Where do things stand on the potential of stem cell treatments for multiple sclerosis (MS)?
MS is an often devastating autoimmune disease in which the immune system attacks its own nervous system. More info can be found at the National MS website here.
MS is a very serious health problem globally as about 400,000 people have MS in just the US alone and more than 2 million worldwide suffer from the disease. Almost all of us know someone who has MS. It’s a common disease with a huge negative impact. New treatments are desperately needed.
In patients with MS, the immune system mistakenly attacks the insulation, called myelin, surrounding nerves. It thus belongs to a class of disorders called demyelinating diseases. This damage basically screws up the function of the nerves in profound ways. The symptoms (see at left) are diverse, but often it first manifests as tingling and numbness, weakness in arms or legs, unusual changes in vision, or changes in cognition.
There is no known cure for MS and while some medications are helpful, they often can have side effects and in many cases the disease progresses anyway. As a result, it is not unusual for MS patients to seek out alternative medicine therapies. One such at least theoretically possible kind of new therapy is based on stem cells.
MS patients regularly contact me and ask about the potential of stem cells to be used for MS.
- Do stem cells have promise for MS?
- Can they be used now for MS?
- What about clinics offering stem cell treatments now?
Great questions.
There is a lot of important stem cell research ongoing for the treatment of MS that have promise. On the government clinical trials website I see 30 studies listed for a search of “stem cells” and “multiple sclerosis”.
However, at this time, although a number of clinics in the US and internationally claim they have effective stem cell treatments for MS via using so-called mesenchymal stem cells (MSCs), the science is just not there to support such claims yet.
Still I see ads on the Internet claiming to provide effective stem cell treatments for MS, but inevitably they lead to dubious clinics.
Nonetheless, there is reason for hope, but patience is required. I know that waiting longer is frustrating and in some cases the disease is so severe that waiting seems impossible. I respect that urgency. Also, I understand the seriousness of the disease and the frustration of being told to try to wait, but many of the so-called “stem cell” treatments for MS offered by for-profit clinics could be dangerous and do far more harm than good. They are also extremely expensive.
More research is needed, particularly FDA approved clinical trials such as those I mentioned above. I would recommend that patients talk to their personal physicians and consider a clinical trial rather than a dubious treatment at a clinic.
Theoretically how might stem cells help MS?
I can see three main ways.
- One way is through immunomodulation. What this means is stem cells, particularly one type called mesenchymal stem cells (MSCs), have the power to potentially turn down the level of activity of the immune system. In that way MSCs may be able to reduce autoimmunity and inflammation that leads to cell and tissue damage. Think of it as MSCs turning down the thermostat of immunity in the nervous system.
- A second, cutting edge approach that is under investigation is to use MSCs as tools to deliver drugs. In this way, kind of like pizza delivery guys, MSCs might home in on areas of damage in MS and squirt out the equivalent of two-liter (of course much smaller volume in reality) bottles of some chemicals that reduce inflammation right in the zone where it is needed most. They may also “feed” the injured cells pizzas in the form of various supportive substances, proteins, etc.
- Finally, nervous system stem cells, rather than MSCs, could be used for cell therapy via transplantation to help re-myelinate nerves and repair the damage.
Bone marrow transplantation (which at its heart is a stem cell therapy) may also be effective to treat or some claim to even cure MS, however this procedure is extremely risky and it alone can kill some patients making it appropriate arguably only for the most extreme cases of MS.
Overall I’m optimistic about stem cell treatments for multiple sclerosis or MS, but it’s going to take some years and the clinics now offering non-FDA approved treatments for MS should be avoided in my opinion.
my friend marty has m.s are stem cells trials going to be available in ca. i only see them being done in other countries for lots of money, i told my friend i would like a dance with her stem cells would make that possible.
Paul there is another company that is working on rebooting the immune system so it haults the attack on the myolin sheath indicative of MS. Ultra-High-Dose Cyclophosphamide.
Revimmune
(High-Dose Cyclophosphamide) for Autoimmune Diseases
Accentia Fast Facts: Revimmune™
(Ultra-High-Dose Cyclophosphamide)
Stage
Phase III Pending
Indication
Multiple Sclerosis and other Autoimmune Diseases
Competitive Products
Believed to be the first therapy in development for multiple sclerosis designed to restore neurological and physical functions with the potential to eliminate autoimmunity
Collaborative Partner
Johns Hopkins University
Market Opportunity
MS affects more than 2.5 million people worldwide, with more than 200 people diagnosed every week in the U.S. alone.
2008 Pending Milestones
File a regulatory application (IND) with the U.S. FDA seeking approval to commence a Fast-Tracked Phase III clinical trial for MS; Secure strategic development/marketing partnership
Relevant Publications
Hopkins Medicine Magazine article featuring Revimmune
Frost & Sullivan Report
Scientific Publications
For scientific publications on Revimmune, please contact us
While Accentia has great expectations for Revimmune based on highly encouraging preliminary study results, this product has not yet been approved by the U.S. FDA or any international regulatory agency. Revimmune is also being developed by Biovest International, Inc., Accentia’s publicly-held, majority-owned subsidiary, for the treatment and prevention of transplant rejection, including bone marrow transplants for the purpose of curing sickle cell anemia.
About Revimmune™
Accentia holds the worldwide exclusive license to Revimmune for the treatment of autoimmune diseases, such as multiple sclerosis. Developed by Dr. Richard Jones, Dr. Robert Brodsky, and colleagues at Johns Hopkins University School of Medicine, Revimmune uses an already-approved active pharmaceutical (cyclophosphamide) in a novel, patent-pending, ultra-high dose, pulsed administration capable of “rebooting” a patient’s immune system. Revimmune therapy is believed to act by completely eliminating mature lymphocytes throughout the body while selectively sparing immune stem cells in the bone marrow. Shortly following a course of Revimmune, a natural “rebooting” process takes place as bone marrow stem cells repopulate the immune system without memory of the autoimmunity.
Investigators at Johns Hopkins discovered that stem cells uniquely have high levels of a particular protective enzyme that can be measured in advance of therapy, which makes them impervious to Revimmune, and allows the surviving stem cells to give rise to the new immune system over 2 to 3 weeks. The newly reconstituted peripheral immune system typically lacks the misdirected immunity to self-antigens, which is characteristic of autoimmune diseases.
Revimmune is the world’s first therapy to propose the restoration of neurologic and physical functions and offer the potential for the elimination of autoimmunity.
Study Results: Revimmune for Multiple Sclerosis
Researchers from Johns Hopkins University have published encouraging results from a two-year study evaluating the treatment of aggressive relapsing-remitting multiple sclerosis with Revimmune.
An article titled “Reduction of Disease Activity and Disability with High-Dose Cyclophosphamide in Patients with Aggressive Multiple Sclerosis” was published in the Archives of Neurology. It concludes that Revimmune was safe and well-tolerated in patients, and that the therapy resulted in a pronounced reduction in disease activity and disability after treatment which was sustained during the course of follow-up for approximately two years. In contrast, existing approved therapies for the treatment of multiple sclerosis are only intended to slow progression of the disease, not improve the patient’s functional status.
Accentia believes these preliminary results to be unprecedented with an average functional score improvement of about 40% in these patients who were tracked for two years after receiving therapy with sustained restoration of their functional improvement. Of those nine patients, eight of them had failed other therapies, and during the course of follow-up, five of them had no signs of disease activity, and the other four showed dramatic improvement over the course of follow-up.
Paul I hope this helps I remember this was released prior to Geron’s haulting their programs and is one of the resons I wasso upset about the change in direction. I would think Prof. Jeffery D. Kocsis, Ph.D., from Yale University School of Medicine or Jane Lebkowski, Ph.D. would be good sources for information on this subject.
Oct. 25, 2011, 7:31 a.m. EDT
Geron Announces Presentation At ECTRIMS/ACTRIMS
Non-Clinical Data Supports Use of GRNOPC1 in Multiple Sclerosis
MENLO PARK, Calif., Oct 25, 2011 (BUSINESS WIRE) — Geron Corporation GERN -0.86% today announced data on the use of GRNOPC1, oligodendrocyte progenitors derived from human embryonic stem cells, for myelin repair in a non-human primate model. The data supports further investigation of the potential therapeutic use of GRNOPC1 in central nervous system (CNS) disorders where the central or contributing pathology is destructive removal of myelin from nerve axons, such as observed in multiple sclerosis (MS), myelitis and spinal cord injury. GRNOPC1 is currently in a Phase 1 clinical trial in patients with spinal cord injury.
The new data were presented at the 5th Joint Triennial Congress of the European and Americas Committees for Treatment and Research in Multiple Sclerosis, in Amsterdam, by Prof. Jeffery D. Kocsis, Ph.D., from Yale University School of Medicine. The work was performed in collaboration with scientists at Geron.
The studies utilized a non-human primate model where demyelinated lesions, such as seen in multiple sclerosis, were induced chemically in the spinal cord. GRNOPC1 was injected into the demyelinated spinal cord lesions one week after chemical induction. The lesion sites from six monkeys were analyzed using light and electron microscopy at various timepoints up to one year after injection of GRNOPC1 to look for evidence of cell survival and remyelination of nerve axons. In the first few weeks after implantation, the injection sites contained maturing transplanted cells indicative of a premyelinating phenotype with evidence of variable numbers and degrees of remyelinated axons. By four months post implantation, GRNOPC1 had induced extensive and thick myelin around the formerly denuded axons. Human cells were detected at the lesion site, providing evidence for survival of transplanted GRNOPC1. There was no evidence of abnormal tissue, tumor formation or other pathologies associated with the injection of GRNOPC1. Neurological exams of the injected animals were normal. The data showed in the non-human primate that GRNOPC1 can survive at the lesion site and progressively promote remyelination of axons.
“These new data confirm and extend previous results showing that GRNOPC1 can promote remyelination in rodents and non-human primates,” said Jane Lebkowski, Ph.D., Geron’s Chief Scientific Officer. “These results provide further support for the potential of GRNOPC1 to provide therapeutic benefit in a number of central nervous system diseases, such as MS and myelitis.”
About GRNOPC1
Oligodendrocytes produce myelin, an insulating layer made up of protein and fatty substances that forms around nerves in the CNS to enable them to conduct electrical signals. Without myelin, many of the nerves in the brain and spinal cord cannot function properly. Oligodendrocytes are lost in CNS disorders such as spinal cord injury and MS.
GRNOPC1 contains hESC-derived oligodendrocyte progenitor cells that have demonstrated remyelinating, nerve growth stimulating and angiogenic properties leading to restoration of function in rodent models of acute spinal cord injury. Non-clinical studies have shown that administration of GRNOPC1 seven days after injury significantly improved locomotor activity and kinematic scores of rats with spinal cord injuries compared to untreated controls. Histological examination of the injured spinal cords treated with GRNOPC1 showed improved axon survival and extensive remyelination surrounding the rat axons. Geron is conducting a Phase 1 clinical trial to assess the safety of GRNOPC1 in patients with complete, thoracic spinal cord injuries. In addition, Geron has established collaborations with academic groups to evaluate GRNOPC1 in models of other CNS disorders. For more information about GRNOPC1, visit human embryonic stem cell-based therapeutics | biopharmaceuticals | cancer | telomerase | human embryonic stem cell | vaccine | Geron .
About Geron
Geron is developing first-in-class biopharmaceuticals for the treatment of cancer and chronic degenerative diseases. The company is advancing anti-cancer therapies through multiple Phase 2 clinical trials in different cancers by targeting the enzyme telomerase and with a compound designed to penetrate the blood-brain barrier. The company is developing cell therapies from differentiated human embryonic stem cells, with the first product in a Phase 1 clinical trial for spinal cord injury. For more information, visit human embryonic stem cell-based therapeutics | biopharmaceuticals | cancer | telomerase | human embryonic stem cell | vaccine | Geron .
This news release may contain forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that statements in this press release regarding potential applications of Geron’s human embryonic stem cell technologies and GRNOPC1 constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and protection of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Geron’s periodic reports, filed with the Securities and Exchange Commission, including the quarterly report on Form 10-Q for the quarter ended June 30, 2011.
SOURCE: Geron Corporation