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  1. #1

    Stem Cell Therapy In Muscular Dystrophy

    HELLO FRIENDS,
    i'm dr rajeev goyal from india.i'm doing md pathology.suffering from musculardystrophy.there is new hope for the treatment.stem cell therapy is going on for the musculardystrophy in india.i'm going to take the treatment from dr geeta shroff.if i am able to get even one % recovery it will be a miracle for me.pls friends give me ur suggestions.give me best of luck.
    DR RAJEEV GOYAL

  2. #2
    Quote Originally Posted by drrajeevgoyal
    HELLO FRIENDS,
    i'm dr rajeev goyal from india.i'm doing md pathology.suffering from musculardystrophy.there is new hope for the treatment.stem cell therapy is going on for the musculardystrophy in india.i'm going to take the treatment from dr geeta shroff.if i am able to get even one % recovery it will be a miracle for me.pls friends give me ur suggestions.give me best of luck.
    DR RAJEEV GOYAL
    Thanks for posting. Please let us know how it goes. Wise.

  3. #3
    Senior Member
    Join Date
    May 2005
    Location
    Pakistan
    Posts
    1,168
    My Dear Friend Rajeev

    Hope you will be fine and sorry for delay.

    You have not to give up hope at any stage and Dr. Young will give you the best advise which we can not give and also believe we should not advise you for treatments which are at trial basis yet.

    So live happy and be optimistic. God will help my friend.

    Sincerely with love

    Jawaid

  4. #4
    Rajeev
    Wish you good luck & a lot of recovery.

  5. #5
    yes keep us posted, i think muscular dystorophy is similiar to denerved /flaccid muscle
    cauda equina

  6. #6
    hello rajiv wish you best of luck . i am also suffering from muscular dystrophy pl send email address of dr. geeta shroff so that i can also get treatment . thanks

  7. #7

    dr geeta email

    here is the email add of dr geeta geetashroff@hotmail.com

  8. #8
    Mannu did you also contacted Dr. Geeta Shroff... What happened?? write few lines of your experience... I too am desperate to try any clinical trials with stem cells, after understanding the concept.

    Hemant
    P.S. Follow my post for Dr. Rajeev

  9. #9

    Dear Dr. Wise, your guidance required

    I am Hemant, from India, suffering from Muscular dystrophy (LGMD-2B) and I really enjoy being on this forum and keep myself updated by reading various discussions especially the stem cell related articles…

    I have these 2 articles and would like to sort your opinion on the following doubts…

    Therapy of Duchenne muscular dystrophy with umbilical cord blood stem cell transplantation
    http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=AbstractPlu s&list_uids=16086277&query_hl=3&itool=pubmed_DocSu m

    Allogeneic umbilical cord blood stem cell transplantation in Duchenne muscular dystrophy
    http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=AbstractPlu s&list_uids=15949330&query_hl=8&itool=pubmed_docsu m

    If supposing these are considered for clinical trials to treat MD then
    (1) What are the chances of improvement as per your view point ??
    (2) What are the risk ?? How can it be minimized ??
    (3) Considering both theoretical and practical aspect of the article do you find any thing that create skepticism or doubt in your knowledge… and would it “work” as suggested in the article

    Please guide me…. I know that these articles are from china and more over the case discuss are “isolated” one but this is one part of truth but other than that can these be taken seriously for further study using clinical trials… and your whole view on them. I am asking your goodself because one of my doctors got interested and wants to collect few experts’ opinions on this before taking a step forward.

    Sir, I am doing all that is possible to help and gather information from my side. I have seen many of your post being explained with simplicity to understand for all those who read them…I hope you shall put your honest opinion to guide me. .

    With regards
    hb179
    hemant_ngp@sancharnet.in

  10. #10
    Quote Originally Posted by hb179
    I am Hemant, from India, suffering from Muscular dystrophy (LGMD-2B) and I really enjoy being on this forum and keep myself updated by reading various discussions especially the stem cell related articles…

    I have these 2 articles and would like to sort your opinion on the following doubts…

    Therapy of Duchenne muscular dystrophy with umbilical cord blood stem cell transplantation
    http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=AbstractPlu s&list_uids=16086277&query_hl=3&itool=pubmed_DocSu m

    Allogeneic umbilical cord blood stem cell transplantation in Duchenne muscular dystrophy
    http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=AbstractPlu s&list_uids=15949330&query_hl=8&itool=pubmed_docsu m

    If supposing these are considered for clinical trials to treat MD then
    (1) What are the chances of improvement as per your view point ??
    (2) What are the risk ?? How can it be minimized ??
    (3) Considering both theoretical and practical aspect of the article do you find any thing that create skepticism or doubt in your knowledge… and would it “work” as suggested in the article

    Please guide me…. I know that these articles are from china and more over the case discuss are “isolated” one but this is one part of truth but other than that can these be taken seriously for further study using clinical trials… and your whole view on them. I am asking your goodself because one of my doctors got interested and wants to collect few experts’ opinions on this before taking a step forward.

    Sir, I am doing all that is possible to help and gather information from my side. I have seen many of your post being explained with simplicity to understand for all those who read them…I hope you shall put your honest opinion to guide me. .

    With regards
    hb179
    hemant_ngp@sancharnet.in
    Hemant, yes, these studies from China are quite exciting but I should point out caveats and cautions before undergoint these therapies. First, as described by Zhang, et al., (2005), the procedure requires chemotherapy (busulfan, cyclophosphamide, rabbit anti-thymocyte antibody) to destroy the bone marrow of the recipient (so that the umbilical cord blood cells will engraft). The patients received immunosuppression therapy (cycloposporin, methylprednisolone, and MMP) after the cord blood transfusion to prevent graft-versus-host reactions (which is less serious with umbilical cord blood transplants but still a real problem if and when it occurs). These are not trivial procedures. Until the cord blood cells engraft, the patient is vulnerable to infectious diseases.

    They also treated an 11-year old boy. A single unit of umbilical cord blood may not provide sufficient cells to ensure rapid engraftment for an adult. In some centers, when they use cord blood transfusions for adults, they give more than one unit.

    Finally, the cord blood cells need to be carefully matched for HLA. These are the antigens that lead to immune-rejection of transplanted cells. Xiao, et al. (2004) had earlier reported the feasibility of obtaining 6/6 HLA matches and found such units for 25% of patients. Note that HLA matching depends on the race of the individual and that the best matches are found between people of the same races.

    Wise.

    • Zhang C, Chen W, Xiao LL, Tan EX, Luo SK, Zheng D, Ye X, Li Z, Lu XL and Liu Y (2005). [Allogeneic umbilical cord blood stem cell transplantation in Duchenne muscular dystrophy]. Zhonghua Yi Xue Za Zhi 85: 522-5. OBJECTIVE: To study the feasibility of treatment of Duchenne muscular dystrophy (DMD) with umbilical cord stem cell transplantation. METHODS: HLA matching was conducted for a 11-year-old DMD boy with family history was underwent umbilical cord blood stem cell transplantation and a sample of umbilical cord stem cells with 5 matched HLA sites was found in the cord blood bank with 27.32 x 10(8) nucleated cells, about 2.6 times that of the treatment dosage for him. After pretreatment with busulfan 14 mg/kg.d, cyclophosphamide 50 mg/kg.d, and rabbit anti-human thymocyte globulin 10 mg/kg.d, the allogeneic cord blood stem cells were transplanted intravenously. Cyclosporin A, methylprednisolone and MMF were used after the transplantation so as to prevent graft versus host reaction. Prostaglandin E1 was used to prevent Budd-Chiari syndrome, and ganciclovir was used to prevent cytomegalovirus infection. At the same time, Gran, granulocytic cell stimulating factor, and gammaglobulin were also used. Biochemistry test, including serum creatine kinase (CK), was conducted. Evidence of reconstruction of blood making, including conversion of blood type, was observed. PCR-STR analysis was used to observe the status of implantation of the donor umbilical cord blood stem cells. RESULTS: (1) 12 days after transplantation, the white blood cells (WBC) of peripheral blood were 0.5 x 10(9)/L, 14 days after, the numbers of WBC and neutrophils were 1.0 x 10(9)/L and 0.6 x 10(9)/L respectively. In 37 days, granulocytic cell stimulating factor was no more used, the peripheral blood WBC fluctuated around 3.34 approximately 12.2 x 10(9)/L. In the 27th day, the number of blood platelets was more than 20 x 10(9)/L and hemoglobin rose to 88 g/L. On the 24th day red blood cells transfusion was stopped. (2) In the 42nd day, the blood type of the patient transformed from type A before transplantation to type AB (the blood type of transplanted stem cells is type B). (3) PCR-STR test of the peripheral blood made 17, 26, and 42 days after transplantation showed that the gene type of the patient was mixed mosaic: The ratio of donor gradually increased from 40% approximately 45% to 55% approximately 65%. (4) In the 38th day I degrees GVHD appeared. (5) serum CK level declined from 6000 U/L to 600 approximately 2200 U/L. (6) In the 42nd day, physical examination revealed obviously improvement in walking, turning the body over, and standing up. CONCLUSION: This is first case of prospective clinical transplantation on DMD by allogeneic cord blood stem cell. Umbilical cord stem cell transplantation helps re-build blood-making function, and improve locomotive function with a mild GVHD reaction. The genotype of rebuilt blood is mosaic but the ratio of gene mosaic gradually turn from recipient gene type > donor gene type to recipient gene type < donor gene type. The serum CK level decreases significantly after transplantation, which may slow down the necrosis of muscle cell. DMD patient will be benefited by stem cell transplantation. Department of Neurology, First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, China. http://www.ncbi.nlm.nih.gov/entrez/q..._uids=15949330
    • Xiao LL, Chen W, Zhang C, Liu ZL, Ye X, Zhang WD and Yi Y (2004). [A probability analysis for HLA matching in adult stem cell transplantation treating nervous genetic diseases]. Zhongguo Shi Yan Xue Ye Xue Za Zhi 12: 845-8. The aim of this study was to investigate the clinical feasibility of adult stem cell transplantation for lethal mono-gene inherited disease, Duchenne muscular dystrophy (DMD). A total of 30 blood samples from DMD patients were genotyped with HLA-A,-B and -DR alleles by means of polymerase chain reaction-reverse sequence specific oligonucleotide (PCR-RSSO). The HLA gene types in 30 DMD patients were compared with those of 668 unrelated donors from Umbilical Cord Blood Center of Guangdong Province and 34 910 unrelated donors from Chinese Bone Marrow Bank. The results showed that HLA gene of the DMD group was inherited in normal distribution. There was no striking difference of HLA-A, -B and -DR alleles expression between the DMD patients group and control healthy group. 25% of the DMD patients got suitable donors for stem cell transplantation, in which 15 patients found donors with >or= 5/6 HLA match at the Umbilical Cord Blood Center of Guangdong Province, i.e. occupying 50% of the total. Eight patients got 6/6 HLA matching donors at the Chinese Bone Marrow Bank, i.e. occupying 26% of the total. It is concluded that stem cells transplantation therapy for DMD patients is feasible, which will benefit these patients suffered from the lethal neuromuscular disease, and create a new way to treat this tough nervous system disease. Guangzhou Blood Center, Guangzhou, 510095 China. lulux-640@hotmail.com http://www.ncbi.nlm.nih.gov/entrez/q..._uids=15631675

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