SCT Futuristic Science or down home bone marrow transplant?

    • Anonymous
      September 3, 2011 at 8:36 pm

      Lately I’ve found myself wondering why I thought a Stem Cell Transplant (SCT) was some new-fangled science. Well, one program discussed on this site is on a Phase 1 Clinical Trial.

      Doesn’t that qualify for Science Fiction or something?

      Doesn’t that automatically make it new-fangled?

      No. it would seem it does not. I asked ‘around’ about this and reached an understanding for myself that a SCT is merely a type of bone marrow transplant (BMT).

      Bone marrow transplant? I’ve heard of those. My neighbor, who has cancer, is going to get one. The guy who sits next to me on IVIG treatment day at the Cancer Center had one last month.

      In fact, I’d wager that thousands of bone marrow transplants are done every year.

      So, then, what is different about a SCT that seperates this procedure from a BMT?

      Consider this word- myeloablative.

      “myeloablation /my·elo·ab·la·tion/ (mi″ĕ-lo-ab-la´shun) the severe or complete depletion of bone marrow cells; as the administration of high doses of chemotherapy or radiation therapy prior to bone marrow transplantation”

      A SCT done at the program we’ve read about on this site is NON-myeloabitive. NON means no, not, uh-uhhhh.

      Therefore it is my understanding that a SCT for CIDP uses a less aggressive chemo treatment than a standard bome marrow transplant.

    • Anonymous
      September 3, 2011 at 9:18 pm

      As I understand it, the Stem Cell transplant works this way: Stem cells are harvested from your blood and prepared for retransplantation. You then get chemo to destroy your immune cells. The stem cells are re-introduced to “grow” a new immune system.

      With different cancers they kill different things with chemo. My brother had Multiple Myeloma and they killed off the part that makes platelets and then used stem cell transplantation to “grow” new ones. In our case the immune system is faulty and needs replaced.

      The technology is similar, I guess it is in “trials” because of the newness of the application. I cannot wait until it becomes more readily available so I can get rid of this CIDP and start over.

    • Anonymous
      September 4, 2011 at 2:17 am

      The Wikipedia article on BMT appears to be very good, if necessarily basic, It is a good place to start for those who want more information.

      In the past, an autologous BMT was used if the disease afflicting the bone marrow was in remission or if the condition being treated did not involve bone marrow. The bone marrow could be treated to remove malignant cells. For cancer, it is clear that the idea is to kill off the malignant cells produced by the bone marrow and then replace them with non-malignant cells.

      I think what is new are two things: as Yuehan points out, the SCT trial is non-myeloablative. The other is the hypothesis that the stem cells reconfigure in such a way as to create an immune system that does not lead to the autoimmune disease again. Per a [I]Science [/I](12 February 2010, p. 772) article, it is not clear how the stem cells do this.


    • Anonymous
      September 6, 2011 at 2:27 am

      I would love not to have infusions every week……

    • Anonymous
      September 6, 2011 at 12:19 pm

      I wish I didn’t need infusions every 10 days also. Don’t we all?
      When I asked my neurologist about SCT: she said “The benefits outweigh the risks”. This is her opinion. She specializes in neuromuscular conditions. She has never heard of Dr. Burt’s clinical study SCT in CIDP. I don’t know what to think. I wish I could eradicate the disease from my body.

    • September 6, 2011 at 1:52 pm

      I contacted the study and received an email back from someone there at the study. I asked if they would ever consider someone who has CIDP but is not at the end for choices with treatment. I am doing ok with my IVIg infusions and don’t have much in lasting damage to my nerves…yet. I wondered why they could not also study the effect on SCT on people like me who haven’t had the disease that long and don’t have long-lasting complications yet. Why not cure us before the disease takes hold of our bodies and causes irreversable damage. Wouldn’t it be cheaper for the insurance companies to pay the large fee once up front to cure us instead of paying $10,000 plus each month just to maintain us where we are at? But she said at this point they are only doing the SCT on people who have failed in all types of treatment and are pretty much at the end of options for treatment. Bummer. I would also love to stop this disease right now before I progress any further and start to live my life without IVIg treatment every 21 days. I hope they approve this as a treatment in the future for all CIDP sufferers.

    • September 6, 2011 at 8:31 pm

      thatg’s discouraging–i thought that we only had to fail 2 treatments to be considered. Hopefully with all these pioneers paving the way it will soon be a standard treatment for cidp. Then hopefully it would be able to be done locally for us instead of having to travel to Northwestern, it sounds like that is a wonderful place but it would be easier to do it closer to home/work, family etc.

    • September 7, 2011 at 8:15 am

      I think it is important to remember that this treatment is highly toxic, high risk in terms of potential complications and the chemotherapy is associated with secondary problems that may appear years down the road. It is also not known actual remission/ cure rate and how long remission may last. For all those who are wishing they would qualify for this study protocol I myself am thankful that I am not a candidate and have a reasonable quality of life on a much safer regimen. For those who have severe disability and life threatening CIDP they have my full support and prayers as they undergo this treatment, as I would if in that position . I think that this treatment will have a role in severe disease but the real future is a non toxic targeted therapy that just adresses the specific autoantibody without harming the rest of the immune system – as others have pointed out, bone marrow transplant is not new technology just its application in autoimmune disease is new. We need to continue to push for funding for safe, effective and non toxic treatments for CIDP and autoimmune disease for all levels of this disease.

    • September 7, 2011 at 8:42 am

      You are right shellbones. I guess I am grateful that I do have good quality of life on the IVIg. I was not aware that the chemo drugs actually had a risk of causing cancer down the way. I do not want that risk at this point since I am able to control my CIDP somewhat with IVIg. I guess I should count my blessings and enjoy each and every day that I am still mobile and able to get along without too much trouble. It just sounded so nice to not have to think about CIDP all the time and how to manage it with a busy life. But hopefully, like you said, they will come up with a better, less toxic way to treat CIDP in the NEAR future so that we all can benefit and get back to living. Best wishes to all who are going through the SCT. You all deserve to get better and back to a normal life 🙂

    • September 7, 2011 at 11:08 am

      I know it seems to be hard to be grateful to be on IVIG- I myself have been on IVIG for almost 3 years combined with low dose azothioprine ( also not risk free but have been able to keep the dose low) for almost one year now – I go weekly for IVIG and it has really stabilized me and I am able to work part time and enjoy generally good functioning , take care of kids etc- I will soon attempt to wean dose to every 10 days. I share the same love/hate relationship with ivig that many here do -thankful for getting me out of the hospital and walking but ungrateful for the burden of having to receive it . I am thrilled for the early initial results of those who received the stem cell transplant protocol with many reporting disease remission and improvement and I hope they continue to fare well !

    • Anonymous
      September 7, 2011 at 11:47 am

      Let’s don’t get hyper scared with terms like high risk, chemo, remission, etc.

      What are the fine print risks of whatever cocktail(s) of drugs being taken now?

      Regardless, no will likely be invited to participate in this trial until they have [I][B][COLOR=”Red”]”…CIDP that has not responded to the standard treatment methods…”[/COLOR][/B][/I]

      Decide for yourself if you know you have failed to respond. Decide for yourself if you are going to take Chemo and Immune Suppression, as some already do, forever…. and then willingly assume the long term risks of those treatments.

      What might those risks be? Cancer, toxic side effects and, yes, eventually and sadly, a failure to arrest the original condition which may result in inexorable decline.

      Life is a gamble. Live it to your fullest.

    • Anonymous
      September 7, 2011 at 8:06 pm

      Well, let’s not overlook that the absolute greatest distinction between a non-myeloablative SCT, like they are performing at Northwestern for autoimmune diseases, and a true bone marrow transplant (BMT) as performed on patients with diseases such as Acute Lymphocytic Leukemia is that a true bone marrow transplant involves the infusion of marrow cells harvested from a DONOR. Additionally, the donor must be a near-perfect match to the recipient, then, the recipient must take heavy immuno-suppressive/anti-transplant rejection drugs for life. The immuno-suppresive dosage these people take to combat organ transplant rejection is of a magnitude far greater than immuno-suppressive regimens prescribed for CIDP. The mortality rate for true bone marrow transplant due to rejection/complications is still fairly high, maybe 25% I believe.

      There are few similarities between SCT and BMT (bone marrow transplant), in reality. BMT is a major procedure, involving significant risk, utilized mainly for the most life-threatening of malignant blood disorders.

    • Anonymous
      September 7, 2011 at 9:15 pm

      [QUOTE=Billt]…absolute greatest distinction between a non-myeloablative SCT, like they are performing at Northwestern for autoimmune diseases, and a true bone marrow transplant (BMT) as performed on patients with diseases such as Acute Lymphocytic Leukemia is that a true bone marrow transplant involves the infusion of marrow cells harvested from a DONOR.[/QUOTE]

      I agree with what I take to be one of your premises which is that a myeloablative BMT has far greater risks than a non-myelobalative BMT/SCT.

      However, I do not agree with the non-referenced statement above.

      Nor do I agree that a SCT is any less a ‘true’ bone marrow transplant.

      Afterall, that was the point of this thead. The two are one and the same, in my opinion and according to some references.

      For example, a Wikipedia article already referred to us to read by another member says-

      “[I]…Of these, 28,901 (57%) were autologous and 21,516 (43%) were allogeneic (11,928 from family donors and 9,588 from unrelated donors).

      The main indications for transplant were lymphoproliferative disorders (54.5%) and leukemias (33.8%), and the majority took place in either Europe (48%) or the Americas (36%).[3]

      In 2009, according to the world marrow donor association, stem cell products provided for unrelated transplantation worldwide had increased to 15,399 (3,445 bone marrow donations, 8,162 peripheral blood stem cell donations, and 3,792 cord blood units).[4]…[/I]”

      So, in fact, according to this article, Autologous and periperhal blood stem cell transplants constitute the largest percentage of Bone Marrow transplants discussed in the article.

    • Anonymous
      September 8, 2011 at 1:38 am

      A clinical trial is not about curing or treating any particular patient, or even a group of patients. It is about determining if the proposed treatment is safe and effective. A clinical trial that shows that a proposed treatment is not safe or not effective is just as valid as a trial that shows the opposite. If you submit to a clinical trial, you are agreeing to be part of a science experiment, and if you will forgive for my being blunt, pretty much at the level of a lab rat.

      All science experiments must be designed before the experiment starts, so that the variables can be considered and controlled. Part of the reason that Dr. Burt should not alter the criteria for selection for any particular patient is that to do otherwise causes the loss of control over one or more of the variables. This loss could invalidate the trial.

      Clinical trials are set up at different phases. Dr. Burt’s CIDP trial is a Phase I/Phase II, meaning that the efficacy and toxicity are being determined on a small group of people. The proposed treatment is thought to be safe and effective, but it has not been proven so. Under the normal standards of medical ethics, it would typically be considered unethical to prescribe the test treatment if other treatments are effective. This is the other part of the reason Dr. Burt has the selection criteria; he can only include those who have effectively no other recourse.

      More of us could be included in a Phase III trial, but the Phase I/II trial must finish first and the results reviewed and approved. While we would all like to hurry the experiment along, you run the risk of ruining it if you do. Unfortunately for us, the experiment has a while to go. Dr. Burt’s study is not expected to finish before the end of 2014. The results then must be reviewed and approved. A Phase III trial will have to be organized. It would be unlikely to end before 2016 and given the risks of any bone marrow suppression, likely a year or two longer.


    • September 8, 2011 at 12:18 pm

      There is an excellent article written by Dr.Burt in 2004 which outlines rationale for SCT in autoinnune disease and compares myeloablative vs. non myeloablative. Also gives a summary of history of SCT in treating autoimmune disease- it is interesting to read to get the “lay of the land” so to speak. He also adresses briefly use of allogenic ( stem cells from donor ie non self) in autoimmune disease. The webadress is:

    • September 8, 2011 at 6:49 pm

      That is a great explanation Markens—really gave me second thoughts on the whole procedure. Is it the phase II trials that some people will receieve placebos?
      I guess with any new medical procedure, there needs to be trials and risks and people willing to go forth with it. One definitely needs to weigh the pros and cons of it.

    • Anonymous
      September 8, 2011 at 11:45 pm

      I am #21 in the clinical trials for CIDP. Dr. Burt’s used this procedure on other autoimmune conditions for much longer, so I feel very comfortable with his knowledge of how to rid our bodies of these diseases.

      Clinical Trial stage 3 is when the randomized control group is used. While I was in Chicago getting my sct, there were 8 others going through this at the same time. 4 with MS, 3 with CIDP, 1 with Lupus and 1 with Crohn’s. It was an amazing group and we remain in contact.

      MS is in stage 3 of clinical trials. One of the patients went through the control group last year and got chemo, but not stem cells. She was disappointed to be in the control group, but was told she could not apply again without going through the control process first. She did and she got worse and so was back for the real deal this time. She told me she did not regret it, as MS was taking her life away and this seemed like the only viable option for her.

      That is one reason why I wanted to do this now – before CIDP goes to stage 3 testing.

      It was the most amazing experience of my life! I trust Dr. Burt and I do believe he knows what he is doing. And, this way, I get to be forever 21!

    • Anonymous
      September 12, 2011 at 6:59 pm

      Linda M mentions several other protocols for Stem Cell Transplant (STC) in the previous post. I wanted to know more. If you do also, here ya’ go:

      Cyclophosphamide and rATG/Rituximab in Patients With Systemic Lupus Erythematosus

      webpage: [url][/url]

      Stem Cell Therapy for Patients With Multiple Sclerosis Failing Interferon A Randomized Study

      webpage: [url][/url]

      Phase I Study of High-Dose Cyclophosphamide and Total Body Irradiation With T Lymphocyte-Depleted Autologous Peripheral Blood Stem Cell or Bone Marrow Rescue in Patients With Multiple Sclerosis

      webpage: [url][/url]

      Immunological Mechanisms of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis

      webpage: [url][/url]

      Autologous Stem Cell Transplantation for Crohn’s Disease

      webpage: [url][/url]

      ASTIC Autologous Stem Cell Transplantation for Crohn’s Disease

      webpage: [url][/url]

      Crohn’s Disease Stem Cell Transplantation

      webpage: [url][/url]

      ok ok I get it, that’s enough. If you are interested check it out. It’s fair to say there are a “few” studies going on. Some are in Chicago, some are not. Some are actively recruiting, some are not.

    • Anonymous
      September 13, 2011 at 5:40 pm

      For the doubters or the skeptics or the curious or the medically challenged and yes, that still includes myself, there is this info from Mayo Clinic:

      [I]”…Although the procedure to replenish your body’s supply of healthy blood-forming cells [COLOR=”DarkGreen”]is generally called a stem cell transplant, it’s also known as a bone marrow transplant or an umbilical cord blood transplant[/COLOR], depending on the source of the stem cells. Stem cell transplants can use cells from your own body (autologous stem cell transplant), or they can use stem cells from donors (allogenic stem cell transplant)…”[/I]

      here- [url][/url]