Not a nice therapy, but looks like it helps if used early and agressively. On a side note, it's kind of sad that they are still discovering new stuff about this drug. It's been used at least experimentally in MS since the late 60's I believe. Better late than never?
High Dose Cyclophosphamide Achieves Rapid Clinical Improvements in Early, Aggressive Multiple Sclerosis: Presented at AAN
April 6, 2006 -- Preliminary data from a study utilizing 50 mg/kg/day of cyclophosphamide (HiCy) for 4 consecutive days suggest it is safe and provides rapid clinical and radiological improvements in patients with early, aggressive multiple sclerosis (MS).
Peter Calabresi, MD, Department of Neurology, Johns Hopkins University Hospital, Baltimore, Maryland, presented the data here at the 58th annual meeting of the American Academy of Neurology (AAN).
"This is a study using extremely high doses of cyclophosphamide, a drug that's been tested for many years, and used mostly in monthly pulse protocols," Dr. Calabresi said during his poster presentation on April 4th. "The concept came from our cancer center group, who has treated approximately 3000 patients with malignancies and about 200 patients with autoimmune diseases, but not MS."
Patients who were entered into the open-label protocol had at least two gadolinium enhancing lesions present on magnetic resonance imaging, and one clinical exacerbation within the past year. They were on conventional MS therapy, yet had sustained increases of >1.0 on the Expanded Disability Status Scale (EDSS).
The protocol requires a 4-day hospitalization period, after which participants became outpatients.
"We've had no deaths on this protocol," Dr. Calabresi said.
"Clinically, the results recapitulate what has been seen in other, aggressive bone marrow protocols, where patients with early, very active disease do very well. Their EDSS goes down, they stabilize for long periods of time," he explained.
An advantage of the HiCy protocol, however, is that it does not require bone marrow stem cell transplantation. "We have found that the morbidity and mortality is much lower or non-existent with this protocol, so it's extremely well tolerated and much cheaper because you don't have to harvest stem cells and give them back to the patient," he said.
The results show reductions in lesions from baseline in all 8 patients. Additionally, 5 of the participants experienced sustained decreases in their EDSS following HiCy therapy, while 3 patients had similar or increased EDSS.
"The patients who went into this study with high EDSS do not do well; they likely already had fixed damage" Dr. Calabresi said.
"There were two patients that started out at 6.25 [EDSS] that continued to slowly progress, which tells us that if the disease is relatively advanced then this treatment is not worth the risk. If, however, it's relatively early stage disease, and worsening quickly, then this may be a therapeutic strategy," he said.
Adverse events (AEs) and side effects related to treatment reported during the protocol include nausea, alopecia, anemia, thrombocytopenia, leucopenia, loss of immunization, febrile neutropenia, and amenorrhea. No serious AEs have been reported to date; however, one patient has been hospitalized due to a suspected exacerbation.
With respect to future plans for the protocol, Dr. Calabresi said, "We're now following the participants for 1 year, and we want to see what happens at 2 years."
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