Stem cells and multiple sclerosis

"For the first time, some of the disability associated with the early stages of multiple sclerosis appears to have been reversed.  The treatment works by resetting patients' immune systems using their own stem cells."

New Scientist

Well, that paragraph certainly caught my eye this morning in the news, thanks to a friend for sending the link over. 

My initial thoughts were two fold - I liked the idea that stem cell technology might possibly help people with MS since I have several friends who suffer from the debilitating condition, but it tempered by the phrase 'resetting patients immune systems'.  Regular readers of this blog will be familiar with the disastrous TGN 1412 trial at Northwick Park, which has been covered here previously see here and here.  That study warned of the dangers of stimulating a normal immune system in homeostasis.

Let's take a look at multiple sclerosis though.  It's an autoimmune disease where the fatty myelin sheath, which wraps around nerve cells and speeds up their rate of transmission, is attacked by the body's own immune system.

Previous efforts with stem cells have tried to reverse that process with no success.  The researchers decided that perhaps waiting until advanced disease when neuro-regeneration had set in was the problem.  The next logical approach was to therefore look at early stage disease before the body is under attack.

21 people with early relapsing-remitting MS who had not responded to treatment with the standard drug, interferon beta, after six months received an autologous stem cell transplant (aSCT).  The patients had stem cells removed from their bone marrow, then receive chemotherapy to destroy all existing immune cells in the body, before re-injecting the stem cells.  The stem cells then developed into naïve immune cells that do not see myelin as alien, and hence do not attack it.

Three years later, 17 of the patients had improved by at least one point on a standard disability scale, while none of the patients had deteriorated.  The study has just been published in The Lancet Neurology.

The authors concluded that:

"Non-myeloablative autologous haemopoietic stem cell transplantation in patients with relapsing-remitting MS reverses neurological deficits, but these results need to be confirmed in a randomised trial."

I have to say that realising the patients received a stem cell transplant rather than some immune stimulant reassured me greatly.  However, SCT is not a walk in the park - it is a known curative treatment for some forms of leukemia, but because it involves wiping out all the existing white blood cells, it weakens the patient and some die because they are unable to fight infection.

Interestingly, the patients received alemtuzumab as part of their SCT regimen, similarly to the Cambridge patients reported in the Guardian article highlighted below.  Alemtuzumab is a monoclonal antibody approved for the treatment of leukemia, but it became a candidate for MS when it was realised that it dampens the immune system.

Larger scale trials are still needed to confirm the initial results, but so far it looks a promising approach for the treatment of early stage MS.

Sources: R BURT, Y LOH, B COHEN, D STEFOSKY, R BALABANOV, G KATSAMAKIS, Y OYAMA, E RUSSELL, J STERN, P MURARO (2009). Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study The Lancet Neurology DOI: 10.1016/S1474-4422(09)70017-1

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Comments

[Thor]

Great article...once again!

The application of stem cells to MS seems like a promising therapy, thought wiping out existing white blood cells is a bit concerning.

The point about the failure of current treaments is an important one, and highlights the importance of new therapeutics that approach the disease in a different way (like stem cell therapy).

I reviewed the new perspectives on MS and therapies recently (not including stem cells). Interestingly, the overall perspective has shifted to MS being a constitutive diffuse syndrome rather than the classical notion of a multifocal disease with periodic heightened immune activation. This shift has allowed scientists to intervene the aberrant immune systems from different angles than before.

http://optimism.thorscave.com/category/health/

[thevoice@voicedup.com]

Let's think of how many Americans are suffering from ailments and what this research can do for them. I'm sure everyone reading this knows someone important to them that is affected by one of these maladies.Research in the U.S.has not progressed since earlier this decade, cures are a very long time away and lets not even discuss the approval requirement by the FDA. At least this can be seen as a huge step forward to join the rest of the world at bringing about cures that could help millions of people including you and I some day.

[Czes Kulvis]

They say "...treatment works by resetting patients' immune systems using their own stem cells"...

You say "...it involves wiping out all the existing white blood cells, it weakens the patient and some die"...

Thank you for clarifying the issue.

YES - I am a MS sufferer.

NO - I don't want to live without immune system !

Thanks again