Stem cell therapy is a controversial area both in terms of ethics and scientific merit. Despite the promise, there has been little progress in the treatment of disease. Asterias Biotherapeutics released the results of the first stage of a phase 1/2 trial of a stem cell-derived therapy called AST OPC-1 in the treamtent of spinal cord injuries. This first stage was conducted in three patients with spinal cord injuries with the lowest of three doses of the treatment.
The Reuters report gave a good background on the therapy, outlining its history at Geron Corp to Asterias’ acquisition in 2013. Where the story falls short, however, is in explaining the research itself, in its incomplete description of the benefits and potential harms, and in its use of a single source, the company’s CEO. An independent expert would have help put this research into perspective. What also is missing is a brief description on the natural history of spinal cord injuries (how much do they usually get better on their own?) and their usual treatments.
Spinal cord damage can be life altering and in far too many cases irreversible. Existing treatments, while helpful, frequently do not allow the patient to undergo a complete recovery. Stem cells, although controversial, have the potential to develop into specialized cell types and to replace damaged tissue. But much work still needs to be done on how stem cells can be translated into meaningful therapies to treat diseases and injuries. Stories shedding light on this area of research could encourage more public discussion, which in turn might lead to more research and subsequent advances.
The therapy, AST OPC-1, is in its early developmental stages – making it difficult to estimate costs in this case.
Since the story is clear about the early state of the research, we rate this one Not Applicable.
We think this report could have done a more thorough job here, especially since its headline claims the stem cell therapy “shows promise.” We think that it is helpful to point out that this is a phase 1/2 trial and is the first cohort of patients to be enrolled. A very early stage in the arduous path to commercialization.
The only mention of benefits in this Reuters story is that “severity of the spinal injury was reduced in the first patient,” but the story doesn’t try to quantify his improvement. To what extent was the spinal injury reduced? And in what time frame? Since there is no control group in this study, it would have been helpful to know if patients normally experience improvements over time. The news release provides a bit more useful information in this regard with a quote from one of the investigators; “This progress in the first patient is very encouraging and is observed in less than 5 percent of our AIS A patients at this stage of their recovery.”
The story does include a restraining quote from the company CEO who says, “It is important to know that we do not expect patients to get up and play basketball. But we do expect the patients to have significant improvements in mobility.” But what does “significant improvement” mean?
AST OPC-1 just completed its phase 1/2a clinical trial, which essentially evaluates the safety of the therapy. The three patients in the trial have not shown any serious adverse events, and the story reports this. However, we think the story could made it clearer that that is very early data, and that harms resulting from the therapy remain to be seen. This is the first stage of a three-step dose escalation trial and the dose used was the lowest envisioned. It’s certainly possible that adverse events not seen at a low dose will occur more frequently with a higher dose of the therapy.
The first sentence of the Reuters report was a bit misleading. Initial data did not indicate that stem cell therapy “could improve mobility in patients” with spinal cord injuries. Instead, the initial trial indicated this stem cell therapy was safe in low doses, or at two million stem cells. Since this was a phase 1/2a clinical trial, the main purpose was to determine the therapy’s safety. Efficacy was a secondary consideration. With such a tiny sample size of 3 patients, it is impossible to draw any conclusions about the therapy’s effectiveness.
The story also could have used some discussion about the limitations of an open-label, single-arm study. In an open-label trial, both researchers and patients know which treatment is being administered. In single-arm or non-randomized trials, everyone enrolled in the study receives the therapy. And in this clinical trial, there was no control group. A comment or two about the natural course of untreated injury would have provided an important context to the story
These study designs may be common during phase 1 and 2 testing, but they provide opportunities to introduce bias into the trial. Until scientists conduct a double-blind, randomized clinical trial, it would be premature to draw conclusions on the therapy’s efficacy.
There is no disease mongering in the article.
Only the CEO of Asterias is quoted in this article. An independent source – who could have discussed the limitations and significance of the trial – would have helped balance the story, bringing a much-needed perspective to readers.
Severe spinal cord damage is unfortunately irreversible, but treatments to improve nerve function exist. These include medications, such as intravenous methylprednisolone for acute spinal cord injuries, and surgery to remove foreign objects that are compressing the spine. Rehabilitation nurses and physical therapists could also work with patients to maintain and strengthen muscle function.
Since the article does not mention any of these alternatives, we give it a Not Satisfactory rating here.
The Reuters story makes it fairly clear that this therapy is in its early phases. The article describes how Asterias acquired the therapy from another research company in 2013 and how this announcement has been the first about the research in more than two years.
The report does a good job detailing the therapy’s history at Geron Corp, which also conducted an early-phase study with five patients suffering from spinal cord injuries prior to 2011. Asterias bought OPC-1 in 2013 and just completed its own early-stage trial, the article said. It also notes that AST OPC-1 “is the first product derived from human embryos to be tested on humans.”
Although the article refers to the company’s news release, the comments from CEO Lichtinger are original. But one-source stories are not ideal: We would have liked to have seen comments from other researchers, especially one who was not involved in the trial.
Since the Reuters story made an effort to go beyond the news release, we give it a Satisfactory rating here.