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The Nobel Prize in Chemistry 2020


 The Nobel Prize in Chemistry acknowledged a remarkable contribution in to health and medicine: Emmanuelle Charpentier and Jennifer A. Doudna received it for the development of a method for genome editing known as CRISPR. 

CRISPR has revolutionized all the life sciences, from plant breeding to genetic therapy.

Charpentier is a French microbiologist, and Doudna is an American biochemist affiliated with UC Berkeley. In 2012, they were studying the immune system of a Streptococcus bacterium. They discovered that they could use "genetic scissors" already present in the metabolism of the bacterium so that they could make precise cuts in the DNA sequences.

Just as one illustration of the ramifications, consider that early this year researchers tested a cancer treatment in which the body's immune cells are CRISPR-edited to get them to hunt down and attack the body's cancer cells. The results were not such as to declare a breakthrough cure, but they have encouraged continued work along those lines.

There is also the matter of ALS. Early this month, pharmaceutical giant Biogen Inc. and start-up Scribe Therapeutics announced a joint project to develop therapies for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Scribe was co-founded by the above mentioned Jennifer Doudna, and CRISPR will be at the heart of this project.

ALS is a progressive condition that attacks the nervous system. It is famously associated with Gehrig, the Yankees great who was part of the "murderers row line-up" that terrified opposing pitchers through the 1920s and '30s. It is associated, also, with Stephen Hawking, the English theoretical physicist who suffered from a slowly developing form of the disease.

This is not Biogen's first press on the ALS front. Years ago it invested a lot of money in dexpramipexole, which seemed to hold promise against ALS. In 2013, though, dexpramipexole failed in a critical trial and Biogen had to stop development. Its return to the front in partnership with Scribe may prove an old truth: nothing is settled until it is settled right!

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