It All Started With An Email...

Have you ever heard of PEG-PAL?  Probably not.  I vaguely knew about it myself... until today.

You see, occasionally I get emails from this PKU Group in my state.  Today I received one of theses emails from them and discovered some very interesting news about PEG-PAL.  I didn't really know anything about it so I did some research.   

PEG-PAL is 2 different things really.

There's PAL which is Phenylalanine ammonia lyase (PAL).  It's a protein (enzyme) found in plants, it converts L-phenylalanine to trans-cinnamic acid and ammonia.  

Then there's PEG (aka polyethylene glycol) which is a water soluble compound that is non-toxic and can be safely cleared from the body. 

They use the PEG to help get the PAL into the body, because PAL is this unknown substance that your immune system wants to get rid of.  The PEG hides the PAL from your immune system.

The PAL is a stable enzyme.  They put the PAL into the body and it metabolizes Phe.  The PAL basically takes over for the PAH (which is the enzyme PKU'ers have troubles with and converts the extra Phenylalanine into trans-cinnamic acid and ammonia.  The body then can easily get rid of the trans-cinnamic acid and there isn't enough ammonia to do much damage.    

How do you get the PEG-PAL into your body?  Injections.  Yuck.  But how often you would take need to take it depends on the person.  From everyday to once a week.

So now you know what I know about PEG-PAL, here's the news: PEG-PAL is going into Phase 3 testing in 2013.  Phase 3 testing is basically the big-group testing.  It's already been proven to work on a wide range of individuals (including lots of Classical PKU'ers).  Some people have been taking it for 2 years now as part of the long term effects testing.

Now here are the reported side effects (I'm going to list them clearly with normal print unlike medication commercials): rashes, and sore joints.

Seriously.  That's it.  Everyone had sore joints for the first few weeks of taking the drug.  But as the body became used to it, the sore joints wasn't there anymore.  People who got rashes reacted to the PEG.  And apparently if your taking any medicine with PEG in it already, you can't use PEG-PAL.

The benefits?  You basically can go off diet.  You have enzymes breaking down the excess Phe.  There is no build up, so a controlled diet is no longer needed.
    
I've tried to be non biased in my research so far... but now it's time for me to share my opinion.  We're getting closer.  The PEG-PAL is not a perfect solution.  Who wants to have injections everyday?  But it's better than KUVAN by a long shot.  I like the theory behind it.  I really do.  It makes sense to me.  There's still some things that need to be fixed during this next phase, but modern medical science has been making leaps forward to a solution.  I might even try it... Not now.  I'm a poor college student with no car, and lives far from the clinic.  But if I can get over the injections idea... I might think about it.  :)

And to think that this super long post started with an email I got today... :)