From Basic Research on Polymeric Protein Models to the Most Used Drug Against Multiple Sclerosis

From Basic Research on Polymeric Protein Models to the Most Used Drug Against Multiple Sclerosis

Michael Sela
Weizmann Institute of Science, Rehovot, Israel

 

I would like first of all to make a couple of comments on the main topic of our deliberation, namely on “emerging basic science toward solutions for people’s wellbeing”. On the one hand, the drug we invented against multiple sclerosis (copaxone) improved the health of many hundreds of thousands of patients and this helped their wellbeing and alleviated the worry of their families.

At the same time the drug, copaxone, became the main export of Israel and definitely improved the economy of the country. Thus, what started as totally basic research ended in significant improvement of health and rise in the level of economy.

As shown previously, in continuation of our work on polymers of amino acids as models of proteins we were able to show immunological cross reactivity with proteins such as collagen or enzymes like lysozyme. We prepared a positively charged copolymer resembling to some extent the myelin basic protein in the brain. This copolymer, which we used to call copolymer 1 or Cop 1, and industry later named Copaxone or glatiramer acetate (GA), is now the most frequently used drug against the exacerbating-remitting stage of multiple sclerosis. Close to 400,000 patients in 50 countries are being treated by the drug, which is specific and does not act as a general immunosuppressive modality. Recent work on an animal model of multiple sclerosis (experimental autoimmune encephalomyelitis), suggests that copaxone not only prevents demyelination but may even promote re-myelination in the brain.

In this study the purpose was the curiosity driven basic research. If the amino acid copolymers were meant as models of proteins, then we expect them to resemble proteins also immunologically. A copolymer meant to resemble this protein appeared to repair the damage done to mice by the original protein. Thus started the translational research that led to the drug against multiple sclerosis.

For most individuals, after many years of research, publication in an impressive journal seems the end of the road, but this is not true, certainly not in translational research, because the next step – the development of the fruits of research – is most important. The translation of research into products is important both for the individual and for humanity as a whole.

Nevertheless, in summary, while both achievements – health and economy – are significant, undoubtedly the greatest satisfaction for a scientist is the unexpected result in basic research.

 

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