New opportunities with biological drugs

Medicines of biological origin have actually been around for a long time, such as blood transfusions and vaccines. New, however, are further developed so-called ATMP, Advanced Therapy Medicinal Products - drugs for advanced therapy based on cells, tissues and genes. An area where development is now fast.

- These are drugs that in many cases are individualized. By taking cells from one's own individual, removing a gene, modifying it and then putting it back, it is possible to stop, reverse or cure things that have not been possible before, for example certain forms of cancer, says Anna Tegnesjö, who works with sustainable precision health at Vinnova.

Can make a big difference in the future

So far, the use of these drugs is limited, but there are high hopes that they will make a big difference for many patients in the future.

Johan Hyllner is Executive Director Cell Therapy at AstraZeneca and leads the work of building their cell-based therapies globally

- We focus on developing cell-based therapies. An example of a project is about improving the lives of patients with heart attacks. Another example where there is currently an approved treatment concerns immunodeficiency diseases, where children have a short lifespan due to a genetic change in an enzyme. By removing cells from patients, modifying them so that they are correct and putting them back, five out of six patients can live a normal life. Another example is the use of immune cells from patients targeting blood cancer cells, which has given dramatically positive results, he says.

However, these drugs are expensive to develop and they also require a lot of resources from healthcare, both when they are developed and used.

- Access to clinical material is needed to carry out large-scale studies and more collaboration is needed with healthcare than with the development of traditional medicines. The cells need to be cultured and introduced into the patients, and this is often done in hospitals, close to the patient. It also requires a lot of research and development and access to skills. It is also about reimbursement models, how do you charge for these drugs? One way is to try to give payment according to the effect that the treatments give, says Anna Tegnesjö.

Industrial capacity is needed

- Sweden has cutting-edge research in academia, but not capacity for industrial production. There needs to be infrastructure for bioprocess development and production, preferably in Sweden so that companies do not disappear abroad. There is a need for collaborations between healthcare, academia and industry and there is also a need for collaborations between different types of academic institutions, from basic research to more applied research, says Johan Hyllner.

Sweden has cutting-edge research in academia, but not capacity for industrial production. There needs to be infrastructure for bioprocess development and production, preferably in Sweden so that companies do not disappear abroad.

Since 2016, Vinnova has invested in a large programme for research and development in biological drugs that finances centers and project in advanced therapy drugs. An example is the Center for Advanced Medical Products (CAMP), where health and medical care regions in Sweden are involved together with large pharmaceutical and biotechnology industry partners, small and medium-sized companies, universities and the RISE research institute. The plan is for it to become an internationally recognized center for advanced therapy with a focus on industry, growth for small and medium-sized enterprises, clinical practice, research and education, advanced production technology and a prominent innovation and business climate.

National infrastructure

The center has contributed to a plan to establish a national infrastructure for advanced therapy drugs in Sweden, based on and in collaboration with a model built at the Center for Commercialization of Regenerative Medicine in Toronto Kanada.

- It is important for Sweden's development in the area and focuses primarily on the development, production and commercialization of advanced therapy drugs. We also need to continue working with the other parts of the system, replacement models, competence development, clinical practice, academic research and the patient perspective to achieve a well-functioning whole and attractiveness for Sweden in the area, says Mats Jarekrans, programme manager biological drugs at Vinnova.

On behalf of the government, Vinnova is also involved in establishing an innovation hub to enable the production of advanced drugs and vaccines in collaboration with the company NorthX Biologics in Matfors outside Sundsvall. Something that will help small businesses, growth companies and academic groups to develop their innovation projects.

What does the future hold then?

- The hope is that cell and gene therapy-based drugs will be able to be manufactured on a large scale so that these treatments will be available to more and cheaper people. My dream is that it will be possible to treat major public diseases such as Parkinson's and diabetes and that with these therapies it will be possible to cure diseases and not just treat the symptoms. There are many clinical trials going on and breakthroughs often come suddenly, but in ten to twenty years' time I think we will see a lot of this. In some cases it will go faster, in other cases it will take longer. But we need to have a long-term perspective, says Johan Hyllner.