Information for Patients

There are currently only a few approved treatments for MS [what is MS?] that are designed to modify the course of the disease: interferon (Avonex®, Betaseron®, Rebif®), glatiramer acetate (Copaxone®) and mitoxantrone (Novantrone®) are likely familiar to most MS sufferers. These drugs are taken on a long-term basis, with the goal of slowing the natural course of MS. For those MS sufferers who have more active or advanced disease, there are even fewer effective treatment options.

The HALT-MS study is investigating an experimental treatment of intensive immunosuppression to determine whether it can stabilize the disease in patients with relapsing-remitting or progressive relapsing MS and a poor prognosis.

What is Halt-MS?

HALT-MS is a clinical trial in which we are studying the use of high-dose immunosuppression (chemotherapy) followed by autologous hematopoietic (blood) stem cell transplantation as a treatment for poor-prognosis MS.

Before high-dose immunosuppression, blood stem cells (those cells that go on to produce all blood cells) are collected or withdrawn from the participant’s own bloodstream. The collected cells are processed in a lab to select the blood stem cells and remove the T cells that are thought to cause autoimmune diseases like MS). The selected blood stem cells are then frozen or stored. The participant then undergoes intensive immunosuppression with high-dose chemotherapy. The selected blood stem cells are returned to the patient as an infusion to restore the blood counts after the treatment. We are investigating if the immune system will be “reset” after the intensive immunosuppression preventing further relapses or loss of neurological function.

Briefly, the study consists of the following phases:

1. Screening:  Evaluations will be done to determine if a participant is eligible for the study.  Tests will include a neurological evaluation, blood work, MRI, lumbar puncture, and an assessment of the lungs (pulmonary function test and chest x-ray) and the heart (ECG and echocardiogram or MUGA scan) .

2. Treatment:

   1. Baseline Evaluations- Screening will be continued  and in addition a bone marrow aspiration and leukapheresis( removal of some white blood cells for research studies) will be done. Patients will also be asked to complete a quality of life questionnaire.
   2. Stem Cell Collection- Patients will receive G-CSF (a blood growth factor) by injection for 4-5 days. G-CSF increases the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis (method of withdrawing stem cells from the blood) will then be done until a sufficient amount of blood stem cells are collected to restore the blood counts and the immune system after high-dose immunosuppression.  The blood stem cells will be processed and then frozen for later use.
   3. High-Dose Immunosuppression and Transplantation- Patients are hospitalized to receive high-dose immunosuppression with a chemotherapy regimen called BEAM, which consists of four different drugs (carmustine, etoposide, cytarabine, and melphalan), and an immunosuppressive agent known as Thymoglobulin. After high-dose immunosuppression, the autologous (from self) blood stem cells will be infused or transplanted after thawing. It is anticipated that recovery of the blood counts will occur by 9-14 days after transplantation. The immune system will take a longer time to recover. Patients will be monitored closely at the transplant center for approximately 2 months.
   4. Follow-up- HALT-MS participants will be asked to attend eight (8) study visits over the subsequent 5 year period. Study visits will take place at study site and will include blood and urine collection, MRI studies, leukapheresis, and MS neurology exams and questionnaire completion.

What is known about this technique?

A pilot study in Europe has tested this technique in 19 MS patients. Eighteen (18) of those treated had their disease stabilize or improve for at least three years following treatment; One patient showed a new lesion on magnetic resonance imaging (MRI) of the brain 4.5 years after treatment.

The HALT-MS study aims to extend the results of that study, to establish the safety and effectiveness of the treatment for advanced MS and to learn more about how the treatment works on the immune system.

Like all medicines, there are risks of side effects and complications involved in receiving an experimental therapy in a clinical trial. Some of these risks are known and others may be unknown. For instance, it is not uncommon to experience flu-like symptoms (nausea, vomiting, fever, fatigue, muscle and joint aches, headaches) related to the medications used in this study and there is a risk of experiencing allergic reactions to these medications. Complications specific to MS can occur after high-dose immunosuppressive therapy and there is a risk of infection. Every attempt is made to minimize each patient's risk of side effects, and each individual's experience will vary in both the type and number of side effects, as well as their severity. If you are interested in participating in HALT-MS, our study staff will explain the potential risks and benefits of the treatment in detail and will be happy to answer any additional questions you might have about the trial.

Find out if you may be eligible to participate...