ASCC Research Portfolio

Therapeutic Programs - Providing Solutions for Unmet Clinical Needs

Treatment with cell-based and regenerative products offers the hope of providing patients with functional improvements to replace the diseased or damaged organs or tissues. The ASCC actively identifies specific therapeutic areas in which to focus their research and development with the aim of delivering therapeutic outcomes. Haematology is currently the most comprehensive of these Programs and comprises four product teams each including multiple research projects being conducted in research Institutes across Australia. This Program represents a cross-disciplinary, multi-institution approach to creating a new paradigm in the supply of blood products such as red blood cells and platelets.

The ASCC Haematology Therapeutic Program -

creating blood from stem cells

The Australian Stem Cell Centre, via our Haematology Therapeutic Platform, has a major investment in the development of blood from stem cells with the aim of creating a manufactured transfusible blood product. The need to create a safe and ready supply of blood is vital and becoming more urgent. Increasing numbers of people are no longer suitable as blood donors due to known and newly emerging infectious diseases, including Jacob-Kreutzfeld disease, hepatitis and AIDS. In third world countries, a blood collection and screening infrastructure is virtually non-existent and many people unnecessarily die from the simple but critical lack of available blood. Given these issues, a safe stem-cell based transfusible blood product would relieve the considerable escalating pressure on this industry and contribute significantly toward Australia’s growing expertise in blood products.

One of the more significant achievements within this Program to date has been the differentiation of embryonic stem cells to relatively pure populations of haematopoietic cells as well as the creation of primitive, or precursor blood cells.  In addition, the program focuses on expansion of adult stem cells from umbilical cord. Considerable progress has also been made in defining the environment, or niche, in which adult stem cells reside in the bone marrow, which in time give rise to blood cells.

The Haematology Program is an innovative approach to the use of stem cell technologies based on world-class expertise in the ASCC. This program has the potential to place Australia at the forefront of cell-based therapeutics and may be the first widespread use of a product based on human embryonic stem cell technologies.

Identifying New Therapeutic Programs

The ASCCs therapeutic programs may change over time according to the discoveries generated from the platform technologies, commercial realities, intellectual property development or clinical advances in disease treatments. The selection of our current and future therapeutic Programs involves an extensive scientific and commercial due diligence process. This evaluation process assesses the likelihood of success in the proposed areas based on the strengths of existing Australian research, the intellectual property environment, regulatory hurdles, international competition, commercialisation, and significant unmet clinical need.

Each therapeutic Program must also address a major medical need, such as a condition in which patients currently have limited treatment options, the condition severely impinge upon a patient's quality of life and/or the disease condition is highly prevalent. In addition, each Program must also represent a critical mass of complementary primary projects interacting to advance current therapies in the short, medium and long term.

More information on stem cells and what we hope to achieve with this technology can be found in the Stem Cell Section. Additional material is also available through the section.

 

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