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.
- Diagnosis criteria of MS fulfilling revised Mc Donald criteria (2010) i.e. people have MS
- Uni-or bilateral optic neuropathy with worst eye VA≤ 5/10 confirmed at 6 months
- 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.
- Informed consent prior to any study procedure
- Patient aged 18-75 years
- 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)
- Normal RNFL at OCT
- Presence of other ocular pathology
- Bilateral visual acuity <1/20
- Visual impairment caused by ocular flutter or nystagmus
- Pregnancy or childbearing potential woman without contraception
- Any general chronic handicapping disease other than MS
- New treatment introduced less than 3 months prior to inclusion or less than 1 month for Fampridine