The first scientific symposium on CDNF/MANF-family proteins

Henri Huttunen 22.5.2018

One integral part of scientific research are international conferences where scientists gather together from all over the world to report their most recent results and discuss future direction of research. The first symposium dedicated to CDNF/MANF-family proteins was organized as part of the International Meeting on Neural Therapy and Repair (INTR) on 25-27 April 2018 in Clearwater Beach, Florida. Drs. Merja Voutilainen and Mikko Airavaara, and professor Mart Saarma from the University of Helsinki, and Dr. Henri Huttunen from Herantis Pharma presented results on CDNF and MANF in the conference, focusing on Parkinson’s disease, ALS and ischemic stroke. The recently started first-in-human study with CDNF protein was warmly welcomed by the audience. Advancement of a new drug candidate from preclinical development to clinical trials is exciting news in any field. In the field of neurotrophic factors, this was the first time for years.

The history of neurotrophic factors includes many failures in the clinical trials, mostly due to challenges in drug delivery. Delivering a biological drug molecule to the target area in the brain is far from trivial, and over the past two decades major advances have been achieved in brain drug delivery. A pioneer in the field, prof. Krys Bankiewizc, a Polish-American neurosurgeon, was awarded with the inaugural Roy Bakay memorial award for his contributions. Prof. Bankiewizc’s work has certainly paved the way also benefitting Herantis. Prof. Bankiewizc presented recent results from a phase 1 clinical study with an intracerebral gene therapy for children suffering from a rare congenital neurometabolic disorder causing severe dystonia (AADC deficiency). The results were simply amazing. Nearly completely wheelchair-immobilized children had come back to life in all meanings of the word, learning to walk and talk. This is science at its best, developing breakthrough treatments for devastating conditions and truly transforming the lives of patients’ and their families.

Other exciting developments were also reported. Stroke is one of the leading causes of death and disability in Western countries, and in the past several decades very few novel treatments have been successful in clinical trials. When stroke hits the brain, every minute millions of neurons and billions of synapses are lost until reperfusion occurs. This massive loss of critically important brain cells is very hard to overcome, which translates into poor prognosis for patients. Stem cells may have been overly hyped about; nevertheless in the field of post-stroke rehabilitation, some very exciting progress has been made. Several companies reported on-going and new clinical trials. However, the mechanisms underlying the beneficial effects of stem cell transplants may relate more to balancing the body’s own repair mechanisms and inflammatory processes, rather than rebuilding new tissue mass. There was also strong criticism raised towards taking stem cell-based therapies to clinical trials too hastily. And rightly so. Patient safety has to come first, even in exciting new fields of science.

The INTR meeting has been organized in the same place annually for the past 25 years. The meeting with less than 200 participants is tiny compared to many other international conferences. This is why it felt more like a family gathering rather than a conference. This highlights one important feature of science. Although there is a constant competition on publications, grants, patents and so on, in the end we are all in the same boat. Every scientist depends heavily on one’s network of collaborators. And when colleagues get together every year for decades, life-long relationships are built. This may well be a precious yet underappreciated force driving scientific research.

In family gatherings food is often prepared together. Before the last night’s beach party dinner, Dr. Airavaara and colleagues went ocean fishing bringing back nearly 8 kg of fresh mackerel and king fish. With the fresh ingredients, the hotel staff was kind and skilled enough to prepare a memorable seafood dinner for all participants. Perfect ending for the 25thINTR family get-together!