Clinical trials (Ct)
Clinical trials are research studies in human volunteers that determine whether potential treatments are safe and effective. All clinical trials have strict guidelines about who can take part (factors that allow inclusion criteria). Clinical trials are usually conducted in four progressive phases which check for safety and efficacy, establish the correct dosage and method of delivery, and assess the drug’s ability to treat the condition it is designed for.
Clinical trials take many years to complete and are often extremely costly. At any stage the drug can be deemed too dangerous, or inefficient, to take into the next phase. To speed up the process, clinical trials are beginning to incorporate a biomarker element (a biological characteristic) into their design. Monitoring levels of specific biomarkers during a trial will help establish if the drug or intervention being tested is influencing disease progression. Researchers are also looking to ‘repurpose’ drugs that have showed an effect for other diseases and have already been shown to be safe in humans, as potential treatments for MND.
Several clinical trials of potential new treatments for MND have been completed and some are still in progress. So far, only one drug, riluzole, has been proven to have enough of an impact on the disease to warrant its licensing for general use in the UK. For a list of MND clinical trials taking place in the UK and around the world, please visit www.mndassociation.org/treatment-trials. For a complete list, see www.clinicaltrials.gov, which gives updated information on clinical trials.
At the Symposium
Session 2B: clinical trials included a talk on phase 2 trial of MN-166 (Ibudilast), which Canada recently approved a new patent for use in ALS treatment (C9). Ibudilast is an anti-inflammatory drug currently used to treat asthma and tested for the treatment of neurodegenerative diseases by Medicinova. It is thought to suppress inflammation, activate neuronal function and reduce the number of toxic glial (support) cells.
NurOwn®, Brainstorm-Cell Therapeutics, is an investigational therapy which reprogrammes bone marrow-derived stem cells into neurone-supporting cells (C10). These are then transplanted back to the same patient so that they can secrete neutrotrophic factors to protect and promote growth of neurones. The Phase 3 trial has fully enrolled 200 participants across centres in the USA to investigate it’s effectiveness, and treatment is now underway. Results are expected by the end of 2020.
The Phase 2 clinical trial results of tofersen, an antisense oligonucleotide (ASO) therapy to reduce SOD1 as a potential treatment for SOD1-mediated MND was also presented this year (C11). Results supported the continued development of tofersen for treatment and is currently being recruited for Phase 3, VALOR.
Within Theme 9: Clinical trials and trial designs, CLT-16 presented information on TUDCA (Tauroursodeoxycholic Acid)-ALS, a novel European-run clinical trial design for disease progression, that recently began recruiting for Phase 3 in the UK and Ireland. CLT-24 presented the clinical trial design for a Phase 2 trial of CENTAUR (AMX0035), which is Tauroursodeoxycholic Acid (TUDCA) and Sodium Phenylbutyrate. The study of 132 participants over 24 wks administration showed significantly slowed progression in people with ALS, but Pahse 3 results are required to see how the treatment pans out in a longer term.
Check out our full updates on the Clinical trials session and some of the posters presented in Perth on our MND Research blog: