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Sunday, March 18, 2012

Ready, Set.... Wait

Today my wife and I are supposed to be in our new apartment in Chicago preparing for my hospital admission and HSCT to begin at 7:00AM tomorrow morning.  Instead, we're in NY doing our regular Sunday routine.

Earlier this week I was in Chicago for final baseline testing and a meeting with Dr. Burt.  All went swimmingly and on schedule.  I caught the late flight home Wednesday with the news I hoped for, all systems go!

I went to work Thursday morning for the last 2 days in the office for a few months.  Early in the afternoon I received a call from Dr. Burt's Nurse Practitioner and from the tone of her voice I immediately knew it was bad news.  The blood work I did while at Northwestern came back indicating a compromised immune system, specifically a low B and T cell lymphocyte count.  This is a result of the Fingolimod I was taking for MS.  Dr. Burt put the procedure on hold for at least one month to clear my system of Fingolimod and allow my body to naturally restore my B and T cell count to a normal level before receiving chemotherapy.  Failure to do this could be dangerous because it will take me longer to rebuild sufficient immunity from antigens in the einvironment. 

While this is very disappointing news I can honestly say this is the first setback in this process and it could have been a lot worse.  The last medication I was taking (Tysabri) required 6 months for metabolic clearance as opposed to Fingolimod with a half life of 6-9 days.  Dr. Burt recommends 4 times the half life so one month will be sufficient. 

So, I get to work a few more weeks before treatment.  I have already received a revised calendar from Kim at Northwestern and the revised start date is April 16th. 

Wednesday, March 14, 2012


Hello and Welcome! 

I decided to follow many of my fellow MS'ers slow foot steps (MS joke) and chronicle my journey through Autologous lymphoablative Hematapoietic Stem Cell Transplantation (AHSCT) for the treatment of my Relapsing Remitting Multiple Sclerosis.  I'm generally a very private person and would ordinarily tend to keep an experience like this to myself.  However, over the past six months I have read, reread and rereread the many blogs of my brave new friends who not only forged ahead to go through this difficult, painful and sometimes deadly procedure seeking relief from this hideous disease, but they also were courageous enough to write about every step of this process in excrutiating detail.  These day by day reports were incredibly helpful for me through the research, application and now soon to be treatment stage of HSCT.

I can't go further without thanking my friend George Goss who has helped me and countless others through the research and contemplation of pursuing HSCT for the treatment of MS.  George received HSCT in Heidelberg, Germany and after 2+ years he continues to enjoy the results of a very successful procedure and even reversal of significant physical deficit.  His inspirational and educational blog can be found at

Some background:  I'm a 42 year old father of two from Long Island New York.  I have a beautiful and incredibly supportive wife without whom this journey would be impossible.  I was diagnosed with RRMS in 2002 while training for the NYC marathon.  Initial symptoms of the disease were present a few years earlier but like many MS'ers, denial was my first treatment choice for MS.  The extensive training for the marathon caused an exacerbation I couldn't ignore.  My left leg and hands lost most feeling making walking and writing difficult and the Lhermittes Syndrome I had experienced intermittently for a few years had increased exponentially.  After a few months of tests and evaluations I was diagnosed with Multiple Sclerosis.

I recovered from this episode within a few months and even completed the marathon.  Over the next 8 years I managed my MS with little interruption to my very active lifestyle filled with a lot of work, a little travel and some occasional sports.  While age and sports activity increased and decreased respectively, I still cherished the occasional tennis match, ski trip or a simple run on the beach.  Previous DMTs include Avonex, Betaseron, Tysabri, Gilenya, Detrol and Baclofan

In May of 2011 I stopped Tysabri due to increased lesion load, the presence of antibodies to Natalizumab, JCV+ and mounting physical deficit.  Since then symptoms have progressed month over month making walking and writing more difficult.  Stairs became an adventure, golf and tennis were no longer on my calendar, buttoning my shirts became a project and writing more than a paragraph or two was no more.  Bladder urgency and frequency became a very frustrating daily symptom and pain in my back and legs from spasticity appeared for the first time since diagnosis. 

By September of last year I started to really worry.  The "Post Tysabri" relapse was unrelenting and I knew it was a matter of time, quickening time that the life I enjoyed was slipping away.  I began researching what was out there beyond the protocol of the many Neurologists I had been treated by.  What was in clinical trials, experimental, ANYTHING that could stop this damn disease!

I came across a web site called TIMS This is a valuable source of information on all topics related to MS.  Its member authored posts are incredibly detailed, scientific and all have the same goal which is to assist others to educate themselves regarding the best method to treat their disease.  This lead me to George Goss's blog and the journey towards HSCT began.  Similar to many others with MS, my treating Neurologists have been very quick to increase the number of little orange CVS containers in my bathroom filled with different size pills to treat different size symptoms but never once did any of them mention the groundbreaking research around the world related to using immunosuppressive treatment to "reboot" one's defective immune system and therefore stopping the unfathomable reality of misguided lymphocytes attacking and destroying live healthy tissue (myelin) and reaking havoc in an otherwise healthy body. 

I found the research of Dr. Richard Burt from Northwestern University in Chicago.  I read his published papers in JAMA and The Lancet Neurology detailing his phase I/II clinical trial from 2003-2005.

Findings below from: Published online January 30, 2009 DOI:10.1016/S1474-4422(09)70017-1 

"Between January, 2003, and February, 2005, 21 patients were treated. Engraftment of white blood cells and platelets was on median day 9 (range day 8–11) and patients were discharged from hospital on mean day 11 (rangeday 8–13). One patient had diarrhoea due to clostridium diffi cile and two patients had dermatomal zoster. Two of the 17 patients receiving alemtuzumab developed late immune thrombocytopenic purpura that remitted with standard therapy. 17 of 21 patients (81%) improved by at least 1 point on the Kurtzke expanded disability status scale (EDSS), and five patients (24%) relapsed but achieved remission after further immunosuppression. After a mean of 37 months

(range 24–48 months), all patients were free from progression (no deterioration in EDSS score), and 16 were free of relapses. Signifi cant improvements were noted in neurological disability, as determined by EDSS score (p<0・0001), neurological rating scale score (p=0・0001), paced auditory serial addition test (p=0・014), 25-foot walk (p<0・0001), and quality of life, as measured with the short form-36 (SF-36) questionnaire (p<0・0001)."

This research and preliminary success has been followed by a phase III clinical trial currently accepting patients at Northwestern University Hospital in Chicago.  The trial is called MIST and can be accessed through the following web link  Because this is a phase III trial the qualification criteria is very specific. Dr. Burt is very accessible, returned my email within 12 hours and we spoke within 2 days.  He works tirelessly pursuing his passion to advance the use of AHSCT for MS and many other autoimmune diseases.  I did not qualify for the trial as my MS does not meet McDonald's criteria for MS.  The criteria is a complicated formula including a ratio of T2 lesion load between brain and C-spine.   Dr. Burt agreed to perform HSCT on me for compassionate need.  I'm very grateful to have the support of an incredible family who has supported and encouraged me through the lengthy application process.  We immediately began making arrangements for child care, airfare and an apartment in Chicago for my wife to stay during the next 2 months of treatment. 

Before acceptance to Northwestern I communicated extensively with University of Heidelberg in Germany applying to receive treatment there. I was invited for an evaluation in Heidelberg but with no guarantee of acceptance. I also applied to India as well. George Goss in his blog has extensive descriptions and contact information about facilities around the world performing myeloablative and lymphoablative HSCT procedures for the treatment of MS.

The process has 2 phases:  Mobilization during which an initial infusion of Cyclophosphamide chemotherapy is administered to reduce the chances of a relapse while another drug Nupogen is used to boost stem cell CD34+ progenitor peripheral blood stem cells.  This takes approximately 10 days before the "harvest" is performed to filter my blood to collect a minimum of 2 millions PBSCs. 

The second phase begins with a lymphoablative 5 day regimen of Cyclophosphamide, Mesna and ATG chemotherapies to reduce the immune system to a level just above requiring repeated childhood immunizations. After chemotherapy my stem cells are thawed and returned to me through a brief 20-30 minute infusion.  This phase requires a hospital admission of  2-3 weeks while my immune system rebuilds to a level that allows exposure to naturally occuring pathogens in the environment.  I will return home to NY and rest for a few weeks before returning to work...without my hair!

There are two protocols for AHSCT.  Dr. Burt practices the lymphoablative method as described above.  The other protocol is called myeloablative which is a much more aggressive chemotherapy protocol that completely destroys the immune system requiring repeat childhood immunizations and a much longer recovery period.  Dr.  Burt believes the additional toxicity of the more aggressive chemotherapy is not required to achieve the desired "reboot" of the immune system.  Other physicians around the world practice the myeloablative protocol e.g. the HALT study in Texas.  While I was actively seeking acceptance to Heidelberg, Germany with the myeloablative protocol I would have received this treatment.  However, a shorter recovery period and remaining in the US with insurance coverage made this decision a no brainer for me.

As most of you know the insurance industry is reticent to pay for any service that is not approved by the FDA. While there are phase III clinical trials in process for the use of HSCT for MS, FDA approval is still down the road. Shockingly, I received authorization from my insurance plan (United) in just a few weeks. I couldn't believe the excellent service I received from the transplant nurse assigned to my case. Karen was very professional, called me frequently and worked to close the case as quickly as possible. I did take a proactive strategy to send my insurer a letter detailing why this treatment was medically necessary for me at this time and I included a graph detailing the ROI of HSCT compared to the roughly $77k per anum current medical and pharma costs of treating my disease. Adding a conservative 4% medical inflation factor, within 3 years United will recover the investment of HSCT as I will hopefully be able to stop my DMT and symptom based medications. I don't know how much they will cover of the lengthy "pre-transplant" testing regimen but any assistance for the procedure will be great. I was very surprised and obviously elated as this procedure at Northwestern begins at $125k and I have heard from others of invoices north of $190k. 

I begin the mobilization phase on Monday 3/19 in Chicago.  I will post periodic updates throughout the treatment.  I encourage you to ask questions that I will answer to the best of my ability.  To those contemplating treatment I wish you the best of luck!