xcell - stemcell treatment for dmd

[koksal]

Dear David,

I visited the www.xcell-center.com They argue that treat DMD with a stemcells method. is it possible now?

I appreciate if you search.

Sincerely.

Koksal Durmus

[David Feder]

Koksal: I think that is possible to treat muscular dystrophy now with stem cells but we need to know 3 informations:
1) Wich source of this cells; in muscular dystrophy it's impossible to use the own cells; only is possible if we treat this cells with gene therapy.
2) I think that is necessary more than one application.
3) It's necessary use of immunossupresive drugs?
If they can give more informations we can discuss more about this therapy,
Best reguards,
David

[yuri]

David,

Do you really think that there is stem cells therapy effective today?

Do you know any case of effective cell therapy?
There are a lot of clinics offering this therapies all around the world. Many of them are just business, I think.

[David Feder]

Yes Yuri, you are right. The clinics that offer stem cell treatment currently charging for therapy does not deserve the slightest confidence. The only interest is financial. But recently I talked to a researcher who studies adult stem cells obtained from dental pulp. The information I got left me optimistic about these cells. I do not see why not now start clinical studies. We do not have time to wait. An ethical protocol with good medical care can provide valuable information on the use of stem cells. She has treated dogs, cats and horses with different pathologies with stem cells with good results as we don't see any advance in DMD therapy.

[hb]

Hi David,
Its long while I haven't posted but I track all comments very closely.
I want to have your suggestion on the following treatment for md.

HLA matched Umbilical cord Mesenchymal SC to be given in IV?


I have tried unmatched M-SC (BM in 2006 and also Adipose in 2008) both IV... The results were very encouraging for a period of 6 months with stamina and good energy levels but no functional improvement. My CK levels came of normal for 4-6 months but again climbed back to higher levels (as before the treatment) after 6 months past. No immeno-suppressor was used in both cases. Anyways.

I would like to know from you or any scientist (special mention Dr M. Zats) that what can be expected with HLA matched MSC?
I understand and know all about risk but I want to know how promising can be the outcome.

Pls let me know ur comments.
Regards
hb

[David Feder]

Hi HB:
In my opinion, stem cell therapy should be repeated monthly for the first six months and every 3 months; the use of adult mesenchymal stem cells avoids the need to use cell HLA compatible.
David

[David Feder]

Liao, Yu
Department of Neurology, Wuxi Second Hospital, Nanjing Medical University, Wuxi 214002, Jiangsu Province, China
Liao Yu, Master, Chief physician, Department of Neurology, Wuxi Second Hospital, Nanjing Medical University, Wuxi 214002, Jiangsu Province, China [e-mail]
Liao Y.Allogeneic cord blood mesenchymal stem cell transplantation for treating Emery-Dreifus muscular dystrophy in one case. Zhongguo Zuzhi Gongcheng Yanjiu yu Linchuang Kangfu 2009;13(27): 5393-5396(China)
Title Allogeneic cord blood mesenchymal stem cell transplantation for treating Emery-Dreifus muscular dystrophy in one case[star operator].[Article]

Source Journal of Clinical Rehabilitation Tissue Engineering Research. 13(27):5393-5396, July 2, 2009.

A male patient, aged 32 years, weighing 38 kg, was recruited at the Department of Neurology, Wuxi Second Hospital, Nanjing Medical University in February 2008. The patient had general fatigue and amyotrophy for 18 years, and recruited in the hospital 3 days following burst dizziness and vomiting. Following genetic analysis and muscle biopsy, the patient was diagnosed as having Emery-Dreifus muscular dystrophy and cerebellar infarction. HLA compatible cord blood mesenchymal stem cell (CB-MSC) transplantation was performed. The grafted CB-MSCs were from Shanghai Cord Blood Bank. The donor was HIA all-identical, Rh blood type-identical, ABO blood type-incompatible. The patient received preconditioning of busulfan + cyclophosphamide + rabbit antithymocyte globulin. The patient then received peripheral venous administration of 40 mL CB-MSCs, 10 drops/minute, with total nucleated cell number of 31.98x108. 5 weeks after transplantation, symptoms of dizziness, swallowing difficulty and regurgitation of fluid were markedly alleviated. His weight increased by 4 kg and he recovered of hand coordination to handling computer operation. 6 months following transplantation, serum creatine phosphokinase decreased from 35.79 [mu]kat/L before transplantation to 9.55 [mu]kat/L; serum creatine phosphokinase isoenzyme reduced from 18.20 [mu]kat/L before transplantation to 4.78 [mu]kat/L. Myoglobin decreased from 413.50 [mu]g/L to 213.20 [mu]g/L. Functional independence measure scores increased from 71 points to 101 points. Immunological rejection or tumorigenesis was not identified. CB-MSC transplantation can evidently decrease serum creatine phosphokinase level within a short period, and improve motor function in Emery-Dreifus muscular dystrophy patient. CB-MSC transplantation is beneficial for Emery-Dreifus muscular dystrophy, and short-term safety is reliable.

[David Feder]

Xu YF, Yang XF, Wu YX, Wang HM, Zhang YB, Lu NW, Shan H, Cui JP, Zhou JX, Yin FH.Transplantation of autologous bone marrow stem cells for limb-girdle muscular dystrophies in 33 cases.Zhongguo Zuzhi Gongcheng Yanjiu yu Linchuang Kangfu. 2009;13(32):6345-6348. [ www.crter.cn http://en.zglckf.com]

Journal of Clinical Rehabilitation Tissue Engineering Research. 13(32):6345-6348, August 6, 2009.

Following the agreement of Ethics Committee of the 463 Hospital of Chinese PLA, a total of 33 limb-girdle muscular dystrophy (LGMD) patients, including 27 males and 6 females, aged 6 to 46 years with the course of disease of 2 to 20 years were enrolled from the Department of Hematology and Endocrinology of the 463 Hospital of Chinese PLA from December 2007 to June 2008. Granulocyte-colony stimulating factor (G-CSF) was infused into patients, and bone marrow was collected following 4 days of mobilization. Bone marrow mononuclear cells were isolated and cultured for 7-10 days by Percoll gradient centrifugation. Cell suspension (2.48x1010/L) was prepared. Stem cells were transplanted into all over the body by intravenous infusion and extremities by intramuscular injection. The amount of mononuclear cells was (6.13+/-2.39)x108 and the amount of mesenchymal cells was (1.92+/-0.98)x108. Six months after transplantation, increased rates of bare-handed muscle force and activities of daily living (ADL) were respectively 54.5% and 81.8%. The decreased rates of serum creatine kinase and lactic acid dehydrogenase were respectively 78.8% and 81.8%. The increased rate of creatinine was 72.7%. It was indicated that transplantation of autologous bone marrow stem cells for LGMD can enhance the bare-handed muscle force and activities of daily living and improve values of serum creatine kinase, lactic acid dehydrogenase and creatinine.

[hb]

Thanks for the journal articles and your reply.
It is correct that MSC's do not require HLA compatibility but I think, we (MD people) need HLA match MSC because the progene of MSC (when MSC gets converted into Muscle cells) are detected by the immune system due to donor HLA incompatibility and are then destroyed.

hb

[David Feder]

Hb: stem cells will synthesize the protein that is lacking in the muscle and the immune system may consider this protein as foreign. Maybe is necessary immunossupressive drugs.

[hb]

Hi David,
As long as MSC’s are present at the muscle site, the immune cells are switched off and the protein needed by muscle can be synthesized and sustain. If immuno-supressor is given it may again prolong and sustain the synthesis process till immuno-supressor drug works… but then we come back to square-one, were we started, as has been my experience.

I know it is difficult to get HLA cord blood MSC, but once this problem is over and above then possible cure or improvement for a longer time frame is possible. Because the protein will not be treated as foreign cell by the body. Secondly it is completely risk free. Only point of discussion is whether it should be taken IV or IM ? Your suggestion about the whole matter and is it a good hope for improvement.

Thanks for your advice and reply. It has been very helpful in understanding the core issue.
With Regards
HB

[David Feder]

Hb: I believe that IV is the most viable to treat all muscles.

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