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

ID: 
16
This submission reflects the views of
Organisation Name: 
National Stem Cell Foundation of Australia (NSCFA)
Personal Details
Questions
1. Is the document in the appropriate format? If not, how could the document be better formatted?: 

The documents are in an appropriate format, the National Stem Cell Foundation of Asutralia (NSCFA) has no specific suggestions on reformatting.

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 language is appropriate for both potential patients and for doctors.  However the NSCFA has several suggestions to improve the clarity and efficacy of the documents, see section 7 below.

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

The NSCFA does not consider any of the information provided irrelevant but does have suggestions, see sections 4 and 7 below.

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

The  NSCFA makes the following important suggestions to clarify and improve the documents:  where words are to be added we have highlighted them in yellow for clarity.

Concerning the first document:

 Frequently Asked Questions- A Resource for Patients

 in paragraph 1 which ends “............All of these cells originate from stem cells.”

 Suggest adding “which may be either pluripotent, meaning they are capable of making every cell type in the body, or unipotent/multipotent, meaning they are not capable of making every cell type in the body but are capable of one or rarely more than one, but generally a few, cell types”.

 Under heading “1. What are stem cells?”

 Suggest replacing the second “stem cells” with “them” in the third bullet point, and adding the following to the  third bullet point:

  • After “..... in the treatment of disease.”  “It should be emphasised that this is a normal (non-pathological) process, for example in replacing cell types that have a naturally rapid rate of aging, death and replacement, for instance blood cells.”

 Suggest adding as fourth and fifth bullet points the following:

  • “Stem cells which are pluripotent are derived from early stage embryos (Embryonic Stem Cells or ESC), or by the manipulation of mature adult cells to become induced Pluripotent Stem cells or iPS cells”
  • “Unipotent or multipotent stem cells are derived from differentiated mature organs of a foetus, a newborn, a child or an adult and are not capable of producing every cell type in the body.  They are known as Adult Stem Cells or Committed Stem Cells”

This means that the stem cell diagram should be changed to show that the pluripotent stem cell on the left has to go through a phase as a committed or adult stem cell before becoming a committed adult cell on the lower right.  These issues are important because ESC are far less developed and proven in both clinical safety and efficacy than adult stem cells.

 Under heading “2. Which diseases/conditions are stem cell treatments proven ‘standard practice’ in Australia?”

 The NSCFA suggests the following changes:

 In paragraph 1,

In  line 1delete “...well-established stem cell..” and add “or overseas” after “in Australia”

In line 2/3 add “ ....bone marrow or peripheral blood stem cell transplantation.”

In line 3 delete “certain”

In line 5 after “for patients with”  add “partial or complete bone marrow failure or”

At the end of the paragraph  add “Transplants can be autologous – where the patient is their own stem cell donor, or allogeneic – where the stem cells are donated by another person”

 In paragraph 2,

In line 2 after “in Australia”  add “or overseas”

 Under heading “3. What is the process for developing a new medical treatment?”

 After the first paragraph suggest adding the following:  “It is important to know that it is not usual for patients to be asked to pay anything to participate in clinical trials.  The fact that you may be asked to pay for an unproven treatment should be discussed with the provider and your usual medical practitioners.”

 In the second bullet point, suggest it read as follows:

  • “Phase 1 – A small ‘first in human trial’ performed in healthy human individuals (most drugs) or cancer sufferers (cancer drugs and treatments) to evaluate....”

 Under heading “4. What evidence should I look for when researching stem cell treatments on line?”

 In the first paragraph, at the end suggest adding the following sentences: “Some stem cell clinics offering unproven treatments have recently started to operate in Australia. This has occurred because the Australian Regulations do allow autologous transplants to be offered for unproven indications.”

 In footnote 3 at the bottom of page 3 add the following: “ 3 the Therapeutic Goods Administration (TGA) is responsible for registering treatments within Australia, the Food and Drug Administration (FDA) is responsible for registering treatments within the USA”

 Under heading “5. How can I seek further information?”

 In page 4, paragraph 2 on lines 2 and 3 suggest adding the followings” It is also recommended that you discuss with your family doctor and specialist medical practitioners any information.......”

 Under “Patient Checklist”

 under bullet point 2 suggest the following:

“NB: Remember that all treatments involve risks even if the treatments are approved by regulatory authorities”

 Under bullet point 4 suggest that “cerebral heart disease” should probably read “cerebral vascular disease” although the author may have had something else in mind?

 Under bullet point 6; suggest that “How many treatments do I need?” should be a separate bullet point and check box

 In the note about the “Patient handbook produced by the Australian Stem Cell Centre-......add ” Whilst it is true that the ASCC is no longer in operation the handbook is available via links to the Stem Cells Australia (SCA) website and to the National Stem Cell Foundation of Australia (NSCFA) website, and SCA and NSCFA have jointly committed to keep the Handbook up to date.  The NSCFA is a charitable foundation which is a legacy of the ASCC and is committed to supporting stem cell research and therapy in Australia and maintaining the handbook as an up-to-date and credible information source for the foreseeable future.”

Concerning the second document:

 Stem Cell Treatments – a quick guide for medical practitioners

 under Question 2: What are stem cells and why are they important?

NSCFA believes it is important to repeat the definitions and descriptions of pluripotent and unipotent/multi[potent stem cell types, and autologous and allogeneic transplants, even though one would expect that the medical practitioners would read the first document  Frequently Asked Questions- A Resource for Patients

under Question 4: What treatments are currently standard practice in Australia?

in paragraph 2  line 3 “is” should be replaced with “are”

 in paragraph 3 line 3 haemopoietic stem cell transplantation is not commonly used for sickle cell anaemia in Australia.  It would be better to include myeloma, lymphoma, aplastic anaemia etc

after paragraph 4 it would be worth adding a new final sentence: “Unproven treatments using stem cells are starting to be offered by private clinics in Australia.”

under Question 5: What is Stem Cell ‘Tourism’ and why should patients be cautious about unproven treatments?

Given that a number of Australian Clinics are now offering these unproven treatments a number of changes are required.

In paragraph 1 the following should be added to the end:  “These kinds of treatments are starting to be offered in Australia”

In paragraph 2 the first sentence should be altered to read: “International and Australian stem cell clinics directly offer.....................”  And in the final sentence “the same treatment is promoted” should be changed to. “the same treatments are promoted”  and the final ) should be deleted

In paragraph 3 the following should be added at the end:”The regulations in Australia do at present allow Australian centres to offer unproven therapies using autologous (patient’s own) stem cells.”

under Question 6: What advice can medical practitioners give to their patients?

In paragraph 2 in line 1 / 2 the following should be added:  “Whilst it is ultimately the patient’s decision whether they decide to accept unproven treatment from a private clinic in Australia or travel overseas.....”

Under “Quick Tips for medical practitioners”, second bullet point, point 4 re types of stem cells the examples should include “(e.g cells from bone marrow, fat, umbilical cord blood....”.

Under the third bullet point, patients should also be directed to the locally produced handbook on the Stem Cells Australia/NSCFA websites entitled:”Patient Handbook produced by the Australian Stem Cell Centre – Stem cells Now and in the Future”.

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

The NSCFA believes that this initiative is sufficiently important that hard copies of the two final documents should be posted to all disease based patient advocacy groups, to every medical practitioner in Australia and to relevant paramedical practitioners such as physiotherapists, occupational therapists, speech therapists, rehabilitation specialists and so on.

It might also be appropriate to publish a précis of the information with the appropriate links in RADAR and the various Australian general and specialist medical journals.

It is important to ensure that the links appear in searches performed by patients, relatives and medical and paramedical professionals.

It would be wise for the NHMRC to apply to present these documents and their approach to relevant specialist societies, especially endocrinologists (diabetes), neurologists and neurosurgeons (stroke, spinal cord injury, MS, cerebral palsy, degenerative disease), haematologists (bone marrow transplantation), paediatricians, cardiologists, nephrologists and general physicians.  This is by no means exhaustive.

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

see responses to point 5 above

Specific Comments
Specific Comments: 
General Comments
Comments: 

NSCFA would like to emphasise its continuing role as a charitable foundation and as an important legacy of the Australian Stem Cell Centre.

NSCFA would also like to emphasise how important and worrying it regards the move to offer unproven treatments within Australia as well as overseas.  This may require changes in the regulations of the TGA and also of the Australian Health Professionals Regulatory Authority (AHPRA).

Page reviewed: 7 February, 2014