An amendment is being planned for an ongoing Phase 1/2 clinical trial for amyotrophic lateral sclerosis (ALS). This intention has just been announced by the cell therapy company Kadimastem. However, researchers don’t expect their amendment to alter their research timeline. Their hope is to submit an IND application by the middle of 2021. If all goes well, a multi-center trial will advance.

The multicenter trial will compare a frozen version of the drug to one that is not frozen. If the frozen one works, manufacturing costs could be significantly reduced. Considering the fact that the average costs for ALS treatment are currently 200,000 dollars per patient per year, any chance of decreasing this cost would be beneficial.

The Current Trial

The current phase 1/2 trial has four cohorts. For cohorts A and B, nothing will change with the amendment.

Preliminary results for cohort A were reported in September. The treatment showed safety and efficacy, with reduced progression of disease in the 3 months after patients received the therapy.

The amendment to the study will allow for testing of a low dose of the therapy in cohort C. Patients will receive 2 consecutive injections every 2 to 3 months. The previous plan was to test a medium dose of the therapy for cohort C. Now cohort D will receive the medium dose, pending results from the prior cohorts.

As of now, full cohort A results are expected by the end of the year; cohort B results are expected by the third quarter of 2020 and cohort C results should be announced within the first half of 2021.

Looking Forward

There is a lot of hope surrounding this investigative treatment for ALS. Researchers believe it has the potential to not only slow disease progression, but improve life expectancy, and patients’ quality of life.

Preclinical studies have shown its promise and the research team is optimistic about its future. Also indicating support for its potential, the FDA has granted Orphan Drug Designation to the treatment.

You can read more about this clinical trial for ALS and its recent amendment here.