#AAN2018 – Stem Cell Transplant is Effective Treatment for ‘Aggressive’ MS, Study Shows

#AAN2018 – Stem Cell Transplant is Effective Treatment for ‘Aggressive’ MS, Study Shows

Autologous hematopoietic stem cell transplantation (aHSCT) has been shown to be safe and highly effective to treat patients with “aggressive” multiple sclerosis (MS). Tested in 19 patients, transplantation of stem cells was found to induce clinically meaningful improvements in disability.

These findings were shared at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, California, in a presentation titled “Autologous Haematopoietic Stem Cell Transplantation in Treatment Naïve Patients with ‘Aggressive’ Multiple Sclerosis.”

aHSCT uses a patient’s own healthy bone marrow stem cells, in combination with a much less aggressive chemotherapy and/or radiation regimen, to prepare the patient for the transplant.

Previous studies have suggested that aHSCT is an effective strategy to treat patients with highly active relapsing-remitting MS (RRMS) who do not respond to available disease-modifying therapies (DMTs), and international guidelines advocate for its use in patients with “aggressive” MS.

To further demonstrate the potential of aHSCT as a treatment for “aggressive” MS, a research team evaluated its safety and effectiveness in MS patients who had not been treated previously with DMTs.

A total of 19 patients were treated across several clinical centers: seven patients were from Sheffield, U.K., seven from Uppsala, Sweden, four from Ottawa, Canada, and one patient was from Florence, Italy. All patients received aHSCT between May 2004 and May 2017.

In addition to aHSCT, patients were treated with BEAM (carmustine, etoposide, cytarabine, melphalan) chemotherapy plus antithymocyte globulin (ATG) to reduce transplant rejection, or with Cytoxan (cyclophosphamide) with ATG, or the triple combination of Cytoxan, ATG, plus busulfan as conditioning regimens.

Patients had a median age of 33 years at diagnosis and received the aHSCT by a median time of nine years after symptom onset. They had a median disability score of 6.5 before the treatment, as determined by the Expanded Disability Status Scale (EDSS).

After a median follow-up period of 30 months, patients had a median EDSS score of 2.0, which represented a median improvement of 2 points (the higher the score, the worse the patient’s disability level).

None of the patients had clinical relapse following the transplant of stem cells.

Only three patients developed new brain lesions detectable by magnetic resonance imaging (MRI) at the first six-month follow-up evaluation, but no additional new lesions were detected in the following scans.

The adverse effects reported during the study were comparable to those previously observed in similar treatments. No deaths related to the treatment were reported.

Based on these preliminary results, the researchers concluded that aHSCT is “safe and highly effective in inducing rapid and sustain remission” in highly active MS, and “was associated with a significant improvement of [patient’s] level of disability.”

“aHSCT should be considered as first line therapy in patients with ‘aggressive’ MS,” the team concluded.

Another study presented at the AAN 2018 meeting further supports these findings, demonstrating the superior effectiveness of aHSCT over conventional DMTs for RRMS.

28 comments

  1. Karen Evans says:

    Hello I have remitting ms now for last 8 years I want to be put forward for the stem cell to halt my ms my neurologist won’t listen to what I say twice they put me on ddt s which made me very ill indeed I have now refused all further tablets etc
    I know I’m alright at the moment but am very wary of the future my health is important to me

  2. Heracles says:

    Great!! FDA, approve stem cells NOW!!! Stop protecting Big Pharma profits at the expense of the health of American patients.

  3. Lon Honzell says:

    If only the USA would stop giving in to the FDA so that they could make more money.
    Then just maybe people affected with MS and other diseases could get some needed help.
    I was diagnosed with MS in 1990, self employed, became disabled in 1997. Taking several MS meds that do not work, I have progressive MS. Holding my own?
    Unable to walk, can walk some with a walker.
    Right foot Burns, Freezes 24/7.
    Have even had the stupid thought of having it amputated but then what if I started having phantom pain that could not be cut out, off.
    So guess we will just have to wait till the US decides to help.

    • Jason says:

      Try Mexico. Russia does it as well. There are several place across the globe; or, you can sit tight and hope that 2020 (or something) the US gets it.

  4. I have spms maybe PPMS all I know I am going down hill fast! Please tell me how to get into stem cell trial?I am not your normal ms pt.I was healthy as an ox,had minor surgery and ened up with MRSA IN MY BLOODSTREAM seeding up in c-3 c-5 on a vent quadparalais 3 months rehab I worked so hard and walked again 2008 as a nurse in PACU I couldn’t go back to work maybe a desk job,but I could walk slow with my grandchildren.THEN BEGINNING OF 2010 FEELING WEAK DRS. TRYING TO FIND OUT WHY FOUND LESIONS AND SPINAL DX WITH MS,I am on Rituxn now… I have gone right back to square one,so depressed I want to throw in the towel….CAN U HELP ME?

  5. Karen J Foley says:

    The Tisch Foundation in NY is doing FDA approved clinical stem cell trials.
    The catch: they are not taking new patients and anyone in their sixties or older.
    So my husband who had an active life style up until a few years ago is being written off. We do not live in a socialized medicine state -yet… Why isn’t this available to everyone? Now drs. say, “Well you are older and can’t do as much….” He is 68 and has been rapidly declining for past two years. Prior to that he could lift heavy weights and use both of his hands and had no trouble with his balance. Now he can barely walk. The medical community is not listening to MS patients. They throw up their hands and order another blood test. Tecfidera did not work. Copaxone did not work and the new “miracle” drug Ocrevus (by infusions) did not work. So are we supposed to just give up. We refuse to lay down and be passive. We need help and we need it now.

    • Harriet Eskildsen says:

      Hello, I too am a senior citizen with PPMS and completely understand your frustration. It’s frustrating enough there are so few options for people with PPMS compared to the RRMS community, but then to be denied access to the cutting edge treatments is at least for me, like throwing salt on an open wound; and feeling my disability issues are unimportant
      should be discarded, makes me very angry. . I understand the researchers want younger, less disabled patients to demonstrate the success and effectiveness of their treatment. The success generates more research dollars to further the research project. The pharmaceutical company wants to see the success of their multi – million dollar investment too. I understand, older people are more frail and can die, and that is the last thing a researcher or pharmaceutical company wants to risk. It’s bad because the research money might be more difficult to obtain and bad for the pharmaceutical company’s stockholders and their bottom line. Too bad, seniors with informed consent, are not offered the opportunity to be included in the research project as another control group.

  6. Karen J Foley says:

    My husband showed no new lesions on his last few MRIs, so why is he worse every single day?

    The drs. have no answers. We do not understand this.
    So frustrated and then the drs. want to know if he feels depressed? Who wouldn’t?
    It would be abnormal to be happy-go-lucky when you are in pain every single day and can barely walk.

  7. Karen LHF says:

    Will we all be dead from old age when this is finally available to everyone? What are they waiting for? Then, when it will be available, they will say you are too old to take it. This is insane. People who were once productive and are now not, due to the disease, are not being given any realistic hope or options. You know what is old age and what is the disease. Since when is the sixties age-wise considered old age in 2018? Someone who worked hard everyday using his hands, and lifting heavy weights as a mechanic can now barely walk and can only use one hand. I am sick of the medical community who thinks someone who worked in a “blue collar” profession is an idiot. My husband is one of the smartest individuals I have ever met. He is well-read and well-spoken. We come to the drs.’ offices asking about things we have researched. Why is the stem cell research being blocked in the US except by one place doing limited clinical trials of only THEIR OWN patients? Isn’t that a conflict of interest? ISN’T THAT ETHICALLY IMPROPER?

  8. Laurie Uherek says:

    What more does the FDA want with regards to results? A person who has been diagnosed with MS is essentially told, you are basically engaged in a slow dying, painful death. My husbands neurologist told him that the doctors just don’t know how to treat secondary progressive MS to where possible reversal can be realized. Another neurologist told him “my goal is to see you don’t progress any further”. We haven’t heard back from him since we visited over a year ago. He’s been through all of the DMT’s and has done the Whole 30, Wahls Protocol, Keto, and a 21 day Daniel Fast. He eats healthy, still tries to workout (once was a body builder), and pushes his wheelchair into work every day because he refuses to sit in it. He has the most positive attitude of anyone I’ve ever met. If cancer patients are approved to be treated with HCST, why can’t MS patients be approved? If statistics over time reveal there’s no change in condition, then deal with it. But to deny it, is criminal!

    • Angie says:

      Keeping you with flare ups,infusions and useless drugs, because once cured then the money gets cut off.If they actually found a cure they would not give it because there’s no money for cures.

  9. Carol says:

    It is exactly the same in the UK. Stem cell therapy is not offered to anyone over 60. Trials are limited to younger people with
    RRMS with a lower disability score.
    Not good news for someone like me with PPMS.
    There needs to be more money and research into MS. It’s hard to believe that we are no nearer finding what causes this condition, let alone a cure.

  10. Karri Brown says:

    My husband has MS. No DMT are working. He is declining fast. We are interested in Stem Cell Transplant. Is there a way we can get in on stem cell treatment?

  11. James Nowicki says:

    It is my opinion that we in the United States WON’T SEE STEM CELL THERAPY because the BIG PHARMACEUTICAL companies will use their money and influence to deny citizens our rights.

  12. Carol says:

    I would like to reply to Jason and James.
    I think you have a point there regarding the pharmaceutical companies (without sounding too cynical.
    I would go to Mexico or Russia for stem cell therapy if I had a spare £65.000! Not being able to work for the last 5 years, has depleted my savings! So can’t afford it
    Carol

  13. WJ Ives says:

    Simply put Big Pharma is scared of stem cells & hasn’t figured a way to patent it, IE exploit those with MS . ($) Congress is not on the public’s side. Look how much cash Big Pharma pumps into lobbying/politics. Both Repubs & Democ should be ashamed. They won’t even adjust the SSI/SSDI resource level for inflation after over 20 years. Write your congressman/senator.

  14. Al says:

    Thank you to the gentleman that continues to say Russia and Mexico. I had a good job, working hard, and saving this money but went through it after becoming disabled with MS, sorry no spare change to go to Russia or Mexico. The problem here in the United States is that the pharmaceutical lobby owns the FDA and Congress. It’s more important for the pharmaceutical industry to charge individuals with MS and other immune disorders upward of $80,000 a year instead of taking the chance on stem cell at a cost of $80,000 to $100,000 therefore, taking a chance on stopping the MS in its tracks. How would the big pharma make their obscene profits if patients were allowed to use stem cell to stop their MS payday, by stopping, slowing or reversing MS in some patient, and they lose their Cash Cow. Congress is owned by large pharma and the large pharma runs the FDA and the only loser is the patient whether they have MS, ALS, Parkinson’s or whatever. Let’s not forget the US taxpayer that picks up the bill for every MS patient that is on disability hence Medicare or Medicaid. And if you’re not on disability it just runs up the cost of private insurance to a point were no one cannot afford to have it. At which point the large pharmaceutical companies will now give you the medicine for free. Figure that one out. The entire situation from start to finish makes no sense.

  15. Xenia says:

    Lemtrada is also super-effective, and it’s much safer. My MS was very aggressive, and it’s completely dormant now, thanks to Lemtrada.

    • Tornado Alley says:

      What kind of MS do you have? I was hit head-on by a drunk driver with NO INSURANCE at at 146 MPH, who was smoking a cigarette, smoking marijuana & drinking alcohol. He left me in the middle of the busy interstate to die like yesterday’s human roadkill. I was cut out of the mangled vehicles by the jaws of life and lifeflighted to ER Trauma Center with over 34 broken bones and a punctured lung & heart. Immediately placed on ventilator and in coma for 45 days. That said, I’ve had 67 major surgeries including one amputation. Subsequently, diagnosed with Multiple Sclerosis. I have taken Copaxone for ten years. Have not walked nor driven in eleven years, using wheelchair.

  16. Harriet Eskildsen says:

    Hello, I too am a senior citizen with PPMS and completely understand your frustration. It’s frustrating enough there are so few options for people with PPMS compared to the the options of therapies for the RRMS community. But, then to be denied access to the cutting edge treatments is at least for me, like throwing salt on an open wound. Feeling my disability issues are unimportant because I am older and
    disposable, makes me very angry. I understand the researchers want younger, less disabled patients to demonstrate the success and effectiveness of their treatment. Successful outcomes generate more research dollars and furthers the research project. The pharmaceutical company has a vested interest for the success of their multi – million dollar investment. While I understand, I disagree with their rationale. Older people are more frail, can die and therefore may be a liability to the project’s success. That is the last thing a researcher or pharmaceutical company wants to risk. Can you blame anyone for setting the parameters for the best possible results? Successful outcomes mean more research money and continued success for the pharmaceutical company’s stockholders and their bottom line. Too bad, seniors with informed consent, are not at least offered the opportunity to be included in the research project as another control group.

    The frustration and anger I read from people writing about the Big Pharmacy lobbyists is another issue, a legal issue, that needs to be addressed and in my opinion, replaced with a more moral and ethical solution. It’s not just the the pharmaceutical industry, it’s also the tobacco industry and especially the NRA that are extremely wealthy and aggressive lobbyists. Paying, in my mind, bribing, congressmen/women to support any legislation that supports a specific interest group and does not benefit its citizens, is a breach in their responsibilities of their sworn duties to represent us, their constituents, the American people. Let me be clear: We vote people into office to give them the power to represent and promulgate OUR interests, and not provide a platform and an opportunity for them to think first of themselves and provide a means for them to generate additional personal income and wealth to the detriment of the people, whose quality of lives they took an oath to improve. Our congress people are there to serve and represent the people’s interests, and nothing else.

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