"A total of 21 patients with confirmed SPMS participated in the study, divided according to the safety (n = 11) and efficacy (n = 20) sets. These patients were in a phase of secondary progression of the disease, as seen from the mean duration of SPMS of 4.3 and 4.6 years, respectively. Their mean MS durations since the first manifestation were 16.6 and 17.2 years, respectively, and they were still under active disease according to the number of attacks they had in the previous year (means, 1.4 and 1.4, respectively). Their mean EDSS scores were 4.95 and 5.05, respectively.
"All of the patients either stabilized or even improved over the observation period of 1 year," Dr Wiendl indicated. The annual relapse rate of 1.5 prior to study entry decreased significantly to 0 by the end of the study year (P <.016).
Within the MRI analyses, there was a trend for a reduction in Gd-enhancing lesions, although, as Dr. Wiendl said, "What we saw by the end of this study was that some of these patients seemed to be having recurring activity, which could be an indication that the 3-monthly maintenance pulses are not sufficient to be anti-inflammatory."
Therefore, the researchers concluded that not only was treosulfan safe and well tolerated, but nine of the 11 patients remained on treosulfan for the complete study period, during which they showed clinical stabilization or improvements, as judged by their EDSS and MSFC scores. Furthermore, no clinical relapses were seen during the treatment period, and the mean numbers and volumes of their T2 and T1 lesions did not change during the year of treatment.
These results have thus moved things forward with the establishing of a randomized phase 2 study, which is currently in recruitment, Dr. Wiendl said."