Two companies, Advanced Cell Technology (ACT) and Geron, both HQ’d here in California, have FDA-approved early stage clinical trials in the works.
Geron’s trial has already started and reportedly now has enrolled at least two patients. The trial is of course for the safety of Geron’s GRNOPC1 oligodendrocyte product made from human ES cells for the treatment of acute spinal cord injury. Patients to be involved in this trial must meet strict criteria based on pre-clinical work and because of the required acute nature of their injury, the trial has to proceed as it can based on when people get this kind of injury.
ACT has two FDA-approved trials and just this week it was reported that patients have started being selected. ACT’s product being tested in both cases is retinal pigmented epithelial cells produced from human ES cells. One trial is for Stargardt Macular Dystrophy, a relative rare progressive disorder, and Dry Age-Related Macular Degeneration, a more common condition. Both cause blindness and neither have effective existing treatments. By the nature of the diseases, ACT can proceed with their trials with already existing patients, likely making their trials far more rapid than that of Geron. In fact, ACT’s press release on Thursday said patients had already enrolled and “Each study will enroll 12 patients with cohorts of three patients in an ascending dosage format.”
So in some sense ACT is already far ahead of Geron even though Geron’s trial started first and ACT’s trials look to be completed first, that is if one views the two as competitors.
But are they competitors or in some sense allies?
They are not aiming to treat the same disease so in that sense they are not competitors. In addition, while their products are both based on human ES cells, the end derivatives are completely different. So their products are really completely different.
So why do many people see them as competitors? Many investors and company insiders view the situation as a competition for history. In that sense Geron has won one battle, getting the first FDA approved trial based on human ES cells, but as mentioned above it seems almost certain that ACT will have the first completed trial. Of course we do not know how the trials will turn out, which is indeed far more important than which one starts or finishes first.
In fact, I would argue that what is good for ACT is good for Geron, and vice versa. Good news for one helps both. Bad news for one, hurts the other. In this way, I see the two as allies. Certainly negative safety outcomes in one of the trials will impact the other from the FDA’s perspective.
I really do not believe that they are competitors in any sense that matters.
What will matter from a historical perspective is which trial, if any of these 3, leads to a safe, effective treatment. We in the stem cell field hope all 3 succeed and that there are more to come soon.
So the reality is that whether they see it that way or not, Geron and ACT are allies and should be wishing each other well.