Multiple Sclerosis Stem Cell Studies

Multiple sclerosis (MS) is widely believed to be an autoimmune condition – the body’s immune system mistakenly attacks, and subsequently damages, the ‘myelin sheath’ protecting nerve cells in the brain and spinal cord. This damage causes messages to and from the brain to be slowed, distorted or stopped altogether. This is what leads to the symptoms of MS.

Damage to the myelin sheath is believed to cause ‘relapses’, or MS attacks. In these attacks, symptoms flare up and last for anything from 24 hours to several months. Over time, if nerve fibres themselves become damaged, or destroyed completely, this can lead to ‘progression’ of the MS and an increase in disability.

• Relapsing remitting MS (RRMS):
  Periods of acute attacks with worsening of symptoms followed by complete or partial recovery (remission)
  Around 85 per cent of people with MS are diagnosed with RRMS.
• Secondary progressive MS (SPMS):
  Following an initial period of relapsing remitting MS, many people develop secondary progressive MS. This is characterised by a gradual accumulation of disability, either with or without relapses (relapsing SPMS or non-relapsing SPMS).
• Primary progressive MS (PPMS):
  A gradual accumulation of disability from the beginning, with no distinct periods of relapse and remission.
  Around 10-15 per cent of people diagnosed have this form of MS.
• Progressive-Relapsing MS:
  In this relatively rare course of MS (5%), people experience steadily worsening disease from the beginning, but with clear attacks of worsening neurologic function along the way. They may or may not experience some recovery following these relapses, but the disease continues to progress without remissions.

People suffering from Multiple Sclerosis often suffer from the following symptoms:

• Fatigue
• Tingling
• Pain
• Numbness
• Problems with balance and walking
• Changes in vision
• Depression
• Emotional changes
• Impaired thinking
• Impaired understanding
• Poor muscle coordination
• Sexual problems
• Slurred speech
• Stuttering
• Bladder and bowel problems

Stem cells are unprogrammed cells in the human body that can be described as "shape shifters." These cells have the ability to change or “differentiate” into other types of cells. Stem cells are at the center of a new field of science called regenerative medicine. Because stem cells can become bone, muscle, cartilage and other specialized types of cells, they have the potential to treat many diseases, including Parkinson's, Alzheimer's, Multiple Sclerosis, Diabetes and more.

StemGenex offers stem cell therapy using Adult stem cells only. There are four known types of stem cells:

• Adult Stem Cells - derived from the adult human body
  The use of adult stem cells in research and therapy is not as controversial as the use of embryonic stem cells, because the production of adult stem cells does not require the destruction of an embryo. Additionally, in instances where adult stem cells are obtained from the intended recipient, the risk of rejection is essentially non-existent. Consequently, more US government funding is being provided for adult stem cell research. This is why StemGenex offers stem cell studies using Adult stem cells only.
• Embryonic Stem Cells - derived from embryos
  These cells require specific signals to differentiate to the desired cell type. If they are simply injected directly, they will differentiate into many different types of cells, resulting in a tumor derived from this abnormal pluripotent cell development (a teratoma). The directed differentiation of ES cells and avoidance of transplant rejection are just two of the hurdles that ES cell researchers still face. StemGenex does not use embryonic stem cells.
• Fetal Stem Cells - derived from aborted fetuses
  Have developed further than embryonic stem cells and are a little more specialized – their options are slightly more limited. However, they can still produce most types of cell in the body. StemGenex does not use fetal stem cells.
• Induced Pluripotent Stem Cells (iPSCs) - from some parts of the human body
  These stem cells are engineered from older, fully specialized cells – for example, skin cells, which are limited naturally to being only skin cells. Scientists encourage these limited cells to act like embryonic stem cells again, with the ability to become any type of human cell. This is a complex technique that has only recently been developed and is the subject of much ongoing research. StemGenex does not use induced pluripotent stem cells.

Stem cell therapy is an intervention strategy that introduces new adult stem cells into damaged tissue in order to treat disease or injury. Many medical researchers believe that stem cell treatments have the potential to change the face of human disease and alleviate suffering. The ability of stem cells to self-renew and give rise to subsequent generations with variable degrees of differentiation capacities, offers significant potential for generation of tissues that can potentially replace diseases and damaged areas in the body, with minimal risk of rejection and side effects.

From what we know so far about stem cells and MS, there are two main ways that potential treatments for MS might be developed:

• Immunomodulation – preventing immune damage to the nervous system
• Remyelination – repairing the myelin sheath that has already been damaged

These are both considered ‘neuroprotective’ therapies because they aim to protect the nerve fibres inside the myelin sheath.

Because MS is a neurodegenerative disease, and its more prominent feature is the damage the disease does to the central nervous system, it is hoped that stem cells may hold the key to reversing the damage done by facilitating the repair of damaged nerve cells.

StemGenex is studying potential ways to directly target the conditions and complications themselves. These studies consist of multiple ways to deliver the highest amount of activated stem cells to the areas patients need them most. When stem cells are studied through StemGenex, as potential therapy for Multiple Sclerosis, there are multiple ways they can be administered:

• Full body IV – directed into the vein
• Intra Nasal – administration to access a highly vascular pathway of the nose to encourage more stem cells to travel past the blood brain barrier
• Direct Bladder Injections – administration to access the bladder directly in attempts to alleviate issues of incontinence in patients
• Injections at the Base of the spine – administration targeting lesions on the spine
• Direct site injections - injected directly into the site that needs repair, i.e., muscles and tendons

Where stem cells are injected is likely to influence how they work. The intravenous and intrathecal methods are so far the only ones used to test treatments for MS, but all might have a role to play as we learn more about stem cells.

Yes. Scientists around the world believe there is enough evidence to suggest that stem cells hold real potential as a therapy for MS. This evidence comes from a multitude of early clinical studies. They believe that it is now time for a concerted effort in stem cell research and an international effort to support clinical studies of stem cells for MS.

No. There are currently no FDA approved stem cell therapies for MS disease. All stem cell therapies for MS disease are currently ‘unproven’, ‘experimental’ therapies. This means that the FDA does not know whether stem cells are effective for people with MS disease. The only way to determine the effectiveness of stem cell therapy is through the type of clinical studies and trials which are currently being conducted in the US.

One of the goals of StemGenex, through our stem cell studies, is to understand what a particular stem cell therapy might be able to achieve. For example, does it have the potential for slowing the disease's progression, replacing damaged cells and memories, or both? With this goal in mind, StemGenex continues to study these diseases and the full effect of stem cell therapy on each disease. Anecdotally, these results have been overwhelmingly positive but there is more that needs to be done to determine the exact effectiveness of these therapies.

After stem cells have been administered into someone’s body they have to make their way to the correct place (e.g. area of damage) and then have their desired effect. This process takes time and although it is difficult to predict exactly how long, it is likely that it will take several weeks or months on average to see the full desired effect.

Yes, a stem cell therapy may be repeated. Current studies indicate the strong possibility of a cumulative effect from multiple stem cell therapies a patient received for their condition. Long-term studies will attempt to better understand this in detail.

We don’t know yet. This will not be studied in early clinical trials, as this would make it very difficult to measure the true effects of the stem cell therapy. However, a combination therapy may be effective for MS and is likely to be studied in the future.