Background
ILB is a pleiotropic molecule which means it is believed to have multiple effects in the body. Its active component, a patented form of dextran sulphate, targets multiple pathways that are involved in the loss of function and death of neurons, which is characteristic of MND and other neurological diseases. (Ref: MND Association website).
There are a limited number of animal studies in neurological disease models that have been carried out to date with ILB, but its preclinical safety profile is well established, and the drug has been shown to restore brain energy metabolism after severe traumatic brain injury in rats. Subcutaneously injected (injected under the skin) ILB induces a rapid release of Hepatocyte Growth Factor (HGF) into the circulation in animals and healthy human volunteers, which may provide a key neurotrophic stimulus (critical in the survival of motor neurons) and a myogenic stimulus (can stimulate muscle growth) to degenerating muscle. (Ref: Logan et al, 2022). Preclinical studies from the ILB programme also demonstrated the drug’s ability to enhance endogenous (within the body) repair mechanisms and rebalance inflammatory responses. (Ref: BioSpace).
On 25 May 2022, researchers from TikoMed AB, Sweden, published a paper in PLOS ONE that showed ILB was safe and tolerable in people with MND, and suggested evidence of potential clinical benefits. The clinical trial was a Phase IIa, single-centre, open label, single-arm proof of concept study in a small number of patients, where the primary endpoint was safety and tolerability of subcutaneously administered ILB. To assess possible efficacy in patients with ALS, the trial was conducted in a heterogeneous ALS patient group of intermediate disease severity. (Ref: Logan et al, 2022).
- ILB administered once a week, by injection, for 5 weeks in 13 people with MND (in Sweden). (Ref: BioSpace).
- Increased functional scores were seen, assessed using ALSFRS-R scores and Norris clinical rating scales (the Norris clinical rating scale consists of two parts – the Limb Norris Scale, which has 21 items to evaluate extremity function, and the Norris Bulbar scale which has 13 items to evaluate bulbar function). (Ref: Logan et al, 2022), (Ref: Hashizume et al, 2015).
- Following the ILB injections patients reported increased vitality, decreased muscle stiffness and increased mobility. (Ref: Logan et al, 2022). A decrease in muscle atrophy biomarkers was also seen. (Ref: Logan et al, 2022).
- Changes in the functional scores had disappeared 3-4 weeks after the last dosage. (Ref: Logan et al, 2022).
Summary
This pilot clinical study demonstrates safety and tolerability of ILB in people with MND. The exploratory biomarker and functional measures potentially suggest clinical benefit and have a bearing on the mechanism of action of ILB. A larger, randomised, placebo-controlled trial is needed to confirm any suggested clinical benefit seen in this pilot trial.
References
- Press release (BioSpace) https://www.biospace.com/article/releases/tikomed-ab-reports-positive phase-ii-clinical-data-for-the-treatment-of-amyothropic-lateral-sclerosis in-data-published-by-plos-one/?s=114
- PLOS ONE paper – (Logan et. al, 2022) https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0267183
- ClinicalTrials.gov – https://clinicaltrials.gov/ct2/show/NCT03705390?term=Tikomed&draw=2& rank=3
- Norris scale definition – (A functional scale for spinal and muscular atrophy: Cross sectional and longitudinal study. Hashizume et al, 2015) https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5608513/
- MND Association website: https://www.mndassociation.org/research/clinical-trials/treatment trials/ilb/
International Alliance of ALS/MND Associations
March 2024
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