Research news

Way Ahead 2009;13(2):4

Stem cell study shows promise in RRMS
Oral formulations show promise in phase III studies
New study links lack of vitamin D with increased risk of MS

Stem cell study shows promise in RRMS

A team of Chicago-based researchers reported on a stem cell study which involved 21 people with relapsing remitting MS who had experienced two relapses in the previous year despite treatment with beta interferon.

In preparation for stem cell transplantation all study participants undertook a conditioning course of immune suppressing drugs (cyclophosphamide and either alemtuzumab or rabbit antithymocyte globulin).

The participants were followed for an average of three years. All 21 showed no worsening of disability as measured by the EDSS scale, and 17 improved by at least one point. 16 people experienced no further relapses following the stem cell treatment.

The researchers recognise that larger randomised trials are needed to confirm these results and pointed to a second, larger, multicentre trial with study centres in North and South America.

Burt RK, Loh Y, Cohen B, et al.
Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study.
Lancet Neurol 2009;8(3):244-253.

Hematopoietic stem cell therapy for patients with inflammatory multiple sclerosis failing interferon: a randomized study.
Available from: URL: http://clinicaltrials.gov/ct2/show/NCT00273364.

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Oral formulations show promise in phase III studies

Fingolimod

A one-year phase III study of the investigational oral drug FTY720 (fingolimod) showed a significant reduction in annualised relapse rate compared with treatment with an injectable interferon beta-1a.

The study had three treatment arms whereby 1,292 study participants were randomized to receive either oral fingolimod 0.5 mg once daily, oral fingolimod 1.25 mg once daily, or the active comparator interferon beta-1a given once weekly by intra-muscular injection.

At one year, patients given fingolimod 0.5 mg saw a 52% reduction in annualised relapse rate compared with those on interferon beta-1a. The fingolimod 1.25 mg dose group saw a 38% reduction in annualised relapse rate compared with the interferon beta-1a group.

The drug manufacturer says regulatory submissions remain on track to be completed in the US and EU at the end of 2009.

Source: Novartis

Cladribine

Results of a two year phase III study comparing cladribine against placebo indicated that cladribine significantly reduces relapse rates in people with relapsing remitting MS. Based on these positive results, the manufacturers, Merck Serono, hope to submit the drug for licensing later this year.

1,326 people with relapsing remitting MS were randomised to one of three different treatment groups. Cladribine tablets were administered in two dosage arms; one treatment group received two courses for the first and second year; the other treatment group received four courses for the first year and two courses for the second year. Each course consisted of once daily administration for four to five consecutive days. The third treatment group received a placebo.

The primary end-point of the study was reduction in clinical relapse rate. A 58% relative reduction in annualised relapse rate was seen in the low total dose treatment group and 55% in the high total dose treatment group. The study will be extended for a further two years to provide data on the long-term safety of cladribine tablets administered for up to four years.

Further clinical trials are looking to determine the safety and efficacy of oral cladribine as a monotherapy in people who have experienced a first clinical event suggestive of MS; and as an add-on therapy to people with relapsing forms of MS who continue to show signs of active disease while on established beta interferon therapy.

Source: Merck Serono.

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New study links lack of vitamin D with increased risk of MS

A study published in the online journal PLos Genetics has provided further evidence of a link between a lack of vitamin D and increased susceptibility to MS.

Researchers at the University of Oxford and the University of British Columbia have demonstrated how vitamin D interacts with a gene variant known as DRB1*1501 - one of the main genes implicated in genetic susceptibility to MS. Presence of the gene is believed to increase the risk of developing MS three-fold.

The study authors claim these results support the use of vitamin D supplementation during pregnancy and in young children.

Ramagopalan SV, Maugeri NJ, Handunnetthi L, et al.
Expression of the multiple sclerosis associated MHC Class II Allele HLA-DRB1*1501 is regulated by vitamin D.
PLoS Genet 2009;5(2):e1000369.

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