2009 Annual Scientific Meeting.StepAhead Australia's Annual Scientific Meeting held over 2 days, from the 12th -13th November, 2009 at St Vincent's Hospital, Melbourne was wonderfully received by those in attendance and a highlight of the year for StepAhead Australia. |
Feb 24 2010 |
PROGRAM SUMMARY
OPENING
Tony Benbow & George Owen
Session I: Basic Biology Spinal Cord Injury
Alex Collie - The economic cost of traumatic spinal cord injury in Australia 2008
Sheree Palmer - Living with Spinal Cord Injury
Sheree Palmer gave the most inspirational and eye-opening talk of the meeting. Speaking from the heart she detailed her story and how it began nearly two years ago and the impact it had upon her life, family and friends. She discussed the role that rehabilitation plays and how she sees the "medical system" working for and against disabled people. Living life with a spinal cord injury is a struggle on a day-to-day basis with normal basic human functions that were taken for granted are now a constant battle. Mrs. Palmer closed by expressing her feeling on what effective stem cell based treatments would mean to her life and that of the lives of all disabled people.
Jesse Owens - Obstacles and Opportunities in Chronic Spinal Cord Injury: An Overview
Dr. Owens is uniquely qualified as an expert in the area of spinal cord injury research. A paraplegic himself and a professor at the University of Alaska, Dr. Owens has dedicated his life to understanding the causes behind the inability of the spinal cord to repair itself and discovering and developing ways to improve the quality of life for those affected with this type of injury. Dr. Owens outlined the immense suffering that occurs following a spinal cord injury and detailed the major obstacles to healing and recovery of function. He is fond of pointing out that spinal cord injury is not a static disease, but rather a degenerative disorder that elicits a number of secondary problems, many which are life-threatening. With the major obstacles to repair having been identified, this raises great hope that these hurdles can be overcome based on emerging laboratory studies that have occurred in the past decade
Session II: Cellular/Matrix Therapy for Spinal Cord Injury Part 1
Brent Reynolds - Introduction to neural stem cells & application for CNS repair
Dr. Reynolds has pioneered the discovery and development of neural stem cells as a source of donor material for cell therapy applications. While numerous stem cells types have been isolated and used for cell repair, there is certain logic in using neural cells to repair the injured brain and spinal cord. In his lecture, Dr. Reynolds compared the similarity of physical engineering problems such as building a expansive bridge or launching a ship to the moon, to that of repairing the injured spinal cord. In both instances, fundamental understandings of the problems that need to be overcome are present and success is dependent on the will and desire to see these problems through. Compared to a decade ago the scientific community has a detailed appreciation of the cellular events that occur following spinal cord injury and the barriers that exist for repair. With the advent of stem cell technology and material science a large number of options exist for engineering repair of the spinal cord, what stands in the way is the means and support to do so.
Giles Plant - Cellular therapies for spinal cord injury
Dr. Plant is one of Australia's most prominent and accomplished spinal cord researchers and one of the few scientists in the world with a focus on studying and developing therapies for chronic spinal cord injury. Central to the research in his lab, Dr. Plant has invested a great deal of time and energy in developing a model of spinal cord injury that reflects the type of injury that occurs in humans. Over the past few years he has tested a number of different types of stem cell populations for their ability to induce repair and summarized these results. By dissecting the effects of transplanted cells, his lab has been able to uncover some of the mechanisms responsible for improved function and determining whether this repair is a direct result of the cells that were implanted or if they induced endogenous repair actions. The rigorous approach taken by his lab, and focus on understanding the mechanisms of action, are important for future development and in resolving the correct cell types to use.
Session III: Cellular/Matrix Therapy for Spinal Cord Injury Part 2
Rob Kapsa - Tailoring synthetic polymers for nerve repair
Following injury to the adult spinal cord a gap between the proximal and distal portion of the injury site is often created. This represents a significant hurdle, as this gap will often develop into a cyst creating a space that axons are unable to grow over. Creating bridges or providing a matrix that axons or cells can attach to is an important therapeutic avenue in developing an effective therapy. Dr. Kapsa's lab has been working on constructing bio-degradable polymers that provide the correct environment for fiber growth, cell attachment and improvements in outcomes following spinal cord injury. Central to his work is the use of “smart polymers” that are constructed with functional parameters that allow the release of growth promoting molecules to further enhance cell survival and fiber extension. Given the importance of bridging the gap and enhancing survival of cells used in transplantation approaches, the use of appropriate synthetic polymers will be key to increasing therapeutic outcomes.
Kathy Traianedes - Matrix application in a spinal cord resection model - Preliminary Results
Following a successful career as a scientist in the biotechnology field in the USA, Dr. Traianedes worked as a Senior Scientist at the Australian Stem Cell Centre. With a strong cell biology and development background, her research is now focused on developing a matrix that can be used to encourage repair in the spinal cord following injury. Building upon the success of a natural matrix called Alloderm, which has been used for a number of conditions and delivered in over 1 million operations, Dr. Traianedes is using a similar approach and matrix for spinal cord injury. Her preliminary results indicate that the newly developed matrix will be applicable for spinal cord treatment. The translational and development experience of Dr. Traianedes, together with the modification of a product that is already in the clinic, raises great hope that her project will soon see application in chronic spinal cord injury trials
Session IV: Moving Towards Translational Therapies
Ann Turnley - Blocking of Eph molecules promotes recovery from spinal cord injury
One of the key barriers following injury is the expression of molecules in the spinal cord to inhibit the growth of axons. Finding ways to inhibit the inhibitors is an intense area of research. Dr. Turnley is expert in the area of cell genesis and the response that occurs after injury. Several years ago, in collaboration with other scientists in Australia, the Turnley lab demonstrated the inhibitory role, following spinal cord injury, of a molecule called EphA4. In her lecture, Dr. Turnely presented data on the use of antibodies, which specifically target EphA4, as a way to block the inhibitors. Following injury, the antibodies are administered to the animal resulting in improved behavior and partial anatomical restoration within the spinal cord. These results are incredibly excited for several reason: (1) they appear to overcome one of the most significant barriers to regeneration within the injured adult spinal cord and (2) the use of antibodies to block cell function is well accepted in the medical community with a clear well established path for clinical development and regulatory approval.
Keynote Speaker:
Stephen Davies - Making the right astrocytes for SCI repair
A highlight of any conference is the lecture given by its keynote speaker; Dr. Davies was approached to give this lecture based on his substantial and important contributions to the field of spinal cord injury. Dr. Davies, a professor in the Department of neurosurgery at the University of Colorado, has a history of seminal findings in understanding the cellular reaction that occurs following spinal cord injury and in developing novel treatments to overcome the hurdles that are created post injury. In his lecture he took us through the events and outcomes that occur within the spinal cord after traumatic injury and the approaches that his lab is taking to over coming these barriers.His recent work has focused on generating and isolating a particular type of nervous system cell, referred to as astrocyte. While astrocytes have traditionally been seen as one of the key negative regulators of spinal cord repair, Dr. Davies research suggests that a specific subtype of astrocyte, which can be derived from a well described drug treatment, can promote efficient axon regeneration and functional repair after transplantation to the injured adult rat spinal cord. For many, his lecture was a highlight of the meeting, providing a rational and scientifically rigorous approach to therapeutic development.
Session V: Clinical & Regulator Considerations
Peter Keller - Translational research of cellular therapies for spinal cord injury: the twin challenges of Regulatory Approval and Costs
Dr. Keller is the CEO of Neuroscience Trials Australia, which plays a key facilitating role in the design and implementation in a broad range of clinical trials in Australia. Dr. Keller’s background as an optometrist, ethicist, and in business administration provides a wealth of practical experience in a challenging field of clinical trial design and tracking one’s way through regulatory approval. In his lecture Dr. Keller outlined the different phases of clinical trials (i.e. phase 1 - safety, phase 2 - dosing, phase 3 - efficacy) and the regulatory environment in Australia. He also detailed the costs to take a product from conception, or early proof of principle, through to market approval. While these costs are substantial, they highlight the effort that is required from multiple levels to develop new, safe and effective therapeutics.
Loane Skene - The ethics of not conducting clinical trials
Professor Skene is one of Australian’s most accomplished and recognized legal ethicists, her work on stem cells has guided and set the stage not only for Australia’s leading efforts in this area but also contributed to the development of guidelines used in North America and Europe. While the need and ethical requirement to protect the safety and interests of patients who are recruited for clinical trials is well recognized and discussed, little attention is paid to our responsibility and ethical duty to develop new treatments for patients who suffer from life-threatening or chronic conditions. Professor Skene brilliantly orated the two sides of the ethical dilemma – on one hand to protect those who may be taken advantage of as new therapies are designed and tested, and on the other hand to advance more quickly, life changing and saving therapies that may carry an element of risk but that may have enormous benefit. Understanding the harm that can be done to the many by protecting the few is an essential element necessary to developing and implementing regulatory policies.
Jim Faed - Development of a clinical trial in spinal cord injury: issues for design and approval of the trial
Dr. Faed is a New Zealand clinician who, together with Spinal Cord Society of New Zealand, has designed a randomized case-controlled clinical trial for chronic spinal cord injury using olfactory mucosal cells. The cellular therapy continues the work of Dr. Lima and is designed to evaluate the safety and efficacy of this treatment. The trial is pioneering in that it is one of the few trials focusing on chronic, as opposed to acute, patients. Dr. Faed shared his experience in seeking regulatory approval for his trial, pointing out the many pitfalls and roadblocks that exist in proposing and implementing novel therapeutic approaches. His lecture was enlightening with a wealth of advice, caution and inspiration that will assist other groups who want to follow along this path.
Session VI: Clinical Intervention(s)
Mark Cook - Opening Remarks
Dr. Cook addressed the importance and challenges that exist in conducting forward thinking clinical trials for treating cSCI and introduced the next set of speakers who are leading in this area and breaking new ground for the field to follow.
Maiyadhaj Samsen - Future of Stem Cell Treatment for SCI in PNI Thailand
Dr. Samsen is the Director of the Prasat Neurological Institute (PNI) in Bangkok Thailand. The Institute has three primary areas of focus, one being spinal cord injury and a strong interest in the application of stem cells for treating neurological diseases. Together with the Mahidol University, which has a strong stem cell group, PNI is planning to invest in pre-clinical and clinical studies of spinal cord injured patients. The incidence of spinal cord injury is high in Thailand due largely in part to the considerable number of people who ride motorscooters. The participation of the PNI, with their clinical base and expertise, together with a mind towards applying a stem cell based combination therapy for chronic spinal cord injury, represents a unique opportunity to accelerate to develop a meaningful treatment for spinal cord injury.
Jean Peduzzi-Nelson - Designing the Safest and Most Efficacious Clinical Trial of a Combination Treatment for Chronic, Severe Spinal Cord Injury
Continuing along with the theme of clinical intervention, Dr. Peduzzi-Nelson tackled one of the more difficult issues facing scientists who are at the stage of wanting to begin testing a promising combination therapy in humans – how does one prepare for and design the best trial possible. Dr. Peduzzi-Nelson emphasized the importance for proper trial design so as to increase the likelihood of regulatory approval and a successful outcome. One of the areas she focused on was the need for safety and the use of large animal models prior to safety studies in humans. Dr. Peduzzi-Nelson went on to summarize some of her recent collaborative data with Dr. Limas on the use of olfactory mucosal cells as a source of donor cells for transplantation into the spinal cord injured patients. She emphasized the importance of combining cellular therapy with rehabilitation to maximize the effect of the treatment.
Gustavo Moviglia - Biological Bases for Combinations of therapies applied to spinal cord injured patients
Dr. Moviglia summarized the results of his group’s most recent clinical trials on chronic spinal cord injured patients. Using one of the most innovative approaches seen in the few clinical interventions that have been published, Dr. Moviglia designed a multistage cellular therapy aimed at first encouraging vascularisation, followed by restoring specific inflammatory activity and then supplying neural precursor cells to rebuild the cellular components. The program was tied together with an intensive neuro-rehabilitation program. The results of his program are impressive with 5 of the 8 patients demonstrating an ASIA A to ASIA D improvement and the remaining 3 showing motor and sensitivity improvement.
Carlos Lima - Olfactory Mucosal Autografts and Overground Gait Training: a Combinatory Therapy for Human SCI Recovery
Dr. Lima has pioneered the application of olfactory mucosal autografts (OMA)in spinal cord injury and has recently published on the use of such cells in patients with chronic spinal cord injury. In his lecture, Dr. Lima detailed the results of using OMA in combination with extensive rehabilitation in 20 patients with chronic, sensorimotor complete or motor complete spinal cord injury. Using a mix of para and tetrplegic patients they reported improvements in the majority of patients on a number of indications including improvements in ASIA scale. Of particular interest was his use of surgical removal of the scar which presents a formative barrier to axon growth in chronic injured patients.
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