Lipitor Possibly Beneficial in Early MS
Although the primary endpoint was not achieved in a recent phase 2 study, high-dose atorvastatin (Lipitor; Pfizer), like the interferon betas, may reduce the likelihood of full-blown multiple sclerosis after a first attack.*
The results of the placebo-controlled trial (N = 81), which were reported last week at the annual meeting of the American Academy of Neurology in Toronto, showed that atorvastatin 80 mg/d significantly reduced the odds of new T2 lesions on brain MR images at 12 months. However, the percentages of patients developing 3 or more new T2 lesions or 1 or more clinical relapses (the combined primary endpoint) were not significantly different between atorvastatin and placebo treatment (49% vs 56%; P = .65).** And while the odds of remaining free of active (ie, gadolinium-enhancing) MR lesions were higher with atorvastatin, this finding also did not reach statistical significance.
The study was originally designed to enroll 150 patients; however, because of slow recruitment, it was stopped after treatments were randomized to 81 patients. The study was sponsored by the NIAID and managed by the Immune Tolerance Network, which receives support from Pfizer and Biogen Idec.
The rationale for assessing atorvastatin in MS relates to its ability to modulate immune reactions and reverse disease in rodent models (ie, experimental autoimmune encephalomyelitis). Open-label studies also suggested clinical benefit with atorvastatin in relapsing-remitting MS.
At MedPage Today, the lead investigator, UCSF's Emmanuelle Waubant, indicated that the results are hypothesis generating—meaning that they warrant phase 3 study.
* AKA clinically isolated syndrome (CIS) manifesting, for example, as optic neuritis, spinal cord syndrome, or brainstem dysfunction.
** Those patients who experienced the primary endpoint were offered weekly IM interferon beta (Avonex; Biogen Idec).
T2-weighted supraventricular horizontal MR image from Harvard's Whole Brain Atlas. Multiple subcortical MS lesions are evident, including a very prominent lesion in the patient's right (left to the viewer) frontal area. The web site also offers a very cool time-lapsed movie of developing MS lesions.
