Stem Cell Research – Where is it Now?

An Interview with Dr. George Kraft and a Look at Current Stem Cell Research and MS

By Laurie Long 12/05

While stem cell transplants have been used for decades for certain types of cancer, it is only recently that researchers have used these techniques, specifically hematopoietic stem cell transplantation (HSCT), to try to stop the onslaught of multiple sclerosis in patients with severe, progressive MS. In the last ten years, HSCT has been used on more than 700 patients with various severe autoimmune diseases in Phase I and II clinical trials in over 20 countries. The results of these studies has been sufficiently positive to offset the inherent dangers – a mortality rate of between 2 – 11% 1, for those suffering from severe autoimmune diseases and for whom all other treatments have failed.

In April 2002 Dr. George Kraft of the University of Washington Medical Center presented results of a stem cell therapy pilot study at the annual meeting of the American Academy of Neurology. The 26 participants in the study all had severe, progressive MS and their disease had not responded to other conventional treatments. Each participant received a transplant of their own blood stem cells after their immune cells were destroyed with chemotherapy, irradiation and anti-thymocytic globulin. “The hope is that these stem cells will eventually reconstitute into healthy immune system cells and the disease process can be stopped,” said Dr. Kraft 2.

The results of the pilot study, compiled after observing the patients for an average of 15 months following the operation, showed that 20 out of the 26 patients appeared to stabilize, although one patient died of complications related to the procedure. In a recent interview with Dr Kraft, he explained that he and his team have continued to observe and test their patients’ stability and have gathered more data on the long-term effects of the stem cell transplant. They plan to present their findings in the spring of 2006, but their research will not end there. “In order to truly study the long term results of this operation,” Dr. Kraft explains, “you have to see how the patients are in 20 years, or 30, or 50. The only way to find out if this procedure can cure MS is to monitor that patient’s health throughout their life.”

At the same time this avenue of research continues, other researchers have been investigating techniques with stem cells that may be able to reverse damage caused by MS. Doctors in Milan, Italy, are using neural stem cells to create glial cells (the cells that produce myelin), to treat mice with an MS-like disease. “Stem cells have the potential to replace the function of damaged nerve cells,” said Dr. Guiseppe Scotti, who leads the team. Neural stem cells can start a chain reaction that leads to myelin production, explained Scotti. In this study the injected neural cells positioned themselves near damaged areas and immediately began the process to create more glial cells, increasing myelin production. The treated mice had almost complete recovery from their disabilities, while the untreated mice continued to decline 3.

In August of 2005, the National MS Society awarded a $3.5 million grant to a team of University of Wisconsin researchers to develop stem cell transplant techniques for the repair of myelin. Dr. Ian Duncan, who heads the team, explained that an important part of the project will be to direct human stem cells to become myelinating cells that could be used in transplants to repair the damage of MS 4.

At the University of Washington, Dr Kraft is currently working to put together a second study on stem cell transplantation for MS. This study, he indicates, will be much larger with perhaps twice the number of participants as the original. Because of the dangers involved, Dr Kraft and his team have worked hard to decrease the risk. “We have used the information we gathered from our first study to modify the treatment and make it safer and more easily tolerated,” he stated. There will be no use of irradiation in the new study and a different group of chemotherapy drugs known as BEAM, which have been widely used in Europe, will be substituted for the previous chemotherapy drugs. The new drugs, Dr. Kraft explains, are safer and more readily tolerated. In addition, this study is aimed at those with relapsing-remitting MS and progressive-relapsing MS with mild to moderate MS symptoms (EDSS 5.5 – 3.0), rather than those with severe, progressive MS. Participants will be those whom standard treatments have failed to help and who have progressed significantly in the last year.

Because this experimental treatment costs somewhere in the range of $150,000 per person, Dr. Kraft and his group have negotiated with insurance companies to help pay for the procedure. The insurance companies, says Dr. Kraft, are hoping this research will mean long-term savings in the costs of drugs and medical treatments. “They are very eager for this to work,” he explains. Dr. Kraft adds,“we will work with people, we will work with their insurance company. We won’t shut people out.”

While Dr. Kraft and his group work to refine their procedure to use a person’s own stem cells to stop their MS, and other researchers begin to follow the path that will hopefully use stem cells to repair the damage caused by MS, the race to develop stem cell treatments continues to build momentum. As Dr. Letterio Politi, co-author of the Milan paper, said, “Cell therapies are a promising true alternative in the treatment of previously untreatable central nervous system disorders.” 5

If you would like to learn more about Dr. Kraft’s new study or would be interested in participating, please contact Bernie McLaughlin, Research Nurse, Fred Hutchinson Cancer Research Center, at 206-667-4916 or bmclaugh@fhcrc.org.

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Further Reading:

Right to Recover: Winning the Political and Religious Wars over Stem Cell Research in America

Cell Therapy is Bound to Become a Part of Medical Practice

Stem Cells (Wikipedia)

AAAS Policy Brief: Stem Cell Research

Endnotes and References

1. Tyndall, A.; Daikeler, T. Autologous Hematopoietic Stem Cell Transplantation for Autoimmune Diseases. Acta Haematologica 2005; 114:239-247.
2. American Academy of Neurology. Stem Cell Transplants May Be Effective for MS Patients. 54 th Annual Meeting, April 13-20, 2002.
3. Hitti, M. Stem Cells May Heal Multiple Sclerosis Damage. Web MD Medical News, December 1, 2004.
4. Devitt, T. $3.4 million directed to key MS study. Embryonic Stem Cell Research at the University of Wisconsin-Madison, August 10, 2005.
5. Dusik, D.; Morley, M. Stem Cells May Hold Promise as Multiple Sclerosis Cure. Radiological Society of North America 90 th Scientific Sessions and Annual Meeting, Chicago, Nov 28 – Dec 3, 2005.

Blanco, y.; et. al. Autologous haematopoietic-stem-cell transplantation for multiple sclerosis. Lancet Neurology, 2002 Jan;4(1):54-63.

Saccardi, R.; et. al. Autologous HSCY for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life. Blood, March 15, 2005, Vol. 105, No. 6, pp. 2601-2607.

Pluchino, S.; Martino, G. The therapeutic use of stem cells for myelin repair in autoimmune demyelinating disorders. Journal Neurol. Sci. 2005 June 15;233(1-2):117-9.

Sykes, M.; Nikolic, B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature, June 2, 2005, 435; 620-627.

Dubois-Dalcq, M.; Ffrench-Constant, C.; Franklin, R.J. Enhancing central nervous system remyelination in multiple sclerosis. Neuron. 2005 Oct 6;48(1):9-12.

Zhao, C.; et. al. Stem cells, progenitors and myelin repair. Journal of Anatomy, September 2005, Vol. 207(3): 251.

Sykes, M.; Nikolic, B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature, 2005 June 2;435(7042):620-7.

Fassas, A.S.; et. al. Hematopoietic Stem Cell Transplantation for Multiple Sclerosis: A Retrospective Multicenter Study. J. Neurology 2002;249:1088-1097.

Ariniello, L. Repairing Damage in Multiple Sclerosis. Society for Neuroscience, Brain Briefings, August 2004.

Multiple Sclerosis Association of America. New Research Strategies and Drugs Under Investigation. www.msaa.com

Taggart, K. Combo drug, stem cell therapy aids in MS. The Medical Post, Neurology Update, May 7, 2002 Vol 38, Issue 18.

Fred Hutchinson Cancer Research Center. Innovative new treatments for autoimmune disease.

Science Daily Magazine. Stem Cell Transplants May Be Effective For Multiple Sclerosis Patients. April 17, 2002.

Kay, L. Stem cells may keep MS from advancing – UW researchers present study. The Seattle Times, April 17, 2002.

Paulson, T. Stem-cell therapy offers new hope for MS patients. Seattle Post-Intelligencer, April 18, 2002.

Research Programs Department. Researchers Report Modest Success Transplanting Blood Stem Cells to Treat Severe Multiple Sclerosis. National MS Society Research/Clinical Update, April 17, 2002.

Stagg Elliott, V. Stem cell researchers see hope in autologous transplant. American Medical News, May 6, 2002.

Paulson, T. Transplant patient an inspiration to others. Seattle Post-Intelligencer, March 22, 2000.

King, M. The Stem Cell Story. National MS Society.

Recer, P. Study: Bone-marrow cells grow into brain cells. Associated Press, The Seattle Times, December 1, 2000.

Wall Street Journal. Study of Transplants of Nerve Cells Yield Encouraging Results. February 1999.

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