Positive results for progressive MS drug candidate presented at AAN

Research into the inflammatory nature of MS has led to profound progress in the treatment of the relapsing-remitting form of the disease. But this leaves us with many unanswered questions, and no treatment options, for progressive MS. Progressive MS bears severe consequences, including pronounced disability, cognitive decline, and extreme fatigue. As the need for treatments grows, so too do research efforts to help understand the onset and nature of progressive MS.

Encouraging results from one clinical trial conducted by Paris-based biotechnology company MedDay could pave the way for a novel, promising treatment option for people with progressive MS. The phase III, placebo-controlled clinical trial – dubbed MS-SPI – included 154 participants who were randomly selected to receive either MD1003 (treatment being evaluated) or a mock treatment (control) over a period of one year.

MD1003 is a high-dose formulation (300 mg/day) of biotin. Biotin – or vitamin H – is an FDA-approved food additive and Health Canada-licensed natural health product which, at concentrations of 100 – 300 mg/day, is around 10,000 times higher than the recommended daily intake as a food supplement. The high concentration of biotin in MD1003 has been previously shown to play a role in stimulating myelin production and improving the transmission of signals throughout the nervous system; specifically, it activates several proteins involved in energy production and the synthesis of myelin.

The results of the clinical trial, presented at the AAN conference in Washington D.C., showed that 13% of participants who received MD1003 experienced improvements in disability as determined by changes in EDSS (Expanded Disability Status Scale) or 25-foot walk time, compared to none (0%) of participants who received the mock treatment. Secondary analyses reported a 67% decreased risk of progression in the treatment group, although this finding did not reach statistical significance. It was reported that MD1003 was well tolerated, and that the occurrence of adverse events was similar between the treatment and control groups.

A separate trial called MS-ON is a randomized, double-blind, placebo-controlled phase III clinical trial that is currently underway to investigate visual improvement following treatment with MD1003 in participants affected by visual loss from MS-related optic neuritis. Results from the trial are expected in late 2015.

9 thoughts on “Positive results for progressive MS drug candidate presented at AAN

  1. Ted Alden

    I can understand what this might do for myelin production. How does this have any effect or correlation to those brain scans I had that indication the presence of MS?

    1. drkarenlee Post author

      Hi Ted,

      Thank you for your question. According to the Clinical Trials registry, one of the additional outcome measures for the study is to examine brain MRI in a subset of participants. So far, the preliminary results released by MedDay (the company that manufactures the drug and conducted the clinical trial) do not include the imaging data; presumably, those findings will be available once the results are published in a peer-reviewed journal.

      Dr. K

  2. Christopher Alkenbrack

    This is very interesting. The sad thing is that once it has gone through the clinical trial phase for MS, the cost will balloon. You can buy biotin supplements at Wal-Mart for about $12. This has been seen before in MS clinical trials where a very inexpensive medication ends up costing in the thousands of dollars per month once it has gone through the clinical trial phase.

    1. drkarenlee Post author

      Hi Christopher,

      Thank you for your comment. MD1003 is not your regular biotin supplement found on pharmacy shelves. It is a high-dose formulation that is approximately 10,000 times the concentration of the daily recommended intake as a nutritional supplement. Bringing a therapeutic from the laboratory to the market, including research into its safety and efficacy through controlled clinical trials, is an enormously expensive undertaking. Vitamins marketed as nutritional supplements, on the other hand, are regulated in Canada under the licensing requirements of the Natural Health Products Regulations, rather than as drugs under Food and Drug Regulations. Licensing criteria for natural health products are generally less strict, depending on the proposed health claim of the product, which allows costs to be kept down. At the end of the day, the hope is that therapeutics for MS will be reasonably priced and accessible to those living with MS.

      Dr. K

      1. Amit

        Stage 1- We are diagnosed with a progressively-disabling illness.
        Stage 2- There is no cure for this progressively disabling illness.
        Stage 3- There is no treatment for progressive MS, when disability accumulates without relapses/exacerbations/attacks.
        Stage 4- Even medical/clinical/scientific researches to find treatment, much less a cure, for progressive MS, are few and far in between.
        Stage 5- Results of any clinical trials will not necessarily confirm the availability of the drug in the territory where the MS Patient resides.
        Stage 6- Even if the MS Patient can cross all the above hurdles, the price of the drug is generally unaffordable implying, even if a treatment is available, it will remain unavailable.
        The Almighty could not have taken a sterner test for patients with progressive MS.

  3. Elaine Newsomr

    I have been left partially sighted with the MS suffer fatigue,walking difficulties. The trials for progressive MS sound very promising and can’t wait to hear the outcome! Have few friends who suffer from a lot of problems described in the piece. I am on Beta-ferone and have for nearly twenty years I am one of the lucky ones it drastically reduced my relapses.
    I wish you luck in this research it could be the answer to a very high number of people’s problem.

    1. drkarenlee Post author

      Hi Elaine,

      Thank you for your feedback, and I’m excited to hear that your relapses have been well controlled. We’re also excited by these preliminary results, and we will be watching this drug closely over the next few months.

      Dr. K

  4. Brad


    Very new to this. My wife may have a diagnosis confirmed next week, one specialist discussed the results of the MRI. I have a couple of questions. She seemed surprised with the MS diagnosis as the fatigue has been accompanied by pain around C1. My wife has always had a problem with Calcium in her body, kidney stones at 17, gall stones at 27, bunion surgery at 42. We were hoping that the dehabilitation was caused by bone spurs blocking the signal. Maybe the pain around C1 was psycho-somatic and in a weird way, wishful thinking. Pre MRI tests were not supportive of MS.

    But if it is primary progressive MS as her steady decline in function suggests.

    1) Are there significant crossovers between AIDS and MS research, both are autoimmune diseases. CD4 receptor cells play a role in both diseases though it appears not quite equally.

    2) Has HGH been used in a mild dose to delay the onset of Primary Progressive MS. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2990924/
    Progressive MS seems to be a failure of the autoimmune system with age.

    Just early stages, pardon my neophyte questions.

    3) To discuss with my teenagers I used the iphone cable, ALS is the disintegration of the cable, spinal damage is the cutting of the cable, MS would be the breaking of the protective sheath that happens at the cable ends, after a while the cable will stop working as signals disintegrate. To continue the analogy, muscles tone and physical fitness are like tape over the broken sheath, it will fix the cable but can delay the inevitable decline in signal. For me muscles , the spine and nerves are like a building construction. Your spine will only take load to support your muscles as rebar to concrete, When the concrete falls away, the rebar becomes overloaded and breaks, or is bent. A strong muscular system will act like the tape on a iphone cable and slow the decline.

    4) To continue that analogy, are their new and clever ways of restoring the myelin sheath. Could HGH, used in combination with other drugs that redirect its focus like AIDs cocktails, play a role in the regrowth of the neural fibre while at the same time improving muscle strength.

    You can respond privately if these questions misdirect the intent of your blog.

    1. drkarenlee Post author

      Hi Brad,

      Thank you for the insightful questions, and I wish your wife and you all the best in these difficult times.

      With regard to your questions:
      1. Although this is not a major area of research, there are a handful of researchers who are investigating the association between HIV/AIDS and MS by looking at related disease mechanisms. Researchers have made the interesting observation that people with HIV appear to have a significantly reduced risk for MS, although it’s not know if this is because of the dampening of the immune system caused by chronic HIV infection, or because of antiretroviral meds. To test the latter, a small pilot trial was started in 2013 to investigate whether raltegravir – an antiretroviral drug used to treat HIV infection – is effective in preventing progression of relapsing remitting MS, based on a single case study suggesting that raltegravir may have therapeutic benefits for people affected by MS. Results of the trial have yet to be published.
      2. There is some evidence to suggest that human growth hormone (and insulin growth factor-1, which is stimulated by growth hormone) can positively affect myelin repair (which feeds into your next question). To my knowledge, there is currently one pilot trail examining the safety of recombinant HGH as a potential remyelination therapy in people living with MS. We’re keeping an eye out for any results of the trial.
      3. and 4. I think that your analogy is fantastic! The only other piece that I would add is that in addition to breaking of the protective sheath around the cable, in some cases there would be a persistent deterioration of the underlying cable, much like people with progressive forms of MS experience damage to their underlying nerve fibres. With regard to remyelination therapies, this is a key area of focus in MS research. The experimental compound anti-LINGO-1 is currently undergoing clinical trials to test its safety profile and remyelination potential, while other approved therapies such as fingolimod and glatirimer acetate are suspected to have remyelinating properties. Finally, researchers are attempting to find new molecular targets that hopefully hold some promise for promoting remyelination

      I hope that answers your questions!

      Dr. K


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