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Thread: Stem Cell Therapy In Muscular Dystrophy

  1. #61
    Dear Dr. Wise

    Please reply to the protocol for DMD posted by SRK1974 in january 2009 about stemcell in india.

    we are very eagerly awaiting your valuable suggestion.

    Thanks,

    Rajesh

  2. #62
    Quote Originally Posted by srk1974 View Post
    Dear Dr Wise Young

    Team of doctors in India has worked out this protocol for DMD . Your comments on this pls . First is it safe . Positive effects may not be there

    1) DMD boy will be given cyclosplorin before the treatment for around 15 days. Dosage will depend upon the age , size weight etc of the host . This course of 15 days can be done by the boy at home . No need for hospitalization or staying in the sterile room in isolation

    2) After immune system is supressed by cyclsploring dosage for 15 days , 3/6 , 4/6 or 5/6 HLA matched allogenic umblicle cord blood stem cells will be injected in the host through IV . Around 2 to 3 units of blood will be transfused . Whole blood ( all stem cells like mesenchymal , hamatopoeitic etc ) without any culturing in the lab or adding any growth factors will be injected in the blood stream of the host .. They will try to get the best nearest match

    3) Transfusion will take around 1 or 2 days of hospitalization .

    4) Before the treatment MRI of the muscles will be done . 3 months after the transfusion again MRI of the muscles will be done to compare the muscle structure and strength .

    5) According to the doctors the new (donor's ) Umblical cord stem cells will infuse with the damaged muscle cells and repair them . They will rush to the site of damage and repair the muscle fibres . . They may also infuse among them selves and create a cell pool , which will be used as a reservoir for future . So hence forth whenever muscle cells of the host will dye because of the defective gene present in them or because of the normal day to day activity , the donor's stem cells from this new pool will shoot out and convert itself into new muscle cell with correct gene in it , which will be producing dystrophine

    6) However it is not known at this point of time how long this pool of new stem cells will last . . Once this pool gets exhausted stem cells from the bone marrow will come into action . However in DMD host it wont be of any use . So recurring transfusions might be required . However the frequency of the transfusion is not known at this point . It may be once in 12 months .So it is beneficial to have maximum number of cells to be infused to create pool so it lasts longer and frequency of the transfusion is less and at greater time intervals .

    7) It is hopeful that maximum number of these new stem cells will infuse and create cell pool till the immune system is kept low . Once the immune system switches on, the remaining cells which could not infuse or could not be be a part of the new cell pool will be killed by the host's immune cells .

    Your valuable comments on this protocol will be highly appreciated

    Warm regards

    Sagar
    Sagar,

    I am sorry that I did not answer your post earlier. In part, it is because this is not my field of expertise and also because I am not sure that you have all the details of the protocol. For what it is worth, I don't think that this protocol will be effective nor would allow engraftment of cord blood cells to treat Duchenne's Muscular Dystrophy.

    At the present, the process of umbilical cord blood transplant requires myeloablation. This is usually a chemotherapeutic or radiotherapeutic process that is designed to damage or eliminate bone marrow cells so that the infused umbilical cord blood cells will replace them.

    When cord blood is being used to treat leukemia, the chemo- and radiation therapy aimed at the leukemia itself is myeloablative. For the treatment of genetic disease, it is probably sufficient to do what they call a mini-myeloablation, which uses a short course of chemotherapy, anti-T-cell antibodies.

    Cyclosporin and steroids are usually used only to treat graft-versus-host-disease (GVHD). The fact that the doctors are using cyclosporin as a primary treatment, presumably to prevent immune-rejection of the cells, and apparently not using myeloblation and chemotherapy (i.e. cyclophosphamide), I get the impression that they are not experienced.

    I don't think that it is appropriate to judge a protocol that comes in this fashion, over internet and probably incomplete. There may be rationale for this protocol that I am not aware of. Is there any data that these doctors have to show the engraftment occurs with such a protocol?

    Wise.

  3. #63

    Update on My Son and Dr. K.

    Dr. Young - Hope you have been well...Please see the email I sent to you on 6/22/09 and respond when you have a second if you don't mind. I think you will find it interesting. Thanks! SG (YDXYRBC)

  4. #64

    Reqeust for Clinnical trails information at Apollo hospital India

    Dear Dr. Wise,

    Please kindly help us to get the clincial trail information at Apollo hospital. I have tried to contact through the email and phone numbers. Nobody dont know the information. So, we are requesting you who is the right person to contact in Apollo for stem cell clinical trails for MD. We ready to participate this clincial trails.

    Thanks,
    Guddati

  5. #65
    I found something in this web http://www.nrrfr.com/bbs_English_nr....e=&classid=566

    China - To go or not to go?

    "I have Becker Muscular Dystrophy. After I started reading about stem cells treatment in China, I heard many doctors in my country trying to persuade me not to go.

    ........

    And now I know that it's possible to get better. It's not easy, it's not fast, but it's POSSIBLE."

    It is not a cure but one more choice.

  6. #66

    Mesenchymal Stem Cells for MD

    Dear Dr Wise,

    I (29 Yrs, M) am from India and was diagnosed with LGMD 2B (Dysferlinopathy) at the age of 24 yrs. One hospital in India is providing a stem cell therapy for muscular dystrophy, which according to them can provide some improvement. Please check the following details of the treatment being offerred by them and give your valued opinion.

    1. Mesenchymal Stem Cells separated from Umbilical Cord Blood are used. No growth factor etc is added to the stem cells.

    2. HLA matching is not done. According to them, mesenchymal stem cells do not express HLA Antigens and themselves suppress the immune system. So they are never rejected by the body and don't need HLA matching. HLA matching is nneded when Hematopoietic Stem Cells are used.

    3. Mesenchymal Stem Cells injected into the femoral artery of the lower limbs.

    4. In total 5 shots of stem cells. There will be a gap of 6 weeks between two shots. One million cells per KG of body weight will be injected each time.

    5. As per the hospital, some muscle dystrophy patients have shown some improvement after this treatment. But even then they do not guarantee any benefits. But are sure that this therapy does not have any side-effects and is worth trying.

    6. Mesenchymal Stem cells are not engrafted into the bone marrow. They will be injected into femoral artery of lower limbs. It is expected that cells will be absorbed into the muscle areas and help in repairing them. Now how long these stem cells will keep helping muscle repair is not known. Whether there will be need of repeating the treatment is also not known.

    Please let me know, whether I should go for the above mentioned treatment.
    Can this treatment provide any benefits? Is it worth even trying?
    And are there any negative effects of this treatment?

    Thanks in advance for any inputs you can provide on this.

  7. #67
    Dear Dr. Wise Young,

    Subject: Clinical Trials in Apollo Hospital,Ahmedabad, (Gujarat, India) for Muscular
    Dystrophy

    There was an article in the Times of India(Ahmedabad edition), dated 7th Aug, 2009, in which it was stated that Apollo Hospital Ahmedabad will start research in stem cell
    therapy for spinal cord injuries and muscular dystrophy. Stemcyte and Cadila
    pharmaceuticals will also be involved.

    You were quoted in the article as saying that the clinical trials will begin soon for
    spinal cord injuries. We have a lot of hopes regarding this development, and are very
    happy that you are involved in the research.

    My 2.5 yr old nephew Khush suffers from DMD.

    I have the following questions
    a. When will the clinical trials for DMD begin?
    b. What is the procedure for participating in the clinical trials?
    c. What procedure will be performed on DMD patients? [that is, a procedure similar to that performed by Dr Zhang Chen, or perhaps, mini-myloablation will be performed]
    d. Who is the contact person at Apollo hospital , Ahmedabad regarding this matter?

    We will be highly obliged if you could provide this information.
    Thanking you,
    Sincerely
    Sagar R. Kapadia,
    Surat, Gujarat
    India

  8. #68
    Quote Originally Posted by srk1974 View Post
    Dear Dr. Wise Young,

    Subject: Clinical Trials in Apollo Hospital,Ahmedabad, (Gujarat, India) for Muscular
    Dystrophy

    There was an article in the Times of India(Ahmedabad edition), dated 7th Aug, 2009, in which it was stated that Apollo Hospital Ahmedabad will start research in stem cell
    therapy for spinal cord injuries and muscular dystrophy. Stemcyte and Cadila
    pharmaceuticals will also be involved.

    You were quoted in the article as saying that the clinical trials will begin soon for
    spinal cord injuries. We have a lot of hopes regarding this development, and are very
    happy that you are involved in the research.

    My 2.5 yr old nephew Khush suffers from DMD.

    I have the following questions
    a. When will the clinical trials for DMD begin?
    b. What is the procedure for participating in the clinical trials?
    c. What procedure will be performed on DMD patients? [that is, a procedure similar to that performed by Dr Zhang Chen, or perhaps, mini-myloablation will be performed]
    d. Who is the contact person at Apollo hospital , Ahmedabad regarding this matter?

    We will be highly obliged if you could provide this information.
    Thanking you,
    Sincerely
    Sagar R. Kapadia,
    Surat, Gujarat
    India

    Sagar,

    I am not sure when they will start. When I was last in Delhi, the doctors at Apollo Hospital were really quite enthusiastic about starting umbilical cord blood cell transplants (with mini-myeloablation) in some children with Duchenne's Muscular Dystrophy. They are already doing umbilical cord blood cell transplants for other conditions (such as post-chemotherapy for leukemia). So, it doesn't seem to be too big a jump for them to start doing some cases for DMD. I think that the initial trials will probably be done at Delhi or Hyderabad rather than Amedhabad.

    Wise.

  9. #69
    Quote Originally Posted by va80 View Post
    Dear Dr Wise,

    I (29 Yrs, M) am from India and was diagnosed with LGMD 2B (Dysferlinopathy) at the age of 24 yrs. One hospital in India is providing a stem cell therapy for muscular dystrophy, which according to them can provide some improvement. Please check the following details of the treatment being offerred by them and give your valued opinion.

    1. Mesenchymal Stem Cells separated from Umbilical Cord Blood are used. No growth factor etc is added to the stem cells.

    2. HLA matching is not done. According to them, mesenchymal stem cells do not express HLA Antigens and themselves suppress the immune system. So they are never rejected by the body and don't need HLA matching. HLA matching is nneded when Hematopoietic Stem Cells are used.

    3. Mesenchymal Stem Cells injected into the femoral artery of the lower limbs.

    4. In total 5 shots of stem cells. There will be a gap of 6 weeks between two shots. One million cells per KG of body weight will be injected each time.

    5. As per the hospital, some muscle dystrophy patients have shown some improvement after this treatment. But even then they do not guarantee any benefits. But are sure that this therapy does not have any side-effects and is worth trying.

    6. Mesenchymal Stem cells are not engrafted into the bone marrow. They will be injected into femoral artery of lower limbs. It is expected that cells will be absorbed into the muscle areas and help in repairing them. Now how long these stem cells will keep helping muscle repair is not known. Whether there will be need of repeating the treatment is also not known.

    Please let me know, whether I should go for the above mentioned treatment.
    Can this treatment provide any benefits? Is it worth even trying?
    And are there any negative effects of this treatment?

    Thanks in advance for any inputs you can provide on this.
    Va80,

    What you describe seems reasonable except the decision not to use HLA-matched cells. It is well known that non-HLA-matched umbilical cord blood cells are rejected and that the likelihood of engraftment is much higher when the cells are matched. It is true that mesenchymal stem cells have anti-immune properties but it is not clear that this is true of the progeny of mesenchymal stem cells.

    Umbilical cord blood itself have relatively few mesenchymal stem cells (probably much less than 1% of the mononuclear cells). In order for the mesenchymal stem cells to contribute to muscle and increase the amount of normal dystrophin in muscle (Source), the mesenchymal stem cells must make myoblasts and these myoblasts need to incorporate themselves into the muscle. It is not clear that myoblasts will be immune-privileged or anti-immune like mesenchymal stem cells appear to be.

    So, while the idea of treating muscular dystrophy with mesenchymal stem cells is a good one, I believe that it makes far more sense to engraft umbilical cord blood cells in the bone marrow and for these cells in the bone marrow to constantly shower the body and the muscles with myoblasts that make dystrophin in muscle.

    If these investigators have done the study in patients, have they measured dystrophin levels in the muscles of the patients that they have treated? What are the results?

    Wise.
    Last edited by Wise Young; 09-03-2009 at 08:06 AM. Reason: Edited some mispellings (in bold)

  10. #70
    Thanks Dr Wise for your inputs. I will discuss the points raised by you with the doctors.
    Thanks.

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