Monday, February 10, 2014

Keep fighting, keep learning

Thanks to my friend Sharon for reminding me about David Bexfield.  Mr. Bexfield had the same form of aggressive MS I did. He was accepted for aHSCT treatment, which halted his MS in 2010.  My own aHSCT treatment concluded in February 2014. It is my hope that my results will be as good as, or even better than his.

Similarly to me, Dave had to pay for the treatment himself because his health insurance would not pay. Dave is still battling with his insurance company to try to get reimbursed for the cost of the treatment.  Here is Dave's video , which emphasizes that aHSCT treatment probably saved his life.

One of the last frames of Dave's video references a scholarly paper that outlines the benefits of aHSCT. That paper will now be added to my State of Minnesota appeal to overturn my insurance company's rejection decision. Thanks, Dave. Good luck to us all.

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The following excerpts include highlights from the scholarly paper, Autologous Hematopoietic Stem Cell Transplantation as a Treatment Option for Aggressive Multiple Sclerosis, Current Treatment Options in Neurology, June 2013. Nikolai Pfender, MS, Riccardo Sac card, MD, Roland Martin, MD

The full text of this paper may be purchased at www.springer.com


Opinion statement
Despite the development of several injectable or oral treatments for relapsing-remitting multiple sclerosis (RRMS), it remains difficult to treat patients with aggressive disease, and many of these continue to develop severe disability. During the last two decades autologous hematopoietic stem cell transplantation (aHSCT) has been explored with the goal to eliminate an aberrant immune system and then re-install a healthy and tolerant one from hematopoietic precursor cells that had been harvested from the patient prior to chemo- therapy. Clinical studies have shown that aHSCT is able to completely halt disease activity in the majority of patients with aggressive RRMS. Research on the mechanisms of action supports that aHSCT indeed leads to renewal of a healthy immune system. Below we will summarize important aspects of aHSCT and mention the currently best-examined regimen.

...Eight drugs are currently available and multiple others either already filed for approval or in late stage clinical development. However, due to the chronic nature of MS, all of these need to be given for long times or forever. Further, all of these have side effects and some of them very serious ones, and, depending on their route of administration, some treatments compromise quality of life. Also, most of these are very expensive, and lead to substantial socioeconomic burden...  

...in recent years, aHSCT has advanced substantially, and transplant-related mortality has been between 1 %1.5 % since 2000 with the BEAM-ATG regimen. Through tight collaboration be tween hematologists/transplant specialists and neurolo-gists aHSCT is now more standardized, mortality is within the range of the most active approved therapy, ie, mitoxantrone, the efficacy is likely superior to all other available treatments, its mechanisms are better under stood, and aHSCT is probably the only treatment of MS that has to be applied only once with no further need for therapy in the majority of patients, provided that they have been selected carefully...

... Some of the MS drugs, eg, mitoxantrone or alemtuzumab, have long-lasting effects on the hematopoietic system or carry the risk of secondary malignancies and cardiac damage (mitoxantrone) or of secondary autoimmune diseases (alemtuzumab). Natalizumab (Tysabri) may lead to PML with increasing risk following 2 years of treatment or even higher risk following prior treatment with chemotherapeutic agents such as mitoxantrone...

...More than 500 MS patients have received aHSCT in Europe alone in the last 20 years, and followup for substantial fraction of these is longer than 10 years. A joint study of the European and American Bone Marrow Transplantation Societies on long-term outcomes after HSCT in MS is currently ongoing...

... existing data indicate that even the most effective available drugs need to be given continuously, that particularly the most effective therapies carry substantial risk, and that they are expected to be inferior to aHSCT regarding their efficacy. Further, aHSCT is a one time treatment with no need for continuing immunomodulation, and, when considering the cost of currently available treatments of up to 45,000 USD/year, will lead to substantial socioeconomic benefit in patients with aggressive MS... 

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