Oncotyrol, an Example That Translational Research Can Work
- Fig. 1: Langerhans cell between collagen fibrils. The topic of this picture is the fight of the immune system against cancer. It shows a vaccine carrying dentritic cell (Langerhans cell) on its journey through the (collagen) connective tissue of the skin. Its destination is the lymph node, where immune cells are being activated against tumors. © K. Pfaller
- Fig. 2: Shown are two proteins that are bound to each other. One protein (BAG-1, white) is involved in the development of cancer. BAG-1 needs a protein, called Hsc70 (grey), to fulfill its function. One research project at Oncotyrol tries to inhibit the interaction of these two proteins, to avoid the development of cancer. Highlighted in green is the part of the protein that is important for the binding of Hsc70. © Marion Enthammer
- Fig. 3: Breast cancer cell. Breast cancer is one subject Oncotyrol is focused on. © K. Pfaller
- Fig. 4: A CD8+ lymphocyte attacks a tumor cell. The T-cells (white blood cells) try to destroy the cancer cells. At Oncotyrol strategies are being developed in different research projects, to help and strengthen the immune system in its fight against cancer. © K. Pfaller
- Fig. 5: Mammary carcinoma. This picture shows a colored histological cut of breast cancer tissue and the molecule HER2 is excessively expressed (dark staining). Meanwhile a target-oriented therapy against HER2-positive cancer exists. The goal of Oncotyrol is the development of such therapies that are target-oriented at the molecular level. © Elisabeth Müller-Holzner
Basic research focuses on many issues that are important for human health. However, despite of permanent scientific progress only a few innovative drugs actually reach the patient. Oncotyrol, the Center for personalized cancer medicine in Innsbruck, has developed an appropriate strategy in order to improve the rate at which research is implemented. Equitable distribution of intellectual property rights and risk minimization for the companies are the key factors.
The distribution of tasks between the state and the private sector in healthcare evolved on a historical basis. The state is positioned at the beginning and the end of the chain as it is in charge of the university-related basic research as well as the efficient patient care. The development, production and commercialization of drugs and medical products by the private sector is encompassed within these two points. Unfortunately research results are not always transferred in an optimal way from the industrial sector to medical practice. Therefore translational research, i.e. basic research, which is focused on concrete economic benefits, is increasingly promoted by the state.
Oncotyrol is a good example to illustrate this: It is a translational research center for personalized cancer medicine and is based on Public Private Partnership (PPP), thus a long-term partnership between public (research) institutions and the private sector. Within the scope of the COMET program of the Austrian Research Promotion Agency (FFG), 55 % of the research is funded by public authorities and 45 % by the industry.
Different Research Philosophies Inhibit Cooperation
There are many reasons, which complicate or inhibit cooperation between basic researchers and life science companies. The different philosophies behind academic or profit-oriented science play an essential role. The gain of scientific knowledge alone is all that counts at universities. Idealistically speaking research is driven by pure intellectual curiosity. Some researchers even condemn the idea of profitable commercialization. Academic research is particularly interested in fundamental questions, for example trying to prove that molecule xy inhibits the expression of gene yz, which is known to be involved in the development of a disease.
With regard to the industrial sector the real work starts at that point. In order to find out whether molecule xy is suitable as a target molecule for the development of active substances, it must undergo extensive testing. This target validation is very costly and time consuming but absolutely essential, since pharmaceutical companies often cannot afford expensive failures in later stages of the development.
Industrial Sector Avoids Major Investments Due to High Risk
Another problem is that industrial research must comply with certified experimental standards such as Good Manufacturing Practice (GMP) or Good Laboratory Practice (GLP) to guarantee the strongest industrial protection rights and good conditions for admission to the market at a later stage. In academic research these experimental and technical conditions are generally not met.
For industrial partners this means that they have to invest significant amounts in the transition from academic to industrial standards. The financial risk is considerable, bearing in mind the relatively high probability of failure in the development. Companies only take this risk in exceptional cases, if they in return receive exclusive Intellectual Property Rights. On the other hand this poses a problem to researchers and public institutions.
Matter of Conscience for the Researchers: Publication or Patent
A fundamental problem for academic researchers in industrial cooperations is the manner in which scientific careers are closely linked to publications. Results have to be published in a most effective way. However this is where the dilemma begins. What is published cannot be patented because you cannot have patented what is published, and what is not protected by patent cannot be brought to the market. Vice versa researchers can often not afford to wait until results are being protected. It is possible that a competitor publishes the same "story" in the meantime and the publication that is so important for the career development is lost.
Oncotyrol‘s Deal: Risk Minimization Hand in Hand With Fair IPR Regulation
Oncotyrol works in research close together with company partners and these partners can make their technical and experimental requirements known from the very beginning. For this reason research is in the first place conducted in an industrially compatible way, allowing companies to avoid the expense of adaptation to industrial standards. In this way the financial risk of the companies is significantly reduced and the additional advantage of state funding makes cooperation more interesting to the sector. The companies benefit from early access to clinical and basic science expertise. Important for the researchers and their institutions is that the high scientific potential and the experience gained over the years remains on site and is not industrially outsourced.
A deciding factor in the success of translational research is a strong strategy on how IPR (Intellectual Property Rights) matters are being handled. Oncotyrol has developed its own solution for this. The reasoning behind this is the following: At least three parties make their contributions, namely the academic institution, the company partner/s and the Center itself, which as a legal entity defends the interests of the public funding authority. Consequently all these parties have rights to the intellectual property arising in the project. The fair distribution of IPR may seem difficult. But Oncotyrol has developed a quantitative method in order to precisely evaluate the material as well as the immaterial, i.e. intellectual contribution of each partner. Experience shows that it is possible to satisfy all partners. But above all it turns out that a strong, trusting and long-lasting partnership is even more valuable than intellectual property - to all partners.
Personalized Cancer Medicine as a Prime Example of Translational Research
In cancer research, previous standard therapies are being more and more replaced by molecular target-oriented therapies. They are directly based on knowledge gained from basic research. Thus they are not empirically discovered but rationally designed. Research has shown that the classification of cancer types into organs and histological findings is only partially useful. The molecular diagnosis of the causes proves to be just as significant, in determining the further course of the disease and the therapeutic decision, as the conventional histological or imaging techniques.
Therefore the expertise of basic molecular researchers is as important for translational research in this area as practical medical experience in the daily interaction of physicians with their patients. Also large quantities of sample material and high-end technologies are necessary to analyze this sample material. The life science location in Innsbruck and Hall in the Tyrol comprising three universities, a hospital with a total of 1800 beds and the local biotech companies fulfill these requirements. The know-how in handling large-scale equipment at an industrial level is of equal importance for the successful implementation of personalized medicine. Oncotyrol's 34 international company partners contribute to this know-how by cooperating in 29 research projects with academic researchers. This cooperation should help to make the results of research more readily available to benefit the lives of those for whom they are intended - the patients.