Haematopoetic Stem Cell Therapy is the best way to deliver NEDA

NEDA status in highly active MS can be more easily obtained with autologous hematopoietic stem cell transplantation than other drugs.Sormani MP, Muraro PA, Saccardi R, Mancardi G. Mult Scler. 2016 Apr 26. pii: 1352458516645670. [Epub ahead of print]

The no evidence of disease activity (NEDA) composite measure has emerged as one attractive new target of therapies in relapsing-remittingmultiple sclerosis (RRMS), consisting of the following features: (1) no relapses, (2) no disability progression, and (3) no magnetic resonance imaging (MRI) activity (new or enlarging T2 lesions or Gd-enhancing lesions). Achievement of NEDA status in patients receiving a disease-modifying therapy (DMT) seems to be an ambitious but ideal goal for therapies in RRMS. Recently, published post hoc analyses of clinical trials reported percentages of RRMS patients maintaining the NEDA status after 2 years of therapy ranging between 13% and 46%. Long-term assessment of NEDA patients in real-life settings showed very low probability to be NEDA in the long run. Against this scenario, immunoablative therapy followed by autologous hematopoietic stem cell transplantation (aHSCT) demonstrated the potential to maintain a much higher proportion of NEDA patients at 2 years (ranging from 78% to 83%) and also at 5 years (ranging from 60% to 68%). This is even more relevant when considering that MS patients who underwent aHSCT are much more active than patients usually enrolled in clinical trials. The emerging evidence of the efficacy of this therapeutic approach in early aggressive and treatment-resistant RRMS calls for the organization of a randomized comparative trial to fully evaluate the risk-benefit profile of aHSCT in patients with highly active MS not responding to DMTs

We have been calling for the desire to achieve NEDA to promote brain health and if you take the ultimate DMT and replace the immune system you get better levels of NEDA than that achieved with any current drug and if you compare this to new data published for beta interferon

Uher T, Havrdova E, Sobisek L, Krasensky J, Vaneckova M, Seidl Z, Tyblova M, Ramasamy D, Zivadinov R, Horakova D. Is no evidence of disease activity an achievable goal in MS patients on intramuscular interferon beta-1a treatment over long-term follow-up?Mult Scler. 2016 May 26. pii: 1352458516650525. [Epub ahead of print]

BACKGROUND:No evidence of disease activity (NEDA) has been proposed as a new treatment goal in multiple sclerosis (MS). NEDA-3 status is defined as the absence of magnetic resonance imaging (MRI; new/enlarging/enhancing lesions and increased whole brain volume loss in NEDA-4) and clinical disease activity.
OBJECTIVES:To investigate the persistence of NEDA status over long-term follow-up in MS patients treated with weekly intramuscular interferon beta-1a.
METHODS:We included 192 patients after the first demyelinating event suggestive of MS, that is, clinically isolated syndrome (CIS) and 162 relapsing-remitting MS (RRMS) patients.
RESULTS: NEDA-3 status was observed in 40.1% of CIS and 20.4% of RRMS patients after 1 year. After 4 years, 10.1% of CIS patients had NEDA-3 status. After 10 years, none of the RRMS patients had NEDA-3 status. Only 4.6% of CIS and 1.0% of RRMS patients maintained NEDA-4 status after 4 years. Loss of NEDA-3 status after the first year was associated with a higher risk of disability progression (hazard ratio (HR) = 2.3-4.0; p = 0.005-0.03) over 6 years.
CONCLUSIONS: Despite intramuscular interferon beta-1a treatment, loss of NEDA status occurred in the vast majority of individuals. Loss of NEDA status during the first year was associated with disability progression over long-term follow-up; however, specificity for individual patient was low.

HSCT is not without risks but the level of NEDA clearly appears better than at least the CRAB drugs. 

Can they recruit to a trial? 

Will this be the death knell of big pharma drugs?. I doubt it, if there are neuros scared to use alemtuzumab, they will be too scared to recommend ablative HSCT.

Wonder how the idea of ZEUS trial is getting on?