Friday, November 11, 2011

Results from the the Canadian MS/BMT Study

On October 19 -22 Chris' doctors attended the European and Americas Committees for Treatment and Research in Multiple Sclerosis and presented their results from the Canadian Multiple Sclerosis Bone Marrow Transplant study.

While Chris is not included in the official study, he underwent the exact same treatment, so it was really interesting to see the results. 

We won't be getting Chris' results until December, so we are using this good news to tide us over until then. 

I have included the official abstract that they presented. I have also added my comments in bold. My comments are based on my understanding, so there may be some errors. 

Neurological recovery following treatment of aggressive multiple sclerosis with immunoablation and autologous stem cell transplantation
M.S. Freedman, H.L. Atkins, M.J. Bowman on behalf of the Canadian MS/BMT Study Group

Background & Objectives: Immunoablation followed by autologous stem cell transplant (ASCT) is being studied as a potential treatment for MS to establish if a long-lasting MS progression free response can be induced for patients with active and progressive disease who are predicted to have a poor prognosis. 

The Canadian MS/BMT trial is a non-randomized (i.e. everyone knows what treatment they are getting) phase II trial of intensive chemotherapy and CD34 selected ASCT in 24 patients (this does not include Chris as he wasn't formally part of the trial) considered at high risk of progression with aggressive MS who failed >1 year of standard treatment and EDSS >3 and <6 (EDSS is the scale used to evaluate the level of disability in MS patients. Chris was about a 3 or 3.5 before his transplant)

Patients underwent stem cell mobilization  following high dose cyclophosphamide (CTX) and G-CSF (this was the stem cell collection stage that involved cyclophosphamide and neupogen injections and then the removal of Chris' excess stem cells). Immunoablation with CTX, anti-thymocyte globulin (ATG) and adjusted-dose busulphan was followed by infusion of an ASCT graft depleted of immune cells (this is when they blasted Chris with busulphan, cyclophosphamide, and ATG to destroy his immune system and then reintroduced his old stem cells minus the T cell.)

Results: The first transplant occurred in October 2001 and the 24th transplant was completed in December 2009 with a median follow-up of 62 months.

Most patients developed expected organ toxicities and febrile neutropenia but these were generally mild and transient. Serious toxicity developed in 2 patients receiving the highest dose of busulphan leading to fatal liver necrosis in one.

After 1,571 patient-months of follow-up
- not a single patient has experienced any further signs of inflammatory disease manifesting as relapses or new MRI lesions. (this is huge!  no more relapses!)

- 16/24 patients have stabilized or improved in their level of disability as measured by their EDSS,

- 7 patients have experienced ongoing progression of disabilities.(this means that while they have not had any relapses, the disabilities that they already had prior to treatment have gotten worse. This supports the idea that earlier intervention when the patient still has relapse-remitting MS has the best results)

Overall survival reaches a plateau of 95%.
The long term event – free (relapse, progression, death) survival reached a plateau of ~70%.

Conclusions: The results to date demonstrate that high intensity immunosuppression with ASCT is a viable treatment option for patients with very poor prognosis and can change the natural history of aggressive MS.

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