Brainstorm Cell

[Lobster]

Lou Gehrig's disease patient: Brainstorm Cell treatment helped me


Avichai Kremer is the first patient to be treated with Brainstorm Cell's drug, NurOwn, on the basis of a special approval.

4 August 11 15:19, Yael Gruntman and Gali Weinreb


A patient with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) has testified to the effectiveness of the stem cell-based treatment for the disease being developed by Brainstorm Cell Therapeutics Ltd. (Bulletin Board:BCLI).

In a letter obtained by "Globes", Avichai Kremer stated, "I was diagnosed (with ALS) seven years ago at age 29. Today I am almost completely paralyzed and can’t walk, talk, or eat (I use a feeding tube). I am writing this letter using my head. ALS/MND has no effective treatment and diagnosis means a death sentence even today, a decade into the 21st century.

"Nevertheless, I was fortunate to be the first to get the stem cells treatment of Brainstorm Cell Therapeutics two and a half months ago (October 2010) at Hadassah hospital in Jerusalem, Israel. The procedure was simple and quick. Within 24 hours I was back home. Two and a half months later, I can testify that the Brainstorm treatment is completely safe and that my condition didn’t deteriorate in that time. In fact, not only did the disease seem to slow/stop in that time period, but preliminary follow-up tests done at Hadassah showed slight improvement in several physical functions such as breathing and speech."

Kremer is the first patient to be treated with Brainstorm Cell's drug, NurOwn, on the basis of a special approval. The treatment is not part of the clinical trials that are conducted to establish statistical analyses of a drug's efficacy and safety. The company is currently conducting a Phase I/II clinical trial of the drug.

Treatment with a drug without US Food and Drug Administration (FDA) or Ministry of Health approval of a single patient is an extraordinary procedure, given in incurable diseases. Such cases are usually handled at the medical center that is also conducting the clinical trial, and the results are not always sent to the company that is developing the drug.

Brainstorm Cell initiated the clinical trial of NurOwn last month, and is currently selecting patients for participation. If the treatment is successful, it could be a historic achievement, as there is no treatment for ALS, and sufferers die within 2-5 years. However, success in a single patient often cannot be replicated, and is statistically meaningless in a clinical trial and have no bearing on approval of the drug.

Nonetheless, Kremer's letter is the first indication of possible success of Brainstorm Cell's treatment - provided that the clinical trial achieves similar results. A successful treatment opens the $1 billion a year ALS market before the company.

Brainstorm Cell declined to comment on the report.

Brainstorm Cell's share price closed at $0.44 on Wall Street yesterday, giving a market cap of $52.6 million.

Published by Globes [online], Israel business news - Globes - Israel business news - on August 4, 2011

 

[helpingpals]

I am glad that this is posted. This article is partially while I decided to join this forum. I have been checking it out for a while but have been too nervous to join or post. I am heavily involved with a few ALS groups / associations / clinics etc in Atlanta and other portions of Georgia. I am not sure if this is the right place to post this but i founf it relevant to the article. I want to know if anyone else has been hearing what I have been hearing.

Not sure how to say it so I will just spit it out. There has been a lot of buzz over the last couple of months that the Stem cell trial at Emory has actually helped one person. I have no proof but have heard several times that there is one gentlemen who has shown sighnificant improvement. Not cured but regained strength in hands, arms, legs and even lungs. From what I understand (second hand) is that even teh Dr's are amazed because they did not expect it to help so many regions of the research subjects body. However the word is that they are keeping it close to the vest because they have only seen this in one of the 12 patients. Evidently while this is a safety based trial they have been tracking efficacy data as while and in the next few months there wil be an article coming out with the efficacy data and that one of the patients numbers will raise eyebrows.

Unfortunately as the article above states until they can replicate the results they are taking a wait and see approach and observing the research subject and measuring progress or regression.

Obviously no cure yet but perhaps if what I have heard is true and the above article is true then perhaps there is hope for the future.

If anyone else has heard more about the above article or the Emory trial I would love to learn more. It warms my heart to hear potential positive news for a change when it comes to this dreaded disease.

God bless all CALS and PALS.

 

[helpingpals]

I failed to mention that they are hoping that once the FDA grants approval and they move the injections to the cervical area of the spine that they will possibly be able to duplicate the results. As long as they have the FDA's approval they plan to begin the next group at the end of August / beginning of September.

I am praying for every PAL and CAL that while this does not seem to be a true cure that they can eventually replicate the results and it can become a viable treatment that helps numerous patients.

 

[trfogey]

Not sure how to say it so I will just spit it out. There has been a lot of buzz over the last couple of months that the Stem cell trial at Emory has actually helped one person. I have no proof but have heard several times that there is one gentlemen who has shown sighnificant improvement. Not cured but regained strength in hands, arms, legs and even lungs. From what I understand (second hand) is that even teh Dr's are amazed because they did not expect it to help so many regions of the research subjects body. However the word is that they are keeping it close to the vest because they have only seen this in one of the 12 patients. Evidently while this is a safety based trial they have been tracking efficacy data as while and in the next few months there wil be an article coming out with the efficacy data and that one of the patients numbers will raise eyebrows.

If you have no proof of the claims you are making, which you readily admit, why speculate and spread what is tantamount to unsubstantiated gossip? The report on the trial will be provided when it's ready and when the evidence is in.

And the raised eyebrows that you speak of should be directed toward establishing that any effects reported are actually caused by the therapy and not due to some other cause.

If anyone else has heard more about the above article or the Emory trial I would love to learn more. It warms my heart to hear potential positive news for a change when it comes to this dreaded disease.

The last official update posted on the Emory website is here:

Emory ALS Center: ALS Research

 

[fba75]

A phase I trial’s purpose is basically to assess the safety and the tolerability of a new drug or treatment. You can not get any information about the effectiveness of a treatment before phase II at least. And usually it is with Phase III that you have a real assessment of the effectiveness.

We all want to hear good news about a promising new treatment but we have to stay realistic. A good phase I result means the treatment seems to be tolerated and not “too dangerous”. That’s it, nothing more. If a patient feel better after this trial, good for him but medically it is not significant.

We have to be aware of that to avoid all sorts of scammer’s doctors pretending to offer new effective treatment. They are both expensive and dangerous.

 

[helpingpals]

trfogey ypu are correct. My apologies. I guess I have been so excited about what I have seen and heard and got carried away. Your other point is extremely astute as well. My understanding is they did go back and re diagnose the patient from the beginning to confirm the original diagnosis which they did and they have been attempting to rule other theories out as well. It is difficulty because they can not truly determine the migration paths of the stem cells and if they survived or multiplied tec until the after the patient has passed away and they can do a spinal biopsy. I hope for the gentlemen that it is a very long time for him.

It is unfortunate that it is only one patient thus far, I pray daily that they can learn and understand more. I apologize again if I offended anyone. All I can say is that this is real. I do not know the patient personally but I do know when I saw him at functions he used to use a cane and walked slowly. I saw him recently without a cane and that was after I had been hearing positive news.

I guess I am being over zealous and hoping for good news and frustrated that nothing more has come out about this even though several of us know what is happening from what we have seen and from what whispers are coming out of people we now from the clinic.

You are both correct it will come out when Emory is ready. Just one other note while it is a safety study tehy are definitely recording the efficacy data. As a matter of fact if you go to clinicaltrials.gov and look up the trial they have all the secondary outcomes they are measuring which is the safety data.

 

[helpingpals]

Here is the link to the trial on the CDC website as you can see while it is a safety study and that is the primary outcome they are recording and reporting to the FDA the efficacy data.
Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis - Full Text View - ClinicalTrials.gov

Thanks again for your time. I apologize again for giving any false hopes, I guess my desire and passion outweighs the rationale portion of me at times.

 

[asantiago]

So that was done nearly a year ago? How is he now I wonder and did he continue receiving treatments? I wouldn't expect much deterioration in two months from a man who has already had ALS for 7 years. Was that just a safety trial?

It seems there are several drugs/treatments in the works that may show potential, I question if it has always been this way with hope dangling the carrot for PALS or could we actually be nearing some effective treatments?

 

[helpingpals]

As a matter of fact if you go to clinicaltrials.gov and look up the trial they have all the secondary outcomes they are measuring which is the safety data.

I meant to say that the secondary outcomes are the efficacy data. The primary outcomes is the saftey data. Both are being measured both prior to the surgery and post surgery.

 

[helpingpals]

So that was done nearly a year ago? How is he now I wonder and did he continue receiving treatments? I wouldn't expect much deterioration in two months from a man who has already had ALS for 7 years. Was that just a safety trial?

It seems there are several drugs/treatments in the works that may show potential, I question if it has always been this way with hope dangling the carrot for PALS or could we actually be nearing some effective treatments?

No from what I understand he was in the last group and was done in 2011. It is not a matter of deteriration it is the fact that he showed considerable improvements.

You are right there seems to always be a carrot out there that never comes to fruition. I pray that this is not that scenario but as others have pointed out they need to be able to replicate it before it becomes a real hope.

One thing to think about which honestly is not helpful is that they do not truly know what ALS is. They know the syndrome but not the root cause. Some people have discussed that some treatments may help some PALS but not others depending on what the trigger was. Of course with only 12 people done so far it is not a big pool to determine that.

 

[asantiago]

The article said "Nevertheless, I was fortunate to be the first to get the stem cells treatment of Brainstorm Cell Therapeutics two and a half months ago (October 2010) at Hadassah hospital in Jerusalem, Israel."

and not to be a stickler about words but the article said slight improvements then it didn't really define how they were measured

Basically what I'm saying here is TELL ME MORE.

 

[helpingpals]

I am sorry asantiago I thought you were referring to the patient I was talking about at Emory. My apologies.

 

[asantiago]

gotcha! That makes more sense. thanks for the clarification.