Biotech company retracts Parkinson’s treatment study after “possible deviation from protocol”

LCTLiving Cell Technologies (LCT), a biotech company headquartered in Australia, has retracted a 2011 paper purporting to show that their product reversed Parkinson’s symptoms in rats after “being unable to reconfirm their reported results and a possible deviation from the protocol.”

LCT is developing NTCELL, which, according to their site:

is a choroid plexus cell product with the potential to treat Parkinson’s disease and other disorders, such as stroke, Huntington’s disease and hearing loss, as well as wound hearing. These cells help produce cerebrospinal fluid as well as a range of neurotrophins (or nerve growth factors) that have been shown to protect against neuron (nerve) cell death in animal models of disease.

(We assume that’s supposed to be “wound healing.”)

Here’s the notice from Regenerative Medicine, which went online yesterday:

Due to the authors being unable to reconfirm their reported results and a possible deviation from the protocol that may have occurred, the following article has been retracted from Regenerative Medicine:

Stephen JM Skinner, Hai Lin, Marilyn S Geaney, Thorsten Gorba, Robert B Elliott & Paul LJ Tan: Restoration of motor control and dopaminergic activity in rats with unilateral 6 hydroxy-dopamine lesions. Regen. Med. 6(3), 319–326 (2011).

The authors and editors of Regenerative Medicine regret any negative consequences this publication might have caused in the scientific and medical communities.

The paper has been cited just once, according to Thomson Scientific’s Web of Knowledge, by a paper that included most of the authors of the now-retracted study.

Because LCT is a publicly traded company, and a retraction is material information that could affect its stock price, this is the rare retraction that was accompanied by a press release. A December 19, 2013, company release about the retraction — which refers in the text, but not a footnote, to the wrong journal — notes that the data will be “withdrawn from all regulatory documentation:”

The publication is being withdrawn following an internal quality assurance (QA) audit which showed that the source data for the study held on file at LCT are incomplete and therefore the efficacy conclusions in the publication cannot be confirmed.

The withdrawal of the rat efficacy data does not in itself present a safety risk with regard to the use
of NTCELL in humans. Nonetheless, as a precautionary measure LCT has placed a hold on any further patient recruitment into the human Phase I/IIa clinical study that is currently underway at Auckland City Hospital. This is to allow the company to work with the New Zealand medicines regulator (Medsafe) and the data safety monitoring board (DSMB) to fully understand the impact of the withdrawal of the rat efficacy data on the Phase I clinical trial.

That hold will cost the company money, at least in the short term:

LCT and Otsuka Pharmaceutical Factory (OPF) are co-developing NTCELL as a treatment for Parkinson’s disease. A second cash payment of A$2m which was due from OPF to LCT as a result of the DSMB authorising the recruitment of the remaining three patients to the study is now not expected to be received until patient recruitment into the trial is recommenced.

The company reported that the subject of the clinical trial “continues to do well since their implant in September 2013.”

Paul Tan, the last author of the paper, retired last week from his post as LCT’s chief science and medical officer. The company’s stock, which has been trading below $1 per share over the past year, fell from 95 cents to 60 cents following the release about the retraction last month. It closed yesterday at 69 cents.

Hat tip: Rolf Degen

6 thoughts on “Biotech company retracts Parkinson’s treatment study after “possible deviation from protocol””

  1. Why would genuine academics and scientists continue to support the publish or perish maxim, when there is no guarantee that what is published has actually been done?

    1. Because in the scientific method, if errors or problems are found out they are also published. In this case the company itself apparently initiated the retraction although it may devastate their business. If the company think someone actually fabricated data, there may be lawsuits.

      Any any case, the publish-or-perish system is often criticized, but rarely does anyone suggest an alternative. As a scientist you are either doing work worthy of publication or … um… or what are you doing?

      1. You might be discovering, inventing, finding solutions to certain problems or perhaps trying to reproduce or verify others work. Why aren’t scientists entitled to protection of their intellects against those who may steal their ideas?

    2. Although, in this case, there is absolutely no suggestion that the results of the PD trial will be released to the public before human trials, since in fact a human trial is ongoing, I wish to seek some advice from bloggers about the general principles underlying rat-to-human trials, or direct rat trials followed by release of a product into the public. My query is a general query, and is not related to this story directly.

      I have some concerns about some substances I see being released into the human population in multiple food stuffs after rat-based studies showed no toxicity and after they obtained GRAS approval. Interestingly, the one particular case I have in mind, GRAS approval was obtained after a commercial company made the bid, in which the “technical and scientific panel” consisted of three individuals, including one company representative (i.e., bias). I can now further state that because of that three-person influence in obtaining (purchasing) the GRAS rights, this “compound” is now “safe” for consumption GLOBALLY. A victory for business, but it still doesn’t take away my nagging feeling that something is horribly wrong when rat-based results are extrapolated to the human population. Recently, concerned about this void between lab and society, and looking at the issue pragmatically, I have been approaching several specialists in the field, who all unanimously seem to invoke the same clichéd response that the substance is not metabolized so thus, cannot be toxic. Yet, to date, I have not yet received a satisfactory explanation why a result derived from a pool of 6 rats can be applied to a pool of hundreds of thousands or even millions of humans just because three people (from a company) decided it should be so. In particular, in theory, would any high level of a substance applied to an organism not eventually be toxic? Or would long-term consumption not eventually lead to toxicity, even if each dose is a low concentration? My fear is that human populations are becoming the real “clinical trials” of some (?) rat-based laboratory findings. Is anyone else seeing or perceiving this?

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