Thoughts on ILB, which may slow ALS progression, per data from small clinical trial

[powerpadman]

Treatment with a high dose of ILB, an experimental injection therapy being developed by Tikomed, was well tolerated and appeared to slow the progression of amyotrophic lateral sclerosis (ALS) in a small clinical trial in the U.K.

That’s according to data from the exploratory Phase 2 study (NCT03705390), in which the therapy was tested in 11 adults with the progressive neurodegenerative disease.

The results were detailed in “A low molecular weight dextran sulphate, ILB, for the treatment of amyotrophic lateral sclerosis (ALS): An open-label, single-arm, single-centre, phase II trial,” a study published in the journal PLOS One.

Ann Logan, scientific advisor to Tikomed and a professor at the University of Warwick, in England, added that the findings “indicate the future potential of ILB to be the first disease-modifying drug to treat both familial and sporadic ALS with minimal side effects.”

ILB, a formulation of the sugar molecule dextran sulfate, is thought to promote nerve health by prompting the release of signaling molecules that encourage nerve survival.

The experimental medication, which is administered subcutaneously, or via under-the-skin injections, is being explored to slow the nerve cell degeneration that drives ALS. It has been named an orphan drug in the U.S. and Europe as a potential ALS treatment.

An earlier Phase 2 study (NCT03613571), conducted in Sweden, explored the therapy’s use in 13 ALS patients. Each received five weekly subcutaneous injections of ILB at a dose of 1 mg per kilogram of body weight (mg/kg). Results after about 15 weeks, or slightly longer than three months, showed the treatment was generally safe and hinted that it could slow or even reverse disease progression.

 

[AnxiousStatsGuy]

It certainly looks good, but there is always reason to pause when looking at a single arm trial - all doctors and patients knew they were getting the treatment, which makes it potentially easy to bias the fairly subjective ALSFRS-R scale.

With that said, little/no progression on that scale over nearly a year would be a) quite uncommon and b) quite egregious to "fake" if it really weren't so

Additionally, there was seemingly no reduction in NFL levels

 

[lgelb]

Please do not submit verbatim text cut and pasted from another content source, nor include the numerous and gratuitous definition hyperlinks that link back to that source. This is not a link farm.

 

[powerpadman]

I was just trying to summarize the text as best as possible in case people didn't want to click to the article.

I'm interested and curious to what people think about this promising treatment. Not trying to create backlinks to improve SEO of other sites.

 

[lgelb]

Since you cut/pasted a good deal of the article, I had to take time to remove the self-referential links. Next time just provide the link, please. People can see for themselves and decide if/what they want to read.

There is nothing to think as yet. 24 P2 patients across 2 trials is not generalizable evidence for safety, tolerability, or efficacy. As for the scientific advisor's bubbliness, she is well-paid by the manufacturer for her role, so her opinion cannot be an objective one.

The spreadsheet of orphan drugs in ALS is always available at the FDA's site. It is littered with failures and abandonments, but it is certainly possible to keep up with the serious programs. This isn't yet one.

Five weekly SC injections is a significant dosing burden, esp. for PALS who lose bulk. And trial conditions, in which compliance is monitored and incentivized, are not real life.