Improvements?

[rcharlton]

I am confused when participants in drug trials claim that their symptoms improve or are less severe.

From my understanding of ALS, actual improvements will only occur when the dead motor neurons are regenerated or replaced with new ones. An incredibly tricky process.

Our bodies cannot regenerate motor neurons on their own. And I am not aware of any of the trial drugs being designed to, or claiming that they can, regenerate motor neurons. I mean, if any of the drugs were designed to regenerate motor neurons they would be tested on and marketed to a larger affected population (maybe spinal cord injuries?).

I can understand trial drugs maybe slowing progression (wouldn't that be great) but I can't understand claims of return of lost function.

I have always understood any cure for ALS as being a two stage affair. 1. Stop progression and 2. Regenerate motor neurons.

Drugs are targeting progression but I think we will need to rely on stem cell therapy to regenerate motor neurons and effect actual improvements.

So how can participants in drug trials be experiencing any improvements?

I would love it if was true - I just don't understand it.

 

[LizT]

This may be a case of wishful thinking.... you know- the power of the mind type of thing.
I shouldnt even be answering this- im not even a PALS.
But its a thought

 

[Danijela]

I was going to write the same thing as LizT. And, isn't it mostly the case of subjective reporting based on perception, rather than an outcome of medical exam/evaluation? (which is presumably being done too and will be included into published results).

 

[TedH5]

I will address the stem cell portion of your question and the part about conducting it on a larger population. 1) A stem cell study was being conducted by a company called Geron. However Geron recently halted the trial because of the cost and and the numerous hurdles the FDA placed before them and the long timeline to clear the FDA's hurdles.

As far as the stem cell trial I am involved in through Emory and Neuralstem, I happaned to see a video of a conference where my Dr's were part of a panel on stem cell's and treating ALS, and they were asked the very question you did. They were very frank and honest and when I saw it, the answer was somewhat chilling. They said the longterm hope is if this proves safe and effective the hope is to use it on all types of Neurological conditions and spinal cord injuries. However they started with ALS patients because as harsh as it sounds it is true, we already have a terminal disease. You see there is an extreme risk with the actual procedure of penetrating the spinal cord and injecting the stem cell to get the cells right next to the anterior horn nerve endings. That is why within the ALS population they started with patients who couldn't walk any longer so that if there was a problem during the operation and they ended up paralyzing the person below the waist, the impact was less because they already could not walk already and we all know about the life span of an ALS patient.

So you see we make good guinea pigs. Obviously I am OK with that because it is just the reality of the situation.

 

[Bad Balance]

Ruthless Reality

 

[rose]

I too don't understand. But,from the vantage point of the simplistic way that MND was first explained to me (by my doctor, not here): MND has chronic as well as active denervation and reinnervation. If the disease was slowed/stopped at the point of denervation, couldn't the reinnervation continue to take place? Because then, the motor neurons could regain some territory, if they weren't continually getting killed off again.

If it was halted before all nerves were dead, and there were some still neruons attempting to 'sprout'... then the person eventually would show improvement.

Ted, again, thank you for your willingness to participate. You're making history, probably not the kind you ever envisioned though.

 

[TedH5]

Rose that is correct there is active denervation and attempts at reinervation. Obviously eventually denervation wins. I will tell you that after I started showing improvement they decided to do another EMG. They could not believe what they saw, while I still had denervation almost every muscle they checked was showing reinervation as well. I heard the Dr's talking in the hallway and they were talking about theories etc but it sounded as though they had never quite seen something like this were there was such wide spread reinervation.

The thing is they do not know how many stem cells to use. They originally wanted to use 40 million but the FDA told them that 1 million was the maximum they could use. Which sounds like a lot but really isn't, so I believe that the Dr's think that the 1 million will not be enough to keep up and protect all my motor neurons that are being attacked.

Thanks for the kind words Rose, I have been blessed, but I am not content. I am pushing the Dr's to do more. Since right now I am the medical outlier yet still have ALS I want them to use me as a Guinea Pig so they can understand and learn what is different about me that allowed it to help me so they can duplicate it for everyone else. They are also considering giving me a second dosage in my neck to see if it helps again. I have told them I will do whatever they need anywhere anytime if it will help them figure out how to beat this stupid disease for all of us!

 

[Al]

Thanks Ted from all of us.

AL.

 

[TedH5]

Thanks Al, but honestly a lot of people thank me and while I APPRECIATE it very much I do not feel I deserve thanks. I have done nothing special and from the very beginning have felt a moral obligation to do whatever I can to help all present and future PALS. That was the main reason I did this trial, the feeling that I could be part of something bigger and hopefully eventually make a difference in peoples lives.

I have a lot of people say what you're doing is great or you're famous...I tell them all I have done nothing special, I was diagnosed with ALS (obviously not something to brag about ), volunteered for a trial mostly for altruistic reasons, but partially as a roll of the dice to see what would happen and finally I have been blessed through faith and science. As you can see I am just along for the ride and hopefully the Dr's and scientists can figure this thing out! I pray for that everyday!

 

[rcharlton]

Ted you are too modest. I just hope you will benefit from your own sacrifice.

I've asked Santa to bring you some fresh cedar chips, sunflower seeds and a new running wheel.

 

[susanf]

I agree with Richard. You sacrificed for the good of all PALS.