Ionis Pharmaceuticals' investigational agent ION464 (BIIB101) has shown promising early results in patients with multiple system atrophy (MSA). The HORIZON trial (NCT04165486), a phase 1/2 single- and multiple-ascending dose study, indicates that ION464 is safe and well-tolerated, with no serious adverse events or deaths related to the drug reported thus far. ION464 is designed to inhibit the production of the alpha-synuclein protein, a potential therapeutic strategy for neurodegenerative disorders including Parkinson's disease (PD) and MSA.
The data, presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), included baseline characteristics of the first 22 participants: 36% female, 91% White, 45% with MSA-Parkinsonism, and 73% with probable MSA. Treatment-emergent adverse events included falls (25), urinary tract infection (12), headache (6), contusion (5), and neck pain (3).
OnabotulinumtoxinA for Essential Tremor
Another study presented at the 2024 MDS Congress detailed the design of the ELATE trial (NCT), a phase 2b clinical trial evaluating the safety and efficacy of onabotulinumtoxinA (onabotA) compared with placebo for the treatment of adult upper limb essential tremor (ULET). The trial aims to enroll 174 participants across 40 sites in the United States and Canada, with completion expected in 2024.
The ELATE study is designed to test four regimens involving onabotA or placebo administered unilaterally or bilaterally over 18 weeks. The primary efficacy measure will be changes on the Tremor Disability Scale-Revised (TREDS-R) score across seven unilateral items. The hope is that the ELATE study results could reveal valuable insights into the effectiveness and safety of onabotA for ULET, potentially offering a better risk-benefit profile than current oral therapies.
Dietary Strategies in Parkinson's Disease
An ongoing crossover study (NCT05469997) is examining the effects of two dietary strategies on patients with Parkinson's disease (PD). Results indicate that while participants responded positively to Mediterranean-style diets, combining them with ketogenic-style interventions presented several challenges.
The trial involved 50 participants following two 8-week dietary interventions, separated by an 8-week washout period: a high-fat, low-carbohydrate Mediterranean diet (MEDI-KD) and a standard Mediterranean diet supplemented with medium-chain triglycerides. Participant interviews revealed positive sentiments toward professional nutritional guidance, increased dietary mindfulness, and Mediterranean-style cuisine. Negative feedback focused on the restrictive nature and social impracticability of the ketogenic diet, as well as the gastrointestinal adverse effects of MCT oil.
