Calling anyone who has had SC treatments

[JerryD]

So Dave and Craig,
What is the hold up for the rest of us to receive / be offered SCT ? What is the 'hold up' for this treatment to become the 'answer'? I am a patient man, but this is ridiculous !

[AMJ]

Jerry, I was part of a clinical trial. My EDSS score has remained the same. I feel much better, but there was no improvement in my walking. See below for details of the study

Immunomodulation
Thursday, October 11, 2012, 15:30 - 17:00
A phase 1b, randomised, placebo-controlled, multiple-dose study of human placenta-derived cells (PDA-001) for the treatment of adults with multiple sclerosis
F. Lublin, J. Bowen, J. Huddlestone, M. Kremenchutzky, A. Carpenter, J. Corboy, M. Freedman, L. Krupp, C. Paulo, R. Hariri, S. Fischkoff (New York, Seattle, Tacoma, US; London, CA; Minneapolis, Aurora, US; Ottawa, CA; Stony Brook, Warrren, US)

Introduction: Infusion of stem cells is a potential approach to the treatment of Multiple Sclerosis (MS). PDA-001 is a preparation of adherent cells derived from healthy, full-term human placental tissue that is expanded in vitro and administered to patients via intravenous infusion. A small number of studies have shown that treatment with certain agents may cause a paradoxical exacerbation of specific autoimmune diseases. This Phase 1 safety study was conducted to rule out this possibility, determine dosing and inform Phase 2 trials.

Methods: Sixteen patients with relapsing-remitting (RRMS) or secondary progressive MS (SPMS) were given 2 infusions of PDA-001 or placebo 1 week apart. Patients were monitored monthly for 6 months with cranial magnetic resonance imaging (MRI). The first 8 patients received either 2 infusions of 2x10^8 cells (Low Dose; 6 patients) or placebo (2 patients) and the next 8 received 2 infusions of 8x10^8 cells (High Dose; 6 patients) or placebo (2 patients). Paradoxical worsening was monitored with Cutter’s Rule (5 or more new gadolinium (Gd) lesions present on 2 consecutive monthly scans), frequency of MS relapse and change in MS brain lesions.

Results: The mean age of the patients was 48.1 years, mean time from diagnosis to study enrolment was 6.8 years; 10 had RRMS and 6 had SPMS. The mean baseline EDSS was 4.6 (range 4-5.5) in the Low Dose group and 5.1 (range 3.5-6) in the High Dose group. After 6 months of follow up, no patient met Cutter’s Rule.

One patient in the high dose group showed an increase in T2 and Gd lesions during an MS flare 5 months after receiving PDA001. Other than this patient, no patient had an increase of EDSS score of greater than 0.5 and most patients had either stable or decreasing EDSS. One patient experienced a Grade 1 anaphylactoid reaction and 1 experienced a Grade 2 superficial thrombophlebitis in the High Dose group. Other adverse events were mild to moderate in severity and included headache, fatigue, infusion site reactions and urinary tract infections.

Conclusion: PDA-001 infusions appear safe and well tolerated in patients with RRMS and SPMS. No paradoxical worsening of lesion counts was noted in either of the dose groups. A Phase 2 study in this patient population is planned.

Robert Hariri is a full time employee of Celgene Cellular Therapeutics and receives stock and stock options. Steven Fischkoff is a full time employee of Celgene Cellular Therapeutics and receives stock and stock options Corri Paulo is a full time employee of Celgene Cellular Therapeutics and receives stock and stock options. James Bowen received funding for clinical trial costs from Celgene Cellular Therapeutics. Adam Carpenter received funding for clinical trial costs from Celgene Cellular Therapeutics. John Corboy received funding for clinical trial costs and honoraria from Celgene Cellular Therapeutics.

Mark Freedman received funding for clinical trial costs and honoraria from Celgene Cellular Therapeutics. John Huddlestone received funding for clinical trial costs from Celgene Cellular Therapeutics. Marcelo Kremenchutzky received funding for clinical trial costs from Celgene Cellular Therapeutics. Laruen Krupp received funding for clinical trial costs from Celgene Cellular Therapeutics. Fred Lublin received funding for clinical trial costs and honoraria from Celgene Cellular Therapeutics.

** Moderator's note - Post broken into paragraphs for easier reading. Many people with MS have visual difficulties that prevent them from reading large blocks of print. **

[JerryD]

AMJ,
I am glad that your stem cell treatment is working for you, but if you follow any blogs from MSers who have had HCST, usually they see improvements in their EDSS along with disease cessation.
From what I can understand from your post, I take it you did NOT receive 'chemo' along with your infusion. I guess what I am wondering is this. Do you think you have gotten all of the benefits that you are going to see?

[AMJ]

Yes I think that HSCT has proven that is effective in stopping and in some reversing MS, but as we all know it is not affordable at this time for most of us. Therefore this was not an option for me. I was very happy to be included in this clinical trial. There was no chemo involved.

To review my EDSS score, When I was diagnosed in 2005 my EDSS was 2. In Jan/10 my EDSS was 4.5, by May it was 5.0. in Nov/10 My EDSS was 5.5. I had the stem cells implanted in 2011. My EDSS has remained at 5.5.

The study report said "no patient had an increase of EDSS score of greater than 0.5 and most patients had either stable or decreasing EDSS." I would like to know where everyone started on the scale and where they ended up. It would be interesting to see if those on the higher dose had better results.

I wish that I would have an improvement in my walking, but I no longer get migraine headaches which is a major improvement in my quality of life. I just feel better overall. I still suffer from fatigue but not to the same extent. Yes I believe that I may have seen all the improvements that I am going to see. Will my condition continue to remain stable? I don't know but I do know that the research is continuing.

I am hopeful that it won't take years for stem cell treatment to become available locally and affordable for everyone.

[AMJ]

Jerry there was another clinical trial that was going on in Ottawa, Canada over the past 13 years. There were 24 patients in this trial. They have not published the results yet, but it was very successful.

They wiped out the entire immune system, and then reboot it with a transplant of the patient’s own bone marrow. There was one death at the beginning of the study but the remainder of the participants had zero disease activity following the transplant.

If you search Ottawacitizen dot com there was an article published April 1, 2013 under the health section you can read more of the details there.

I hope you can find it easily because I understand that I cannot put the website or copy and paste the article.

[JerryD]

Thanks, AMJ. I know there is something to this Stem Cell Therapy. Robin Roberts from ABC had a stem cell treatment. And you can't discount George Goss's HSCT. It is coming to the masses, just not fast enough for me. Keep posting. Good luck

[cgarri]

Some evidence for recovery of the CNS after HSCT

Hi all,

Last week, I had a mini-stroke from which I've nearly completely recovered. My neuro wanted to completely rule out a recurrence of MS, so he had me go in for an MRI of the cervical spine to go with the brain MRI done for the stroke.

The good news, no active lesions in the head or neck, so no MS. The better news, my neuro had previously described my cervical spine as "looking like a battlefield". Today, he said it "no longer looked like Swiss cheese". I've had a number of MRIs of the brain, and on the last one previous to this one, my neuro had said my brain had improved and now looked like someone who had a mild case of MS. This is only the second MRI of my spine, the first being right before the transplant. So, the transplant and physical therapy are promoting healing in my CNS. I'm not sure the doctors thought that would happen, but I'm glad.

Craig

[JerryD]

Craig,
I am glad that you are recovering. The MRI is a wonderful miracle. Now, if we could all receive HSCT and eradicate this MonSter. What a wonderful world it would be. Good luck Keep posting