Tag Archives: Clinical Trial

Mitochondrial Disease Treatment in Phase 3 Trial

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Elamipretide

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!

Cutting Lamotrigine Dose Back

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Well, I’ve tried taking an extra Lamotrigine dose in the morning for about a month now, hoping that would take care of the jerk. I seem to invariably start jerking as soon as I get in Sunday School and continue through church. But the extra dose has not stopped them.

I visited our daughter this last weekend and went to church with her. I only jerked a little in Sunday School, but it was a very small class with only a few women in attendance. But, as soon as we got in the large church sanctuary, my shoulder and foot went crazy. So it must be something to do with being in a crowd.

So, if it’s not going to help, there’s no reason to take the extra pill. I cut the extra one out this morning, and I’ll see how I do for the next few weeks. Getting these doses right is like doing a personal Clinical Trial, as I’ve mentioned in the past. You have to try to compare apples to apples and only change one variable at a time. And I’m careful to do that as much as I can.

I have done better with my motivation to exercise, although right now I’m feeling the effects of the Daylight Savings Time time change, so I didn’t feel like exercising yesterday and today. It always takes me a little while to adjust to the different sleep pattern, more so now that I’m older and on meds that I take on a regular schedule than when I was younger and not on meds.

A Clinical Trial of One??

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In a real Clinical Trial, doctors use a very large number of people, usually, but not always, divided into two groups. One group gets the medicine being tested, and the other group gets fake medicine, called a placebo. At the end of the trial, if the people getting the real medicine have improved considerably more than the group getting the placebo, the medicine is assumed to be the cause of the improvement. That’s a good thing!

They use such large numbers of people to conduct these trials, because there are always going to be unforeseen situations that influence the effectiveness of the medicine for some people. Maybe a few of the subjects have an undiagnosed disease that makes even the best of medicines not work. Or maybe some of them are under a lot of stress that ruins their results.

When you see a new medicine being talked about with glowing praise of its effectiveness, you have to be very cautious about getting all excited about it. I have seen reports like that where the trial only had 16 people in it. That only gives doctors a hint that a certain medicine might be helpful. It’s just not enough people to tell you much.

That’s the problem with trying to find the right medicine for me, or any other PWP. We are, in a very real sense, our own Clinical Trial. The last time I was on Requip, I was terribly bloated, with horrible stomach cramps and gas. So, the Neurologist took me off of it, and he put me on Zelepar. That medicine dissolved under the tongue, so it doesn’t bother the digestive system. My stomach improved tremendously. The question is, was that because of the Zelepar, or was it because, that same week, my Gastroenterologist changed the prescriptions I was taking for my stomach? Also, my symptoms were not alleviated as well when I was on the Zelepar — BUT I had strep throat most of the time I was trying it, without knowing I was that sick.

There’s no way to be sure, is there, with more than one medicine being changed at the same time, and with me being sick, too? I talked with my Neurologist yesterday about this, and the fact that I had gone back to 1 Requip pill a day for several days, because I ran out of the Zelepar samples before my appointment. Even though I had been on the Requip for several days, my stomach was doing just fine.

So, Dr. S. has prescribed another month of Zelepar, to give it a fair trial at helping my PD symptoms. If I am still not getting as good a results with it as I was the Requip, I am to add the old dosage of 3 times a day of Requip to the Zelepar, which I take 2 times a day. I don’t see Dr. S. for 6 more weeks, to try to give me a chance to tell what is going to work best for me.

Parkinson’s Disease is different from many diseases, where there is some MRI or blood test that will tell the doctor what is helping, and what is not. With PD, it really is up to me. I have to be the judge for myself if the Neurologist has prescribed the right medicine and the right dosage. Then he bases my prescriptions on his vast experience with many other PWP he has treated.

But it still boils down to a Clinical Trial of one – ME!