Did NurOwn work for you?

[Kitchener]

Hello PALS
I have been doing some research into NurOwn as a therapy for ALS. The problem I have is that I'm not a scientist, and the published reports leave me a little confused as to what is actually going on. I was hoping that a few people on this forum may have undergone the trials to date and I would love any comments from them about the experience. Specifically did you experience an actual improvement in your symptoms, or a deceleration in the rate of decline, or did it have no effect at all. For anyone who had an improvement, did this wear off after time? If so, how long? I'm also interested in understanding if it's an invasive procedure, did it hurt a lot and was there much hospital time involved. I'm doing this research as there is an outside chance that I will be able to access the therapy for myself. My problem is that I've had ALS for over six years and now have an ALS FRS close to zero. I am trying to figure out whether following this up is worth the physical, emotional and financial investment, or if I should just sit back and pump pina coladas through my peg. Any input from anyone will be most gratefully received.

 

[AKNate]

Hi Kitchener,

I'm so sorry to hear about your progression but excited that you may have been asked to be part of the trial!

I don't know anyone personally who has tried it but a friend from Melbourne said he knows someone on the trial who claimed it slowed down their progression. I can try find out more information for you.

From what I have read, it's stem cell therapy using bone marrow I think, which will be invasive - maybe it's administered through a lumbar puncture?

I'm in Sydney. Just wondering how you got access to the trial? That's amazing!

 

[GregK]

Do a search.
One of our members got her husband into the PhII trial.

 

[Lkaibel]

It looks like NurOwn is coming back into Trials in the states and here in Minnesota, Rochester Mayo clinic. We will get more details at out clinic appointment this month (our clinic is University of Minnesota but they do much cooperative research).

I read about a participant in an earlier U.S. Trial of NurOwn who had positive results.

 

[KW1234]

My clinic has also applied for the Phase 3 trials. From what I understand the phase 3 trials will be 3 injections, one at 2 mos following the first and then six months. They just got approval on a process to freeze the stem cells so they will only have to harvest once.

There is a second clinical trial starting later this year at another clinic in my area with copper astm that also looks promising though I hope to be accepted into the Brainstorm trial.

 

[lgelb]

We have discussed Brainstorm in other threads, but this is the last published report of the early trials. Patients who have received the treatment thus far have been in early disease, and it is unlikely that the upcoming trial will differ in those respects since consistent inclusion criteria are necessary to the approval process.

There are actually more ALS trials recruiting/pending recruitment than you might think, so here is a current list.

For those whose disease is too advanced to qualify for intervention trials (in all honesty, Kitchener, you are probably one of those), you may want to contribute to science via diagnostic/monitoring types of trials, which are also listed, and also keep in mind the means by which members here have found optimal quality and quantity of life, which include optimal nutrition, hydration, respiratory support, passive range of motion exercise, positioning in bed/wheelchair, social interaction and judicious supplement use.

Best,
Laurie

 

[Lkaibel]

Just had to add that I saw something on an American Phase II participant. He was apparently in a wheelchair at the start of the trial and experienced a great response - he was able at one point to walk unassisted again. However, as soon as the trial ended and he no longer had access to treatment his progression put him back in the same condition he had been in and worse. He was upset he was unable to continue and also with the plan for Phase III trials because he felt it was already a proven treatment and further research was just withholding and delaying availability.

There was also a woman named April who ran a site for a long while, the ALS Express. Unfortunately the site is gone now, but she discussed her phase II trial experience at length and felt also that it was an effective treatment that was being needlessly held up.

I would hesitate to jump to conclusions based on two perspectives, but the drug trial process in the U.S. is challenging for sure.

Given that NurOwn is a very popular study with a lot of publicity around it, I imagine a lot of people will want to be involved and Brian's odds of getting in are not huge but honestly I can see some advantages to not getting into the trial.

 

[lgelb]

We can trash the FDA for a lot of reasons, but not yet approving a dosing regimen that hasn't yet been formally tested in anyone (multiple stem cell injections of NurOwn's recipe) probably shouldn't be one of them.

 

[Lkaibel]

There is a "right to try" argument however for terminal illnesses, and I have some sympathy for that for sure.

 

[lgelb]

Philosophically, yes, but mfrs do not have the financial and human resources to do one-off trials, esp. when they are still legally liable [no waiver will be enough] and these would also siphon eligibles out of the approval trials. Nor will anyone get reimbursed without regulatory approval.

 

[Lkaibel]

Well, it remains my personal opinion that the FDA extends the process of drug approvals to sometimes ridiculous extremes, and it is not just philosophically that I think I right to try can and should exist, but that is just me.