GRNOPC1：世界上第一个胚胎干细胞治疗制品

RESEARCH & DEVELOPMENT
ISSN 1746-075111
Regen. Med. (2011) 6(6 Suppl.), 11–1310.2217/RME.11.77 © 2011 Future Medicine Ltd For reprint orders, please contact: reprints@
12Lebkowski
Regen. Med. (2011) 6(6 Suppl.)future science group
Q How was the patient population for the clinical trial determined?
In this open trial, we are recruiting
patients who have a complete ASIA A
thoracic injury, meaning that these
subjects have essentially no motor or
sensory activity below the level of the
spinal cord lesion. We are targeting
subjects whose last fully preserved neurological level is T3 to T11. These patients have severe injuries with significant clinical consequences and are paraplegic – they have no sensory or motor function in their legs. This patient population was chosen after discussions with our clinical steering committee, the investigators in the trial, and the US FDA. The key goal of this first trial is to establish the safety of GRNOPC1 while reducing the potential for harm. GRNOPC1 is administered at the injury site within 14 days of injury. In the selected thoracic injury population, there are relatively large distances between the neurological levels of the spinal cord. If some serious adverse activity such as ascending deterioration of the spinal cord or the development of ectopic tissue were to occur, it would have to progress considerable distances up the spinal cord before clinical consequences were observed, allowing time for intervention. By focusing on patients with complete thoracic injuries we hope to minimize the risks to patients in this first clinical cohort. In recruiting patients for the trial, do physicians face difficulties in terms of patient expectations for this new technology, which has been so widely discussed in the media?That is an issue we have put considerable thought into and have discussed extensively with our investigators in the trial. Any patient who is a candidate for the trial first must go through a considerable amount of screening to ensure that they meet the inclusion/exclusion criteria for the study. After initial screening, subjects must review and sign informed consent for participation in the trial and long-term follow-up. The informed consent form has been approved by our expert committees and each institutional review board at the clinical trial sites. The informed consent document explains in detail that potential benefits from GRNOPC1 are unknown and makes it clear the potential risks of this product.
The informed consent document also clearly spells out the subject long-term follow-up commitments for the trial. Physicians and, in many cases, patient advocates are present to council candidate patients at any time during the consent process. The subjects are not just committing themselves to going through surgery to receive the cells, but they are committing to follow-up for 15 years. During the first year, patients in the trial are followed with nine MRIs to look for evidence of any adverse events happening in the spinal cord. So there is a serious commitment for each of the individual patients. We do not have direct contact with patients, but based on physician feedback, candidate subjects are taking the trial and commitment very seriously. what is the latest news from the trial?We are actively recruiting for the trial; we have seven sites open in the USA, recruiting patients from a total of 48 states. To date, we have seen no serious adverse events in the first two patients to receive GRNOPC1. Once we have enrolled our first cohort in the trial, we want to expand the trial in two ways. First, we want to dose escalate. Currently, we are administering a low dose of 2 million cells but, pending agreement from the FDA, we want to escalate the dose up to as many as “ To date, we have seen no serious adverse events in the first two patients to receive GRNOPC1. ”“The subjects are not just
committing themselves to going through surgery to
receive the cells, but they
are committing to follow-up for 15 years. ”
13GRNOPC1: the world’s first ESC-derived therapy
future science group。