HD Blog

And, though mithramycin is not an easy drug to work with, it is already FDA approved. And accordingly the authors make the argument that mithramycin should be among the top priorities for testing in patients.

First: A Heartfelt Thank You

These researchers (and all those laid the groundwork to make this possible) are to be saluted for the long process that went first from scientific creativity, grant writing, the later through the long hours of laboratory work (that put this drug through a multitude of cell studies and more than a hundred HD mice) then through careful interpretation of results then finally to the report of this promising drug.

And this HD activist couldn’t agree more that it certainly makes good sense to promote this “best so far” drug for trials in HD people.

Next: A Heartfelt Ache

But, I’d go further: “Why hasn’t “best so far” gone farther than a thinking stage? The neuroprotective benefit of mithramycin (in combination with another drug gives even better survival numbers) in the R6/2 was known by these (and other HD research leaders) in early 2002. This data was not made public, but instead (presumably) the drug was taken back to the lab where attempts were made to elucidate molecular mechanism. The present article describes the many experiments done to show that it works (somewhere) far downstream in a broad neuroprotective way that remains to be defined.

From the research perspective, this is very good science. From the patient perspective it is downright exciting that researchers have found a drug that have virtually prevented nerve damage in the R6/2 mouse model. Of course a drug this good in the mouse model should be brought to clinical trial. The “heartache” part is that it was not brought to clinical trial closer to 2002. Is there an explanation that I have missed for why this drug is a better clinical trial candidate for HD people in 2005 than it was in 2002?

HD Needs “Sydney Farber” Docs

The Huntington’s community would benefit from docs like Sydney Farber who would do for HD what he did for childhood leukemia a generation ago. Sydney Farber wasn’t very popular with the traditional leukemia researchers of his day when he “bucked the system” and started small clinical trials in children with leukemia. But those same trials moved first successful treatments for childhood lymphocytic leukemia that began the success story of full treatment for this generation.

I bet an HD “Sydney Farber” type of doc would have taken a drug that looked as promising as mithromycin to a pilot clinical trial. But even if we had “Sydney Farber” docs (and I know we do), they don’t have a chance if information is withheld. Smart Docs can’t pick up the research ball and “run with it” if the researchers are doing “private “thinking” and don’t make promising results available to smart docs taking care of HD patients. And if the “Sydney Farber” docs don’t have a chance of picking up the treatment ball, then HD people have lost the game before the buzzer has sounded.

Maybe HD research is too good; maybe the researcher too powerful. It is the researcher, not the clinical docs, making (or not making) decisions on clinical trials for people. So it’s been back to the lab screening for the more perfect drug or homing in on molecular mechanism, etc. Good science for sure. But it is heartache for HD people that our disease has more drugs and fewer clinical trials than any other disease in the history of disease.

Thrilling research; suffering HD people. How good does a drug have to be before the powers (who are these people) that control HSG acts urgently? Though HD research may see HD “time” from the perspective of “large therapeutic window” HD people have a different perspective: The first year’s loss is devastating, the second year’s loss is devastating, and so on for the long, long duration of this illness.

Desperately Seeking . .

If a “Sydney Farber” doc would have had the chance to start closer to 2002, we wouldn’t still be at the stage of “thinking about thinking about” testing mithramycin in people: We would probably have had an answer. And maybe even a first treatment. More heartache: the same thing could be said for several other agents out there.