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June 9, expert reaction to trial of stem cell transplantation with chemotherapy and multiple sclerosis A paper published in the Lancet journal has looked at treatment of multiple sclerosis and reports the efficacy of chemotherapy to ablate the immune system followed by a stem cell transplant.
The complete suppression of clinical relapse and the lack of development of new brain lesions in 23 of the 24 patients, after long-term follow-up of autologous Haematopoietic Stem Cell Transplantation HSCTand in the absence of any disease-modifying drugs, is a remarkable finding.
This is a clear advantage. This is particularly relevant since MS is classified into four main subtypes: This study has shown improved measures across both relapsing remitting 12 patients and secondary progressive 12 patients disease course profiles.
As the authors of the study have acknowledged, aside from the small sample population 24 and the lack of appropriate controls, the complications treatment-related risks and mortality associated with this more aggressive chemoablation regimen necessary in order to ensure a more complete renewal of immune cells does limit widespread adoption.
The risks need to be carefully weighed when compared with the beneficial outcomes.
The present study indicates a need to examine this further. The study shows that long-term follow-up after transplantation is essential for managing the health of MS patients.
Haematopoietic stem cell transplantation HSCT has been anticipated, for more than twenty years, as a credible means of treating this devastating illness.
Pathological changes, particularly at more advanced stages of the disease, also correlate progressively with devastating neurological disability including movement, co-ordination, cognitive impairment and memory loss. Bakhurayasah et al  give an excellent overview of the current clinical therapeutic usefulness of this approach for MS, as well as some drawbacks.
An intense pre-conditioning regimen does increase the risk of treatment-related infection, could exert a toxic effect on the CNS, and increases the risk of mortality.
Moderating the conditioning may prove to be a less toxic outcome for MS patients treated with HSCT, but clearly there is a fine balance to be met, with a conditioning that supports maximal reconstitution and improvement in neurological and quality of life measures, without unnecessarily jeopardising health concerns and risks associated with toxicity.
Although no direct comparisons are available with standard therapy, none of the drugs that are currently approved or in late phase clinical trials has been reported to achieve a similarly profound control of the disease .
The busulfan-related death occurred early in the trial when only oral and not intravenous busulfan was available the latter enables control of the actual dose absorbed by the body much more precisely, thus reducing risks.
Clinical trials and analysis of a European registry data have shown a steady decrease over the years of treatment-related mortality, likely related to more appropriate patient selection criteria and increased use of low- and intermediate intensity chemotherapy schemes.
Treatment-related mortality has decreased from 7. It is not suitable for people with progressive forms of MS who have accumulated long-standing disability. For these people, the risks outweigh any potential benefit. For all these reasons I would reiterate that aHSCT should only be offered in clinical trials or otherwise rigorously selective clinical programmes in specialised environments, and that patients interested in AHSCT should seek expert advice rather than the un-selective access that we hear is provided, for a fee, at some overseas private facilities.
Nevertheless the clinical results are truly impressive, in some cases close to being curative, though we need longer-term follow-up to know for certain whether the patients continue to do well or if there is a chance of relapse.
And of course this trial will need replication by other groups too. Still I would consider it a breakthrough therapy and the clinical group and the patients should be congratulated for this success. Declared interests Dr Payam Rezaie: Research collaboration grant from Biogen.expert reaction to trial of stem cell transplantation (with chemotherapy) and multiple sclerosis A paper published in the Lancet journal has looked at treatment of multiple sclerosis and reports the efficacy of chemotherapy to ablate the immune system followed by a stem cell transplant.
Jan 04, · Discussion of the bioethics of human stem cell research has transitioned from controversies over the source of human embryonic stem cells to concerns about the ethical use of stem cells in basic and clinical research.
7 days ago · Stem cell: An unspecialized cell that can self-replicate, and transform itself into a specialized cell, like a heart cell or skin cell. Pluripotency: The ability of a cell to become any other cell . March 19, expert reaction to using stem cells to treat macular degeneration.
A study published in Nature Biotechnology describes a phase 1 clinical trial of an embryonic stem cell-derived retinal pigment epithelium patch for age-related macular degeneration..
Dr Carmel Toomes, Associate Professor, Leeds Institutes of Molecular Medicine, said. Stem Cell Therapy Risks And Benefits Outlined In Journal Report: Shots - Health News Three patients were blinded after getting stem cells from fat at a Florida clinic.
But a research study showed. Stem Cell Metabolism. Alessandro Prigione, Heather Christofk, and Michael Teitell discuss the metabolic regulation of pluripotency and reprogramming.