Timothy C. Hain, MD Page last modified: June 18, 2014
For more general information about cerebellar disorders see here.
These medications are all close relatives. They all are sedating, and all sometimes improve balance. In older people, they can increase falling.Acetazolamide (Diamox) (1-3 tabs/day -- 250 mg). This diuretic helps some persons with periodic ataxia. We have tried this medication often with a reasonable (20%) success rate for periodic ataxia.
Baclofen (Lioresal). This medication has been shown to reduce periodic alternating nystagmus, which may be a consequence of damage to the cerebellar nodulus. We have tried this medication on many occasions, with minimal results.
3,4,Diaminopyridine. This is an "orphan drug" that has been reported to increase cerebellar purkinje cell excitability. Little information is available about long term effects or side effects. Perioral paresthesias (numbness), headache, fatigue, difficulty sleeping, and (in excessive doses) seizures can occur. This drug can also be used to increase release of ACH as in Eaton Lambert syndrome. Typical dosage is 5 to 10 mg, 3 to 5 times/day. This medication has been used to reduce downbeating nystagmus. (Strupp et al. 2003).
4-Aminopyridine has also been shown to improve patients with episodic ataxia type 2 (Strupp et al, 2004) as well as many other cerebellar disorders. We have tried this medication in our practice on many occasions, with relatively little success.
INH (100 mg TID, increase by 300 mg every 3 days to a toal of 1200 mg. Liver function test every week. Pyridoxine along with INH dose of 50 mg TID). This drug has helped some persons with Multiple Sclerosis and unsteadiness. We have not tried this medication in our practice.
TRH (0.5 mg IM every other day). TRH is an "orphan drug". It costs $18.50/ampoule. It must be given by injection. Results on average are about 1/3 improvement in balance (according to Japanese literature). Improvement starts quickly, and lasts about 2-3 weeks after last injection. This drug is obviously expensive -- costs about $70/week. We have not tried this medication in our practice.
Varenicline (Chantix). This drug, approved by the FDA for smoking cessation, has recently been reported useful for treatment of cerebellar ataxia (Zesiewicz et al, 2009). It is extremely surprising to find an agent that will ameliorate disorders caused by genetic damage to neurons. It is also disturbing that the number of individuals in the trial (7) was exactly the same as the number of authors on the study. We are frankly very dubious. Nevertheless, this drug might be worth a try.