In this article, we shine the spotlight on Dr Paula Salmikangas. Paula is the Director of Biopharmaceuticals and ATMPs and has been at NDA since 2017. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and the CMC aspects of biopharmaceuticals.
Making a footprint in the future of medicine
Career paths can look quite different. Some people seem to know from birth what they were put on earth to do, while others walk a more crooked path.
Paula Salmikangas´ path has been a straight conceptual line, like a brick road built on past experiences and leading her to the answers she is looking for. Paula has contributed to the world of medicine in many ways, as a researcher, as a regulator and as a consultant. By positioning herself where her knowledge about cell- and molecular biology meets the most innovative Advanced Therapy Products, Paula is in part responsible for many patients receiving breakthrough treatments.
Paula started out as a clinical biochemist, working at Helsinki University Hospital in Finland. At the same time, she was involved in research, investigating different gene defects behind certain rare diseases. A research group in the Department of Pathology had discovered a new unknown DNA fragment which became Paulas PhD project. After identifying the full DNA sequence, she found that the respective gene was associated with a rare muscle disease, limb girdle muscular dystrophy 1A (LGMD 1A) and contacted a research group in the US investigating the disease. This collaborative research discovered that the mutations in this gene were the sole cause of the LGMD1A disease.
“ I was quite fortunate ”, Paula says. “ My PhD project turned out quite successful, and it was fantastic to get new results almost every day. ”
Paula’s early research set the tone for the rest of her career. Due to her background in cell and molecular biology, she soon became a CMC assessor for biologicals at the Finnish Medicines Agency, with a host of very interesting review projects on cell-based products from the European Medicines Agency (EMA). This, in turn, led to her becoming a member, and later also Chair of the Cell Products Working Party (CPWP) at EMA. In 2009, when the Committee for Advanced Therapies (CAT) was established at EMA, Paula became a Member and Vice-Chair of the Committee. In 2014 she was elected as the Chair of the CAT, leading the CAT discussions on ATMP processes including Marketing Authorisation Applications (MAAs), scientific advice, PRIME designations, classifications and certifications. Holding these positions Paula has been involved in the preparation of most of the guidelines available today for Cell and Gene Therapy Products in the EU.
Research is curiosity formalised
Paula is a scientist at her core. After working for many years as a regulator she decided to take a seat on the other side of the table and joined NDA Groups Advisory Board. Although the step from the regulatory world to consultancy services was big and a bit scary, the possibility to work on a wide array of ATMP projects with many ex-colleagues from EMA Committees made the decision easier. Even if research sometimes can be unpredictable and challenging, she is happy to be closer to science when supporting the developers bringing new products on the way to the market, the challenges faced by the developers really motivate Paula to help find suitable solutions. By being heavily involved when the ATMP regulation was negotiated in the EU, she has an unparalleled understanding of the system, which is a valuable asset to her as a consultant.
“ I am always on the lookout for new information ” Paula explains. “ Now, when I’m no longer generating it myself, it is fantastic to get new data and information from clients and literature and see how things are moving forward ”.
ATMPs are highly promising new medicines, some of them could even be curative, but they have also very different safety issues than standard medicines and may pose high risks to the patients. Working with novel products where, in most cases, the whole picture is still not fully understood is exactly where Paula wants to be. She sits in the front row when new ATMPs are developed and introduced to the market to help severely ill patients.
“ Every new product entity brings something extraordinary ” she emphasises. “ Many of the ATMPs are really ground-breaking, giving treatment options for patients that have nothing. It is very motivating to work with these novel products and help bringing them to the patients ”.
When risk is a sure thing
The field of Cell and Gene Therapies is full of unknowns and Paula has spent decades of her life to deal with the outcomes of these unknowns. While innovation moves forward in warp speed, we are still in a place where the long-term consequences are not fully identified. The possibilities to study these products before introducing them into patients are limited, an enigma difficult to side-step.
As Paula has greater insights than most, her knowledge is highly desired when it comes to Cell and Gene Therapy advice. She has learned over time that her work usually peaks periodically and tries to be realistic about keeping her schedule. But for someone who easily gets excited about new and thought-provoking data, it sometimes happens that she accepts more work than planned.
“ I try to keep weekends free from work to have a full break and take my mind off work. Just to get out to the nature and have a change of scenery can recharge my batteries ”
The landscape in medicine is also changing thanks to the new Cell and Gene Therapies. Effective products have been put on the market and their safety issues are managed; there are now treatment options even to some of the more severe side effects caused by ATMPs. It is a big step from traditional fully controlled chemical drugs to ATMPs using cells and genes as the starting material. Cells as viable entities have tens of thousands of genes and proteins, own organelles and signalling systems, which makes things more unpredictable and the products are difficult to control.
“ It all boils down to ensuring safety for the patients ” Paula concludes. “ For these new ATMPs we have to find ways to identify the risks early on and to control them on the product level ”.
And being Paula, her face lights up as she adds: "But of course, if you never try, there will be no new treatments"
