Our aim at the Australian Stem Cell Centre is to keep you
informed about the latest developments across the stem cell field
including recent highlights in research, clinical translation and
relevant policy and regulations.
SEASONS GREETINGS & ASCC CHRISTMAS TRADING HOURS
On behalf of the the ASCC, we wish you and your family a happy and safe festive season. We look forward to working with you all in the New Year.
The ASCC will be closing over the Christmas to New Year period, resuming normal business as of Monday, 4 January 2010.
Contents:
STEM CELL RESEARCH NEWS
PUBLICATIONS
INDUSTRY NEWS
POLICY & REGULATION NEWS
NEWS IN FOCUS
Stem Cell Research News
USA APPROVES FUNDING FOR USE OF NEW HUMAN EMBRYONIC STEM CELL (hESC) LINES
The USA Government through the National Institute of Health (NIH) has approved 40 new human embryonic stem cell lines for use in federally funded research under reduced restrictions announced by President Barack Obama in March 2009. The lines were made by researchers at the Children's Hospital Boston, Harvard University and Rockefeller University using private funds. These are the first new lines to be approved since former President Bush placed restrictions on the funding of hESC research. President Bush's restrictions meant researchers were limited to a very small number of lines that were created before August 2001. During his two terms in office, President Bush went on to famously veto two bills which would have allowed funding for the use of newer lines.
One restriction that may never be removed is that set by Congress in 1996, known as the Dickey-Wicker amendment, which forbids the use of federal funds for any research involving the creation of human embryos for research purposes or for research in which human embryos are destroyed. Therefore the creation of hESC lines will still require alternative sources of funding.
The NIH has established guidelines which ensure strict ethical criteria for acceptance of the stem cells into its registry for funding. The cells, for instance, must have been made using an embryo donated from leftovers at fertility clinics, and parents must have signed detailed consent forms.
More information, the guidelines and the list of eligible lines can be found here http://stemcells.nih.gov.
TECHNICAL CHALLENGES IN USING HUMAN INDUCED PLURIPOTENT STEM CELLS TO MODEL DISEASE
A recent paper in Cell Stem Cell by Krishanu Saha and Rudolf Jaenisch both of the Whitehead Institute for Biomedical Research, discusses an often overlooked use for stem cell technology which is to better understand disease. Traditionally animal models have been used to study diseases, the interactions of drugs and how they work but these models have limitations. Stem cells offer the potential to study diseases and test pharmaceuticals in a human model which would clearly be advantageous. This article examines the challenges and assumptions in creating a disease model from a single cell of the patient. Human induced pluripotent stem cells (iPS cells) provide researchers in the laboratory critical access to patient-derived biomaterials, which constitute the principal input for disease studies. However, there are many technical challenges in generating and manipulating iPS cells before they can be thought to be faithful models of specific diseases.
Read more - Technical Challenges in Using Human Induced Pluripotent Stem Cells to Model Disease. Cell Stem Cell (4 December 2009). DOI 10.1016/j.stem.2009.11.009 (subscription may be required).
STEM CELLS COULD CREATE NEW SKIN TO HELP BURNS VICTIMS
Researchers from the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases in Evry, France, have successfully completed a preclinical study in mice using human embryonic stem cells to create skin which could help serious burns victims. The study published in the journal The Lancet demonstrates that the stem cells grew into fully formed skin just 12 weeks after grafting. This could solve the common problem of rejection that burns victims face while they wait for autologous skin grafts which usually takes at least three weeks during which the patient is at risk of dehydration and infection.
Read more - Human embryonic stem-cell derivatives for full reconstruction of the pluristratified epidermis: a preclinical study. The Lancet, (21 November 2009). DOI:10.1016/S0140-6736(09)61496-3 (subscription may be required) with commentary from BBC News.
STEM CELL INDUCTION MADE SIMPLER
Mouse iPS cells can now be made by inserting genes at just one location, avoiding the use of multiple genetic insertions using viruses. A key challenge for iPS cells has been the fact that to induce pluripotency, four reprogramming genes must be inserted into the genome — Oct4, Sox2, Klf4 and c-Myc, using multiple retroviruses, meaning the genes end up in random locations in the genome, which can interfere with the function of the genes. Using a mouse model, two teams of researchers, led by Rudolf Jaenisch at the Massachusetts Institute of Technology and Whitehead Institute for Biomedical Research and Konrad Hochedlinger at Harvard University, describe a technique in Nature Methods that avoids these difficulties. The technique may also help to answer lingering doubts about the differences between iPS cells and embryonic stem cells as it will now be possible to compare two genetically matched cell types and ask if iPS cells are as useful as embryonic stem cells.
Read more - Single-gene transgenic mouse strains for reprogramming adult somatic cells. Nature Methods (13 December 2009) DOI:10.1038/nmeth.1410, and A reprogrammable mouse strain from gene-targeted embryonic stem cells. Nature Methods (13 December 2009) DOI:10.1038/nmeth.1409 (subscription may be required) with commentary from Nature News (published online 13 December 2009) DOI:10.1038/news.2009.1140.
STEM CELLS AS THE NEW HIV VACCINE
Researchers from the UCLA AIDS Institute and colleagues have demonstrated that human blood stem cells can be engineered into cells that can target and kill HIV infected cells - a process that potentially could be used against a range of chronic viral diseases much like a genetic vaccine. This type of approach can be used to engineer the human immune system, particularly the T-cell response, to specifically target HIV infected cells. These studies lay the foundation for further therapeutic development that involves restoring damaged or defective immune responses towards a variety of viruses that cause chronic disease, or even different types of tumors.
Read more - Engineering Antigen-Specific T Cells from Genetically Modified Human Hematopoietic Stem Cells in Immunodeficient Mice. PLoS ONE 4(12): e8208.DOI:10.1371/journal.pone.0008208 (subscription may be required).
STEM CELLS AND BLINDNESS
A recent clinical trial by investigators at the North East England Stem Cell Institute has demonstrated the potential of stem cell therapy to improve and potentially restore the sight of patients suffering Limbal Stem Cell Deficiency (LSCD) or similar ocular diseases. LSCD, commonly caused by chemical burns, requires long term, costly treatment with frequent hospital visits.
The sight of eight patients with LSCD was improved by cultured stem cells taken from their own eyes. This study demonstrates that transplantation of cultured corneal stem cells without the use of animal cells or products is an effective method of reconstructing the corneal surface and restoring useful sight in patients with unilateral LSCD. A larger study involving 24 new patients is currently underway with funding from the UK's Medical Research Council. Future research will ascertain whether this therapy could be used to treat conditions such as age related macular degeneration.
Read more - Successful Clinical Implementation of Corneal Epithelial Stem Cell Therapy for Treatment of Unilateral Limbal Stem Cell Deficiency Stem Cells Online Publication (10 December 2009) DOI: 10.1002/stem.276, with commentary from Top News.
Another recently published paper demonstrated that iPS cells could be used to rescue vision in blind rats. The iPS cells were turned into retinal pigment epithelium (RPE) cells, which are essential for proper vision. These cells were transplanted into rats that were blind due to having inherited dysfunctional RPE cells. The transplanted cells rescued the blind rats’ vision. These findings suggest that RPE cells derived from iPS cells, could be used to treat blindness in humans, such as blindness caused by age-related macular degeneration, a leading cause of blindness in the western world.
Read more - Protective Effects of Human iPS-Derived Retinal Pigment Epithelium Cell Transplantation in the Retinal Dystrophic Rat 4(12): e8152. DOI:10.1371/journal.pone.0008152, with commentary from The Independent.
Back in Australia, Dr Nick Di Girolamo and Dr Stephanie Watson were recently winners and people’s choice award recipients on the ABC’s New Inventors for their invention, a pioneering technique to treat corneal blindness. The condition affects approximately 10 million people worldwide. The technique has already been trialled to establish safety and efficacy in a small pilot study of three patients. The ASCC supports this work with a grant from our Strategic Development Fund to allow the investigators to expand to a larger trial of 33 patients.
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Some Recent Key Publications
Stem cell researchers from across Australia (including those funded and
not funded by the ASCC) continue to make significant contributions to
the field in diverse areas. Below is a selection of recent key
publications.
Human DNA methylomes at base resolution show widespread epigenomic differences.
Lister, R., Pelizzola, M., Dowen, R. H., Hawkins, R. D., Hon, G., Tonti-Filippini, J., Nery, J. R., Lee, L., Ye, Z., Ngo, Q. M., et al. (2009).. Nature 462, 315-322. DOI: 10.1038/nature08514
www.nature.com/nature/journal/v462/n7271/full/nature08514.html
Direct cell reprogramming is a stochastic (random) process amenable to acceleration.
Hanna J, Saha K, Pando B, van Zon J, Lengner CJ. Creyghton MP, van Oudenaarden A & Jaenisch R. Nature 462, 595-601 (3 December 2009). DOI:10.1038/nature08592.
www.nature.com/nature/journal/v462/n7273/full/nature08592.html
Forcing cells to change lineages.
Graf T, Enver T. Nature 462 (3 December 2009). DOI:10.1038/nature08533
www.nature.com/nature/journal/v462/n7273/full/nature08533.html
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UK's ReNeuron AWARDED TWO GRANTS
ReNeuron has been awarded two grants under the UK Governmen backed Technology Strategy Board’s Regenerative Medicine Programme. This funding programme was launched earlier in 2009 to underpin and enable the best regenerative medicine businesses to flourish in the UK. ReNeuron will use the funding provided under these initial awards to complete the remaining steps to commencement of the Phase I clinical trial for its ReN001 fetal neural stem cell therapy for disabled stroke patients, as well as progressing certain late pre-clinical activities with its ReN009 therapy for peripheral arterial disease.
For more information see www.reneuron.co.uk.
MESOBLAST SUCCESS WITH DIABETES PRECLINICAL TRIAL
Mesoblast has moved one step closer to human trials in diabetes after announcing successful preclinical trial results in their adult stem cell platform. The collaborative study performed with Dr Ravi Krishnan, senior scientist at the Queen Elizabeth Hospital and Mesoblast showed that the injection of adult mesenchymal precursor cells into mice diabetes helped restore their damaged pancreas and reduce blood glucose levels for a three week follow up period. Read Mesoblast's Media Release.
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Policy and Regulation News
AUSTRALIAN STEM CELL CENTRE PUBLISHES PATIENT INFORMATION HANDBOOK
Overseas stem cell therapies: are they safe and how do you know?
In response to increasing concerns surrounding unregulated and unproven stem cell therapies offered to patients overseas, the Australian Stem Cell Centre has published a Patient Handbook. The marketing of overseas stem cell therapies via the internet has been around for some years, but there is increasing interest from Australians in accessing these therapies. Often clinics use the testimonials of patients as a marketing technique, hiding the fact that much of what they offer is not supported by clinical trials and is therefore untested.
There is a clear need to assist the public in understanding what is involved and to guide individuals on how to fully investigate treatment options before travelling or participating.
The Patient Handbook is designed to help individuals understand what stem cells are, which stem cell treatments are considered safe and effective by specialists, which are considered experimental and which are unproven and the safety of the treatment is unknown. The Handbook does not seek to advise patients, evaluate individual treatments, or comment on an individual's reasons for travelling for treatment, but aims to provide the patient with the necessary information prior to considering any therapy.
Visit our Patients page and download the handbook.
iPS CELLS: MAPPING THE POLICY ISSUES
Since their discovery in 2007, there has been an explosion of research on iPS cells. In association with the 2009 annual meeting of the International Society for Stem Cell Research, a workshop of stem cell scientists, bioethicists and ethical, legal, and social issues specialists discussed various ethical, legal, social, and policy issues associated with aspects of iPS cell procurement and basic research. In this fascinating commentary issues such as privacy, consent, intellectual property, potential uses, clinical translation and regulations are considered.
iPS Cells: Mapping the Policy Issues. Cell 139:6 (11 December 2009), DOI 10.1016/j.cell.2009.11.039 (subscription may be required).
TGA COMMENCES PUBLIC CONSULTATION ON PROPOSED STANDARDS FOR HUMAN TISSUES AND CELLULAR THERAPIES
The Therapeutic Goods Administration (TGA) has commenced public consultation on proposed standards and Code of GMP (Good Manufacturing Practice) for human blood and blood components, human tissues and human cellular therapies. The consultation period is from 7 December 2009 to close of business 12 February 2010. Submissions are invited on the following documents:
- Australian Code of Good Manufacturing Practice human blood and blood components, human tissues and human cellular therapies
- Draft Therapeutic Goods Order - Standards for minimising infectious disease transmission via therapeutic goods that are human blood and blood components, human tissues and human cellular therapies
- Draft Therapeutic Goods Order - Standards for banked human cardiovascular tissue
- Draft Therapeutic Goods Order - Standards for banked human musculoskeletal tissue
- Draft Therapeutic Goods Order - Standards for banked human ocular tissue
- Draft Therapeutic Goods Order - Standards for banked human skin.
These six consultation documents, as well as details on how to make a submission, can be found here.
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NEWS IN FOCUS
STEM CELL THERAPIES AND MULTIPLE SCLEROSIS
Almost 16,000 Australians suffer from multiple sclerosis (MS). MS is an autoimmune disease whereby the immune system attacks its own myelin, causing disruption to nerve transmission affecting the central nervous system and stopping nerve impulses travelling to the brain, spinal cord and eyes. MS is unpredictable with varying symptoms and little is known about what causes the disease. Recent news reports have focused on the case of a young Australian man with severe MS who appears to have made a substantial recovery from MS after receiving stem cell therapy in Australia.
The reported experimental treatment involves the extraction of stem cells containing bone marrow from the patient followed by a type of chemotherapy to suppress the immune system and purge all the autoreactive cells from the body. The bone marrow cells are then transplanted back into the patient in the hope that they will reset the immune system. However, this treatment which was first reported in 1997 is not currently routinely used as the risk of the patient developing a serious infection or complications immediately after the chemotherapy treatment while the immune system is suppressed is high.
However, a phase 1 clinical trial of stem cell treatment for MS using a less toxic type of chemotherapy was reported earlier this year in the medical journal The Lancet. The trial carried out in the USA treated 21 patients was small and according to The Lancet editorial 'Although this study is small and had no control procedure, the results show that the technique is feasible and worth further investigation as a potential way to reverse disability in patients with MS'. Details of this trial can be found at: Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. The Lancet Neurology Vol 8, Issue 3 p244 - 253, March 2009. DOI: 10.1016/S1474-4422(09)70017-1 (subscription may be required).
More information on the processes of experimental treatments and clinical trials can be found in the ASCC’s Patient Handbook, or visit MS Australia
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Australian Stem Cell Centre Publishes Patient Handbook
Overseas stem cell therapies: are they safe and how do you know?
2010 ASCC International Travel Awards for ISSCR - Applications Open
The next round of travel awards is for the ISSCR Annual Meeting in San Francisco, June 2010. Deadline for applications is 26 February 2010.
Call for Papers: SBE Stem Cell Engineering Conference
DEADLINE EXTENDED: Submit an abstact by 05 February 2010 for this conference that brings together the bioengineering and stem cell biology communities.
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