The only other hES cell company to have had a horse in this race was pioneering Geron, which in 2010, after a long struggle to win permission from the Food and Drug Administration, started the first clinical trial of a therapy derived from embryonic stem cells in spinal cord patients.
In the Geron trial, nervous system cells derived from the company’s flagship product, GRNOPC1—a population of living cells containing oligodendrocyte progenitor cells (OPC)—were injected into people with severe spinal cord injury.
Geron CEO John A. Scarlett, M.D., said that while there were “no signs” that the treatment was helping the patients, signs of efficacy that were not expected in the safety-focused initial trial. And as he noted, so far, he said, there had been no sign of safety problems.
The company shut down its stem cell therapeutics programs in 2011 to conserve funds, planning it said to lay off 38% of its 175 person staff and to seek partners for the program’s assets. Geron abandoned the $25 million effort, after its clinical trial had continued long enough to establish the safety of an ES-cell based product, but no signs of clinical benefit.
The Geron trial discontinuation was perceived as a major setback for the eHS cell field because of the company’s pioneering role. The company had helped support the initial derivation of human embryonic stem cells at the University of Wisconsin in the late 1990s, giving the company fundamental patent rights in the field.
Geron said that its move did not reflect a lack of promise for the controversial field, but that with scarce resources it had decided to focus on its experimental cancer therapies, which are further along in development. “I deeply believe in the promise of stem cells,” Dr. Scarlett said in an interview. “I don’t think that promise is in any way, shape, or form changed by what we’re doing.”
Meanwhile, ACT says that one of its investigative partners filed an Investigative New Drug (IND) application with the FDA to initiate a Phase I/II human clinical trial using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat a severe form of myopia.
For the moment, only ACT remains the real player in the hES cell business. And as Geron exited stem cell therapeutics to become a commodities company, Robert Lanza, M.D., the CSO of Advanced Cell Technology, said Geron’s move was “very unfortunate for the field” and “puts the pressure on us now.”