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Stem Cell Treatments: A quick guide for medical practitioners and FAQ: a resource for patients submission

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1. Is the document in the appropriate format? If not, how could the document be better formatted?: 

It is not clear to me whether the two documents will be linked together in some way.  Presumably it is the intention for the medical practitioner to convey the information in the Q&A resource during a spoken consultation and then provide the patient with the separate document ‘frequently asked question-a resource for patients’.  If this is the case, this should be made explicit to the medical practitioners and indeed the two documents might reasonably be connected into one document separated by a new page so that the ‘patient resource’ can be printed out separately.  It may also be worthwhile offering to print out copies of the document for circulation to medical practitioner’s consulting rooms and disease-based organisations with an offer to provide multiple copies on application so that the information is disseminated effectively.  It is important that medical practitioners be able to readily access this information on the internet as well so that a link is provided within the document to obtain further copies.

2. Is the language appropriate for the target audience (i.e. medical practitioner for the quick guide on stem cell treatments and patient for the FAQ resource)?: 

The following is a series of comments specifically related to the content of both documents in a linear format and comprises answers to question 2, 3, 4 and 7. 

At the close of the opening paragraph I think there is a strong potential for patients to be misled about the generic term ‘stem cell research’.  With over 50 years of ‘stem cell research’ in bone marrow and peripheral blood stem cell transplantation it is not technically or factually correct that the majority of the research is at a very early stage of treatment development.  I think the sentence at the end of the first paragraph should be qualified with a phase like ‘aside from stem cell therapies for blood cancers’ then the rest of the sentence should be ok. 

On the fourth dot point on page 1 it is important to also include ‘unproven stem cell treatments inside or outside of Australia’.

On page 1, question 2 first paragraph the final sentence is not technically correct.  There is no one stem cell that is capable of dividing into an erythrocyte or myocyte.  At best, this sentence could be misinterpreted and may be misleading.  Easily it can be corrected by placing a full stop after the phase ‘a more specialised function’.  The following sentence could then be “Blood stem cells can become red blood cells (and other mature blood cells) or muscle stem cells can become mature muscle cells”.

On page 2 question 3, first sentence I think it is very important at each opportunity to emphasis what existing stem cell therapies are already of proven efficacy. In this first paragraph of question 3 I think that it is important to state that there are many different types of stem cells that can be obtained from different tissues in the body.  I think it is important to state explicitly that ‘blood forming stem cells obtained from the bone marrow, peripheral blood or umbilical cord are of proven therapeutic value for various cancers of the blood, immunodeficiencies, serious inherited disorders as well as diseases like testicular cancer’. 

In the second paragraph of question 3, reference is made to case reports that are supposed to be of relevance to ‘safety and efficacy’ but in fact neither of these examples is relevant to the question of efficacy of stem cell treatments.  I think it is very important to take great care with these examples and not over state their importance.  I suggest you delete the words ‘and efficacy’ in the context of these examples.

Page 2, question 4, third paragraph, to this list I think should be added ‘metastatic testicular carcinoma’.  I think it is also important to add ‘rare genetic diseases’ to the list of standard practices because diseases like adrenoleukodystrophy and mucopolysaccharidoses should often be treated with stem cell transplantation. 

Top of page three, unfortunately, I just do not think that the distinction between proven and investigational treatments is a very helpful one.  The simple fact is that investigational therapies are, by definition, a subset of unproven treatments.  The use of the term treatments in this is also questionable because it implies that the procedures are intended as ‘treatments’. The same distinction was made for many years when we preferred the term ‘gene transfer’ trials instead of the more widely-understood term ‘gene therapy’.  I understand what the author of this document was trying to achieve, but I am not comfortable with the actual terms that have been chosen.  All potential therapies that are not ‘proven’ should be considered unproven and a subset of the latter will be under investigation through approved clinical trials.  Therefore the distinction is quite artificial and I think this whole approach should be reconsidered in question 4 and when it is used subsequently.  I think more effort should be placed on emphasising the importance of ‘approved clinical trials’ for any therapy that is not considered proven or a standard of care in Australia. In short Im suggesting that the focus on ‘unproven’ and ‘investigational’ is distracting from the main message which is that we should encourage awareness and participation in ‘approved clinical trials’.

Page 3, question 5, this is an important section and is very helpful I think.  There is an addition closed bracket at the end of paragraph 2 which should be deleted.  I would suggest that you also add at the end of page 3 addition dot points in relation to upfront fees or very high costs that are not clearly explained.  An additional dot point should be relation to a lack of transparency in the actual clinical therapy or cell type being administered.  Attention should be paid also to the source of the cells being administered.  This topic has been extensively reviewed in relation to the Asia Pacific Region in a paper by Janet Macpherson and myself.  It was published in the October 2011 edition of Pathology 43(6) pp616-626.  In this paper we have provided a background for cellular therapies in the Asia Pacific Region as well as an explicit discussion of the regulatory environment of many destination countries as well as ongoing clinical trials in the region (I have sought to attach it).  It may be worthwhile including this reference as part of the list of sources used.

Comments on ‘Frequently Asked Question-A Resource for Patients’.

This document should have page numbers indicated.  On the first page in figure 1 it is not necessarily technically correct that the figure has solid lines connecting the single cell to its progeny.  The implication here is that the single cell is capable of differentiating into different cell types where as in fact it is the descendants of the single cell that can differentiate into many cell types.  The figure could be made to be more technically correct by simply replacing the solid lines with arrows into dashed lines with arrows

3. Does the document contain relevant information? If not, which sections do you consider to be irrelevant?: 

See above

4. Does the document exclude any important information that would be useful to medical practitioners or patients?: 

See above

5. How could this information best be disseminated to patients and medical practitioners?: 

Hard copies of the two documents could be mailed to general practitioners and disease-related organisations with a cover letter that could direct them to obtain new hard copies or download their own copies for printing in their own offices and distribution to their clients.  It should also be the case that the PDF’s of the documents be made available on the TGA website and links to these documents should be placed strategically on many publically accessible government websites.  It would also be helpful if this web resource and hard copies were distributed to all health-related organisations that might have stakeholders who would consider undertaking stem cell tourism for any number of disease – for example organisations responsible for caring for the intellectually disabled or suffering from autism or neurologically degenerative diseases.

Ultimately I think the Department of Health could add a ‘helpline’ telephone enquiry service as part of a broader resource for health-professionals and clients.

6. How could general practitioners best access this information (i.e. an appropriate website)?: 

See above

Specific Comments
Specific Comments: 
Frequently Asked Questions - Q2

Page 2, question 2, the first paragraph is only partly correct in terms of the aims of haematopoietic stem cell transplantation.  In allogeneic stem cell transplantation the aim is to achieve a graft versus leukaemia effect as well as reconstituting haemopoiesis.  I would suggest the final sentences be updated to the following: ‘haematopoietic stem cell transplantation aims to rebuild the blood and immune system, in addition to achieving anti-leukaemia effects when the cells are donated from another person.  The cells used for haematopoietic stem cell transplantation can be obtained from one’s own body or from donated bone marrow, peripheral blood or cord blood.  Haemopoietic stem cell transplantation is often performed for patients with cancer of the blood or bone marrow whose immune system has been compromised by radiation and/or chemotherapy. 

Frequently Asked Questions - Q3

Page 2, dot points.  I do not think that this series of five dot points describing the phase of a clinical trial are really necessary.  What use is this information to the general public?  I think it would be sufficient to simply state that ‘various stages of clinical trials (Phase 1, 2, 3 and 4) are structured to obtain increased evidence of safety, tolerability and effectiveness of a potential therapy.’

Frequently Asked Questions - Q4

Page 3, in the third paragraph under question 4 the sentence is not very good English grammar.  I would suggest that it be replaced with the following: ‘as a first step, it is important to know how you can inform yourself about whether there are stem cell treatments approved by the TGA.’

In the fourth paragraph I would suggest the first two sentences be slightly modified to the following: ‘the only stem cell treatment however which is proven, standard practice in Australia is for the treatment of some cancers, genetic diseases, blood and auto-immune diseases.  This includes haematopoietic stem cell transplantation (see question 2 for definition).’ 

In the fifth paragraph the reference to ‘peer review’ begs the question of how a patient can know this.  It is one thing to state that ‘treatment should also have been peer reviewed in scientific journals’ but how will any given patient be able to verify some treatment that may be offered to them?  The right way to proceed in this paragraph would be to direct a patient to certain websites such as clinical trials.gov on the internet or another source of information.

Frequently Asked Questions - Q5

Page 4, in the patient checklist there are a series of boxes but in fact the question do not really provoke a tick of any box.  Obviously the implication is for the patients to consider each one of the dot points.  I would suggest that the heading ‘patient checklist’ be replaced by the following: ‘have you the considered the following checklist of questions?’ or something similar.

In the second dot point of page 4 in relation to the question ‘could the cells harm me or not be accepted by my immune system?’  How is a patient able to assess this?  Some comment should be added as to what this question is implying and why they need to consider this.

Dot point 4, page 4, I presume there may be a typographical error in ‘cerebral heart disease’ which makes no sense to me otherwise.

General Comments

In regards to your comments do you have any conflict of interest?

Yes. I am employed as a pathology practitioner at Genea Pty Ltd and have provided consulting services for Applied Precision and Rarecyte . I have been on the Executive for the International Society for Cellular Therapies until mid 2012. I am the Chair of the Gene Technology Technical Advisory Committee of the Office of the Gene Technology Regulator, Australian Government.

Q7. Are there any other questions or comments you have?

This is a very helpful initiative and will be of assistance to general practitioners who are increasingly receiving enquires from their patients + families. I would also suggest that it may be helpful to establish a website approved by the government of clinics overseas that are undertaking questionable practices. Although this exposes the government to potential complaints for these clinics, it is better to expose them for the benefit of an unwary public. Perhaps specifying or naming them may not be entirely helpful as that might attract attention or interest. However, there are alternative ways of highlighting specific unproven, untested or highly questionable so-called therapeutic stem cell procedures.

You may also wish to establish a panel of experts to update the government of developments. Perhaps this could be achieved via the Advisory Committee on Biologicals.

Page reviewed: 17 February, 2014