Where is stem cell therapy, today?

[JerryD]

I have been patiently waiting and watching stem cell treatment development for about 3 years. There have been many clinical trials at prestigious clinics in the US. I haven't heard much about the outcomes and I wonder what has been going on. There seems to be 'black hole' of information on this subject and it bothers me. If Dr.Burt at Northwestern conducts a clinical trial doing stem cell replacement, why is it difficult to get the results to the population with MS? If the Cleveland Clinics' MS center does a trial using stem cell replacement, where are the results? These boards are the perfect conduit to spread the outcomes of these trials. Where are the reports?

[Marco]

Jerry,
I believe the problem you are experiencing is one of control.
Since the trial is still under way, the results will be delayed until the conclusion of the trial. The trial doctor's do not want to prejudice, or even discourage, future participants. The stem cell patients are quite different than the trial administrators. I'm believe if a patient had experienced a life changing experience no non-disclosure agreement would be able to contain them. They would be talking, their friends would be talking, everyone in their lives would be talking.

Just recently, I had an ITP-Baclofen pump installed. Since then, people are coming up to me to inquire what has changed. These are not just my friends and family, but people at the grocery store, at the pharmacy, and anywhere I frequented over the past few years. Many have asked about the absence of my wheelchair, or my new capacity to perform "X" where such ability existed before. These people have gone on to tell their own social circles about my pump and additional people have been inspired.

The final report will contain the information you seek. Until then, you will need to survive on the crumbs of data that come from a potentially melancholy experience. It's possible that what benefits have been received are from nuking the immune system and not from stem cell. If true, than stem cell is much more akin to Lemtrada, and much less like a cure. I hope that I am wrong, but the lack of enthusiastic results or testimonies might suggest otherwise.

I personally believe that stem cell therapy will become a silver bullet, but at this point I'm not sure it's even silver, much less a bullet. Continue to have hope, especially on treatments tailored to controlling your MS. The cure will eventually present itself, but don't miss the fix, while waiting for the cure.

[fairpace]

Some have previously referred to Facebook for some stem cell groups and information. When my curiosity led me there, I found that the groups were closed and I'd have to join (the Facebook groups for said transplant topic) in order to stick my nose in the dialogue.

Since I'm currently in the "MS closet" and don't care to have any MS footprint on my Facebook page, I really wish some of those enthusiastic transplant anecdotes (I'm assuming) would share here on MSworld (not to mention for the sake of many who forego Facebook altogether.)

Maybe it's overrated for now... Otherwise, as Marco said, we'd be hearing about it.

[Marco]

I not only went into some of those groups, but I even completed Dr. Burt's screening application. I received the following email.

Hello Mr. Martinez,
Thank you for your interest in our stem cell therapy program. Unfortunately, you do not meet the criteria for our study and it is unlikely that you could benefit from our therapy. I am sorry that I could not have been more helpful.

I responded,

"Thank you for the response. I know I am, and I am sure others would be, interested in what criteria we were disqualified for. Nevertheless, thank you again for your response and the work that you do. Much appreciated."

I did not receive a response. The selection criteria for the stem cell trial is strict and would undoubtedly disqualify the majority of MS patients. So even if the news was awesome, it would not apply to the majority of patients reading it. If you're young, healthy and fit, then stem cell, like Lemtrada, may keep the MonSter at bay, but if you already have damage, the treatment may not be of much help. The first treatment that may help repair damage is anti-Lingo1
and it will be a number of years before that medicine may become available. There are already people lining up claiming we do not have a remyelination problem, we have a continual damage problem. If true, then maybe Lemtrada is the first, best answer we have. Let's just hope there aren't a number of "gotchas" found in the first Lemtrada wave. Like with stem cell, I have not been able to obtain any real Lemtrada post-marketing data any of the countries where it has been previously approved.

Of course, the Lemtrada 'cash cow' could be here in the US (where we subsidy medical care for the rest of the world), so if bad news was to come out prior to the US Lemtrada launch the results could be catastrophic. The first Lemtrada patient will be from San Antonio, Texas and then the turnstiles will be open full-tilt. By summer 2015, Lemtrada will be in too short of supply, or collecting dust with the other therapies that overpromised and underdelivered (CCSVI, cladribine and others quickly come to mind).

[JerryD]

Thanks for the responses. Marco, I was pretty sure that you would 'chime in'. And I was counting on it. I value your opinion. And while you received a letter from Dr. Burt explaining that weren't qualified for his treatment, I received a phone call from one of the doctor's team explaining why I wasn't qualified.
Yet, none of his patients that were successfully treated, have come forward and fully publicized their 'miracle'. What goes on ? There should be nearly 1000 successes by now. I mean, these patients or former patients are unusually tight lipped, don't you think. What goes ? I know that the day that we have a proven remedy for MS, there will be plenty of news stories. I'll know it when I see it. I hope it will be soon.

[Marco]

Jerry,
I've been pulling for you for a long time. I know you've had a rough go and have been looking for the silver lining in future therapies. With all due respect, maybe improving your present might be the more prudent task.

You might consider to writing down the Top 5 activities, tasks or whatever that you need help with. Then find a resource to help you with those Top 5. This isn't "I want to walk the Great Wall or scuba dive the Great Barrier Reef." This is more, "if I could reliably walk 35 feet, I would be self-sufficient walking at home." Then you could get a home health PT person to help you with your gait, removing obstacles in your home, strengthening your legs or stabilizing your core strength. This exercise could improve your daily quality of your life and rid you of some of your most frustrating tasks.

When goal setting, it's important to use a framework that works well for both you and the goal. Here are some framework examples:

• SMART: Significant, Measurable, Achievable, Relates to person, Time based
• ABCD: Audience, Behavior, Condition, Degree
• FEAST: Function, Expectation, Action, Specific conditions, Timeline
• RHUMBA: Relevant, How long, Understandable, Measurable, Behavioral, Achievable

Most of the speech, physical and occupational therapists are encouraging, but what about the rest of your life? For me, I have been working with a Stephen Minister(SM) for just under 2 years. A SM is a lay person trained to provide one-to-one, Christ-centered care to hurting people. My SM dutifully comes for one hour/week and will simply be present. During our time, there will be a prayer, but I believe this is optional. He has never pushed 'religion' on me, but as a Christian we have talked about how it impacts my life and struggles.

Again, just having a few resources to use has made a tremendous difference in my life. So I would implore you to get help when and where you can. This could improve your current QoL, and make waiting for the next breakthrough to be a bit easier. Rehab really enforced energy conservation so I would have the energy needed for important things in life. Their little tips & tricks made a difference and I'm so grateful to them.

I am thrilled to hear you have a new neurologist that is still willing to try things. With Tecfidera, your lymphocyte count will hopefully go down reducing the amount of daily damage that you may incur. I hope it works wonders for you, but it might take a 4-6 week adjustment period for your body to tolerate it.

Just remember, you eat an elephant one bite at a time. I wish you well ...

[Marco]

This update on mesenchymal stem cells was updated earlier this month.

http://www.ncbi.nlm.nih.gov/pubmed/25385295

The purpose of the study was to evaluate the therapeutic effects of human umbilical cord-derived mesenchymal stem cell (hUC-MSC) transplantation in MS patients. Twenty-three patients were enrolled in this study and 13 of them were given hUC-MSC therapy at the same time as anti-inflammatory treatment, whereas the control patients received the anti-inflammatory treatment only. Treatment schedule included 1,000 mg/kg of methylprednisolone i.v. daily for 3 days and then 500 mg/kg for 2 days, followed by oral prednisone 1mg/kg/day for 10 days. The dosage of prednisone was then reduced by 5mg every two weeks until reaching a 5mg/day maintenance dosage. Intravenous infusion of hUC-MSCs was applied three times in a 6 week period for each patient. The overall symptoms of the hUC-MSC treated patients improved compared to patients in the control group. Both the EDSS scores and relapse occurrence were significantly lower than those of the control patients. Inflammatory cytokines were assessed, and the data demonstrated a shift from Th1 to Th2 immunity in hUC-MSC treated patients. Our data demonstrated a high potential for hUC-MSC treatment of MS. This manuscript is published as part of the International Association of Neurorestoratology (IANR) special issue of Cell Transplantation.

[CaroleK]

I need to chime in here...

Hi Jerry and Marco: I completely understand the frustration with the lack of testimony/information on the stem cell trials going on in the US. Marco is right: Dr. Burt's study which is in Phase III, will not be complete until probably 2019 or so (that is 5 years' out from his treating the transplant arm of the study) and then the results have to go through the FDA for final approval as a therapy.

Personally, and you probably know this Jerry, I just underwent HSCT with Dr. Burt in September. Yes, I was extremely fortunate to qualify for the study. I will never use the word 'cure' as the damage has been done, but I am very hopeful that the disease's progression will be halted. I won't, of course, know for sure if it has until I have follow up MRIs and as the years go by, but I remain hopeful. Overall, I feel pretty good. My legs are a bit achy and stiff but they were always the area most effected by MS and if they never improve, so be it.

Please don't discount, just because you may not want to join one of the Facebook forums dedicated to Dr. Burt's treatment or other facilities world wide who perform HSCT, the positive results so many patients have experienced by undergoing this procedure. There are people in the forums (I include George Goss's FB forum also) who are 4 to 5 or more years out and still progression free. This is very encouraging.

Because clinical trials are so time consuming, and the FDA approval process is not quick either, the larger MS community as a whole won't see HSCT as an FDA approved treatment until probably 2020 or so. I know so many people do not have the time to wait and it saddens me that this is, unfortunately, the reality. Many people have had to turn to overseas facilities (Russia, Germany, Italy) because of this, which, of course, can be quite costly. I hope someday this treatment will be an option to ALL MS sufferers.

[JerryD]

Again, thanks for the responses. I continue to follow this thread and I appreciate all of the good thoughts.
CaroleK, thanks for staying in touch after your treatment with the venerable Dr. Burt. Like I said before, there are probably 500 or 600 patients that have had HSCT at Northwestern (and other clinics) and you ( and George Goss ) seem to be the only patients that will come out of the closet and discuss the results.
As you know, George's treatment was in Germany. I don't know about every person that had the treatment and if these were successes. It would be nice to have access to that information. I have to wonder what is the problem with hearing all of the info ? I know about the Facebook groups. I find Facebook cumbersome. Thanks, again, good luck to all.

[CaroleK]

Hi again Jerry: I don't think it is just me and George that are willing to come out of the closet to talk about HSCT; I just think many many patients prefer to do it in one of the Facebook forums dedicated to HSCT.

To be honest, I have read several posts in these FB forums that people have tried to spread the word in other MS forums (including MSWorld) and have not felt supported talking about HSCT due to many naysayers (just read George Goss's post and the numerous doubtful comments posted in response to it here and you can see why people might not feel overly supported with their choice to undergo HSCT).

If you are looking for statistical data related to Dr. Burt's Phase I/II study for MS, please google it using the words 'Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study' and you will find his very promising results that were published in The Lancet.

And, for what it's worth, I will keep updating periodically as I progress through recovery.

[Marco]

Carole - I am so grateful for your comments. Please do not be a stranger.

[JerryD]

CaroleK, I cannot speak for the rest of the community, but I am so grateful for your continued updates. Best wishes for your continued recovery.

[dyin_myelin]

Since I'm currently in the "MS closet" and don't care to have any MS footprint on my Facebook page, I really wish some of those enthusiastic transplant anecdotes (I'm assuming) would share here on MSworld (not to mention for the sake of many who forego Facebook altogether.)

Maybe it's overrated for now... Otherwise, as Marco said, we'd be hearing about it.

I am also in the closet as far as my employment goes. I have joined George Goss' closed facebook group and have not had any indication that anyone is now aware that I have MS. George, despite having a degree in physics/career, has been unable to find employment despite a halting of his disease activity and some significant improvements. That could be why not as many people are "loud and proud" as are claiming benefits per Jerry's question. I chose to take the calculated risk of joining and I am glad I did. However, it is easy to set up a spoof FB account which would give you an added layer of privacy. Set up a throwaway email account, fake city, name, picture of a sunset and you are good to go.

I regret somewhat using my real name, and I'm sure a master hacker could divine that I am a member of the HSCT closed group. However, being that I teach art vs. working the design pipeline at Lockheed Martin's Skunkworks, I choose not to flatter myself and feel I have jeopardized any future employment opportunities or my current job. I think the greatest threat to those things right now is MS, and I will avail myself of any resources or information that I can. Looking forward to meeting you there, hopefully.

[aminfla]

Dr Burt HSCT Not A Big Scary Secret

The trial does have strict guidelines because it has been found that RRMS(inflammation stage is when HSCT works) is the type of MS that is most effectively stopped by HSCT. There is a treatment center in Russia that treats some other forms of MS and is effective to varying degrees. Russian is not a trial and if you qualify you get the treatment. It is booked through 2015 I believe. Dr Burts office in Chicago is swamped with applicants and enrollees.

In regards to the Dr Burts HSCT FB forums being closed: This is to provide privacy and open discussion pro and con of the subject. It is very easy to join simply by asking. Want to be anonymous? Create a FB profile with a fake name(it is done all the time). Disclose only what you want then! There is also an international forum on FB concerning all the locations HSCT is being done, Russia, Italy, Sweden, Germany, Brazil, Australia(soon) and more to come. So please don't get bent out of shape about this big secret! It isn't one, except the doctors(who do not want to lose money), Drug companies (who do not want to lose money). Follow the money and you will see who and what wants HSCT to be the big scary, dangerous thing that it is not. How do I know all this? My wife who "HAD" CIDP which is a cousin to MS had HSCT done almost 3 years ago rolled into Chicago on a rollator and out carrying a cane. I live on the HSCT forums on FB and the facts are there if anyone really wants to know, ask to be added to Dr Burts forum on FB . So I posted URL's to this message but haven't posted 5 posts so cannot post them LOVE THEM RULES!

[JerryD]

I am sorry to say that I have been searching the Dr. Burt HSCT facebook site to read posts from patients and I have not been able to access any. I admit that I am somewhat of a 'dummy', but I don't have any problem posting on this site. The Dr.Richard K Burt etc. site is 'closed'. How does one read posts from the successes? I must be missing something. and I'm frustrated with FB.