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.
We value your suggestions and contributions. If you wish to make a
contribution please email us at info@stemcellcentre.edu.au.
STEM CELL RESEARCH NEWS
DISEASE FOCUS: MULTIPLE SCLEROSIS
INDUSTRY NEWS
POLICY, REGULATION & ETHICS NEWS
Stem
Cell Research News
BONE STEM CELLS LOCATED
Researchers at the U.S. National Institutes of Health, working with mice, have pinpointed the location of bone generating stem cells in the spine, at the ends of shins, and in other bones. The team also has identified factors that control the stem cells' growth and studies of this stem cell population may give insight into the formation of bone tumors. The researchers have suggested that eventually, it may be possible to use these newly identified bone stem cells to repair damaged or malformed bone.
Tsang KM et al (2010), Alternate protein kinase A activity identifies a unique population of stromal cells in adult bone, PNAS, advanced online publication, doi:10.1073/pnas.1003680107 (subscription required) with commentary from HealthDay and press release from NIH.
ADDITIONAL 13 EMBRYONIC STEM CELL LINES ELIGIBLE FOR US FEDERAL FUNDING
The NIH has approved a further 13 human embryonic stem cell (hESC) lines to be eligible for US federal funding. The federal approval includes nine lines that had never before been eligible for federal funding and four long used lines derived by researchers at the University of Wisconsin, known as H7, H9, H13 and H14 of which H9 is the most widely used. This recent approval of stem cell lines has been greeted with delight by many researchers who have been waiting for such approval.
See NIH press release with commentary from The Washington Post.
GENE SILENCING RESPONSIBLE FOR LIMITATIONS ON iPS CELLS
Harvard researcher Konrad Hochedlinger and colleagues have found that an important cluster of genes is inactivated in induced pluripotent stem (iPS) cells meaning that the iPS cells do not have the full development potential of hESCs. Generated from adult cells, iPS are thought to be similar to hESC cells in their ability to form every cell in the body. The researchers found that, in mouse, a segment of chromosome 12 containing genes important for fetal development was abnormally shut off in most iPS cells. The researchers have suggested that these findings indicate that improvements are required in the way iPS cells are made. The study received advance online publication from Nature, and will appear at a later date in the journal's print edition.
Stadtfeld M et al (2010), Aberrant silencing of imprinted genes on chromosome 12qF1 in mouse induced pluripotent stem cells, Nature, 465, 175-181 Advanced online publication (25 April) doi: doi:10.1038/nature09017 (subscription required) or visit the Harvard website for more information.
AIMING TO CURE DEAFNESS, SCIENTISTS FIRST TO CREATE FUNCTIONAL INNER-EAR HAIR CELL-LIKE CELLS
Deep inside the ear, specialised hair cell-like cells detect vibrations in the air and translate them into sound. Once damaged, these cells cannot be replaced resulting in hearing loss. US researchers at Stanford University have reported that hESCs and iPS cells can be coaxed into becoming these hair like cells.
This experiment in mice raises the possibility of treating many types of deafness and hearing loss. More importantly, further study showed that the cells also responded to mechanical stimulation by producing currents just like the hair cells in the ear. The scientists hope to take the technology for creating mouse ear hair cells and apply it to humans. Recreating the hair cells from patients with hearing loss could also assist in drug discovery in this area.
Oshima K et al (2010), Mechanosensitive Hair Cell-like Cells from Embryonic and Induced Pluripotent Stem Cells, Cell 141, 704–716 (subscription may be required), with commentary from Reuters and Bionews.
SCRIPPS RESEARCH TEAM PROVIDES GROUNDBREAKING NEW UNDERSTANDING OF STEM
CELLS
Published in Nature Chemical Biology and led by Scripps Research Associate Professor Sheng Ding and Senior Director of the Scripps Research Center for Mass Spectrometry, Gary Siuzdak, the researchers used a unique approach to better understand stem cells and the process by which they differentiate into other cell types. Whilst previous studies have focused on genes and proteins, this study used metabolomics to understand cell regulation. Metabolomics is the systematic study of the unique chemical fingerprints that specific cellular processes leave behind known as metabolites. By comparing the stem cell profile to mature nerve and heart cells the researchers found about 60 previously unidentified metabolites associated with the progression of stem cells to mature cell. In addition stem cells were found to have metabolites that are chemically flexible.
Yanes O et al (2010) Metabolic oxidation regulates embryonic stem cell differentiation, Nature Chemical Biology 6, 411-411, published online 2 May 2010, doi:10.1038/nchembio.364 with commentary at Scienceblog.
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Disease Focus: Multiple Sclerosis
Multiple sclerosis (MS) has perplexed medical science since it was first described by the French neurologist Charcot in 1868. The disease affects the central nervous system and can, to varying degrees, interfere with the transmission of nerve impulses throughout the brain, spinal cord and optic nerves. MS affects an estimated 18,000 Australians. Since its identification, MS has been the subject of intense, world-wide research but still its cause and cure remain elusive.
Considerable media attention has recently been focused on the role of stem cells as a treatment for MS. In particular there has been focus on a young man from Canberra who was recently been featured on ABC Online and on the 7.30 Report who underwent successful stem cell treatment in the form of chemotherapy followed by a bone marrow transplant for severe MS. You can read more about his treatment and the science behind it on the ASCC website. This story follows recent media reports from the Bristol in the UK on a phase I clinical trial using a similar technique.
PHASE I MS STEM CELL THERAPY TRIAL AT UNIVERSITY OF BRISTOL
A Phase I clinical trial at Frenchay Hospital, Bristol, UK using autologous bone marrow stem cells to treat multiple sclerosis conducted by researchers from the University of Bristol has shown encouraging results. In this trial six patients were injected with stem cells harvested from their own bone marrow with the study aiming to determine whether the treatment is safe. There were no serious side effects from the procedure and patient's conditions remained stable over 12 months. The researchers hope to further investigate the effectiveness of the treatment later this year.
Rice CM et al (2010) Safety and Feasibility of Autologous Bone Marrow Cellular Therapy in Relapsing-Progressive Multiple Sclerosis, Clinical Pharmacology & Therapeutics, 87, 6, 679–685 with commentary at Bionews.
MS STEM CELL THERAPY GUIDELINES LAUNCHED
An international consensus has been published on the future of stem cell transplantation research for people with MS, paving the way for more co-ordinated global research efforts and potentially better, and quicker, patient access to stem cell clinical trials. The guidelines, written and approved by some of the most well respected international MS researchers, as well as MS Society's from around the world, spell out hope for the future of MS stem cell research and debunk myths about overseas stem cell clinics claiming to cure the condition. The guidelines outline the promise stem cell transplantation has shown in early stage clinical trials and ways they could be used to treat MS in the future; describe the different types of stem cells that might be used to treat different types of MS; detail methods of delivering these stem cell therapies into patients; and highlight best practice in conducting clinical trials to evaluate the safety and efficacy of stem cell therapies in MS.
Martino G et al (2010), Stem cell transplantation in multiple sclerosis: current status and future prospects, Nature Reviews Neurology 6, 247-255,(subscription may be required) with commentary at Bionews.
The MS Society in the United Kingdom recently released a detailed document relating to stem cell therapies and research in MS. A copy can be downloaded here. For more information on MS please visit the MS Society Australia website.
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CELLULAR DYNAMICS
Cellular Dynamics a company founded by Professor James Thomson of the University of Wisconsin has closed a US$40.6 million round of private equity financing. Professor Thomson discovered hESCs in 1998 and was then one of the first to reprogram human cells into iPS cells. The financing will allow the firm to increase production of human heart cells derived from iPS cells (iCell Cardiomyocytes) which will be sold to pharmaceutical companies to assist in drug discovery and screening.
In an interview with The Times Professor Thomson commented that human tissue grown from iPS cells could be used to weed out poorly-performing drugs before they moved into animal trials, reducing the number of animals used in these procedures. He also added that it may also be a better model for assessing drug safety and effectiveness.
See press release and commentary from Milwaukee Business Journal.
Interview with The Times.
MESOBLAST COMPLETES ENROLMENT FOR FIRST SPINAL FUSION TRIAL AND GAINS CLEARANCE FROM FDA TO COMMENCE PHASE II TRIAL
Mesoblast's trial investigating the use of its proprietary allogeneic stem cells (NeoFuse) in posterolateral lumbar spinal fusion at the New York Hospital for Special Surgery has completed enrolment. Results so far indicate that the treatment was safe and associated with an earlier generation of new bone formation and fusion. The company has also received clearance from the US Food and Drug Administration (FDA) to begin phase II trials of NeoFuse for the fusion of the cervical spine (neck). The trial will compare two separate dosages of NeoFuse versus the current standard-of-care in 36 patients requiring bony fusion at two or more levels in the cervical spine. The trial will recruit 24 patients at multiple sites in the United States, and 12 patients will be recruited at multiple sites in Australia.
See Mesoblast press release.
MESOBLAST JOINS UP WITH ANGIOBLAST AND RAISES FUNDS
Mesoblast will acquire all remaining shares of its USA associate company, Angioblast Systems Inc via a share exchange offer. The acquisition was also accompanied by a capital raising of $37 million from new and existing international and domestic investors. The merger will give Mesoblast full commercial and intellectual property rights to the Mesenchymal Precursor Cell technology platform. The combined entities will have a broad product pipeline including orthopaedic applications, congestive heart failure, heart attack, eye disease and diabetes.
See Mesoblast press release.
GERON ANNOUNCES POSITIVE STUDY DATA ON GRNCM1
Geron reported positive results from a preclinical safety study that demonstrated that GRNCM1, a cardiomyocyte product derived from hESCs, does not cause cardiac arrhythmias in an animal model of chronic heart injury. The current study assessed whether transplantation of GRNCM1 would increase the incidence of cardiac arrhythmia, which is a potential safety concern for cardiac cellular therapies. An arrhythmia is an abnormality of the heart rhythm, which can cause the heart to pump less efficiently.
See Geron press release.
OSIRIS' TYPE 1 DIABETES TREATMENT RECEIVED ORPHAN DRUG DESIGNATION FROM FDA
Osiris Therapeutics has been granted orphan drug designation from the FDA for Prochymal as a treatment for type 1 diabetes mellitus. Prochymal is currently in Phase II clinical trials in the USA for this indication. Orphan drug designation offers a number of advantages such as market exclusivity for seven years and a reduction in registration fees. Orphan drugs must target a disease that affects fewer than 200,000 new patients per year in the United States. Prochymal is a formulation of adult mesenchymal stem cells.
See Osiris press release.
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Policy, Regulation & Ethics News
INTERNATIONAL TELEVISION SPECIALS ON STEM CELL TOURISM
On 19 April 2010 the US 60 Minutes program on CBS aired a story "21st Century Snake Oil" on stem cell tourism. The program used hidden cameras in a clinic offering to cure patients with motor neuron disease and features leading stem cell scientists. To find out more and watch the program online click here.
On 13 May 2010, Irish television station RTE One screened a story called "Hope Hype" investigating the growing number of Irish people travelling overseas for unproven stem cell therapies. The program can be watched online here.
€80 MILLION FUNDING FOR iPS CELL RESEARCH CENTRE IN NORTH RHINE-WESTPHALIA, GERMANY
The Center for Advanced Regenerative Engineering (CARE) was initiated by Professor Dr Hans Schöler, Director of the Max Planck Institute for Molecular Biomedicine in Muenster. The Lead Discovery Center GmbH, in Dortmund, is also on board to collaborate in the development of pharmaceuticals. With initial funding of €80 million (€60 million coming from the local state government of North Rhine Westphalia) CARE is expected to become a flagship project in stem cell research and applications in the area.
Learn more from the Stem Cell Network North Rhine Westphalia or the Max Planck Institute.
STEM CELL PATENT BATTLE CONTINUES
The ongoing battle between the Wisconsin Alumni Research Foundation (WARF) and the Public Patent Foundation has seen one of WARF's three stem cell patents declared invalid by the US Patent and Trademark Office. The patents in question relate to methods for deriving and growing primate and human embryonic stem cells. The Public Patent Foundation has been arguing that the discoveries were not novel enough to be patented and that the claims were too broad and thus impeded stem cell research. The Foundation now claims that the invalidation of one patent effectively invalidates the other two.
See discussion at Nature Blog The Great Beyond.
CANADIAN STEM CELL FOUNDATION WEBSITE WINS AWARD
In May the Canadian Stem Cell Foundation won the People's Voice Award for the Best Activism Website at the 14th Annual Webby Awards. Hailed as the "Internet's highest honour" by the New York Times, The Webby is the leading international award honouring excellence on the Internet. James Price, the foundation's CEO and President commented, "Not only has the public made it clear that stem cell science is important, they have taken this cause as their own". The Foundation website went public last September and opens with "Rock Star Scientists" a 90 second video featuring leading scientists making a passionate case to do more to support stem cell science.
See the website at www.stemcellfoundation.ca
EU AGENCY PREPARES TO ASSESS FIRST STEM CELL DRUG
The European Medicines Agency (EMEA) has been informed about the intent of a European manufacturer to submit the first application for marketing authorisation of a stem cell-based product. Drug makers usually send a letter of intent to the London-based watchdog four to six months before a formal application which would imply a filing toward the end of 2010. The EMEA declined to name the company involved. In preparation for the first applications, officials from the EMEA met recently with drug company officials, regulators from the United States and Japan, and academic scientists to discuss guidelines for approving such treatments. To address some of the issues, the EMEA has drafted a "reflection paper" on the process for approving stem cell-based therapies, which will be finalised by the end of 2010.
Read the reflection paper here or see story at Reuters.
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Stem cell therapy for multiple sclerosis – update
Back in January 2010 the ASCC reported on an ABC news story which focused on a young Canberra man with severe MS who made a substantial recovery after receiving stem cell therapy. Here is an update.
Training Australian Scientists in Latest Stem Cell Discoveries
For the first time in Australia, young researchers will be trained in the techniques of growing and using human induced pluripotent stem (iPS) cells in research.
Can Rabbit Stem Cells Cure Your Ills?
Recent media attention has focused on an organisation known as Stem Cell Biomedics who are advertising information seminars in Perth and Sydney on unproven risky rabbit fetus stem cell 'therapies'. Stem cell experts warn that therapies offered by visiting European practitioners are unproven, potentially dangerous and illegal in Australia.
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