The Human Genome (Part 3)

Anyone with children probably knows that when Harry Potter broke his arm playing Quidditch one of his professors botched the spell to fix it and instead removed the entire bone.  In order to grow a new bone, Harry had to drink a bottle of “Skele-Gro” and about 12 painful hours later he was ready to get back on his broom.  Of all the fanciful creations in the magical world of Harry Potter, this one might one day be our reality.  Research into stem cells and their promising regenerative properties may one day bring this idea from science fiction to science fact.

We wrap up our discussion of the 10^th anniversary of the Human Genome Project with a look at stem cells and their potential clinical applications.  Although not directly related to deciphering the genetic alphabet contained in a human cell, the area of stem cell research has benefited from the genomic revolution.  While genomics gets the most attention, the stem cell revolution is an important component of what many have called Biology 2.0.  Moreover, interest in the therapeutic potential of stem cells was greatly stirred about a decade ago when in 1998 researchers at the University of Wisconsin-Madison derived the first human embryonic stem cell line. 

This ignited strong public interest, but many of the great hopes of stem cells were dashed a few years later when President George W. Bush poured a bucket of cold water on stem cell research by severely limiting federal funding in the area.  In the end, however, this move was a boon to the field because it forced researchers to become more innovative, eventually figuring out how to transform any cell from the body back into a stem cell.  The idea that cells can turn into any other type of cell in the body is very exciting and research is being conducted on the application of stem cells for treating everything from damaged body parts – wound healing and heart, brain or spinal cord damage – to the correction of congenital problems such as blindness, deafness and baldness, to the treatment of diseases such as diabetes and even... Want to read more?  Log in to our subscriber area or subscribe to the Medical Technology Stock Letter.

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Whereas gene therapy was once the province of scary stories – about parents engineering their perfect children or laboratory created abominations – the technology has now progressed to the point that meaningful clinical trials are being attempted.  When talking about gene therapy in medicinal terms, we are usually talking about somatic gene therapy, meaning therapy for the living bodily, or somatic cells.  This is opposed to germ line therapy, which involves changing the DNA of sperm or egg cells to (possibly) alter the traits of offspring.  Unlike somatic therapy, changes in the germ line DNA will be passed from generation to generation.  Explaining the difference between germ-line and somatic gene therapy will be important for the industry as it will allay the worst fears of uncontrolled genetic changes replicating throughout the environment...

The Gene Desert
As if deciphering the gene-coding region of the human genome wasn’t enough work, genomic studies are finally giving more insight into the vast majority of DNA that doesn’t contain genes.  This area – junk DNA or the gene desert – no doubt contains more useful information than is yet realized.  A large portion, it turns out, transcribes many different types of RNA, which – we are discovering – play a much larger role in the cellular machinery than was imagined.  Other portions contain information telling genes when to turn off or on, or regulate cells under sub-optimal conditions.  Although not all biologists have come around to this view, we firmly believe that there is far too much evolutionary pressure on DNA for any more than a tiny fraction to actually be ‘junk’.

 
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Ten Years After is one of the greatest blues bands of all time and featured the maniacal Alvin Lee whose frantic guitar solo on “I’m Going Home” at Woodstock is now part of rock n’ roll legend.  Unfortunately for the human genome project, ten years after its completion, its legacy is still in question.  Most of the recent discussion about the 10 year anniversary of the human genome being sequenced focused on the dearth of any new drugs resulting from the completion of this important milestone.  However, we learned a lot from the Human Genome Project (HGP), and it accelerated the identification targets for potential drugs and was a worthwhile step forward. 

In their excitement, scientists promoted their visions of what genomics might possibly bring and the media exaggerated those claims to the point where the public expected personalized medicines at their next visit to the doctor.  Biologists imagined that the letters of the genetic code that were being written down would create a great encyclopedia of life, complete with footnotes.  What they got instead was more beautiful – a spectacular hieroglyph revealing life’s enormous complexity, yet no Rosetta stone to decipher it.  

In the past few weeks there has been a lot of looking back on the ten years since the HGP.  We therefore thought it was only fair to turn our inquisitive eye back on ourselves and see what we wrote back then.  In Issue No. 392 from March 30, 2000, we said:

 ‘In short, genomics promises to increase the number of targets for drug development ten- to a hundred-fold. It is a major step forward and will provide the foundation for most drug development in the future. The bucket of cold water to throw on overly enthusiastic investors is that finding out what each gene does and validating it as a drug target requires years of work. That’s why we have been cautious about genomics picks in the past.’

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The FDA's Endocrinologic and Metabolic Drugs Advisory Committee on Thursday voted 10-6 to recommend against approval of Vivus Inc. (VVUS) obesity drug Qnexa.  The San Diego Union-Tribune talked to MTSL Editor John McCamant, who correctly predicted that the panel would reject the drug because of safety concerns, for their story:

 http://www.signonsandiego.com/news/2010/jul/15/fda-panel-turns-down-vivus-diet-drug/