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The heading of Jeff Coller’s New York Times article (April 9) is rather bombastic: “This may be the most important medical story of the decade.”

What is this bombshell story?

Little K.J. Muldoon was born with a fatal genetic ailment.  He lacked a gene that generates a crucial compound, ‘carbomyl phosphate synthetase 1’, that enables the nitrogen from protein metabolism to be metabolized.  As a result, ammonia  NH3  accumulates – and ultimately kills.  This is a very rare genetic disease – one in 1.3 million births.  It is named after the missing key compound:  CPS1.

Was little K.J. out of luck?

Fortunately, not.  A brilliant team at Children’s Hospital, in Philadelphia, Pennsylvania, along with Penn Medicine, put together a daring plan.

Step one: Use CRISPR (acronym for clustered regularly interspaced short palindromic repeat), a widely used method for snipping ailing genes and inserting healthy ones. (It was invented by two brilliant female scientists, Emmanuelle Charpentier and Jennifer A. Doudna; they won the Nobel Prize for Chemistry.)

Insert the healthy gene into a molecule.

OK, now what?  The body rejects foreign substances.  You can’t just inject the healthy genetic material.

Step Two:  use messenger RNA (mRNA) technology, used to create the amazing COVID vaccine.  Package the molecule with the healthy gene with a lipid (fat) covering, and use the mRNA to slip it into the liver, where metabolism of protein occurs, without the immune system rejecting it.

Fast forward:  Two-year-old KJ Muldoon won the lottery. He is growing and thriving.  Thanks to this amazing team of medical pioneers.

Now, the downside.   Gene therapy is really really expensive:  Running anywhere from $850,000 per treatment to over $4 million.

Just to keep things in proportion:  One Tomahawk cruise missile costs $2 m. to $3.5 m. Over 850 of them were fired during the six weeks of Epic Fury.   That’s 850 gene therapies.  850 young lives.

I live in Israel.  We face Iran’s nuclear threat.  For us it is very real and imminent.  This does not keep me from perceiving the total insanity of firing Tomahawks rather than giving life to kids.  A wealthy country like the US can, if it so chooses, do both.

But apart from KJ and a handful of others who win the lottery, it is either/or.  This is why, in the grand scheme of things,  Coller’s greatest medical story may actually be a tragedy, rather than a triumph.

© The Times of Israel (Blogs)