Stem cell transplants show incredible promise in treatment of multiple sclerosis

Stem cell research is making giant leaps that are revolutionizing the way we treat diseases. New and promising uses for umbilical cord blood stem cells (a source of hematopoietic stem cells) and umbilical cord tissue stem cells (a source of mesenchymal stem cells) continue to be discovered. In seeking out potential treatment for multiple sclerosis, current trials show the benefit and promise of the use of hematopoietic stem cells and mesenchymal stem cells to treat the symptoms of and potentially cure MS. 

Multiple sclerosis can rob a person of essential functioning and disrupt quality of life. But today, though there is no cure for this chronic condition, treatment for the disease has come a long way. In fact, an experimental treatment that uses a patient’s own stem cells may offer new hope for people with MS.

MS is a progressive disease that damages the central nervous system. It affects about 250,000 to 300,000 people in the U.S., and 2.3 million worldwide. It is classified as an autoimmune condition, in which a person’s own immune system attacks myelin, that fatty coating that covers and protects nerves of the spine and brain. Symptoms of MS can range from mild to severe, and may include numbness and tingling, loss of vision, chronic fatigue, balance and coordination problems, and sometimes a decline in memory and thinking skills. Sometimes damage from the disease can be permanent and lead to disability such as paralysis.

Results of a three year trial show incredible promise in the treatment of this debilitating disease.

The trial

Researchers at Presbyterian–St. Luke’s Medical Center in Denver treated 24 patients with the relapsing/remitting form of the disease (patients have points when their disease is active followed by periods when they do not experience any symptoms) with a combination of high dose immunosuppressive therapy (HDIT) followed by a stem cell transplant using their own blood derived stem cells, known medically as an autologous hematopoietic cell transplant (HCT). This type of cell is responsible for the formation and constant renewal of blood and immune cells in the body. This type of stem cell can be harvested from bone marrow, mobilized peripheral blood apheresis and umbilical cord blood. Umbilical cord blood is considered to have distinct advantages of the three due to the ease of harvest, low Graft Versus Host Disease probability and the young naive nature of the cells. At the end of the trial, the safety, efficacy, and durability of MS disease stabilization through five years after HDIT/HCT will be evaluated. The recent report, featured in JAMA Neurology¹, is a 3-year interim analysis of the trial.

Results

Dramatic improvement was seen in every area the trial sought to measure: improvements were noted in neurologic disability, quality-of-life, and functional scores. Patients experienced long-term disease remission after the administration of high-dose immunosuppressive drugs and undergoing a stem cell transplant.

1. For the three years following the treatment, more than 90 percent of patients did not experience disease progression and 86.3 percent did not have any periods of relapse.
2. Additionally, patients in that time period did not develop any new lesions related to their disease.
3. Treatment was associated with few serious early complications or unexpected adverse events.³ Though a small number of patients did have side effects from the immunosuppressive drugs, they were no different than the side effects typically experienced by MS patients taking the drugs who haven’t undergone stem cell therapy.⁴

Another preliminary study conducted at Northwestern Medicine further corroborates the findings of the St. Luke’s trial. The study, published in the Journal of the American Medical Association, included 145 patients with MS who underwent non-myeloablative hematopoietic stem cell transplantation, a low-intensity stem cell therapy that uses a patient’s own blood-forming cells to reset the immune system.

This study found that the stem cell transplantation may reverse disability and improve quality of life for patients with relapsing-remitting multiple sclerosis.⁵

“The stem cell therapy gets patients off lifelong treatments and gives them results that have never been seen before with this disease,” writes said Richard Burt, MD, chief of Medicine-Immunotherapy and autoimmune Diseases at Northwestern Medicine.

Future studies

A new trial by two Canadian research centers is about to launch to determine if mesenchymal stem cells (“MSCs”) can help alleviate the symptoms of multiple sclerosis. Researchers at the Ottawa Hospital and Winnipeg’s Health Sciences Centre will test whether MSCs can reduce inflammation and even help repair damage already caused by the disease in 20 patients suffering from MS.

Mesenchymal stem cells are primarily found in bone marrow, fat, skin and umbilical cord tissue. “MSCs have the ability to modify the immune system and reduce inflammation,” said neurologist Dr. Mark Freedman of the Ottawa Hospital Research Institute, who is leading the clinical trial. Freedman said researchers want to determine if these stem cells can demonstrate anti-inflammatory properties in patients with MS. “The ultimate hope is that we will be able to…promote repair.”

While mesenchymal stem cells have the ability to give rise to virtually any cell type in the body, Freedman said it’s their tendency to migrate to areas of injury that researchers hope to capitalize on. “And it doesn’t matter where the injury is in the body,” he said. “So they have a potential obviously for treating many different types of illnesses.”⁶

The three trials described above all require the patients to go through an invasive treatment (such as apheresis or liposuction) to get the stem cells. It would be much easier for the patients if the doctors had a ready, waiting source of stem cells such as from a cord blood bank. By banking cord blood now, the need to undergo an invasive treatment in the future to treat a disease like MS could potentially be eliminated.

Cord blood already treats nearly 80 diseases including leukemia, immune deficiency and genetic disorders that otherwise have devastating results. And the number of treatments using stem cells is increasing as the technology continues to evolve. Currently there are close to 350 clinical trials underway investigating the use of umbilical cord blood and umbilical cord tissue for stem cell transplantation. These trials include the treatment of Cerebral Palsy, amyotrophic lateral sclerosis (ALS also known as Lou Gehrig’s Disease), as well as neurodegenerative diseases of the brain and spinal cord. No one can predict what the future might bring, but one thing is for certain: the use of stem cells to treat diseases is only going to become more prevalent and successful.

¹ High-Dose Immunosuppressive Therapy and Autologous Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS)A 3-Year Interim Report. Richard A. Nash, MD; George J. Hutton, MD; Michael K. Racke, MD; Uday Popat, MD; Steven M. Devine, MD; Linda M. Griffith, MD, PhD; Paolo A. Muraro, MD, PhD; Harry Openshaw, MD; Peter H. Sayre, MD, PhD; Olaf Stüve, MD, PhD; Douglas L. Arnold, MD; Meagan E. Spychala, DrPH; Kaitlyn C. McConville, MS; Kristina M. Harris, PhD; Deborah Phippard, PhD; George E. Georges, MD; Annette Wundes, MD; George H. Kraft, MD, MS; James D. Bowen, MD. JAMA Neurol. Published online December 29, 2014. doi:10.1001/jamaneurol.2014.3780. ² “Three Years After Stem Cell Transplant Therapy Most MS Patient Subjects Still In Remission.” Multiple Sclerosis News Today. N.p., 01 Jan. 2015. Web. 11 Feb. 2015. ³ Ibid. ⁴ Firger, Jessica. “Multiple Sclerosis Treatment: Stem Cell Transplant May Help Patients with MS.” CBSNews. CBS Interactive, 29 Dec. 2014. Web. 11 Feb. 2015. ⁵ Dunne, Nora. “Stem Cell Therapy Shows Promise for MS Patients.” Bioscience Technology. N.p., 4 Feb. 2015. Web. 11 Feb. 2015. ⁶ Ubelacker, Sheryl. “Researchers Test Whether Stem Cell Can Alleviate MS Symptoms.” CTVNews. N.p., 30 Jan. 2015. Web. 11 Feb. 2015.