High Dose Chemo/Radiotherapy and Hematopoietic Stem Cell Transplant for Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 2

Summary: 
      Multiple Sclerosis is a disease that may be caused by the immune system reacting against the
      nervous system. It is possible, that by changing the immune system we can modify the
      progression of this disease. In this study, we will try to learn whether treatment with a
      bone marrow transplant (BMT) can help patients with multiple sclerosis. We will also try to
      learn what the side effects are of this treatment in patients with multiple sclerosis.
    

Detailed description: 
      To participate in this study, patients will need to have a central line (a thin plastic
      catheter or tube that is placed during surgery into one of the large veins in the neck or
      chest). Central lines are used to give intravenous medications or to draw blood. Before the
      transplant we will test the patients blood for viruses which can cause problems after the
      transplant. These viruses include Hepatitis B, which causes liver damage, cytomegalovirus,
      which causes lung disease and HIV which causes AIDS. If a patient is positive for the AIDS
      virus, they will not be able to undertake the transplant. In addition to these blood tests
      patients will also have an MRI (where pictures are created using magnetic rather than x-ray
      energy) of the brain and other evaluations that are standard for any patient before having a
      transplant.

      Before the transplant patients will receive daily G-CSF (Neupogen). This medicine will help
      to stimulate the production of white blood cells (WBC) that will be used for the bone marrow
      transplant. In addition, 6 tablespoons of blood will be collected to look at how the immune
      system is functioning before and after transplant. After the white blood cells have reached a
      certain level, patients will undergo leukapheresis. Leukapheresis is a procedure where blood
      is removed from a patients arm, pumped into a machine where the white blood cells are
      separated from most of the other cells, then returned to the patient through the same needle
      or through a needle in the other arm. After collection of the white blood cells, we will use
      a device in the lab to select out certain types of white blood cells (CD34+).

      After leukapheresis, patients will receive an antibody from horses called Atgam (ATG) to help
      destroy the immune system and also a drug called cyclophosphamide. After this, radiation
      treatment will be given to the entire body. This will be done 2 times a day for 3 days. This
      treatment will kill most of the blood-forming cells in the bone marrow. We will then give the
      CD34+ white blood cells that were collected during leukapheresis.

      Blood will be collected for immune reconstitution studies monthly for 3 months and at 6, 9,
      and 12 months after transplant to look at how the immune system is functioning. The amount of
      blood taken will be no more than 90ml (6 tablespoons). After the first year of treatment
      patients will continue to have 90ml (6 tablespoons) of blood taken for immune reconstitution
      studies every 6 months for 2 more years.
    

Intervention name(s): Cyclophosphamide; ATG; MESNA; Radiation therapy
Intervention type(s): Drug; Drug; Drug; Procedure
Intervention MeSH terms: Cyclophosphamide
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment