Pharma Strategy Blog

Commentary on Pharma & Biotech Oncology / Hematology New Product Development

“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:
ResearchBlogging.orgR 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

Reblog this post [with Zemanta]

6 Responses to “Stem cells and multiple sclerosis”

  1. 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/

  2. 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.

  3. 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

  4. Jessica Forester

    After 6 months of offering stem cell therapy in combination with the venous angioplasty liberation procedure, patients of CCSVI Clinic have reported excellent health outcomes. Ms. Kasma Gianopoulos of Athens Greece, who was diagnosed with the Relapsing/Remitting form of MS in 1997 called the combination of treatments a “cure”. “I feel I am completely cured” says Ms. Gianopoulos, “my symptoms have disappeared and I have a recovery of many functions, notably my balance and my muscle strength is all coming (back). Even after six months, I feel like there are good changes happening almost every day. Before, my biggest fear was that the changes wouldn’t (hold). I don’t even worry about having a relapse anymore. I’m looking forward to a normal life with my family. I think I would call that a miracle.”Other recent MS patients who have had Autologous Stem Cell Transplantation (ASCT), or stem cell therapy have posted videos and comments on YouTube. www.youtube.com/watch?v=jFQr2eqm3Cg.Dr. Avneesh Gupte, the Neurosurgeon at Noble Hospital performing the procedure has been encouraged by results in Cerebral Palsy patients as well. “We are fortunate to be able to offer the treatment because not every hospital is able to perform these types of transplants. You must have the specialized medical equipment and specially trained doctors and nurses”.  With regard to MS patients, “We are cautious, but nevertheless excited by what patients are telling us. Suffice to say that the few patients who have had the therapy through us are noticing recovery of neuro deficits beyond what the venous angioplasty only should account for”.Dr. Unmesh of Noble continues: “These are early days and certainly all evidence that the combination of liberation and stem cell therapies working together at this point is anecdotal. However I am not aware of other medical facilities in the world that offer the synthesis of both to MS patients on an approved basis and it is indeed a rare opportunity for MS patients to take advantage of a treatment that is quite possibly unique in the world”.Autologous stem cell transplantation is a procedure by which blood-forming stem cells are removed, and later injected back into the patient. All stem cells are taken from the patient themselves and cultured for later injection. In the case of a bone marrow transplant, the HSC are typically removed from the Pelvis through a large needle that can reach into the bone. The technique is referred to as a bone marrow harvest and is performed under a general anesthesia. The incidence of patients experiencing rejection is rare due to the donor and recipient being the same individual.This remains the only approved method of the SCT therapy.

  5. Robert Taylor

    “Unnecessary risks are being taken by patients seeking the liberation treatment.” says Dr. Avneesh Gupte of the CCSVI Clinic. “It has been our contention since we started doing minimally invasive venous angioplasties nearly 6 years ago that discharging patients who have had neck vein surgery on an outpatient basis is contra-indicated. We have been keeping patients hospitalized for a week to 10 days as a matter of safety and monitoring them for symptoms. Nobody who has the liberation therapy gets discharged earlier than that. During that time we do daily Doppler Ultrasounds, blood work and blood pressure monitoring among other testing. This has been the safe practice standard that we have adopted and this post-procedure monitoring over 10 days is the subject of our recent study as it relates to CCSVI for MS patients.”

    Although the venous angioplasty therapy on neck veins has been done for MS patients at CCSVI Clinic only for the last 18 months it has been performed on narrow or occluded neck veins for other reasons for many years. “Where we encounter blocked neck veins resulting in a reflux of blood to the brain, we treat it as a disease,” says Gupte. “It’s not normal pathology and we have seen improved health outcomes for patients where we have relieved the condition with minimal occurrences of re-stenosis long-term. We believe that our record of safety and success is due to our post-procedure protocol because we have had to take patients back to the OR to re-treat them in that 10-day period. Otherwise some people could have run into trouble, no question.”

    Calgary MS patient Maralyn Clarke died recently after being treated for CCSVI at Synergy Health Concepts of Newport Beach, California on an outpatient basis. Synergy Health Concepts discharges patients as a rule without in-clinic provisions for follow up and aftercare. Post-procedure, Mrs. Clarke was discharged, checked into a hotel, and suffered a massive bleed in the brain only hours after the procedure. Dr. Joseph Hewett of Synergy Health recently made a cross-Canada tour promoting his clinic for safe, effective treatment of CCSVI for MS patients at public forums in major Canadian cities including Calgary.

    “That just couldn’t happen here, but the sooner we develop written standards and best practices for the liberation procedure and observe them in practice, the safer the MS community will be”, says Dr. Gupte. “The way it is now is just madness. Everyone seems to be taking shortcuts. We know that it is expensive to keep patients in a clinical setting over a single night much less 10 days, but it’s quite absurd to release them the same day they have the procedure. We have always believed it to be unsafe and now it has proven to be unsafe. The thing is, are Synergy Health Concepts and other clinics doing the Liberation Treatment going to be changing their aftercare methods even though they know it is unsafe to release a patient on the same day? The answer is no, even after Mrs. Clarke’s unfortunate and unnecessary death. Therefore, they are not focused on patient safety…it’s become about money only and lives are being put at risk as a result.”

    Joanne Warkentin of Morden Manitoba, an MS patient who recently had both the liberation therapy and stem cell therapy at CCSVI Clinic agrees with Dr. Gupte. “Discharging patients on the same day as the procedure is ridiculous. I was in the hospital being monitored for 12 days before we flew back. People looking for a place to have the therapy must do their homework to find better options. We found CCSVI Clinic and there’s no place on earth that’s better to go for Liberation Therapy at the moment. I have given my complete medical file from CCSVI Clinic over to my Canadian physician for review.” For more information Log on to http://ccsviclinic.ca/?p=866 OR Call on: +1 (404) 461-9560.

  6. Smokie1756

    My partner has just been told her neck stems cells are busted. She has had a lumbar puncture what other treatment will happen. She is 46 , had MS for 20 plus years and is now what would be classed as RR MS. We are in Aberdeen so what may happen next??

Comments are closed.

error:
error: Content is protected !!