This Is MS Multiple Sclerosis Knowledge & Support Community

CVfactor,

You're welcome. I did not ask Dr. Burt why other types of MS are excluded from the trial.

Al

Here is a phase II trial that compares the BEAM protocol with a six dose chemotheapy protocol (mitoxantrone).
http://www.astims.org/multiple-sclerosis-trial.html
This trial does not exclude SPMS.

CVfactor wrote: It looks like the trial run by Dr. Burt does not really distinguish between the types of MS but only requires an EDSS score between 2 and 6.

I would imagine he would have some people in the trial that are PPMS, SPMS and PRMS with EDSS scores near 6.

Any form of progressive MS is automatic disqualification from the trial. Furthermore, Burt will not treat any PPMS cases under any circumstances, even outside of the trial. He said that he "may" on occasion treat someone outside the trial that has 'just" transitioned to SPMS, but not someone that has been SPMS for a longer period of time. This is why I was rejected by Burt in 2009 (I was SPMS for four years at that time).

But still, following my HSCT shows that even a progressive patient can experience tremendous benefit. My disease is 100% stopped and I have further experienced a 50% reduction (improvement) of my EDSS.

CVfactor wrote: So far there are only problems with a small portion who relapse that require additional chemotherapy, but is there any indication of how the people with an initially high EDSS score are doing? Has progression stopped?

23% of patients fail to enter remission and require additional retreatment doses under Burt's non-myelo ablative protocol. That doesn't seem to me to be a small nor insignificant number. (The myeloablative BEAM protocol has not shown this effect and never requires retreatment).

However, following retreatment in the non-myeloablative protocol virtually 100% of all RRMS treated patients go into complete remission (which is also characterized by a 100% stopping of all underlying MS disease activity). The EDSS at time of transplantation does not seem to affect this, so even high EDSS patients that are RRMS are extremely likely to experience this good result. However, patients that have a high EDSS and are progressive do not have anywhere near this good result as shown in this single page summary:

http://2.bp.blogspot.com/-PvejGH-NIG4/T ... al%2B2.jpg

the ASTI trial is an international trial. where are the procedures taking place?

do you think dr Burt is trying to achieve the best results he can in order to move this thru FDA approval and in doing so, with rrms patients, the percentage of successful procedures will be higher and therefore look better on the stats sheet?

Hi all (Ciao!) First of all, please accept my apologies for my poor english, I am from Italy and I will try my best. I am here because my wife has been diagnosed ten years ago and after some years with Betaferon she's facing a really bad period (8 relapses in the last 13 months) and has been just shifted to endoxan (cyclophosphamide). Indeed she could not get tysabri being positive to JCV and fingolimod is not yet available in her ms department here. Until the end of 2010 her edss was 1 and now it is 6.5. We are so scared, it seems that cyclophosphamide is not working much, and we are trying to understand whether hsct can be a good way. Next week we'll meet an important neurologist who is leading a trial on hstc, my wife is scared about the procedure but I strongly believe it can be the only way to stop her ms activity. About a month ago we had a meeting with a neurologist involved in a mesenchymal stem cell trial (the trial announced last autumn involving several departments in Europe and Canada(I am not so sure about Canada)): my wife could join the trial (she's ok with all the criteria) but we don't know if this is the right time to get into a phase I/II trial with much higher uncertainty about the results. We are very confused...ms is running so fast now and we can't waste time. I would be so grateful if you could share your opinions, suggestions.

Thank you so much for your replies, this is such a beautiful place to talk about science.

Ciao
Step

Hi Step,

First-off your English is quite good (in my humble opinion). From what you have mentioned your wife's MS has taken an aggressive turn despite her treatment on a disease modifying drug (not uncommon); is she diagnosed with RRMS? The cyclophosphamide may help, however much may depend on the dosages given as cyclophosphamide dosing is very precise based mainly on the patient's weight. If she can get into an HSCT trial I would strongly suggest it. As you may know there are mainly two HSCT protocols for MS in the world; myeloblative and non-myeloablative. I had Dr. Burt's non-myeloblative protocol on 8/22/11 at Northwestern Memorial Hospital in Chicago, Illinois. This protocol's primary 'conditioning' drugs are cyclophosphamide and rATG (rabbit antithymocyte globulin) administered in combination through IV. This international trial is also taking place in Sweden (Uppsala University).

The website is: http://clinicaltrials.gov/ct2/show/NCT0 ... sis&rank=2. Please note this is a Phase III trial.

Dr. Slavin in Israel is also performing non-myeloablative HSCT for MS.

The myeloblative HSCT protocol for MS can be performed at various hospitals throughout Europe including Heidelberg University Hospital in Germany. However, it is very important to select a very reputable hospital, assuming she is accepted. Based on her rapidly increasing EDSS score, indicative of a highly inflammatory MS course, the HSCT may be a good course of action. HSCT is generally very effective in combatting a high degree of inflammation resulting from MS-mediated auto-immune responses.

Al

Step,

I'm really sorry to hear about your wifes deterioration but hopefully you can halt progression through HSCT treatment. There are a lot of knowledgable people here so hopefully they can give you good advice.

Good luck.

Chilax wrote:Hi Step,

First-off your English is quite good (in my humble opinion). From what you have mentioned your wife's MS has taken an aggressive turn despite her treatment on a disease modifying drug (not uncommon); is she diagnosed with RRMS? The cyclophosphamide may help, however much may depend on the dosages given as cyclophosphamide dosing is very precise based mainly on the patient's weight. If she can get into an HSCT trial I would strongly suggest it. As you may know there are mainly two HSCT protocols for MS in the world; myeloblative and non-myeloablative. I had Dr. Burt's non-myeloblative protocol on 8/22/11 at Northwestern Memorial Hospital in Chicago, Illinois. This protocol's primary 'conditioning' drugs are cyclophosphamide and rATG (rabbit antithymocyte globulin) administered in combination through IV. This international trial is also taking place in Sweden (Uppsala University).

The website is: http://clinicaltrials.gov/ct2/show/NCT0 ... sis&rank=2. Please note this is a Phase III trial.

Dr. Slavin in Israel is also performing non-myeloablative HSCT for MS.

The myeloblative HSCT protocol for MS can be performed at various hospitals throughout Europe including Heidelberg University Hospital in Germany. However, it is very important to select a very reputable hospital, assuming she is accepted. Based on her rapidly increasing EDSS score, indicative of a highly inflammatory MS course, the HSCT may be a good course of action. HSCT is generally very effective in combatting a high degree of inflammation resulting from MS-mediated auto-immune responses.

Al

Hi Al,

thank you so much for your reply. My wife has been diagnosed with RRMS, when two months ago I asked the neurologist if it is turning into secondary progressive he said that now it is still RRMS but with rapid disability accumulation. If I am not wrong they're giving her 1g of cyclophosphamide every 28 days, the problem is that now she's feeling all the collateral effects but not improvements on ms.
I will try to get all the informations about the HSCT protocols and the trials we could join. Thank you so much.

Step

CVfactor wrote:Step,

I'm really sorry to hear about your wifes deterioration but hopefully you can halt progression through HSCT treatment. There are a lot of knowledgable people here so hopefully they can give you good advice.

Good luck.

Hi CV,

Thank you for your kind reply, reading here I am getting much information to make a good choice.

Step

Chilax wrote:Hi Step,

First-off your English is quite good (in my humble opinion). From what you have mentioned your wife's MS has taken an aggressive turn despite her treatment on a disease modifying drug (not uncommon); is she diagnosed with RRMS? The cyclophosphamide may help, however much may depend on the dosages given as cyclophosphamide dosing is very precise based mainly on the patient's weight. If she can get into an HSCT trial I would strongly suggest it. As you may know there are mainly two HSCT protocols for MS in the world; myeloblative and non-myeloablative. I had Dr. Burt's non-myeloblative protocol on 8/22/11 at Northwestern Memorial Hospital in Chicago, Illinois. This protocol's primary 'conditioning' drugs are cyclophosphamide and rATG (rabbit antithymocyte globulin) administered in combination through IV. This international trial is also taking place in Sweden (Uppsala University).

The website is: http://clinicaltrials.gov/ct2/show/NCT0 ... sis&rank=2. Please note this is a Phase III trial.

Dr. Slavin in Israel is also performing non-myeloablative HSCT for MS.

The myeloblative HSCT protocol for MS can be performed at various hospitals throughout Europe including Heidelberg University Hospital in Germany. However, it is very important to select a very reputable hospital, assuming she is accepted. Based on her rapidly increasing EDSS score, indicative of a highly inflammatory MS course, the HSCT may be a good course of action. HSCT is generally very effective in combatting a high degree of inflammation resulting from MS-mediated auto-immune responses.

Al

Thank you for the information. One of the Principal Investigators, Roumen D. Balabanov, is Bulgarian as me, and I've just wrote him an e-mail. Hope he can give me some directions if it is possible to participate in the trial in Europe. I'm getting nowhere with local neurologists on the stem cell topic. I'm getting in to phase 3 trial of Ocrelizumab next week, will have the drug or Rebif, but I know it is not a cure and will look for ways to get HSCT treatment one way or another, while I'm still in good condition.

shaight wrote:the ASTI trial is an international trial. where are the procedures taking place?

Hi Shaight,

Sorry for my delay. Taking care of new baby things.

The Phase II randomized ASTIMS trial in affiliation with the European Bone Marrow Transplantation (EBMT) group evaluating autologous HSCT in severe cases of MS was terminated due to difficulty in accruing patients and lack of funds. So the authors transformed the study into something else as reported here, with the same authors in 2005 and 2006 papers. . . . .

Autologous stem cell transplantation for progressive multiple sclerosis: Update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database

http://www.futurehealthbiobank.it/uploa ... ltipla.pdf

Haematopoietic stem cell transplantation in the treatment of severe autoimmune disease: results from phase I/II studies, prospective randomized trials and future directions

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1809422/

Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis

http://www.nature.com/bmt/journal/v45/n ... 9305a.html

Bottom line is that the partially-completed study indicates a definite role for HSCT in the treatment of MS (including SPMS as was my case), especially considering the cost-effective economics of the treatment and end-result. But it’s sad that the authors looked at only the limited view of these aspects of HSCT-for-MS because what is clearly being overlooked here is how much improved people’s lives become following HSCT. As mine is following HSCT.

Patient-Reported [Quality of Life - QoL] Outcomes in Multiple Sclerosis Patients Undergoing Autologous Stem Cell Transplantation

http://www.stemcellms.ru/doklad11.php

step wrote:
Hi Al,

thank you so much for your reply. My wife has been diagnosed with RRMS, when two months ago I asked the neurologist if it is turning into secondary progressive he said that now it is still RRMS but with rapid disability accumulation. If I am not wrong they're giving her 1g of cyclophosphamide every 28 days, the problem is that now she's feeling all the collateral effects but not improvements on ms.
I will try to get all the informations about the HSCT protocols and the trials we could join. Thank you so much.

Step

Hi Step,

I agree with everything that Chilax states. Especially that HSCT treatment for MS earlier is better than later.

Just FYI. . . . I am aware of these facilities that will perform HSCT for MS outside of a trial. I am also communicating now with a facility in Russia that will also perform the procedure in Moscow for a relatively reasonable price and will add it it to this listing page next week:

http://themscure.blogspot.com/2011/06/g ... -have.html

George,

once again, thank you for providing all this information. your knowledge and resources are truly incredible. this quote was one of many that stood out to me,

"Limited case series have reported a dramatic reduction in relapse in patients with SPMS following HSCT;24 however, there is currently no controlled evidence to show the magnitude of this potential benefit. This assumption may therefore underestimate the benefit of HSCT in reducing MS relapse."

and please don't ever worry about not replying to a post! focus on Kai!

sean

georgegoss wrote:

step wrote:
Hi Al,

thank you so much for your reply. My wife has been diagnosed with RRMS, when two months ago I asked the neurologist if it is turning into secondary progressive he said that now it is still RRMS but with rapid disability accumulation. If I am not wrong they're giving her 1g of cyclophosphamide every 28 days, the problem is that now she's feeling all the collateral effects but not improvements on ms.
I will try to get all the informations about the HSCT protocols and the trials we could join. Thank you so much.

Step

Hi Step,

I agree with everything that Chilax states. Especially that HSCT treatment for MS earlier is better than later.

Just FYI. . . . I am aware of these facilities that will perform HSCT for MS outside of a trial. I am also communicating now with a facility in Russia that will also perform the procedure in Moscow for a relatively reasonable price and will add it it to this listing page next week:

http://themscure.blogspot.com/2011/06/g ... -have.html

Hi George,
thank you so much for your reply. I just read your blog and I found it really helpful. Thank you for sharing your experience and your knowledge.
I would like to ask you your opinion about mesenchymal stem cell. My wife could join a trial (she's ok with the criteria of the trial) but we thought at this very aggressive stage of her ms it wouldn't be the right choice.

Thank you again for everything you are doing for ms community.

Step

P.S.: Congratulations for the baby!!

Step,

If you have a facebook account, check out this group. Many people that have gone thru or are going thru HSCT are members (including George) and there is a wealth of information available.

http://www.facebook.com/groups/149103351840242/