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MND Treatment trials

The only proven treatment for the underlying cause of MND is riluzole. This drug has a modest effect on survival.

There is a worldwide research effort underway to develop new and effective treatments for MND and the drugs listed below are currently undergoing clinical trials.

Introduction

People with MND and their carers play the most important role in the successful completion of a trial. Without their support and commitment these trials couldn’t be carried out.

“We need to find out whether a drug works and find out whether it’s the drug working or the mind playing ‘mind games’. If the placebo has got to prove if the drug works it makes it as important as the actual drug.” – participant in previous MND clinical trial

While the results of previous clinical trials have been largely disappointing, a great deal has been learnt about how trials should and should not be carried out, paving the way to future trials.

It is important to remember that trials are not treatments but scientific experiments. Due to the design of clinical trials, participants may not receive the new drug under investigation and may experience some side effects. Find out more in our Clinical trials – what are they and how are they organised? information sheet.

Overview of current or very recent clinical trials

Ionis SOD1Rx

Current status: ACTIVE (recruiting)

  • USA, Canada, UK (Sheffield), Belgium, Germany

Background

SOD1Rx is specifically designed to treat familial MND caused by mistakes in the SOD1 gene. It uses an approach known as ‘antisense’, in which the drug directly interferes with the faulty instructions for making SOD1 protein, thus stopping the production of the disease-causing substance.

Updates

Phase I/IIa (double-blind, placebo-controlled)

This trial began in early 2016, and is recruiting 72 people with MND in USA, Canada and Europe (including one centre in the UK). The main aim of the study is to evaluate the safety and tolerability of this treatment in people with the SOD1 form of inherited MND. They will also investigate how the drug gets to where it needs to be in the body (known as pharmacokinetics). It is being run by Ionis Pharmaceuticals in partnership with Biogen.

NP001

Current status: ACTIVE (recruiting)

  • USA, Canada

Background

NP001 is being developed by Neuraltus Pharmaceuticals Inc. It acts on particular cells within the immune system in the bloodstream, which in turn influences inflammation within the brain and spinal cord. The study is focusing on people with MND who are also showing signs of an elevated immune response. By ‘damping down’ the immune system, the aim is to reduce the inflammation which damages motor neurones.

Updates

Phase II (randomised, double-blind, placebo-controlled, intra-venous administration)

Results reported in late 2012 suggested that the treatment was safe, but did not significantly slow the progression of MND. However, further analysis raised the possibility of some beneficial effects in a small subset of participants, which has led to another study. The trial is currently recruiting up to 120 participants with evidence of increased inflammatory activity in the blood (C-reactive protein). Recruitment takes place in centres across USA and at one centre in Canada. Results are anticipated in late 2017 or early 2018.

MIROCALS

Current status AWAITING RECRUITMENT

  • UK (Brighton, London, Sheffield), France

Background

This is a joint clinical trial between clinicians in France and the UK that will test a molecule that occurs naturally in our bodies called interleukin-2 (IL-2) as a potential new therapy for MND.

Inflammation in the brain and spinal cord is thought to contribute to MND progression. Studies on animals and humans have shown that IL-2 can control a type of immune cell called a Regulatory T Cell (Treg). Tregs fight diseases and infections and help to control inflammation.

High doses of IL-2 are already used to treat certain forms of cancer. At lower doses, treatment of diseases of the immune system with IL-2 are promising. However, low-dose IL-2 has not yet been tested in MND. The MIROCALS (Modifying Immune Response and Outcomes in ALS) project will test the effectiveness and safety of a low dose of IL-2 in people with MND, to find out what effects, good or bad, it has on people with MND and, in particular, if it may slow down the advance of the disease.

Updates

Phase II (randomised, double-blind, placebo-controlled)

216 participants will be recruited into the trial which is planned to open for recruitment in spring 2017. Only patients newly diagnosed with MND and not yet treated with riluzole, will be eligible to take part. Half of the recruited patients will receive the study drug and half will receive the placebo.

The study will take place at 11 centres across France and the UK. The five centres in the UK include Brighton, Sheffield and London. As well as taking the study drug or placebo, participants will also be asked to take part in a biomarker and genetics study.

Neuralstem

Current status: ACTIVE (not recruiting)

Background

Neuralstem is a stem cell treatment that involves several injections of foetal-derived neural stem cells into the spinal cord of patients.

Updates

Phase II

This study involves giving injections into the lower regions of the spinal cord, at increasing doses (number of cells), to help work out the optimum number of stem cells to give (these injections are given as a surgical procedure). Results from this study confirmed that the transplantation can be safely performed at high doses, to both lumbar (lower back) and cervical (neck) regions.

Brainstorm

Current status: ACTIVE (not recruiting)

  • Israel, USA

Background

Brainstorm reprogrammes bonemarrow-derived stem cells into neurone-supporting cells. These are then transplanted back to the same patient so that they can secrete neutrotrophic factors to protect and promote growth of neurones (known as NurOwn).

Updates

Phase II in Israel (single-arm, repeat-doses, spinal cord administration)

A phase II trial of NurOwn in Israel is now being considered by the Israel Ministry of Health. If it is approved, the trial is planning to recruit around 24 people with MND to receive repeat doses of NurOwn into the spinal cord.

Phase II in USA (randomised, placebo-controlled, double-blind, single dose, spinal cord and muscle administration)

Brainstorm’s Phase II randomised, placebo-controlled trial of NurOwn in USA looked at the safety and effectiveness of stem cell transplantation in 48 people. The injections were given into the spinal cord (intrathecally) and also into the muscle.

Results published in July 2016 revealed that NurOwn treatment was safe and well-tolerated. While not a primary objective of this trial phase, the study also showed beneficial effects of NurOwn as assessed by lower levels of inflammatory biomarkers in the cerebrospinal fluid (CSF).

Tirasemtiv (CK-2017357)

Current status: ACTIVE (not recruiting)

Background

Tirasemtiv is a muscle activator that increases force (contraction) and power of skeletal muscles and therefore delays muscle fatigue. Instead of aiming to slow down progression of MND, the aim of this drug treatment is to increase quality of life by increasing muscular strength.

Updates

Phase III (randomised, double-blind)

A Phase III clinical trial (known as VITALITY-ALS) recruited 600 people with MND in the USA, Canada and Europe (including the UK). The trial tested the effectiveness of different doses of Tirasemtiv on breathing function over a period of 48 weeks. The trial was designed to try and reduce the side effects of the drug experienced in previous studies.

Update on the study enrolment was presented by Dr Jeremy Shefner at the 27th International Symposium on ALS/MND in December 2016. Altogether, 744 people were enrolled in the study, spanning over 81 centres in 11 countries. Finalised study results are expected in late 2017.

Phase IIb – BENEFIT-ALS (multi-national, double-blind, randomised, placebo-controlled)

The Phase IIb study tested safety and tolerability in 605 people with MND across 70 international centres. The results revealed that the treatment itself was safe, however, it did not significantly decrease disease progression, failing its primary objective. However, significant improvement of Slow Vital Capacity, measurement of lung function, was observed in people who were given Tirasemtiv as opposed to those who were on placebo.

Levosimendan (ODM-109)

Current status: ACTIVE (not recruiting)

Background

Levosimendan was developed by Orion Pharma. It is licensed to treat some forms of congestive heart failure by strengthening the action of heart muscle. The trial is investigating whether the drug might also have some usefulness in MND by helping to improve the function of the diaphragm muscle, one of the main muscles involved in breathing.

Updates

Phase II – LEVALS

A Phase II trial (known as LEVALS) is taking place in Europe, including centres on the UK. The trial recruited up to 54 people with MND and specifically looked at the effects of the drug on respiratory function.

The results of Phase II revealed that levosimendan did not improve respiratory function when compared to placebo, failing to meet its primary objective. However, further development of the study is currently underway, studying levosimendan in 66 patients with MND in Europe.

Masitinib

Current status: ACTIVE (not recruiting)

Background

Masitinib inactivates a cellular enzyme called tyrosine kinase, which is believed to be involved in tissue inflammation. While inflammation of the central nervous system may not be a direct cause of MND, it is likely to play a role in the speed of disease progression. Reducing the activity of inflammatory cells in the brain may reduce the damage to motor neurones.

Updates

Phase II/III (randomised, double-blind, placebo-controlled)

Phase II/III trial of this clinical trial examined the safety and effectiveness of masitinib in combination with riluzole in 394 people with MND, when taken for nearly a year. AB Science, the company behind masitinib, has recently published promising results, showing improved ALSFRS-R score when given 4.5mg/kg of masitinib per day. However, no benefit on overall survival was observed. A further clinical trial that will aim to confirm the previously-found results is planned to run through 2017-2018.

See the ALS Association website for a more detailed article.

Edaravone (also known as Radicut)

Current status: ACTIVE (not recruiting)

Background

One theory about why motor neurones die in MND is that they are affected by oxidative stress. Edaravone acts as a ‘free radical scavenger’ helping to reduce the effects of oxidative stress (as explained in a post on our research blog).

The drug is currently available in Japan .

Updates

FDA Approval

In early May this year, edaravone (Radicava) was approved to treat MND by the FDA (Food and Drug Administration), the drug licensing authority in the USA. Discussions are underway with regard to licensing it in Europe. Read more on Radicava on our blog.

Phase III

The first Phase III clinical trial of edaravone in MND found no overall benefit in people with MND, but showed an improvement in breathing function in a subgroup of people with an early form of the disease. Two studies have been conducted in this subgroup of patients. Results of the second study, announced at the 27th International Symposium on ALS/MND, revealed that after 24-week intervention patients showed some signs of beneficial effect.

GM604

Current status: COMPLETED

Background

GM604 is a regulator peptide that monitors and corrects the nervous system. Prior research revealed that, when administered intravenously, it has neuroprotective effects in people with MND diseases.

Genervon, the company behind the drug GM604, have already shown through Phase I and Phase II clinical trials that the drug is safe in people living with MND and Parkinson’s disease.  Read more on our website.

Updates

Phase IIa

The Phase IIa trial, taking place in the USA, recruited 12 patients with ALS, out of which 8 received the active agent and 4 received placebo. Results of this study reported in Genervon’s research article showed that there was no difference in ALSFRS-R ratings between patients receiving GM604 and those receiving placebo. The research article was posted on the public publishing platform F1000 in March 2017 and is currently awaiting peer-review.

No information on FDA approval of GM604 for ALS has been released, with the last updated information on the FDA website dating back to 2015.

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