As we were coming to grips with the fact that I had MS, Scott started reading about treatments available to help offset or cure the disease.  In his effort to become educated, he came across articles about HSCT and found that it could actually cure Multiple Sclerosis.  

What is HSCT?

HSCT stands for Hematopoietic stem cell transplantation.  It is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood. It may be autologous (the patient's own stem cells are used) or allogeneic (the stem cells come from a donor).

Benefits of stem cells

Stem cells would be considered the "raw materials" of our bodies.  These are the cells from which all other cells are created.  The body takes the stem cells and form more cells called "daughter cells". These daughter cells turn into specialized cells with specific functions such as heart cells, brain cells, bone, etc.  So in essence, they are undefined then turn into what the body needs them to be.  These are many diseases for which transplants of blood-forming stem cells (Hematopoietic Stem Cell Transplants, HSCT) are a standard treatment such as Leukemia, Lymphoma, Sickle Cell Disease, and many others. 

The HSCT procedure for MS essentially is as follows:

  • Stem Cell Collection
  • Central Line Placement
  • Chemotherapy to basically kill your current immune system
  • Stem Cell Reinfusion 
The new re-infused stem cells basically reboot your immune system.
In the United States, there are also several clinical trials for HSCT ongoing around the following specific diseases or issues: 
  • Neurologic Disorders  
  • Auto-Immune Disorders (including MS)  
  • Cardiovascular  
  • Gene Therapy for Inherited Disorders  
  • Orthopedic Repairs

We found that there were hospitals around the world that used HSCT to treat Multiple Sclerosis and started sending in applications to see if I would be accepted.  We sent emails all over including:  
  • US: Northwestern University 
  • Israel: International Center for Cell Therapy & Cancer Immunotherapy (CTCI) 
  • Italy: Careggi University Hospital University of Florence 
  • Mexico: Clinica Ruiz 
  • Russia: The A.A. Maximov Department of Hematology and Cellular Therapy
It didn't take too long, and I starting hearing back from a few, but the one I was most excited to hear back from was Northwestern University, since travel costs would be much less that going overseas.  

My Journey with HSCT Clinical Trial at Northwestern

While some of the other hospitals offer HSCT as standard treatment for MS, we found that Northwestern University in Chicago was in their Phase III trial (trial #NCT00273364)  for evaluating HSCT compared to standard treatment with one of the conventional drugs (ie -  Avonex, Betaseron, Copaxone , Novantrone , Tysabri, Gilenya or Tecfidera).  To qualify for this trial, you needed to have current inflammation and have failed two of the conventional drugs. I definitely qualified.

The clinical trial at Northwestern is ran by Dr. Richard Burt.  We looked up all we could find about his work and was very impressed.  He has a website called DIAD: Division of Immune Therapy and Autoimmune Diseases http://www.stemcell-immunotherapy.com/ which really walks you through what the trial is about and the work at Northwestern.  

Below you will find my timeline and the events that have occurred with Northwestern.

Clinical Trial Communications:

  • July 30, 2015 - I sent an email to NW stating that I was interested in their trial
  • July 31, 2015 - I received a response from Kim Yaung, Dr. Burt's nurse, asking me to complete a screening form which I promptly completed and sent back.
  • August 7, 2015 - Kim sent an email stating that Dr. Burt had read my screening form and decided that I possibly qualified for the study.  At that point, it was requested that I complete a patient intake form and send in copies of my MRI's.  Kim explained that the next steps would be to travel to Chicago for the initial evaluation.  From there they would decide if I was a good candidate.  The evaluation would consist of: 
    • MRI of brain and cervical spine with/without contrast at Northwestern Memorial Hospital
    • Evaluation by study neurologist- Dr. Roumen Balabanov at Rush Medical Multiple Sclerosis Center
    • Evaluation by Dr. Richard Burt at Northwestern Medical Faculty Foundation
  • October 6, 2015 - I received the preliminary calendar (shown below) for the evaluation and several forms to complete along with information on how the trial is completed and what to expect. It was explained that after the evaluation, if accepted, I would be randomized into either the Transplant Arm or the Control Arm.  The Transplant Arm would mean I would receive HSCT and the Control Arm would mean that I continue on one of the approved MS drugs and be re-evaluated periodically by NW. Of course - I was hoping to be randomized into the Transplant Arm.

Pre-Transplant Evaluation Schedule

  • October 19, 2015 
    • Labs Drawn
    • EKG/ Chest X-Ray
    • MRI
  • October 20, 2015
    • 2D Echo
    • Pulmonary Function Test
  • October 21, 2015
    • Meet with  Dr. Roumen Balabanov (blind portion review for the study)
    • Meet with Dr. Richard Burt (trial portion review for the study)
  • October 22, 2015
    • Meet with Dr. Deborah Zuskar (mental evaluation)
  • October 23, 2015 
    • Fly back home
While I was there, my Echo came back abnormal, so I had to repeat the test.  It came back a second time as abnormal, so on 10/21/2015 - I had to go through a more thorough stress exam called a Dobutamine Stress Echo, where they gave me medication that simulated exercise to evaluate how my heart reacted.  It came back normal, so I was approved to be in the study. 

Post Evaluation 

After flying home, NW worked with my insurance company to ensure that the costs would be covered.  I was approved by my insurance company and assigned a Transplant Case worker which would be 100% covered
Kim, Dr. Burt's nurse, worked with me to set up a preliminary calendar for the transplant to occur in December IF I was randomized into the Transplant arm. 

On November 4, 2015 - I received the call from Dr. Burt stating that I was put into the Control Arm.  I was DEVASTATED to say the least.  While I understand that all studies require a control arm, it doesn't make it any easier to hear the bad news.  I was told by Dr. Burt, that they would need me to come back in 6 months for evaluation and then once a year for 5 years for additional evaluations.  He did state that if my EDSS score goes down by 1 point, then they can transfer me over to the Transplant arm, so still a sliver of hope.   

Hematopoietic stem cell transplant, HSCT, Multiple Sclerosis, MS, Myelin, Inflammation, Clinica Ruiz, Northwestern University, Chicago, Mexico, Israel, CTCI, Italy Careggi, Russia, Maximov, Avonex, Betaseron, Copaxone , Novantrone , Tysabri, Gilenya ,Tecfidera, DIAD, MRI, EDSS

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