Friday, February 27, 2015

WOW!



Roy, Richard and Linda at the beach, 1947
I wish I had known Roy
Wow!  That’s all I can say: Wow!  The research team of J & R Ingwall has sent me a link to an article in the NY Times on a development in cancer therapy that gives promise of being a genuine breakthrough.  I urge you all to read it; it will certainly be on the next test.
Traditionally, cancer therapy has been specific to the affected organ: if you have ovarian cancer, for instance, you get one kind of drug, if you have prostate cancer you get another, and so forth.  Lately it has become recognized that organ-specific treatment may not be maximally effective, because – with many cancers – a single organ may have multiple “varieties” of tumor.  With ovarian there are four, I believe, which vary in their response to the traditional therapeutic protocol. 
However, we have known for a long time that cancer ultimately is the result of one or more mutations.  The new research described here looks at the mutations, not to where the tumor happens to reside.  For instance, a drug already in use for melanoma absolutely cured an elderly woman of Langerhans disease, which is a rare cancer of the white blood cells.  It appears that Langerhans disease (in this case, at least) is caused by the same mutation that in other people results in melanoma.  It was possible to determine that this is the case because sequencing of genes known to be involved in cancer has dropped in price from the high thousands to the low hundreds.  (Of dollars, obviously, although the same would be true nowadays if we were speaking in euros, thanks to the strong dollar.  Time to take that trip to Europe.)
The FDA seems gratifyingly cooperative for once.  Instead of insisting on a traditional trial, involving many hundreds of subjects, double-blindness with a control group, heaps of money and years of time,  they will simply look at the data (from smaller, less controlled studies) and ask the question:  “Is the American population better off with this drug than without it?”  Eminently sensible, for once.
So, the ongoing research goes something like this:  Oncologists from around the country will send tumor samples to various central labs, where the mutated gene(s) are determined.  The patient then is assigned to a “basket”, which “contains” all those with a tumor caused by that particular mutation, regardless of where the tumor may reside.  All patients in this basket will receive the same treatment set, and the results are scrutinized.  Marked improvement without severe complications will be taken as affirmation that the drug/treatment is efficacious.  The program under which this is being done is called “Match”, and is funded by NCI.  To me, this makes good sense.   I take back all the bad things I have written in the past about both FDA and NCI.  Well, some of them. 
As an example of what is possible, consider the “basket” that contained Mrs. Hurwitz, the lady with Langerhans disease.  Of the 70 individuals in her basket, 18 had one of two rare blood cancers.   Fourteen of these are cured, and the remaining four have only just started the treatment.
Of course, failures also have occurred.  One of them is described in the article:
 
 
 
 
 


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