Whyare people so uninterested in having SCT?

[CaroleK]

Katje, I'm glad you started this thread...

It's just the nature of life that we all won't agree and the nature of a very complex and poorly understood (medically speaking) disease that makes people suspect about HSCT. I'm with you: I have aggressive MS and have failed on two drugs already. I am going to try for HSCT and hope for the best. It's a personal decision and I don't begrudge anyone who doesn't agree with it. To each his/her own. I, too, have found a lot of support and valuable information from the Facebook forums dedicated to this subject. You did a great service posting about this: you've encouraged some people here, intentionally or not, to think outside the Big Pharma box. Even if, in the end, they don't agree, it was still a discussion worth starting.

[ActiveMSers]

As someone who has been through a stem cell transplant as part of an NIH-sponsored clinical trial, I thought I would clear up some confusion as this thread has become popular. I’ve researched the procedure extensively and collected the abstracts (and in some cases the full studies) of all of the research published on stem cell transplantation for multiple sclerosis over the past 10 years. Regretfully, most information published on the internet is incomplete and only partially correct.

1) Insurance. It is uncommon for insurance to cover the treatment outside of a clinical trial, and even then it is unlikely unless perhaps the patient’s life is at risk and all available drugs have failed. Clinical trials involving stem cell transplants are often paid for by the patient. Unlike most clinical trials, they are rarely free as government funding is lacking and there are no pharmaceutical companies to support the research.

2) Studies. Phase I studies evaluate safety. Phase II studies look at potential effectiveness to justify a larger study. Phase III studies look at effectiveness on a larger scale and will have a control arm, so some patients will be randomized into an FDA-approved treatment instead of getting the transplant. But for stem cell transplantation to get approved by the FDA, these studies have to be completed.

3) Cost. The cost of getting a transplant ranges from as little as $60K (say, if done overseas in India) to over $200K if done here in the States. There are three sites in the US that do the procedure outside of a clinical trial: Chicago (Northwestern with Dr. Burt), Seattle (The Hutch), and Denver (Cancer Center with Dr. Nash). These sites are doing so because of the documented effectiveness of HSCT in MS.

4) Risk. The risk of death is about one percent, which is why the treatment is typically recommended for those with aggressive MS failing conventional therapy. I have known people who have died, lost their hearing, or had to have digits amputated due to severe infection. While the risk of infection is most severe in the first weeks and months, there is also an increased risk for blood cancers in later years.

5) Effectiveness. The treatment is most effective within the first five years of the RRMS phase, in patients 40 and younger, with active lesions. The effectiveness of HSCT in SPMS is questionable and in PPMS unlikely, the success dropping further without active lesions. There are no official published numbers by researchers and those found on the internet (“an XX% cure rate!”) are wishful. The procedure does halt MS progression in most cases, but the key is for how long. For example, in PPMS it is rare for progression to be halted for longer than a couple of years. There are cases that have seen the disease halt for a dozen years... and counting.

6) Improvement. Based on previous research, fewer than half of those treated will see an improvement in his or her EDSS scores, and those improvements tend to be modest, 0.5 to 2.0 points. Some will improve dramatically. Gains may or may not hold over time.

7) Research. More than five dozen studies have been published over the last 10 years on HSCT and MS, and all have shown the treatment to be effective and potentially life-saving in aggressive cases. It works, but there are lots of asterisks. I would strongly encourage anyone considering getting a transplant to purchase the long-term studies and read them thoroughly. Do not trust the “research” on the internet. Heck, including everything I just wrote.

More information on my experience can be found at ActiveMSers, in particular on the forum where I have a dedicated section to follow my progress and recent research. I hope this helps.

Dave Bexfield

[Katje]

I am glad I started the thread as well. It took me a year to be mentally prepared for this, it was not an overnight decision. I also am blessed to have so many awesome people in my life and a large support network of people that are growing everyday. I think that's part of the battle, the rest is up to me alone.

I want to make it clear I never once stated this was a cure nor does Dr. Burt. I am not seeking a cure and people who had this does still consider themselves to still have MS. However what it has shown to do is put our MS into remission. Stats don't lie and the people who had this done years ago are proof that is does work. I even saw a woman with PPMS walking.

I find it doubtful we'll ever find a cure, a cure most think is something that repairs damage and stops the disease. This is why this treatment is key to having it done early, they can't reverse or repair but they can stop it. that is good enough for me and many others. With that said some have had their EDSS scale lowered after the treatment. They are the lucky ones.

FWIW, Lemtrada has a higher mortality rate than HSCT and more side effects. HSCT has a less than 1 percent mortality rate for pwMS, that less than 1 percent were done in it's infancy using radiation on people with PPMS. Neither is done anymore.

I see fear and lack of knowledge in this thread. That alone is more disabling than the disease itself and in like in general.

I have my FB link in my profile, if any of you are on FB send me a message on FB and I will add you. My FB is set up where people can only add me if you know a friend of mine, so you will have to message me first.

~Kat

[misslux]

ActiveMSers, thank you for the write up and clearing up some issues and questions that people had.

[Myoak]

“Lemtrada has a higher mortality rate than HSCT”. I'm not sure Genzyme would agree if I read their press release correctly.

I don’t know how many deaths there have been in MSers who underwent HSCT but with Lemtrada there was one death from sepsis and one from accident during the Phase 3 studies CARE-MS 1 (581 patients) & CARE-MS 2 (840 patients) or in the year following those studies. That would put the mortality rate for Msers taking Lemtrada at far less than 1 tenth of 1% if we attribute that case of sepsis as being caused by Lemtrada.

Genzyme’s press release march 21, 2013:

“Safety results from the first year of the extension study were reported for patients who received LEMTRADA in the Phase III pivotal studies... The most common adverse events during this period of time were infections, including predominantly mild to moderate upper respiratory and urinary tract infections. There were two deaths. One, as previously reported, was from sepsis. The other death was presumed accidental and deemed unrelated to study treatment...”

http://news.genzyme.com/press-releas...tal-ms-studies

Some may want to include cancer patients in safety data for HSCT and Lemtrada, others may not. Perhaps, excluding terminal cancer patients and just looking at Msers would alter the safety data for both treatments and provide better clarity for comparing mortality risk.

Lemtrada does have potentially serious side effects, as Katje said, and as Genzyme acknowledges in the above press release.

[AMJ]

Great thread Katje

You have let everyone weigh in with their thoughts. As has been previously stated not everyone is going to agree on any given treatment.

I personally think that stem cell treatment is the best option right now to halt the progress of this dreadful disease. That is just my opinion and I know others have their own thoughts on this. You have thought about this and done your own research. I wish you all the best with your treatment.

Also great post by ActiveMSers, thank you for posting this information.

[KatieAgain]

As someone who has been through a stem cell transplant as part of an NIH-sponsored clinical trial, I thought I would clear up some confusion as this thread has become popular. I’ve researched the procedure extensively and collected the abstracts (and in some cases the full studies) of all of the research published on stem cell transplantation for multiple sclerosis over the past 10 years. Regretfully, most information published on the internet is incomplete and only

Dave Bexfield

Dave, I just read your Blog on Active MSers. Well worth the read! Your sense of humor was also refreshing. I am so glad this worked out for you...I really am.

I am also very appreciative of the Executive Version you put on MSWorld. Thank-you for sharing your incredible journey with everyone.

Anyone who is interested in the HSCT Procedure...Dave's Blog is a must.

[ActiveMSers]

Dave, I just read your Blog on Active MSers. Well worth the read! Your sense of humor was also refreshing. I am so glad this worked out for you...I really am.

Thanks for the compliments, Katie, but I do have to be clear. Using present tense is very important in this treatment. To date I would say HSCT is working out for me, but that could all change tomorrow and I am quite aware of that. I take nothing for granted and am fortunate to have had my aggressive disease be on holiday for the past four years. We'll see what tomorrow brings, and the day after that. I'm always optimistic, but I am also realistic.

-Dave

[Katje]

Thanks for the compliments, Katie, but I do have to be clear. Using present tense is very important in this treatment. To date I would say HSCT is working out for me, but that could all change tomorrow and I am quite aware of that. I take nothing for granted and am fortunate to have had my aggressive disease be on holiday for the past four years. We'll see what tomorrow brings, and the day after that. I'm always optimistic, but I am also realistic.

-Dave

Did you have myeloablative or non myeloablative? I know the long term side effects with myeloablative are greater.

[Outdoorslover]

Dave, Thanks for your post!

Dave,

I was about to suggest that those interested follow your blog. Thanks for updating all of us on SCT's.

Outdoorslover, Ron

[Dwayne]

Been there, done that!

Hi Katje,
I went to Costa Rica in 2009 for SCT. I was newly diagnosed and had the resources to go for it. It was $30K for a month of treatment.
I don't know where you are looking to go. If it is Panama, that is Dr. Neal Riordan, same Dr. that I had.

In hind site, it did not help me, turns out I have PPMS not RRMS, but it was one of the greatest experiences of my life.

If you have the resources, GO FOR IT!

If you have questions I also live in Denver and would be glad to share my experiences with you.

Dwayne

[Katje]

Hi Katje,
I went to Costa Rica in 2009 for SCT. I was newly diagnosed and had the resources to go for it. It was $30K for a month of treatment.
I don't know where you are looking to go. If it is Panama, that is Dr. Neal Riordan, same Dr. that I had.

In hind site, it did not help me, turns out I have PPMS not RRMS, but it was one of the greatest experiences of my life.

If you have the resources, GO FOR IT!

If you have questions I also live in Denver and would be glad to share my experiences with you.

Dwayne

HSCT in Chicago is not the same procedure that you had done.

[Tawanda]

In hind site, it did not help me, turns out I have PPMS not RRMS, but it was one of the greatest experiences of my life.

I have to ask...how was a failed medical treatment "one of the greatest experiences" of your life??

[Tawanda]

I take nothing for granted and am fortunate to have had my aggressive disease be on holiday for the past four years. Dave

Hi Dave,
Strictly my own .02, but 4 years without this M.S.-B.S. Is a success, IMHO (at least at my age)!

[Jules A]

Thanks for the compliments, Katie, but I do have to be clear. Using present tense is very important in this treatment. To date I would say HSCT is working out for me, but that could all change tomorrow and I am quite aware of that. I take nothing for granted and am fortunate to have had my aggressive disease be on holiday for the past four years. We'll see what tomorrow brings, and the day after that. I'm always optimistic, but I am also realistic.

-Dave

I would also like to thank you for taking the time to add the detailed information.

I especially appreciate your candor with regard to prognosis. One of the most frustrating things for me about this disease is the unpredictability.