Old 02-05-2019, 04:57 PM #1
jaclyn's Avatar
jaclyn jaclyn is offline
New Member (Say Hi)
 
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
jaclyn jaclyn is offline
New Member (Say Hi)
jaclyn's Avatar
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
Default Brainstorm Nurown 'Right to Try' participant shares progress

As some of you may know, Brainstorm granted one PALS free access to NurOwn as a 'thank you' for the work he did on Right to Try. He is not in a trial but is being given the course separately.

His update is on Facebook -- matt.bellinski.7

Mod note -- not linking directly due to fundraising solicitation on page.

Last edited by lgelb : 02-06-2019 at 02:02 AM Reason: duplicate content/removed link to page w/ fundraising
jaclyn is offline   Reply With QuoteReply With Quote
Old 02-05-2019, 11:32 PM #2
Jesse35's Avatar
Jesse35 Jesse35 is offline
New Member (Say Hi)
 
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Jesse35 Jesse35 is offline
New Member (Say Hi)
Jesse35's Avatar
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

If the therapy is accepted, how much would it cost??
Jesse35 is offline   Reply With QuoteReply With Quote
Thanks from
jaclyn (02-08-2019)
Old 02-06-2019, 06:22 AM #3
Nikki J's Avatar
Nikki J Nikki J is offline
Extremely Helpful Member
Forum Moderator
 
Join Date: 2012
City: Boston
State: MA
Country: US
Diagnosed: 04/2014
Interest: I have been diagnosed with ALS.
Posts: 7,058
Nikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond repute
Nikki J Nikki J is offline
Extremely Helpful Member
Forum Moderator

Nikki J's Avatar
Join Date: 2012
City: Boston
State: MA
Country: US
Diagnosed: 04/2014
Interest: I have been diagnosed with ALS.
Posts: 7,058
Nikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond reputeNikki J has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

I think I saw 300 000 US per treatment quoted in a press release
Nikki J is offline   Reply With QuoteReply With Quote
Old 02-06-2019, 10:47 AM #4
jaclyn's Avatar
jaclyn jaclyn is offline
New Member (Say Hi)
 
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
jaclyn jaclyn is offline
New Member (Say Hi)
jaclyn's Avatar
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

Someone in the facebook thread says it's $500k for 6 months.

It's worth noting that while Matt has had big improvements in one month, someone else says that their own progression slowed to nothing during the Brainstorm trial but no actual improvements. Then progression returned 2 months after the treatment stopped. That person believes they did get the real treatment.

The same person says it looks like mid 2020 for availability for NurOwn, if it comes to market. [mod note: incorrect, see later post]

Another person is on their 10th visit with 4 to go in the Brainstorm trial and still has seen no improvement (of course, they could have the placebo).

Someone else says that their husband was in the second clinical trial and saw improvement.

Last edited by lgelb : 02-07-2019 at 09:11 PM Reason: removed quoted text
jaclyn is offline   Reply With QuoteReply With Quote
Thanks from
Idaho2790 (02-24-2019)
Old 02-06-2019, 04:23 PM #5
lgelb's Avatar
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator
 
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator

lgelb's Avatar
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

If the treatment is approved and the PALS is within the indicated group, it will be paid for by private/public insurance in the countries it's approved in, though as with any surgical procedure, preauthorization would be required (which in some countries will be subject to budgets) and hospital stays subject to normal coinsurance/deductibles.
lgelb is offline   Reply With QuoteReply With Quote
Old 02-06-2019, 06:14 PM #6
Par220 Par220 is offline
New Member
 
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Par220 Par220 is offline
New Member
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

https://www.bloomberg.com/news/artic...s-it-s-working
Par220 is offline   Reply With QuoteReply With Quote
Old 02-06-2019, 06:37 PM #7
Jesse35's Avatar
Jesse35 Jesse35 is offline
New Member (Say Hi)
 
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Jesse35 Jesse35 is offline
New Member (Say Hi)
Jesse35's Avatar
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

When you say within the indicated group, do you mean the group of PALS that have to meet the criteria to be eligible for the phase 3 trial? My mother is 68 years old which is not within the age group they ask for, and by the time the therapy gets approved she will probably have a feeding tube, which is also an exclusion for the trials
Jesse35 is offline   Reply With QuoteReply With Quote
Old 02-06-2019, 06:49 PM #8
KimT's Avatar
KimT KimT is offline
Extremely Helpful Member
 
Join Date: 2014
City: The Beach
State: South
Country: US
Diagnosed: 08/2015
Interest: I have been diagnosed with ALS.
Posts: 3,152
KimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond repute
KimT KimT is offline
Extremely Helpful Member

KimT's Avatar
Join Date: 2014
City: The Beach
State: South
Country: US
Diagnosed: 08/2015
Interest: I have been diagnosed with ALS.
Posts: 3,152
KimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond reputeKimT has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

There is an ongoing discussion over at ALSTDI and Matt is posting, FYI.
KimT is offline   Reply With QuoteReply With Quote
Old 02-06-2019, 08:27 PM #9
lgelb's Avatar
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator
 
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator

lgelb's Avatar
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

The trials, esp. in something as devastating as ALS, tend to be more restrictive than what payors ultimately allow. Radicava is an example. Still, the current stem cell experience in cancer is that Medicare payments that do not cover full costs, especially those of harvesting the cells used, are more of a barrier than disapproval based on patient status.

In trials, excluding certain interventions like a feeding tube and BiPAP is needed to rule out the possibility that the intervention is slowing progression more than the stem cells, since we don't have the numerical evidence of benefit that we can factor out, as we can with riluzole. And there are practical considerations such as the ability to lie flat during procedures.

Since there are bad actors out there relative to ALS and stem cells, just a reminder that Matt is describing one experience in an "unblinded N of 1 trial." The Phase 3 trial will reveal more.

So don't book a ticket to get transplanted anywhere (and possibly harmed/killed -- both have happened) on the basis of a Facebook post. Spend that time, energy and money on some of the approaches here, including experiences that you love, that there are reasons to believe help, and that you know exactly what you are paying for, every step of the way.
lgelb is offline   Reply With QuoteReply With Quote
Thanks from
vltsra (02-07-2019)
Old 02-07-2019, 07:54 PM #10
Jesse35's Avatar
Jesse35 Jesse35 is offline
New Member (Say Hi)
 
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Jesse35 Jesse35 is offline
New Member (Say Hi)
Jesse35's Avatar
Join Date: 2018
City: Trois-Rivière
State: Quebec
Country: Ca
Diagnosed: 08/2018
Interest: I am a family member of someone with ALS/MND.
Posts: 9
Jesse35 is on a distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

Thank you for the information. Lets hope they can make the therapy available sooner than mid 2020
Jesse35 is offline   Reply With QuoteReply With Quote
Old 02-07-2019, 09:07 PM #11
lgelb's Avatar
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator
 
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
♥ lgelb lgelb is offline
Extremely Helpful Member

Forum Moderator

lgelb's Avatar
Join Date: 2009
City: Seattle
State: WA
Country: US
Diagnosed: 09/2009
Interest: I lost a loved one to ALS/MND.
Posts: 7,082
lgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond reputelgelb has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

They really can't jump the timeline past the consideration they already received (Fast Track, Orphan Drug Designation and Priority Review), and the hearsay you quoted is incorrect. Brainstorm's estimate is for topline (preliminary) data availability in mid 2020, not treatment availability. Not too long ago, they were planning top line data a year earlier. Things slip.

Then they will need final data before they can finish filing for registration, which the Fast Track allows them to do in installments. Next, there is an expedited review process that can still take up to 6 months, instead of the usual 10. Of course, if the trial is positive, the FDA will try to act as quickly as possible.

I have attached one of their recent decks.

Again, we don't know the results of the trial as yet. This dosing regimen is new, in an effort to improve the results. That means there could be more side effects.

Also remember they are requiring "rapid progressors" for this trial, based on the Phase II results, and no one >60.

So only time will tell for whom it works and how well, even if approved. It could all be wonderful, but we have no way to know as yet.

Because a lot can get garbled, we don't usually host secondhand reports of threads elsewhere. Let's leave it to our readers, Jaclyn, to check out the threads they're interested in.
Attached Files
File Type: pdf BCLI - Non-Confidential Presentation_Nov 6.pdf (1.79 MB, 54 views)

Last edited by lgelb : 02-07-2019 at 09:13 PM
lgelb is offline   Reply With QuoteReply With Quote
The Following 2 Users Say Thank You to lgelb For This Useful Post:
blitzc (02-24-2019), sassy (02-07-2019)
Old 02-08-2019, 10:42 AM #12
jaclyn's Avatar
jaclyn jaclyn is offline
New Member (Say Hi)
 
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
jaclyn jaclyn is offline
New Member (Say Hi)
jaclyn's Avatar
Join Date: 2018
City: NYC
State: NY
Country: US
Interest: I am a friend of someone with ALS/MND.
Posts: 7
jaclyn is on a distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

So sorry, I didn't mean to confuse people! There are a lot of comments in that thread and I wanted to make sure people who were too busy to go through all of it could see those that appeared to be more relevant. I won't do that again.

Thanks for clarifying with a more accurate timeline.

Last edited by jaclyn : 02-08-2019 at 11:19 AM
jaclyn is offline   Reply With QuoteReply With Quote
Old 02-08-2019, 02:12 PM #13
affected's Avatar
affected affected is offline
Extremely Helpful Member
 
Join Date: 2013
City: lala land
State: home
Country: OZ
Diagnosed: 05/2013
Interest: I lost a loved one to ALS/MND.
Posts: 11,459
affected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond repute
affected affected is offline
Extremely Helpful Member
affected's Avatar
Join Date: 2013
City: lala land
State: home
Country: OZ
Diagnosed: 05/2013
Interest: I lost a loved one to ALS/MND.
Posts: 11,459
affected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond reputeaffected has a reputation beyond repute
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

Jaclyn I appreciated that you posted the information because now I wouldn't have a clue where to find what you started this thread with - instead I only have all the information against it without knowing what it was about. JMHO
affected is offline   Reply With QuoteReply With Quote
Thanks from
jaclyn (02-08-2019)
Old 02-10-2019, 06:11 PM #14
Par220 Par220 is offline
New Member
 
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Par220 Par220 is offline
New Member
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

Here is something I have in mind for Matt and the other ALS advocates meeting with the FDA this week. Now we have the Phase 2 results, the multiple phase 3 accounts, and now with an unblinded participant with improvement in strength and an undeniable 23% improvement of FVC after a single treatment. I would use the FDA commissioner Scott Gottlieb's own words with him at the meeting considering he is a lymphoma survivor. Here is a segment of his own speech on June 2, 2018 at an oncology conference:

"There are critics who say we should hold drugs back from the market, and demand more pre-market studies proving overall survival endpoints, before we consider approving new drug.

I disagree. And I suspect some of the patients who face long odds, for whom available therapy gives them just a slim chance of long-term survival, might also disagree.

I had Hodgkin lymphoma. I had a very curable tumor. At the time of my diagnosis, I was told my odds of a cure were 90 percent or better. For me, available therapy was promising.

But to help me make better decisions on how to use the available drugs, I was searching for pristine studies that could get that 90 percent up a few more points.

So, I understand why demanding large, pristine studies ultimately serves the interest of patients like me.

But my situation was very different than being diagnosed with a cancer and being told your chance of surviving five years is 50 percent, or 30 percent, or just 10. Available therapy isn’t very promising if that’s your circumstance. And the ability to access novel treatments becomes more urgent in these circumstances.

Waiting three more years for another large, prospective, randomized trial to be completed – to confirm highly promising results already observed in an earlier clinical trial -- may not sound as compelling to the patient who faces these long odds."
Par220 is offline   Reply With QuoteReply With Quote
The Following 2 Users Say Thank You to Par220 For This Useful Post:
KimT (02-10-2019), Narrowminded (02-10-2019)
Old 02-12-2019, 08:17 PM #15
Par220 Par220 is offline
New Member
 
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Par220 Par220 is offline
New Member
Join Date: 2015
City: Southern California
State: CA
Country: US
Diagnosed: 07/2011
Interest: I have been diagnosed with ALS.
Posts: 31
Par220 is on a very distinguished road
Default Re: Brainstorm Nurown 'Right to Try' participant shares progress

Does anyone have an update from the ALS advocates meeting with the FDA?

The FDA approved Radicava with only minimal results only in early ALS in a Japanese trial, not to mention a high cost. I have yet to hear results from anyone using it that come even close to Nurown.

(Radicava wikipedia- It was approved for ALS in the US in 2017 based on a small randomized controlled clinical trial with people who had early-stage ALS in Japan, who were administered the drug for 6 months; it had failed two earlier trials in people with all stages of ALS).

I hope they take Nurown more seriously considering the diagnosis and the multiple significant accounts even from Matt who was diagnosed in 2012.
Par220 is offline   Reply With QuoteReply With Quote
The Following 2 Users Say Thank You to Par220 For This Useful Post:
jaclyn (02-13-2019), Lkaibel (03-01-2019)
Reply

Tags
clinical, family, free, fvc, hope, improvement, lung capacity, medical, night, nurown, speech, swallowing, therapy, treatment, trial, update, work


Currently Active Users Viewing This Thread: 1 (0 members and 1 guests)
 
Thread Tools Search this Thread
Search this Thread:

Advanced Search

Posting Rules
You may not post new threads
You may not post replies
You may not post attachments
You may not edit your posts

BB code is On
Smilies are On
[IMG] code is On
HTML code is Off

Similar Threads
Thread Thread Starter Forum Replies Last Post
Brainstorm considering Right to Try for NurOwn access jaclyn General Discussion About ALS/MND 4 06-26-2018 03:44 PM
NurOwn by Brainstorm fast-tracked anderkling ALS Research News 2 09-07-2015 03:28 PM
Mayo Brainstorm Nurown Stem Cell BlueandGold ALS Drug Trials 8 04-24-2015 08:45 PM
New Forum Participant Calvin-CALS Current Caregivers (CALS) 32 01-24-2015 02:32 PM
ALS shares same gene mutation as Degenerative Myelopathy in dogs Julie N ALS Research News 4 04-13-2009 11:41 AM


All times are GMT -5. The time now is 03:27 AM.

 

Who we are

"We are a volunteer-driven online community that originated in 2003. We are a resource for anyone affected by ALS and MND. We are a community of like-minded and compassionate individuals. Together we are the ALS Forum and together we build this online resource and online community."

 

Want to help?

Have you found our website helpful? Do you enjoy having a place to talk to people that are knowledgeable about ALS and MND? If you have found it helpful to chat with people at different stages of the ALS/MND journey we encourage you to get involved.

Copyright © 2016