Advanced Cell Technology (OTCBB: ACTC) 1st Patients undergo hESC Transplantation Treatment for Stargardt’s Disease and Macular Degeneration
The dosing of the 1st patients in each of its 2 Phase 1/2 clinical trials was initiated for Stargardt’s macular dystrophy and dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).
The patients were treated at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute. Both patients successfully underwent the outpatient transplantation surgeries and are recovering uneventfully;
- Both the Stargardt’s trial and the dry AMD trial will enroll 12 patients each, with cohorts of 3 patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt’s and dry AMD at 12 months, the studies’ primary endpoint
The Bottom Line: The dosing represents an important milestone for treating the many forms of macular degeneration. The patient(s) in each clinical trial has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. Early indications are that the patients tolerated the surgical procedures well. Remember, the primary objective of these P1/2 studies is to assess the safety and tolerability of these stem cell-derived transplants. Investors should recognize ACT’s ability to finally get respect for its trial initiation, a big cost but, the only validator of years of Lanza’s lab work and corporate development. ACT is still the 2nd company to get the FDA to approve a (potential) hESC trial. Their Orphan Drug designation also validates their position in the stem cell universe however, their capitalization and financing structure’s inability to realize a share-price value and does not position ACT to a broader investor base. It has been a long road traveled by ACT and travails are many as they advance their product candidates. Although reversal of the macular degeneration condition is not possible, their are still a number of treatments to hold the condition in check but, the market is HUGE. ACT’s financial position is still suspect as they pay off the debts of the past; but, trial advancement should/must stimulate a joint venture or a partnership to advance their funding, R&D candidates and industry standing. However, their extremely loyal retail base or ”true believers” is offset by a lack of following in the institutional arena. News has always played a part in ACT’s upward and downward share pricing but, it unfortunately fades with no support other than retail. The only momentum indicator, to me, is the level of interest of day-traders! Outlook to the future is insecure but, $0.21 to maybe $0.24 is my prognostication without any joint-venture or partnering catalyst validation.
- ACTC.OB is trading at $0.1930 up 3.43% up $0.0064 with a 4.1 share volume.