Today – October 14 of 2014 is a very important day in “stem cell history”. US-based company Advanced Cell Technology (ACT) released results of the clinical trials, where embryonic stem cell-derived product was tested in patients with macular degeneration. The first ever peer-reviewed results of FDA-approved clinical trials, involved use of embryonic stem cells (ESC), published online in Lancet (keep renewing your browser window until Lancet will put abstract in). You can download article here. This is a big milestone in stem cell research and cell therapy industry! I think, it’s probably the most important event of the year so far! Here is why:
- Out of few, approved by federal regulatory agencies, ESC-based trials, none results were published in peer-reviewed literature until now (I don’t count Asterias PR and ACT two cases report).
- Everybody were eagerly awaiting for results of safety profile of ESC-based therapeutic. ACT’s study concluded that ESC-derived product is safe during observation time of 2 years. Study is feasible.
- Even though, trials were not design to assess efficacy, more than half of patients demonstrate different degree of improvement of sight.
- There were some signs of engraftment of ESC-derived cells in more than half patients. That’s what we are missing in “adult stem cell studies”.
- Despite criticism and some critical financial periods, ACT was able to conduct/ complete the trials and report results. ACT shows – it’s possible! What a courage and inspiration for others! No matter how the next phase will progress, ACT is blazed the trail for other developers.
- With positive data, this study is triggering a new wave of interest to embryonic stem cell-based therapies.
Now, some considerations against premature hype:
- The report is not final, but interim. Patients from 2 separate trials (NCT01345006 and NCT01344993) were combined for analysis. Each trial has an enrollment goal of 16 patients by the end of this year. So data could change when all patients will be analyzed.
- Two years is mid-term observation, but long-term follow-up (10-15 years) is required to conclude safety of ESC-derived products. And… yes – we still don’t know the efficacy until Phase 2 will be concluded.
- Some complications were reported in the study (but not in official company’s PR and mass media coverage): 4 eyes had significant progression of cataract, requiring surgery 6-12 months after cell therapy, 1 patient developed bacterial eye infection 4 days after cell therapy, 1 eye – “sterile inflammation”, 1 eye – subretinal bleb. All complications were resolved and did not lead to enucleation. There was no evidence for direct relation to a product, but seem like complications were procedure-related (surgery).
- Instead of paying much attention to PR and mass media coverage, I’d encourage you to read an article and do your own assessment.
PS: I do not have any conflicts, related to ACT.