What the heck is PEG-PAL??

Hey all! I’ve been seeing lots of posts on my PKU groups on Facebook asking about PEG-PAL, so I decided to write a post about my experiences with this PKU wonder-drug. If ever there was a time to have PKU….IT’S NOW. This drug is a REVOLUTION! Here we go:

The Drug

PEG-PAL is, in a nut-shell, an injectable enzyme that replaces the one that’s deficient in people with PKU. Though it’s not the exact same enzyme we’re missing, it’s similar. PKU people are lacking phenylalanine hydroxylase, an enzyme that converts phenylalanine to tyrosine. Without this enzyme, phenylalanine we acquire from our diet is not broken down by the body, and builds up in our bloodstream and brain, causing brain damage. Also, since phe isn’t converted to tyrosine by PKU people, tyrosine becomes an “essential” amino acid in us, meaning tyrosine is something we have to acquire via our diet since it is not made by our bodies. PEG-PAL on the other hand is a phenylalanine ammonia lyase. Fancy words saying it breaks down phenylalanine, but to different harmless components (not tyrosine). The PEG in PEG-PAL stands for pegylated. Pegylating something means coating it in a substance called Poly Ethylene Glycol (PEG), which basically masks the substance from the host’s immune system, preventing an immune/allergic response, and helping the substance last longer. For those of you who are familiar with the Depo-Provera birth control shot you can get every three months, it is also pegylated.

Background

I’m 25 and currently live in Omaha, Nebraska where the only PKU (metabolic) clinic in the state is located. Even more convenient, I’m a third year medical student at the University of Nebraska Medical Center. I’ll be a doctor in a year, yay! So it’s fair to say I’m at the hospital fairly frequently. I tried Kuvan, another FDA-approved Biomarin drug for PKU, and unfortunately didn’t respond. Since then, I’ve been bugging my doc about this PEG-PAL thing and involving Nebraska in one of Biomarin’s (the PEG-PAL creators) clinical trials. Hallelujah they listened! Last winter I was notified UNMC’s proposal to become a study site for the PEG-PAL trials. Side note… props to my physician for making this happen. There’s an unbelievably complex time-consuming, tree-killing, paper-dense process sites must go through to become “approved” for such a study. The man-power and time it takes to make this happen is crazy…but that’s a whole separate story.

Initial Trial

I was recruited to be one of two initial study participants at the Nebraska site. The first “visit” consisted of signing about 20 pages of consent forms basically stating what the trial was about, the risks involved, and if I consented to things like blood sample donation and skin biopsies if I had a reaction to the drug.

Next, I went through a battery of tests. Probably 10 tubes of blood were drawn initially to check base phe levels, kidney function, electrolytes, etc. If you’re not a fan of blood draws, like I was, you’ll quickly get used to them while in this trial. I also had a physical exam done, chest X-ray, and EKG to look at my heart function. All were normal, so I started the trial.

The first few months consisted of me coming in to clinic and receiving a low-dose injection of PEGPAL given to me in the fat layer of my arm by a nurse. Then I sat….for 30 boring minutes…to monitor for any reactions I might have to the drug. They slowly increased the dose of PEG-PAL I was receiving over the weeks, and the frequency I’d get the injections. I finally found a dose that kept my phe levels in a “goal-range” (less than 6 I think).  By that time, I was trained to give myself the injections and was allowed to take the drug home, where I’d give myself the shot once a day, five days per week. I also kept strict diet journals to turn in before my weekly blood draws.

Since then, I’ve been through a few more subcategories of the trial where my injection schedules or doses have changed, each time having to go through the million blood tubes (or so it seems), physical, X-ray, and EKG. I’ve slowly liberalized my diet over the last year and now it’s largely a “normal person diet!” I’m including starches, dairy (cheese, milk, yogurt), and MEAT (chicken, beef, seafood, etc) and my phe levels continue to be UNDETECTABLE! I feel the best I’ve felt since my mom was controlling everything that went in my mouth at age 2.

Injections!

Here’s some pics of tonight’s injection so you can see how the process works. Although a daily shot with – shudder – needle may sound a bit scary, it’s a small price to pay for perfect phe levels! The shot itself barely hurts. It goes into the subcutaneous (fat) layer just below your skin, and uses a small, short 25 gauge needle. Easy as pie after a couple practice sessions!

Here's PEGPAL in it's individual vials. The small black-and-white screen is called a TagAlert and indicates if the environment of the drug (AKA my fridge) is the appropriate temperature. I have to check it before drawing up the drug.

Here’s PEGPAL in it’s individual vials. The small black-and-white screen is called a TagAlert and indicates if the environment of the drug (AKA my fridge) is the appropriate temperature. I have to check it before drawing up the drug.

My PEGPAL log book where I keep track of where and when I give my injections, my tyrosine supplements, and any reactions I've had.

My PEGPAL log book where I keep track of where and when I give my injections, my tyrosine supplements, and any reactions I’ve had.

In my current sub-study, I'm supposed to take three 500 mg capsules of tyrosine per day. I keep track of my tyrosine in the booklet.

In my current sub-study, I’m supposed to take three 500 mg capsules of tyrosine per day. I keep track of my tyrosine in the booklet.

The supplies I use every night I do an injection. 2 alcohol swabs (for cleaning off vials and my skin), one cotton ball (for any small amount blood or drug that leaks out after injection), a 3 mL syringe with big needle (to draw up drug), a small needle (to inject drug), and 2 vials of PEGPAL.

The supplies I use every night I do an injection. 2 alcohol swabs (for cleaning off vials and my skin), one cotton ball (for any small amount blood or drug that leaks out after injection), a 3 mL syringe with big needle (to draw up drug), a small needle (to inject drug), and 2 vials of PEGPAL.

I first draw up the drug from the vials into the syringe. My current dose is 2.67 mLs.

I first draw up the drug from the vials into the syringe. My current dose is 2.67 mLs.

Once drug is drawn up, I switch the syringe to the SMALL needle, then clean off my injection site with an alcohol swab. You can inject PEGPAL into your abdomen, thighs, or upper arms...pretty much anywhere with a fatty layer.  I have no problem finding one of these areas.   :)

Once drug is drawn up, I switch the syringe to the SMALL needle, then clean off my injection site with an alcohol swab. You can inject PEGPAL into your abdomen, thighs, or upper arms…pretty much anywhere with a fatty layer. I have no problem finding one of these areas. 🙂

Poke and sometimes a burn! It's kind of hit and miss regarding if the injection hurts or not. Some spots of my abdomen are more sensitive than others to the poke of the needle. Usually it's painless!

Poke and sometimes a burn! It’s kind of hit and miss regarding if the injection hurts or not. Some spots of my abdomen are more sensitive than others to the poke of the needle. Usually it’s painless!

Voila! I massage the injection site for a second to help the drug absorb, cover it with a cotton ball for a minute or two, and am done till tomorrow!

Voila! I massage the injection site for a second to help the drug absorb, cover it with a cotton ball for a minute or two, and am done till tomorrow!

 

Side Effects

I’ve been very fortunate when it comes to side effects for PEG-PAL. I initially  had none, then began getting occasional hives on both of my upper arms. The were itchy and warm to the touch, but subsided after an hour. I now take a Claritin or Allegra (non-drowsy antihistamine) everyday to prevent the hives and have no problems with them! This is the most common side effect and usually subsides after a few months on its own or with an anti-histamine.  Other things I noticed: the lymph nodes in my groin area seemed a bit swollen after I started the injections. Makes sense. You’re injecting a protein with a protective coating your body isn’t used to seeing, so my lymph nodes (part of immune system) got a bit bigger after introducing PEGPAL. I also get headaches occasionally, but so does everyone else right? Who knows if this is related to PEG-PAL or if it’s just me.

The other more serious side effects I’ve heard of other trial members having are worse, more extensive rashes than mine, rigors (or shakes), and one person with syncope (fainting spells). These are RARE and you’re monitored closely throughout the trial with blood tests and checkups to make sure your body is tolerating the drug well.  You’re also given an Epi-Pen should you have a serious allergic reaction call anaphylaxis, which can affect your airway. I haven’t heard of any PEGPAL users having an anaphylactic reaction.

From what I’ve read, it sounds like you can predict how you’ll react to PEG-PAL by looking at how your immune system acts normally. People who have an overactive immune system, or who are “atopic,” are more likely to have more reactions than those with less active immune systems. An atopic person is usually someone with severe allergies (seasonal, food, etc.), eczema, and/or asthma. I myself am fairly atopic as I have some pretty significant seasonal allergies. Despite my happy-go-lucky immune system, my side effects are virtually non-existent with my daily anti-histamine.

The End

I guess my goal for this post was just informing anyone who wants to know more about PEG-PAL what it is and how it works. Hope this was helpful! Please feel free to contact me via comments on this post or on Facebook with any comments or questions you have! I love to hear from people, especially about anything PKU!  🙂

-Jenn

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136 thoughts on “What the heck is PEG-PAL??

  1. Andee w says:

    Hi there, I randomly came across your wordpress when I was looking to send some info to a friend to explain what I am going through. I’m in wisconsin and I was one of the first people here to go on this medication. I also was one of the people who had a super tough time but stubbornly I made it through. Infact I was told a lot of adjustments on how and when to dose have been made based all the stuff I went through. I looked like I had the measles and had to take steroids , the first time it was for a month, the second time it was for quite awhile and I had to take steroids for nearly 6 months. And even after that I had some flair ups. I stuck with it with the wonderful team placement of brilliant and caring doctors dieticians and coordinators that I am lucky to have.

    Interestingly I also have these giant painful at times “lumps” you stated you get them in your groin area I get them under my arms, and initially when they start I can’t even put my arms down or lay on my side, and it seems that when one goes away another boils up.

    Everything has been pretty steady for awhile, as I am also on tyrosine which is much easier to swallow than the lovely shake mix. I have noticed, as I’ve been religious about the tyrosine and even sometimes taking the shake, that my hair has even started to come back. After my third child I lost 2/3 of my hair and kept it off for atleast 5 years now.. And now it is actually starting to come back- I am beyond excited about this! Being able to eat food and not cheat has been mind boggling to me after almost 30 years of not being able to. At first I had to drink a proteins shake just to get to the recommended protien level. It’s nice being able to read another persons story. Even though we have went through a lot I’m glad to be able to pave the way for all these other children and adults, even if it started with what not to do.

    Thanks! Andee

    • Hi Andee! Thank you so much for your post. I’m learning there’s been more adverse reactions to PEG-PAL than I originally thought, and you definitely sound like one of those people. Props to you for sticking with it throughout and I’m glad things have gotten better for you!

  2. rhodri says:

    Jenn
    I have a 6 year old daughter who has PKU (also a niece and a nephew with it). Sadly the UK is so much slower in its grasp of medical advances as Kuvan is still not available over here. However some good news today, it looks like they are going to allow it for children between 6 and 16. A huge step forward, but more importantly in my mind is that it is clearing the path for PegPal in the near future.

    You also brought a smile to my face when I read that you were studing medicine (good on you). When we were first given the diagnosis all we could see on the internet were words such as “reduced IQ”, “brain damage etc.

    My daughters grandad and great grandad were doctors so who knows (assuming she doesnt follow me for brains!!).

    Good luck with everything. please keep us posted on how it is all going and thank you for taking the time to put this site together.
    Regards
    Rhodri

    • Rhodri, Thank you so much for your comment! Sure hoping your sweet daughter gets to try out the Kuvan soon! I’m happy UK is making some progress!! Kuvan can sure be a wonderful drug!! It would be great to have another PKU doc around. It sounds like she’s got some good genes for it! 🙂 Best of luck to you and your family. Let me know how she responds if the Kuvan thing works out!!

  3. Hello Jenn, i’m the mother of a boy old of 11 years. We are french, we leave in PARIS.
    He is a classic PKU. He has been tested for the BH4 but it has not been successfull., Last monday, I learnt at the Necker hospital (the referent PKU center in Paris), that US is progressing very well wtih a new treatment . And indeed, I can say yes , when i read your blog . It’s very enthusiast for the futur. So, i’m very curious, to know more about this PEG-PAL. Do you hear anyhting about a final medicine for all the PKU people ? Do you know if it will be BIOMARIN who will trade the product ?
    Do we have an idea of the timeframe to be at the final stage for trading ? Thanks a lot for your response PS : You can imagine how our hope it is 🙂

    • Hi Vanessa! Thank you so much for your comment. It’s exciting to have the privilege to speak with so many PKU families world-wide. My knowledge about the future of PEG-PAL is very limited since I am a study participant. A lot of the drug development is kept confidential within the company. It sounds like I’ve been pretty lucky in my response to PEG-PAL. As you can see from Andee’s comment a few posts above yours, some participants have had some significant reactions to the drug. I’d expect at least 3 more years before PEG-PAL is approved here. And yes, I think it would be BioMarin who would trade PEG-PAL. Sorry this isn’t more helpful! There may be a bit more information available at BioMarin’s website as well. Wishing you and your son the best in Paris!!

  4. Steve says:

    I’m in Indiana. I have been on PegPal for a little over a year now. My last appointment my Phe level was around 16. I had been doing most of my injections in my belly. My doctor had me switch from my belly to other areas like my thighs, buttocks, and arms. I took a blood dot the next week and my level was down to 6ish and then another a week later because they were surprised and my level was down to 3ish. I don’t know if it has been the switchng the injection site or the fact that I have been doing the injections for a year. My doctors have said that some people have been taking a year to respond. Another interesting fact is that I have never noticed when my levels have been high nor did my parents notice when I was younger. For the past couple of years my levels have been in the upper teens to low 20’s and I have never had any ill effects. I have also not noticed any change in how I feel or act since my levels have dropped so much. I am happy to have much healthier levels now though.

    • Hey Steve! Thanks for your comment! Glad to hear you’re responding to PEG-PAL! I wonder if some scarring from using the same injection site or not having enough fat tissue on your abdomen could hinder the absorption of the drug and that’s why your levels have gone down so much?

    • Hey Kellie! I don’t think there’s a definite timeline in place. When I’ve asked my study nurse, doc, and dietician, they suspect at least another 3 years before FDA approval in the United States. Sorry this isn’t a more specific answer for you!

    • I’m sorry to hear about your not-so-pleasant experience!! However, I’m thankful you wrote about it as it’s very important we are aware of all possible side effects, good and bad. Hope things are getting better!

  5. Rachel Schreiner says:

    Hi Jenn,

    First off, congratulations on your academic accomplishments. Could your career direction be in the field of genetics maybe?
    I am a 42 year old mother of 3 girls, twins who are 12 and a 10 year old. All miracle babies and my life’s biggest accomplishment. I worked in healthcare for 18 years as a patient care technician ,the physical demands of that job were catching up to me so I took a leap of faith into property management. At the same time I enrolled into the peg pal study. This has been the best thing I ever did. I too started out the same procedures as you but had several reactions.
    Itching yes but also extreme joint pain. I believe this is going to be the price I pay for low Phe levels that have not quite lowered enough.. I am currently on pre filled injections totally 40 mg. and will be a candidate for a increase to
    60 mg. I take the 3 tyrosine pills daily, a zyrtec in a.m and a Zantac at night for joint pain but has stopped working. I tried Predisone which helped temp.
    And now trying Tramadol. The joint pain is so severe at times but still worth it! Even though I still don’t have the lowest levels yet I feel the best I have ever felt mentally and emotionally.
    Peg pal has been allowing me to live a normal life without concentration , comprehension or emotional depression issues. I am sharper than I have ever been and not afraid to try and learn new things. The needle sticks are worth it and I would rather hurt physically then allow a condition a rob me of life. Thank you for your post and
    Look forward to getting in touch. Btw, I am a patient at Tampa General under the care of the best physician ever!! Dr Amarills Valle Sanchez.😊 I am smiling today cause of peg pal
    Rachel

    • Rachel, Thank you for your response! I’m happy peg pal is working for you and sorry to hear it’s been a struggle. Since writing this post I’ve heard a LOT of people have suffered from the joint pain you’re speaking of. Hopefully it improves! You sound like a very optimistic and hard working person and your kiddos are lucky to have you! Congrats on the PKU success and have a great holiday season!

  6. Gordon Lester says:

    Hello Kellie, My name is Gordon from Kentucky U.S.A. I have recently started Peg-Pal and was wondering when your side effects begin and when they began with others? I am entering week 4 and so far so good! Just a little breaking out around the injection sites. Best wishes to you!

  7. Esther says:

    Jenn,

    We live in California. My Daughter has been trying to get back on diet and the Kaiser Doctor in Los Angeles will not subscribe any Supplement until me daughter gets her levels consistently below 600 ml. (we don’t use the same scale of measure that we did when she was little). She brought her levers down to a little over 700ml and her tyrosine is just about out of range. (40-60 – hers is 41). It is impossible for her to maintain that level. Her dietician doesn’t know how much Tyrosine she should take (pretty bad) We got an invitation to become part of the study here through the California Coalition for PKU and Allied Disorders. I was so excited to read your post and my daughter is checking into getting into the program.

    A little history; after High School she went to Culinary School. (Told absolutely not by the Kaiser Dr. not her choice) Her levels dropped while in school to near normal. She got an AA degree in Culinary Arts. She wanted to be able to create medically necessary foods, so she took her credits and went into Food Science and Technology with a Certificate in Alternative Nutrition. She has finished her first quarter with a 3.90 grade average. She has documented learning disabilities and has a 504 on file with the State. We are extremely proud.

    This has been in my prayers for so long, you have no idea. If she get’s in, I’ll keep you posted.

    God’s Blessings and thank you to those that have gone before to make this possible.

    • Ester, Your daughter sounds incredible. I got to attend a PKU conference at a culinary school in Denver my senior year of high school. It was THE most incredible experience. The professors created specialized dishes and teaching demonstrations specifically for us and we even had an Iron Chef PKU competition. I swear if I didn’t go into medicine I would’ve done culinary school. We NEED more people like your daughter in the PKU community! I’ll cross my fingers she is accepted into the trial and PEG-PAL is a success for her. Keep us updated! Happy Holidays!

  8. Ann says:

    Jenn your explanation and details of how you are feeling is great. I have two boys waiting to get their chance to get the pegpal shot. I see that you said that your clinic feels that it will be about three more years. God I hope that it Is quicker than that…. Time is slipping away from my children’s teenage years. As a parent I can’t wait to see my kids eat normal, feel normal, and just be happier children. I wish that the FDA would allow younger children to participate. My oldest is seventeen. Our clinic in Pittsburgh told us that they will be looking for more people for the next leg of the study. However we have to wait until he turns eighteen. It is so hard to wait. He struggles in school, is tempormental, and easily upset. I worry that he is losing brain cells as we wait for FDA approval.
    Ann

    • I hear you Ann. I know it’s tough but there are reasons for the prolonged trials and age restrictions. Safety is first and foremost and I’d suspect Biomarin wants to ensure PEG-PAL is maximally helpful and minimally harmful. We are so much closer than we were 25 years ago! And just think how things will be once your children are adults. It’s a very exciting time and it’s only getting better!

  9. Sarah says:

    Hi Jenn. It’s been a while since I checked in to see how your trial is going? Hope all is well. Any updates on the trial?

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