efficacy

#AAN2018 ā€“ Stem Cell Transplant is Effective Treatment for ‘Aggressive’ MS, Study Shows

Autologous hematopoietic stem cell transplantation, also known as aHSCT, has been shown to be safe and highly effective to treat patients with "aggressive" multiple sclerosis. Tested in 19 patients, transplantation of stem cells was found to induce clinically meaningful improvements in disability. These findings were shared at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, California. aHSCT uses a patientā€™s own healthy bone marrow stem cells, in combination with a much less aggressive chemotherapy and/or radiation regimen, to prepare the patient for the transplant. Previous studies have suggested that aHSCT is an effective strategy to treat patients with highly active relapsing-remitting MS (RRMS) who do not respond to available disease-modifying therapies (DMTs), and international guidelines advocate for its use in patients with "aggressive" MS. To further demonstrate the potential of aHSCT as a treatment for "aggressive" MS, a research team evaluated its safety and effectiveness in MS patients who had not been treated previously with DMTs. A total of 19 patients were treated across several clinical centers: seven patients were from Sheffield, U.K., seven from Uppsala, Sweden, four from Ottawa, Canada, and one patient was from Florence, Italy. All patients received aHSCT between May 2004 and May 2017. In addition to aHSCT, patients were treated with BEAM (carmustine, etoposide, cytarabine, melphalan) chemotherapy plus antithymocyte globulin (ATG) to reduce transplant rejection, or with Cytoxan (cyclophosphamide) with ATG, or the triple combination of Cytoxan, ATG, plus busulfan as conditioning regimens. Patients had a median age of 33 years at diagnosis and received the aHSCT by a median time of nine years after symptom onset. They had a median disability score of 6.5 before the treatment, as determined by the Expanded Disability Status Scale (EDSS). After a median follow-up period of 30 months, patients had a median EDSS score of 2.0, which represented a median improvement of 2 points (the higher the score, the worse the patient's disability level). None of the patients had clinical relapse following the transplant of stem cells. Only three patients developed new brain lesions detectable by magnetic resonance imaging (MRI) at the first six-month follow-up evaluation, but no additional new lesions were detected in the following scans. The adverse effects reported during the study were comparable to those previously observed in similar treatments. No deaths related to the treatment were reported. Based on these preliminary results, the researchers concluded that aHSCT is ā€œsafe and highly effective in inducing rapid and sustain remissionā€ in highly active MS, and "was associated with a significant improvement of [patientā€™s] level of disability.ā€ ā€œaHSCT should be considered as first line therapy in patients with ā€˜aggressiveā€™ MS,ā€ the team concluded. Another study presented at the AAN 2018 meeting further supports these findings, demonstrating the superior effectiveness of aHSCT over conventional DMTs for RRMS.

Lemtrada Prevented Progression of Multiple Sclerosis for Five Years, Study Shows

Two short courses of Lemtrada prevented multiple sclerosis from becoming active and progressing for five years, a study reported. Lemtrada's maker,Ā Sanofi-Genzyme,Ā said the study covered the two-year CARE-MS II Phase 3 clinical trialĀ (NCT00548405) and a long-term extension (NCT00930553) trial of people with relapsing-remitting MS. In addition to demonstrating Lemtrada's effectiveness, the study showed that it was safe, researchers said. The Phase 3 trial participants had had an active disease, with at least two relapses in the two years before the study and an inadequate response to earlier treatment. The trial compared Lemtrada's effectiveness with that ofĀ Rebif. The Lemtrada group receivedĀ 12-mg doses for five consecutive days at the start of the study and three consecutive days a year later. Ninety-three percent of the 435 patients who completed the trial enrolled in the extension, which followed patients for another three years. Remarkably, 60 percent of patients required no additional treatment after the two years of the Phase 3 study. Among the 376 patients who required more treatment, 30 percent had one additional Lemtrada course, 10.4 percent had two, and 1.6 percent had three. A small proportion of patients also received other disease-modifying treatments. The most common reason for additional treatment was relapse. Nevertheless, Lemtrada reduced annualized relapse rates to only 0.18 of patients by the fifth year. In addition, during the five years, 75 percent of patients experienced no worsening of their disability over six-month cycles. And 49Ā percent of patients' disability improved. Researchers also tracked patients' scores on the NEDA ā€” or No Evidence of Disease Activity ā€” index. The composite measure takes into account relapses, disease activity detected in MRI scans, and disability progression. In year five, 58 percent of patients achieved NEDA, slightly more than the 53 percent in year three. Another important finding was that patients' loss of brain tissue slowed in the first two years, and dropped further during the extension. Researchers also noted that adverse events dropped during the extension trial. Ninety-six percent were mild or moderate, and no patient left the study because of side effects. The rate of infusion-associated reactions was lower in the extension study than in the Phase 3 study. Patients who did have a reaction most often experienced headache, fever, or rash. Infections did not become more common with accumulating Lemtrada doses and, again, were less common in the extension trial. Patients most often developed colds or urinary tract infections. Autoimmune reactions against the thyroid gland were relatively common, however. Thirty-eight percent of patients developed them over the five years. Most were moderate in severity. Four patients developed various types of cancers. Researchers also examined Lemtrada in the CARE-MS I clinical trial and its extension trial. TheyĀ reportedĀ long-term outcomes and safety findings similar to those in the latest study. Overall, the newest results demonstrated that Lemtrada slowed disease progression over five years inĀ relapsing-remitting MSĀ patients who failed to respond to previous therapy.

Long-term Lemtrada Treatment Benefits Demonstrated in Extension Study

A five-year study demonstrated that Sanofi-Genzymeā€™s Lemtrada (alemtuzumab) provides long-term benefits forĀ relapsing-remitting multiple sclerosis patients, reducing relapse rates and preventing the progression of the disease. Importantly, most patients required only the standard two-phase treatment course. Few needed additional courses because of relapse or new brain lesions. The study,…

New Innate Analysis Fails to Show Clinical Benefit of MIS416 for Secondary Progressive MS Patients

A new analysis of Phase 2 clinical data on Innate Immunotherapeuticsā€™ investigational drug MIS416 to treat secondary progressive multiple sclerosis has confirmed that the drug failed to improve neuromuscular function or patient reported outcomes. The initial evaluation of data obtained from the one-year trial, announced in June, showed disappointing results. These results, gleaned from 70 patients who were randomly designated to receive either weekly injections of MIS416 or a placebo control, failed to demonstrate significant differences or clinically meaningful improvements in patients treated with MIS416 compared to those in the control group. After this initial setback, the Australian company sponsored an additional analysis of the trial results to identify any potential subgroup of clinical responders that could benefit from MIS416 and who might have been masked in the first population-based analysis. However, this post-hoc analysis also failed to show any positive effects of MIS416. Although the detailed report of this second analysis has not yet been released, the Sydney-based company conceded that the final outcome will not change. "All previous reports of MIS416 making a meaningful difference in the lives of many patients must either be dismissed as a very robust placebo effect or the trial failure is attributable to some other reason. It is my view that there may be other reasons," Innate CEO Simon Wilkinson said in a press release. "Patients with SPMS have a complex mix of symptoms and their disease can't be monitored by a simple blood test or MRI scan," he added. "We used the best assessment tools available as recommended by expert practitioners in MS, but we suspect they weren't sensitive enough to pick up the small but potentially significant changes that can lead to a substantial impact on patients' activities of daily living and quality of life." The lack of efficacy of MIS416 shown by the trial results is inconsistent with previous clinical experience, and the benefits reported by those receiving MIS416 for the past eight years.

If We Took a Holiday (from Our Meds) It Could Be So Nice!

I was a teenager during the 1980s and cannot say the word ā€œholidayā€ without Madonna’s song playing briefly in my head. For most holidays the perky music seems okay, but for discussion of a “drug holiday”Ā it feels a bit off. Sort of like, ā€œYay! You have a chronic…

Generic Form of Copaxone, GTR, Safe and Effective, Study Confirms

An extension trial assessing generic glatiramer acetate (GTR) treatment in multiple sclerosis (MS) patients found that the formulation is as safe and effective as Copaxone (branded glatiramer acetate), and that switching to GTR is well-tolerated. The findingsĀ were in theĀ study, ā€œSwitching from branded to generic glatiramer acetate:…

#ECTRIMS2016 – Lemtrada and Tysabri Seen as Most Effective Therapies for MS in 5-Year Study

A five-year study comparing theĀ efficacy of different treatments for relapsing-remitting multiple sclerosis (RRMS) foundĀ that, in general, Lemtrada (alemtuzumab) and Tysabri (natalizumab) are more effective asĀ therapies than Gilenya (fingolimod) and interferon Ī². StudyĀ results were presentedĀ in an oral presentation, ā€œComparison of 5-year treatment outcomes between alemtuzumab versus natalizumab, fingolimod and interferon Ī²-1a,ā€…

Patients Need To Know What Drugs Do Inside Our Bodies

Oral therapy Tecfidera (dimethyl fumarate) seems to be emerging as a first-line treatment for relapsing multiple sclerosis ā€“ according to Biogen, the company that developed it. Thatā€™s good news, and something we need to know. The company agrees that for some time, the long-term effectiveness and safety of this oral…