Brainstorm Nurown 'Right to Try' participant shares progress

[ThroughThatValley]

God, 500k / 6 months treatment.... obscene paywall. So if you are an als patient and not within the 200 wealthiest persons worldwide, you are doomed.

 

[lgelb]

Again, if it's approved, Medicare and other insurance will pay, as for any medical intervention. And I assure you, they will not be charged the "list price" -- everything goes under contracted prices.

 

[Par220]

Matt Bellina wrote this yesterday after his second dose of Nurown:

"meanwhile a week and a half after my second treatment and I have regained the ability to extend three fingers on my left hand"

 

[Jesse35]

Are you kidding me? what are they waiting for to approve this treatment. If they approve it in the states, how long do you think it will be approved in canada afterwards?

 

[lgelb]

What they are waiting for, Jesse, is proof it works to slow down the disease (in most people in the trial, who are rapid progressors) and is safe. The new dosing regimen has not yet completed testing.

Once those two things are clear, they can complete filing with the FDA for the US and regulatory authorities in other countries as well.

 

[Par220]

I actually wrote the leaders of the US FDA and posted the response on the ALSTDI. For anyone who is not on that forum I would like to put it here because I wholeheartedly agree with Jesse35. I asked them as part of my letter :

"I know all diseases have heterogeneity and everyone may not have the same response, but this is the case even with chemotherapeutic agents in cancer treatments. I strongly believe you have the authority and moral obligation to allow expedited access of Nurown, which has already completed its Phase 2 safety trial, to this unique ALS population which faces extreme suffering, extremely poor prognosis and no options."

The only person who was kind enough to respond was Dr. Janet Woodcock. She wrote the following:

"Thanks for writing. Very sorry to hear about your illness. I will defer response to Dr Marks as this is regulated by the Center for Biologics. I certainly hope that effective interventions are in the pipeline. We don’t expect or require new interventions to work for everyone." Janet Woodcock

 

[lgelb]

Mod note: Those who wish to follow Matt Bellina's one-off use of NurOwn via ALSTDI and Facebook are of course welcome to do so there. Many people here work too hard to read to require them to ponder what is duplicated from other sites and to scroll past retransmissions when they recognize them.

Moreover, retransmissions without original context can be viewed as commercialism.

If we start allowing one, we must do for all and we do not want to dilute access or Google's propensity to refer to the original content here. Thank you for understanding.

Best,
Laurie

 

[Lkaibel]

A lot of people have been trying to understand why NurOwn is held up and Radicava was approved with extremely few test subjects.

 

[lgelb]

Any comparison between NurOwn and chemo for cancer overlooks that every chemotherapeutic agent reimbursed in any form of cancer has been approved by the FDA for some other type. That means that safety and efficacy were shown in some type/stage of cancer, before it could be legally used and reimbursed for another.

That is how Radicava is being used in some PALS who wouldn't have been allowed into the trials. And that's very typical for new treatments.

But the way that NurOwn is being dosed in the Phase 3 trial has never been done before in PALS. Safety and efficacy haven't been shown. That's where the chemo comparison doesn't hold up.

 

[lgelb]

Radicava had a Phase 3 trial completed, Lenore, before approval. NurOwn's is still going on. They don't even have topline data yet, and won't, until next year.

In re Matt-- according to his YFALS bio, he showed signs of ALS as early as 2006 but was not diagnosed until 2014, at the age of 30. That would make him an atypical case (now in year 13 if the bio is accurate) in both progression and age.

 

[ThroughThatValley]

I believe some of us have been so deeply frustraded seeing all the promising trials go south and leading to broken hopes that we are not willing to take good news without a pinch of salt. I sepak only for myself, but I've come to the point I don't have the smallest confidence in what Labs and pharma have in stock for the near future. I'm better off faithless than beaten down by dead hopes.

 

[Nikki J]

This is very interesting I have to say my late sister regained some finger movement when she was far more advanced than Matt. She wasn’t getting nurown. She had been taking off label retigabine for a number of months. We hoped it was that and increased the dose but no further improvement. I think it just happened by itself.

I am not saying this is the case with Matt. I hope it isn’t

Nurown isn’t going to allow more Compassionate use I don’t think at this point they have interest in going to the fda and asking for approval either If they don’t the fda isn’t going to call them up and ask them to.

I get that is incredibly hard to see something that might help you and you can’t have it! Even more when you see the timeline and think it will be too late even if it goes through the process.

 

[Par220]

It's hard to have a discussion in this thread because frankly Laurie will delete what she doesn't like or agree with. I have ALS so I feel my thoughts pertinent... One can argue maybe even more valid regarding a current upcoming treatment than someone who lost a loved one to ALS. If your loved one was actively in front of you deteriorating and suffering would you be worried it hasn't finished phase 3 trial when something might help?

What's the disadvantage? Potential suffering and death? That's happening already.

What is a phase 3 trial? - Treatments that have been shown to work in phase II studies usually must succeed in one more phase of testing before they’re approved for general use. Phase III clinical trials compare the safety and effectiveness of the new treatment against the current standard treatment.

What I feel Iqelb fails to consider in her comments is there are no effective current standard treatments for ALS. If we were talking about a drug for high blood pressure I agree with her.

 

[KimT]

I didn't think my post offered old information because it was less than 24 hours when Matt posted. If I'm wrong I stand corrected and am sorry for the duplicate post.

I think all PALS want two things in the way of support, maybe more. The first is help with existing technology, therapies, and other things to help symptoms. The second is current information of promising therapies that will slow, stop, or reverse progression. I have no idea if the current stem cell therapy will help me, but when I see positive comments, it gives me hope. Hope, in turn, gives me a much better quality of life.

I like to follow people who believe they are finding some form of help. I believe, for FALS, gene therapy will be the magic bullet. It doesn't matter if I'm FALS, I would be jumping for joy if there were a breakthrough for FALS.

I see this thread going the way of RCH4 so I'll stop posting.

 

[Nikki J]

I agree hope is important. This is a therapy in a legitimate trial with some positive anecdotal reports. We have a way to go before we can start cheering I think so remembering that phase 3 trials do fail even when we hear good reports during the trial is important ( dex as I mentioned being a sad example).

The current mod position

Links to pages with donation links are still not ok.

Describing how to find a post or video such as those on Matt’s page is ok.

A brief update of a factual development such as posted by Par in post 33 is also ok. ( said post was restored last night your duplicate was not Kim because it was a repeat of Pars)


I find this hopeful for SALS. I understand the need for any crumbs of positive news. In the FALS community we feel like that with the gene therapy trials.