New Leukaemia treatments; the academics perspective.

A fortnight ago, the world’s media extensively reported a new targeted therapy some types of blood cancer. This therapy tries to use the patient’s own immune cells to kill the leukaemia cells and has been hailed as a “paradigm-shift” in the treatment options for leukaemia.  

The idea of this blog post is to give you an idea of what this development actually means, how it will change things for patients and what the next steps in development will be. I am sure many of us are unaware of the rigorous process that new discoveries face in order to become published and viable in the medical world, Professor Ken Mills has kindly given us some insight below. 

This treatment is in a very early stage of development as the results from the initial clinical trial show.   It involves tagging immune cells from the patient with receptor molecules that target a specific cancer and giving them back to the patient. 

And yes, the trial has shown some positive results so a high percent of acute lymphoblastic leukaemia (ALL) patients did enter remission and similar treatments are being examined for other lymphoid blood cancers.  But as mentioned earlier, these are in an early stage and some patients didn’t respond to the treatment whilst others had a range of side effects so the mechanism and use of this type of immunotherapy needs more research before it could become an effective therapy for those patients for whom it would work.

Some of our caution about this study is not that it holds an exciting promise, but that it has yet to be published.   The data was presented at a conference in the United States and as such the details are not available for further examination.   Presentations at conferences are important for the dissemination of our research to the wider scientific and medical community but are rarely reported as extensively as this study has been.     The questions that I and others have got relate to what stage of the disease were the patients when treated, how long did the remission last, what was the extent of the side-effects and many others.  

We have to publish our results in good-quality medical and scientific journals to show what our research has identified.   This adds to the overall knowledge of the disease and topic as well as demonstrating value to the funding body, such as Leukaemia & Lymphoma NI and the organisations involved in the research.   

The publication process can be a long one but can also add clarity to the way that the data is explained in the manuscript.   The results are submitted as a manuscript to the journal; the manuscript not only includes the results, but an introduction to the topic, how the research was carried out and then a discussion or conclusion on the impact of the results.    The manuscript is sent out by the editor of the journal to a number of referees who are experts in the areas around your results.   The referees will read the manuscript and submit back to the editor whether they recommend acceptance, revision or rejection – hopefully not the latter although it often means that the editor thinks that is might be better suited in a different journal!     If revisions are required the referees will indicate what these are and we as authors have to try to address them before we can resubmit the manuscript back to the editor for another round of reviewing then acceptance! 

This peer review process, although long and circuitous, has the benefit that the data has been examined by several independent scientist or clinicians and questions confirming reproducibility, validation or perhaps over-interpretation  can be addressed before the manuscript is published and available for global research community to read.  It is usually at this stage that a press release is made available to the media as the organisations and funders are reassured that it has been through an extensive review process.   

I am not saying in any way that the results reported in the immunotherapy study are inappropriate or wrong, just that the scientific community haven’t been able to fully examine them in more detail.   Even if they are preliminary, immunotherapy will one day become an important weapon in the therapeutic armoury against some, but not all, types of leukaemia, lymphoma and other cancers. 

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