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Interim Results Reported from Clinical Trial of Stem Cell Transplantation in People with Relapsing-Remitting MS

A nationwide team of researchers report on interim results from a small, five-year study of transplantation of the individuals’ own hematopoietic (blood cell-producing) stem cells combined with high-dose immunotherapy in 24 people with relapsing-remitting MS. This procedure aims at “rebooting” the immune system to prevent MS immune attacks against the brain and spinal cord. At three years, 78.4% of participants experienced no new disease activity. When this trial has completed its five-year duration, it will be an important addition to research needed to determine whether this approach to stem cell transplantation is safe and effective in people with MS. Richard A. Nash, MD (Colorado Blood Center Institute) and colleagues report in JAMA Neurology (Published online December 29, 2014). This study was sponsored by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health.
Background: One type of procedure that has been explored for many years in MS is called “autologous hematopoietic (blood cell-producing) stem cell transplantation” – or HSCT. This procedure has been used in attempts to “reboot” the immune system, which launches attacks on the brain and spinal cord in people with MS.
In HSCT, these stem cells (derived from a person’s own bone marrow or blood) are stored, and the rest of the individual’s immune cells are depleted usually by chemotherapy. Then the stored stem cells are reintroduced back to the individual’s bloodstream. The new stem cells migrate to the bone marrow and over time produce new cells. Eventually they repopulate the body with immune cells. The goal of this currently experimental procedure is that the new immune cells will no longer attack myelin or other brain tissue, providing the person, what is hoped to be, a completely new immune system.
The Study: Investigators enrolled 25 people who had experienced an MS relapse involving loss of neurologic function while taking disease-modifying therapies during the previous 18 months. Participants received HSCT along with high-dose immunosuppressive therapy (a regimen of treatments that profoundly suppress the immune system), and followed for five years. The primary endpoint of this study is whether participants experience “event-free survival,” meaning that they did not die or have an increase in disease activity. Disease activity is defined as any one of the following outcomes occurring: confirmed loss of neurologic function, clinical relapse, or new lesions observed on MRI scans. The current publication presents a planned analysis after three years of follow up.
Results: One individual experienced a pulmonary embolism induced by heparin (administered as part of stem cell collection), and withdrew from the study. Event-free survival at three years was 78.4%, down from 95.8% after one year. Treatment failed in five individuals. Scores on clinical scales measuring disease activity and quality of life, including the EDSS, improved significantly at three years after HSCT. Immune system analysis showed prolonged depletion of the immune cells that drive the immune attack, indicating that the immune system was indeed “rebooted.”
Two deaths occurred, one from complications due to MS progression and another due to asthma. One person experienced an MS attack, an individual who had not complied with a prednisone regimen designed to reduce this risk during collection of stem cells. There were 130 adverse events that were severe or life-threatening, mostly cytopenias (blood cell reductions) and infections.
Comment: Rigorous clinical trials of stem cell therapies are crucial to determining their safety and effectiveness in people with MS. “We look forward to seeing the completed results of this important study,” says Bruce Bebo, PhD, Executive Vice President of Research at the National MS Society. “There are significant risks involved in hematopoietic stem cell transplantation, and it’s important to ensure that this will be a safe solution for people with MS, with significant clinical benefit.”
With the urgent need for more effective treatments for MS, particularly for those with more progressive forms of the disease, the National MS Society believes that the potential of all types of cell therapies must be explored. The Society is currently supporting 15 research projects exploring various types of stem cells, including cells derived from bone marrow, fat and skin, and has supported 70 stem cell studies over the past 10 years.

Live Webcast: Research Advances from the World’s Largest MS Meeting

September 13, 2014

On Saturday, September 13, 2014, join a panel of experts for a live nationwide Webcast on “Research Advances from the World’s Largest MS Meeting

Saturday, September 13, 2014
2 – 3 pm ET (1 – 2 pm CT, Noon – 1 pm MT, 11am – Noon PT)

To register for the live webcast please click here.

Topics include:

  • Progressive MS
  • Genetics
  • Wellness and rehabilitation
  • Repair and protection

Moderator: Kate Milliken, founder, Milligrace Productions. Kate has been living with MS since 2006.

Panelists include:

Dr. Philip DeJager, Brigham and Women’s Hospital, Boston, MA

Dr. Don Mahad, University of Edinburgh, UK

Dr. Ellen Mowry, Johns Hopkins University, Baltimore, MD

Dr. Timothy Coetzee, Chief Advocacy, Services and Research Officer for the National MS Society

Register

We want to thank Genzyme, a Sanofi Company; Novartis Pharmaceuticals; Questcor Pharmaceuticals; and Walgreens Specialty Pharmacy for providing generous educational grants to make this program possible.

*For people who cannot participate in real-time, we will post the recorded version with transcript for viewing shortly after the event.

New Guideline from the American Academy of Neurology

The American Academy of Neurology, an association of neurologists and neuroscientists dedicated to promoting high-quality care for people with nervous system disorders, has released a guideline on the use of complementary and alternative medicine (CAM) in MS. The guideline was created by a panel of medical experts who evaluated all available published research studies. Through a comprehensive process, the panel carefully assessed the outcomes of relevant research studies, and also whether study findings could be generalized to the larger population of people with MS, and whether any safety concerns should be considered by physicians and patients who might use these treatments.

The widespread use of CAM by people with MS highlights the unmet need for more research and better therapies that will help people with MS live their best lives. Summaries of the guideline are available for patients and for clinicians.

Specific Guideline Information

Among studies of several forms of marijuana and its derivatives that were evaluated by the panel, the guideline reports that evidence suggests that:
Oral cannabis extract and synthetic THC (tetrahydrocannabinol, a major active component of marijuana) are effective for reducing patient-reported spasticity and pain, but not MS-related tremor or spasticity measured by tests administered by the physician;
Sativex oral spray (GW Pharmaceuticals) is effective for improving patient-reported spasticity, pain, and urinary frequency, but not bladder incontinence, MS-related tremor or spasticity measured by tests administered by the physician;
Smoked marijuana research studies have not produced enough evidence to assess its safety or effectiveness for treating MS symptoms including spasticity, pain, balance, posture, and cognition.

Oral cannabis extract, THC and Sativex are not currently approved by the FDA for use by people with MS. The guideline notes that, as with any therapy, along with potential benefits come potential side effects, and for these cannabis derivatives the most commonly reported side effects were dizziness, drowsiness, difficulty concentrating and memory disturbance.

The guideline also points out that the long-term safety of marijuana use for MS symptom management is not yet known.

Read about the National MS Society’s position statement and more about the use of marijuana for MS symptoms.

Among other CAM therapy studies in MS evaluated by the panel, the available research evidence suggested that:
ginkgo biloba is ineffective for improving cognitive function, but might reduce fatigue;
magnetic therapy might reduce fatigue;
reflexology might help ease unusual skin sensations such as tingling, but there is insufficient evidence to evaluate its potential for treating pain, fatigue, and other symptoms;
the use of omega-3 fatty acids with low-fat diet to reduce relapses or disability, or improving quality of life is not supported by current evidence.

The guideline includes a table of all of the CAM therapies that were evaluated. For many of these, the panel concluded that there was not enough evidence from research studies to advise as to their effectiveness in treating MS or its symptoms. The guideline also indicates that there is little known about how CAM therapies interact in the body with each other or with disease-modifying therapies that individuals may be taking. The effects and side effects reported from non-MS studies were not evaluated.

While many people with MS report that they experience benefits from CAM therapies, this guideline confirms the need for more research to demonstrate the effectiveness of these treatment strategies. The National MS Society is committed to ensuring that no opportunity is wasted in our mission to stop MS progression, restore function lost to MS, and end MS forever so that people with MS can live their best lives. We have supported, and will continue to support, CAM research and will continue to advocate for additional government funding for CAM research.

Ms Society

As part of the National MS Society’s commitment to support the training of physicians in the comprehensive care of people with MS, we are launching a new training program offering five-year awards to mentors and institutions to provide training for board-certified/eligible neurologists and physiatrists in MS comprehensive care. The program was developed in response to the critical need to increase the supply of physicians with expertise in the diagnosis and treatment of MS. The goal is for fellows to acquire the skills and knowledge necessary to provide the highest quality of care for individuals with MS.

Read more about the new program, who is eligible, and how to apply

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