Information has come to light regarding the US Food and Drug Adminstration (FDA)'s freeze on the clinical trails of GRNOPC1, a groundbreaking therapy for spinal cord injury derived from human embryonic stem cells being undertaken by biotech company Geron. The FDA approved the commencement of clinical trails of GRNOPC1 in January 2009 amid much excitement: this was to be the first trial of its kind.
The halt, which was announced on 18 August, was not fully explained at the time by either the FDA or Geron. It appears that the FDA instigated the halt in order to review new information put before them by the company. Despite positive pre-clinical trials in animals, there have always been fears that the cells will differentiate uncontrollably causing tumours to develop.
These fears were allayed following new statements issued from Geron: the information being reviewed related to recent tests that revealed the formation of cysts in some (animal) trail subjects. Although cysts had been detected in earlier trials, the high frequency of cysts in recent trials caused concern. Geron has given assurances that the cysts are not dangerous - they are non-proliferative, and show no adverse effects, or increased health risks. Furthermore, the company stated that a batch of the GRNOPC1 drug, tested according to new markers and assays showed no cysts at all. No evidence of teratomas (a type of tumour) was found.
Geron's share price dropped 14 per cent following the latest suspension of the clinical trials, however it has since rallied. Joseph Pantginis of Merriman Curhan Ford explains: 'I believe that since the worst case scenario (teratomas) did not occur, investors are relieved.'
However, there is no indication of how long the review will take or when the clinical trials are likely to recommence. Pantginis rationalises the less-than-smooth drug trial process experienced so far: 'Geron is especially vulnerable because it is at the forefront, by far, of the embryonic stem cell space, and being on the cutting edge means the company must also be the first to hit the hurdles and learn as they go,' he said.However, not all commentary has been positive. Analyst Adam Feurerstein is on record saying that 'the only thing Geron has done exceedingly well in its 13 years as a public company is surf the waves of stem cell hype and use that momentum to raise lots of money.'