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Old 03-21-2011, 09:15 PM
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Exclamation Long-term results of stem cell transplantation for MS

This was released today from the Journal Neurology and reinforces that this treatment is, in its current form, best for highly aggressive disease. It appears there is a nearly 50% chance that my MS will remain stable for 15 years or longer. I'll definitely take those odds, although it is disappointing it is not more effective in SPMS or primary progressive, which the study focused on. (I am also hopeful the benefits and improvements I've seen will last longer than 2 years with the evolved protocol and RRMS phase.) — Dave

Stem cells may show promise for people with rapidly progressing MS

ST. PAUL, Minn. – A long term study reports about the effectiveness of replacing bone marrow, purposely destroyed by chemotherapy, with autologous (self) stem cell rescue for people with aggressive forms of multiple sclerosis (MS). The study is published in the March 22, 2011, print issue of Neurology®, the medical journal of the American Academy of Neurology.

For the treatment, chemotherapy drugs are used to kill all of the patient's blood cells, including the immune cells that are believed to be attacking the body's own central nervous system. Bone marrow stem cells removed from the patient are purified and transplanted back into the body, which saves life by replacing the blood cells and also is proposed to 'reboot' the immune system.

The study followed 35 people for an average of 11 years after transplant. The study involved people with rapidly progressive MS who had tried a number of other treatments for MS with little or no effect. All were severely disabled by the disease, with an average score of six on a scale of disease activity that ranges from zero being a normal neurological examination to 10 meaning death due to MS. A score of six means able to walk with a cane or crutch; a seven is mainly in a wheelchair. All had worsened by at least one point on the scale in the year prior to the transplant.

After the transplants, the probability of participants having no worsening of their disease for 15 years was 25 percent. The probability was higher-44% percent–for those who had active brain lesions, which are a sign of disease activity, at the time of the transplant.

For 16 people, symptoms improved by an average of one point on the scale after the transplant, and the improvements lasted for an average of two years. The participants also had a reduction in the number and size of lesions in their brains. Two people (six percent) died from complications related to the transplant at two months and 2-1/2 years post-transplant.

Study author Vasilios Kimiskidis, MD, of Aristotle University of Thessaloniki Medical School in Thessaloniki, Greece noted that more research is needed on this treatment, including studies that compare people receiving the treatment to a control group that does not receive the treatment.

"Keeping that in mind, our feeling is that stem cell transplants may benefit people with rapidly progressive MS," he said. "This is not a therapy for the general population of people with MS but should be reserved for aggressive cases that are still in the inflammatory phase of the disease."

ABSTRACT

Long-term results of stem cell transplantation for MS
A single-center experience
A. Fassas, MD, V.K. Kimiskidis, MD, I. Sakellari, MD, K. Kapinas, MD, A. Anagnostopoulos, MD, V. Tsimourtou, K. Sotirakoglou, PhD and A. Kazis, MD
+ Author Affiliations

From the Department of Hematology, Bone Marrow Transplantation and Gene & Cell Therapy Unit (A.F., I.S., A.A.), and the Department of Neurology III (V.K.K., K.K., V.T., K.S., A.K.), Aristotle University of Thessaloniki Medical School, George Papanicolaou Hospital, Thessaloniki, Greece.
Address correspondence and reprint requests to Dr. Vasilios K. Kimiskidis, Department of Neurology III, George Papanicolaou Hospital, 57010 Exokhi, Thessaloniki, Greece kimiskid@med.auth.gr
Objective: To report long-term results of a phase I/II study conducted in a single center in order to investigate the effect of hemopoietic stem cell transplantation (HSCT) in the treatment of multiple sclerosis (MS).

Methods: Clinical and MRI outcomes of 35 patients with aggressive MS treated with HSCT are reported after a median follow-up period of 11 (range 2–15) years.

Results: Disease progression-free survival (PFS) at 15 years is 44% for patients with active CNS disease and 10% for those without (p = 0.01); median time to progression was 11 (95% confidence interval 0–22) and 2 (0–6) years. Improvements by 0.5–5.5 (median 1) Expanded Disability Status Scale (EDSS) points were observed in 16 cases lasting for a median of 2 years. In 9 of these patients, EDSS scores did not progress above baseline scores. Two patients died, at 2 months and 2.5 years, from transplant-related complications. Gadolinium-enhancing lesions were significantly reduced after mobilization but were maximally and persistently diminished post-HSCT.

Conclusion: HSCT is not a therapy for the general population of patients with MS but should be reserved for aggressive cases, still in the inflammatory phase of the disease, and for the malignant form, in which it can be life-saving. HSCT has an impressive and sustained effect in suppressing disease activity on MRI.

Classification of evidence: This study provides Class IV evidence that HSCT results in PFS rates of 25%. PFS rate was significantly better in patients with active MRI lesions; HSCT also resulted in a significant reduction in the number and volume of gadolinium-enhancing lesions on MRI.

Footnotes

EDSS
Expanded Disability Status Scale
HSCT
hemopoietic stem cell transplantation
MS
multiple sclerosis
PFS
progression-free survival
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Old 03-22-2011, 04:03 PM
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A cavalcade of stories are coming out on this study (which I have not read in full) and they are interesting. I guess there are a number of ways to read between the lines.

Some are hopeful, such as this article which includes a short interview with Dr. Nash, who is running HALT-MS:
http://www.webmd.com/multiple-sclero...sive-ms?page=1

Some less so. Apparently the head doc for the NMSS is less than enthusiastic that this would ever be a mainstream therapy:
http://health.usnews.com/health-news...ssive-ms-study
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Old 03-24-2011, 03:07 PM
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I've had a chance to review the study in full. There are some interesting items not in the published stories. Here's a bit of what I discovered:

There was only one RRMS patient in the study. There were three RPMS (relapse progressive) patients and one with aggressive "malignant" RPMS. 19 had secondary progressive and 10 had primary progressive.

Some of the SPMS patients had active brain lesions and some did not. Those that had disease activity (active brain lesions) fared far better. PPMS and SPMS patients with no disease activity fared poorly and the treatment was minimally effective, typically failing within a few years.

"In patients with active MRI lesions, PFS was 44% at 15 years (median 11 years; 95% CI 0–22), in contrast to 10% in patients without active lesions (median 2.3 years; 95% CI 0–6) (p  0.01)"

It is interesting to note that none of the deaths in the study came after BEAM conditioning. Deaths were associated with the other conditioning regimen.

Another note: In terms of EDSS improvements, some are ongoing, and those people were not identified, only that they have continued through 7 and 8 years.

With only one RRMS patient treated, the jury is out as to how effective HSCT can be in that population (hopefully the study I am in can help in that regard). But it appears that even among SPMS, as long as there are active brain lesions, that it has the potential to put the brakes on MS for 10 years and longer.
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Old 03-25-2011, 02:38 AM
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Thanks for that analysis Dave!
More evidence that the key is to treat early.

Regards,
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Old 07-09-2012, 08:16 PM
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Just a reminder: for anyone seriously considering HSCT, this is a must read article, as it is one of the few long term studies released. You can purchase it below for $30 from the journal Neurology.

http://www.neurology.org/content/76/12/1066.long
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