Friday, 24 April 2015

Times are changing as this years AAN had some good news

So this week at the AAN we have seen the birth of  neuroprotection and maybe remyelination and the our data will be presented today, so not bad 2015 so far in my books. 

The grumpies will say this is all well and dandy but they want access to the treatments now! 

I will have to be  grumpier and say that this is not the process and you know that!


The sneeky news was the results of high dose Biotin.

[PL2.002] Effect of MD1003 (High Doses of Biotin) in Progressive Multiple Sclerosis: Results of a Pivotal Phase III Randomized Double Blind Placebo Controlled Study
Ayman Tourbah,Christine Lebrun Frenay,Gilles Edan,Michel Clanet,Caroline Papeix,Sandra Vukusic,Jerome De Seze,Marc Debouverie,Olivier Gout,Pierre Clavelou,Gilles Defer,David Laplaud,Thibault Moreau,Pierre Labauge,Bruno Brochet,Frederic Sedel,Jean Pelletier

OBJECTIVE: To evaluate the efficacy of Biotin 300 mg/day over placebo in patients with progressive multiple sclerosis (PMS).

BACKGROUND: Biotin is a water-soluble vitamin acting as a coenzyme for carboxylases involved in key steps of energy metabolism and fatty acids synthesis. Among others, biotin activates acetylCoA carboxylase, a potentially key-enzyme in myelin synthesis. In one previous open-label pilot study involving 23 patients, it was found that high doses of biotin (100 to 600 mg/day) resulted in progressive and sustained improvement of disability in primary and secondary PMS patients.
DESIGN/METHODS: This is a randomized, double-blind, multicenter, placebo-controlled (2:1) trial of oral biotin 300 mg (3 x 100mg / day in patients with secondary or primary PMS with EDSS between 4.5 and 7 and evidence of EDSS progression within the past two years. Treatment duration was 48 weeks. The primary endpoint was the proportion of patients who improved at M9 and confirmed at M12, defined as decreased EDSS (by at least 1 point for EDSS ≤5.5 and .5 point for EDSS ≥6) or improved TW25 of at least 20%. Other endpoints included MSWS, CGI, % patients with stable or worsened EDSS, SF36, FIS, 9HPT.
RESULTS: The last patient is scheduled to complete the study January 2015. Baseline characteristics: 154 subjects from 16 sites across France were randomized; mean age 51.4; mean disease duration 16.6 years; 41% had PPMS and 59% had SPMS. The mean EDSS score was 6.1. The database will be locked by March 2015. Detailed results from primary and other outcomes will be presented.
CONCLUSIONS: This trial will evaluate the efficacy of Biotin 300 mg/d in a randomized, placebo-controlled trial. Effects of treatment with high doses of biotin in patients with PMS will be discussed in the context of future development of high doses of biotin as a novel potential treatment in PMS.



You are asking what next. The answer is at least on more trial.


Estimated Enrollment:105
Study Start Date:October 2013
Estimated Study Completion Date:January 2016
Estimated Primary Completion Date:June 2015 
(Final data collection date for primary outcome measure)

So here is the trial

Inclusion Criteria:
  1. Diagnosis criteria of MS fulfilling revised Mc Donald criteria (2010) i.e. people have MS
  2. Uni-or bilateral optic neuropathy with worst eye VA≤ 5/10 confirmed at 6 months
  3. Worsening of visual acuity during the last three years the anti-LINGO suffered because they waited three weeks, so at 3 years its a tall ask and says this is not neuroprotection it is either repair or symptom control.
  4. Informed consent prior to any study procedure
  5. Patient aged 18-75 years

Exclusion Criteria:
  1. Optic neuritis relapse within the three months before inclusion (So if the drug works it is probably working on inactive disease so symptom control or repair)
  2. Normal RNFL at OCT 
  3. Presence of other ocular pathology 
  4. Bilateral visual acuity <1/20
  5. Visual impairment caused by ocular flutter or nystagmus
  6. Pregnancy or childbearing potential woman without contraception
  7. Any general chronic handicapping disease other than MS
  8. New treatment introduced less than 3 months prior to inclusion or less than 1 month for Fampridine
Outcome Change from baseline of the best corrected visual acuity at 100% contrast

11 comments:

  1. Time is brain - Prof G's words. Some of the results at the AAN look promising. However, results of trials are not treatments. I'm guessing pills and injections from some of the trials will still be 5-10 years away. It's great to see that Lemtrada benefits are sustainable. I also like the early stem cell results reported by the Tisch MS centre. Lots going on. So I'm less grumpy. I'm also looking forward to Charcot Project t results and signing up to the spasticity trial (I can see the $ signs in Prof Mouse's eyes). I hope Prof G brings you back something nice from Washington. As he reclines on his first class seat on the flight home, perhaps he could list six big things that came out of the AAN meeting and share these with us. He needs to vote for the Tories in the election to retain his Non Dom status. Have the weekend office mice doctors - you upped your game in recent months.

    ReplyDelete
    Replies
    1. I bet he has been "Doing take me Home toto" as this meeting in particular is very long. Looking forward to my West Wing gift a life size replica of Pres Bartlet but I suspect the gift we will get is "I had an idea".

      Unfortunately we never get the weekend off being an MD is 24/7/365 those are the rules

      Delete
    2. Bartlet for America!

      Delete
  2. So I wonder what the downside would be to mirroring this study at home ? There doesn't seem to be anything hidden, ie no secret ingredient.

    Regards as always.

    ReplyDelete
    Replies
    1. Today we will find out as they will present the data including the side effects, I've been to health food shop to ask about sales.

      Delete
    2. Will watch with interest. Thanks.

      The results of our un blinded, un randomised, no placebo arm, one center trial on a patient with ppms and a EDSS of 8.5 with a mean age of 43 ( soon to be 44) will not be published in a peer reviewed journal. ;-)

      Regards as always

      Delete
    3. Do let us know Andy and good luck!
      All the best
      MD2

      Delete
  3. Any increase in sales?
    I've been taking 120mg a day for a couple of weeks working gradually up to it
    No real difference that I can notice, I have had additional issues so maybe progression?
    Only side effect not sure if it's noted is increased urination and itchy scalp dry skin? I've read people who take Biotim for hair treatment have similar symptoms

    ReplyDelete
    Replies
    1. The paper published in March indicated no benefit from 100mg and it took 8 months for benefit to be reported based results presented today 90% did not improve.

      Delete
  4. Where are the tisch results? And are these about the mesenchymal stem cells?

    ReplyDelete
    Replies
    1. Just google tisch ms centre and the results (very early results are there).

      Delete

Please note that all comments are moderated and any personal or marketing-related submissions will not be shown.