a mitochondrial disease treatment protocol. We also know that such a treatment will not be cheap. And we have to face the fact that other medicines will cost more to offset the research and development costs over years of efforts to achieve that treatment.
I look forward to reading your comments!