Table 1.
Medications with potential benefits in ataxia
| Therapy (Class Evidence) | Trial Dosing of Oral Medication | Study Population (No. of Patients) | Study Type | Rating Scales | Clinical Ataxia Outcomes | Common Adverse Events |
|---|---|---|---|---|---|---|
| Riluzole4 (level B)5 | 50 mg BID/day | Mixed (38) | Randomized, double‐blind, placebo‐controlled | ICARS | Improved static (gait), kinetic (tremor), and dysarthria subscores | Elevated liver ALT, vertigo |
| Varenicline 9 (level B) 5 | 1 mg BID | Only SCA3 (18) | Randomized double‐blind placebo controlled | SARA, 25‐foot timed walk test, BDI | Improved gait, stance, and rapid alternating movements on SARA, lower BDI, and faster times on 25‐foot walk test | Nausea, vivid dreaming, leg tingling |
| Amantadine 7, 8 (level C) 5 | 7 mg/kg per day | Only AT (13) | Open label, prospective |
1. ICARS 2. UPDRS 3. Abnormal involuntary movement scale |
Improved total composite scores and improved static, kinetic components of ICARS and bradykinesia of UPDRS | Constipation, sleepiness, headache, rash |
| 200 mg/day | Mixed (OPCA vs. FA) (57) | Double‐blind, randomized trial |
1. Simple visual and auditory mean reaction time 2. Movement time |
Improvements in both reaction and movement time for OPCA, but not FA | Appetite loss, nightmares, mood changes | |
| Gabapentina, 22 | 400 mg × 1 then 900 to 1,600 mg daily | Mixed (13) | Open‐label study | ICARS | Improvements in gait and body sway | Mild transient giddiness, abdominal pain, and impotence |
| Pregabalina, 23 | 75 mg TID | Only CCA (2) | Single‐blind, placebo‐controlled study | SARA | Improvements of overall SARA scores | None reported |
| Zolpidema, 24 | 10 mg/day | Only SCA2 (5) | Case series | No formal scales | 4 of 5 had at least mild improvements in ataxia, tremor, and titubation | None reported |
| Lamotriginea, 25 | Not reported | Only SCA3 (6) | Open‐label, self‐controlled |
1. One leg standing test 2. Tandem gait index |
Tandem gait and balance (such as standing on one leg) | None reported |
| Topiramatea, 26 | 150 mg/day | Multiple Sclerosis (1) | Case report | Expanded Disability Status Scale | Improvements in total distance walked, speech, cerebellar tremor, and fine motor skills | None reported |
| Acetozolamidea 31 | 500 mg/day | Only SCA6 (6) | Open‐label prospective |
1. ARS 2. Stabilometry |
Improvements in total ARS, gait, and diminished body sway | Mild transient hand dysesthesia, asymptomatic mild hypotension, ureteric calculus |
| IGF‐1a 32 | 0.05 mg/kg per BID | Mixed (13) | Open‐label prospective | SARA | Improved total SARA in SCA3 by 8 months and no clinical change in SCA7 | None reported |
| Acetyl‐dl‐leucinea, 38 | 5 g/day | Mixed (13) | Open‐label case series | SARA | Decreased total SARA scores with specific improvements in gait, finger chase, finger‐to‐nose, heel‐to‐shin, speech, and rapid alternating movements | None reported |
a
No existing level of recommendation, insufficient data for recommendation.