KICK-START YOUR T-cells, and hope for the best

These bears had the misfortune to cross the path of Paul Kelly.
Never mess with a kid from Wisconsin

1987

Dick Ingwall again has furnished me with an excuse to write a blog-bit.  Again, the article is in the NY Times, and its web address is http://nyti.ms/VxuHXB.  Again, since I know you won’t read it even if I ask you to, I will summarize.

It appears that a 7-year-old girl in Pennsylvania who was near death a year ago, from something nasty called acute lymphoblastic leukemia, is now healthy, happy and bouncing around (see video recommended in the appended "comment").  She owes this truly remarkable result to a new treatment developed at the University of Pennsylvania.  The treatment is still regarded as experimental but has been used on several leukemia victims who had nothing left to lose.  The results have been mixed: several adults and our little girl have obtained what seems to be complete remission, several others have had partial success, and several more got no benefit whatsoever.  Naturally, they (the U. Penn people) are trying to figure out why. 

The treatment consists of extracting a few million T-cells from the patient, then using a dead HIV virus as a “vector” to insert a new gene into them, whereupon they are re-injected back into the patient.  The idea is  to alert the T-cells (part of the immune system) to the presence of an enemy, whereupon they go to work.  (Sometimes they are referred to as “Killer T-cells”.) 

The results can be horrendous.  In the case of our little girl, death very nearly ensued.  She ran a high fever, became swollen “beyond recognition”, was on a ventilator, and unconscious for a week.
Through the good offices of a drug called (well, it’s unnamed) she recovered.  May she never relapse.

This treatment so interested the Swiss drug company Novartis that they are giving $ 20 million to found a new research facility at U. Penn.  The economics of this is interesting.  This T-cell modification technique calls for personalized treatment: no drug can be put on the market to be taken by millions.  The cost of the treatment is said to be about $20,000.  (This, however, contrasts favorably with the cost of an alternative treatment – bone marrow transplant – which costs significantly more and, I seem to gather, is less effective.  It is not clear to me how Novartis (full disclosure - I own a few shares of their stock) hopes to profit from this research.  Being Swiss, I'm sure they have figured it out.

There is some hope that similar methods can be used to attack solid tumors.  Maybe even ovarian cancer, I hope. 

I stumbled on the NY Times “comments” appended to this article.  They are interesting.  Naturally, most consisted of something on the order of “Thank God”, or “I am so happy for the family.”  A minority, however, took umbrage at the notion that a drug company would seek to profit from a life-saving treatment, whereas an equal number replied “How else would they have enough money to develop new drugs and endow research facilities, you blockhead?”  No doubt the first group would suggest that the government do the work, design the drugs, and distribute them at cost.  There are examples of this (well, minus the distributing part) that work:  The NCI, for instance.  However, having once worked for a Federal scientific agency, I really don’t think this is the way to go.