WASHINGTON -- Some of the hottest research presentations were presented in "data-blitz" style at the Emerging Science session of this year's American Academy of Neurology meeting.
Those included findings from an early-stage trial of aducanumab for Alzheimer's disease, the first evidence of remyelination with a monoclonal antibody, and the potential utility of phenytoin in optic neuritis.
鶹ý picked a handful of additional presentations from this session to highlight in this round-up.
Action Points
- Note that this study was published as an abstract and presented at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal.
No Help for Progressive MS
Oral fingolimod (Gilenya) didn't slow disease progression in primary progressive multiple sclerosis in a phase III trial.
The INFORMS study enrolled 970 patients to either the Sanofi drug or placebo to look at a composite primary endpoint based on change from baseline in Expanded Disability Status Scale (EDSS), 25-Foot Timed-Walk Test (25-TWT), and 9-Hole Peg Test (9-HPT).
At 3 months, neither the primary composite endpoint nor the EDSS endpoint alone was met, , of Mount Sinai Hospital in New York, reported.
"Different therapeutic strategies may be required to treat primary progressive multiple sclerosis," Lublin said.
Tysabri Shot as Good as Infusion
A subcutaneous formulation of natalizumab (Tysabri) worked just as well as the standard infusion in relapsing-remitting multiple sclerosis patients.
In the REFINE study, monthly subcutaneous dosing was comparable to the standard 300-mg intravenous dosing in terms of both clinical and MRI efficacy, according to Maria Trojano, MD, of the University of Bari in Italy.
But a 12-week dosing regimen of the subcutaneous injection was stopped early because it was associated with an increase in relapse activity and in MRI activity, Trojano said.
She added that the safety of the Biogen drug is consistent with the "well-established safety profile of intravenous natalizumab" in previous studies.
Pot May Help Epileptic Kids
Cannabidiol (Epidiolex) reduced seizure frequency in pediatric epilepsy patients who'd had little success on previous therapies.
In an open-label study of 137 patients, patients had a 46% reduction in seizures over 12 weeks, Orrin Devinsky, MD, of NYU Langone Medical Center, reported.
They also found that 9% of patients had no seizures while being treated with the drug, and that it had good safety and tolerability with a low rate of withdrawal.
Devinsky called for controlled trials to fully characterize safety and efficacy.
Gel for Severe Migraine
A topical NSAID may provide relief for migraine pain, , of Duke University, reported.
In the randomized, crossover, double-blind, placebo-controlled study of 48 patients, those who applied topical ketoprofen (ELS-M11) directly to the face had better symptomatic relief and diminished pain compared with those on placebo.
Specifically, 45% of headaches treated with the drug had sustained pain relief from 2 to 24 hours compared with 15% of headaches treated with placebo, they found.
Liedtke said the results support the idea that topical NSAID therapy may be an effective treatment for acute migraine. The drug is being developed by Achelios Therapeutics.
Hope for Rett Syndrome
A new drug for Rett syndrome showed promising efficacy and safety data in an early study reported here by , of Texas Children's Hospital in Houston.
The phase II study of NNZ-2566 -- a [1-3] IGF-1 analog -- enrolled female 56 patients, with a mean age of 25.3, to either a 70 mg/kg dose of the drug or to placebo for a total of 28 days.
Glaze said those on the drug had a significant clinical benefit as measured by clinician and caregiver measures of symptom severity (P=0.023).
The drug, being developed by Neuren Pharmaceuticals, also appeared to be well-tolerated, he said, and there were no time- or dose-dependent adverse effects.
Drug Diminishes Huntington's Chorea
An investigational drug for Huntington's reduced chorea in an early study, according to , of Boston University.
The drug, SD-809, is a deuterated form of tetrabenazine, a VMAT-2 inhibitor used for hyperkinetic movement disorders. Deuterium, a nonradioactive, naturally occurring form of hydrogen, can alter the metabolism of small molecule drugs without changing their pharmacology, Frank said.
The randomized, double-blind, placebo-controlled study randomized 90 patients to either the drug or placebo over 12 weeks, at a dose of 6 to 48 mg.
They found that the drug was associated with a 4-point improvement in the Total Maximal Chorea (TMC) scale compared with placebo, translating to a 21% reduction in chorea (P<0.0001).
"In my clinical experience, treating Huntington's disease for 10 years, this reduction in chorea is meaningful," Frank said. The drug is being developed by Auspex Pharmaceuticals.
Drug Quells Limb Spasticity
OnabotulinumtoxinA improved ankle plantar flexor muscle tone in patients with post-stroke lower limb spasticity.
In a phase III trial, 468 patients were randomized to the Allergan drug or to placebo over a 12-week period.
Those on the drug had significantly greater improvements in ankle muscle tone as measured by the Modified Ashworth Scale than those on placebo (P=0.01), , of McGill University in Montreal, reported.
He said the drug was also well tolerated and had no new safety signals. The most common adverse events included injection site pain and pain in the extremity.
Disclosures
The authors disclosed various relationships with industry.