ILB – Tikomed
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 (a complex
form of polysaccharide or sugar), is thought to target multiple pathways that are
involved in the loss of function and death of neurons, which is characteristic of
ALS/MND and other neurological diseases.
There are a limited number of animal studies in neurological disease models that
have been published to date with ILB, which have established its pre-clinical safety
profile. These studies have also shown the drug can 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 neurotrophic stimulus and a myogenic stimulus, i.e., boosts the health
and/or survival mechanisms for neurons and muscle cells, to degenerating muscle.
Preclinical studies from the ILB programme also demonstrated positive effects on
endogenous (within the body) repair mechanisms and rebalancing of inflammatory
Other clinical trials are currently in progress that are assessing the benefits of HGF
administered via injection into the arm and leg muscles of people with ALS/MND.
Trail Design and Results
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 ALS/MND, and
suggested evidence of some clinical efficacy (Ref: Logan et al, 2022). The clinical trial
was a Phase 2a, single-centre, open label, single-arm proof of concept study of a
very small number of patients, where the primary endpoint was safety and
tolerability of subcutaneously administered ILB.
- As a single-arm open-label study, there is no placebo group to compare
outcomes with. In such studies patients often report improvements which
can also be due to improved care and access to clinical services which trials
provide. It can therefore be difficult to definitively conclude that such trial
effects are solely due to the drug being tested.
- ILB was administered once a week, by injection, for 5 weeks in 13 people with
MND (in Sweden).
- A number of clinical benefits were reported in the trial participants including
slowing of disease progression, increased mobility and biomarker changes.
- The reported benefits decreased 3-4 weeks after the last dosage with disease
measurements returning to pre-trial levels by week 7.
- It has been reported that further trials are planned although details have yet
to be released.
The SAC believes this pilot clinical study appears to demonstrate safety and
tolerability of ILB in people with ALS/MND. The exploratory biomarker and
functional measures must be cautiously interpreted due to small study numbers
and the open-label nature of the trial. A larger, randomised, placebo-controlled trial
is needed to confirm any suggested clinical benefit seen in this small pilot trial.