MPSIIFund News

One of the beautiful pieces of MPS is that we regularly get to meet amazing people. MPS definitely draws a line. People who fear difference and atypical fall away and people who are open and inclusive draw closer. A day after the article in The Province about Trey getting IT doses locally (if you haven’t read it, the rest of this blog won’t make sense until you do, click here to read it), a woman left a message on our voicemail saying she was from The City of North Vancouver, she read the article in The Province, and would I call her back… I was perplexed. Why would a woman from The City of North Van be calling me about the article in the newspaper? I returned her call and left a message.
When we finally connected, she let me know that a man who works for the City, Brad McRae, read the article in The Province and has a red Jeep he wanted to donate to us for a day, to make Trey’s dream come true. I spent most of the rest of the phone call a blubbering mess.
When your kid is diagnosed with a progressive disease and you are given a definite end point for your child’s life… things change. One of the things it changes is dreams. They get pushed to the forefront. You no longer have time to waste or wait. Here we were, with a random stranger reaching out to us to make Trey’s life happier, to make his dream come true. Brad reached out and touched my heart.
Our red Jeep day was yesterday. Trey had an absolute blast. He was on cloud nine. Everyone we drove past (it’s a convertible) he hollered ‘I’m in a cool red Jeep!’ He waved at the people on the buses and he couldn’t wait to show our neighbors and family. Eventually we made it out to Steveston for fish and chips and ice cream.
I will forever be grateful for not only the red Jeep, for Trey could not have been happier for this dream to come true, but for Brad’s kindness and generosity. My heart has been forever changed as a result of his act of kindness. Thank you Brad, for making the world a brighter place.

It’s done. 30mg of Elaprase floating around in Trey’s brain in Vancouver, BC, Canada. There were a few hiccups, but it’s done. That’s all that matters. The doctors at BC Children’s worked hard and formed a team that I look forward to working with monthly for Trey’s intrathecal doses. They listened, respected our routines, adapted, and I really appreciate that. This is a long-term treatment and we need to make it work for Trey, so that this treatment stays ‘okay’ for him. The IT team at BCCH seems to get that.
Next month we’re back to UNC for the every 6 month tests, then back to BCCH for September’s dose, where we will try again to have Dr. Muenzer and Joey Howell’s BWBH documentary film crew come up and be a part of the second local dose.
Above are the friends we will miss by not traveling to UNC monthly: Case, who is in the trial with Trey, and his brothers, Tyson and Brock. Melissa, it’s about time you visited Vancouver and we visited Tennessee!

Trey’s first local intrathecal dose was re-scheduled. That makes it sound simple. It was supposed to be today. Dr. Muenzer was booked to fly in, Joey Howell’s Boys with Bigger Hearts documentary team was coming up to film… We went to the hospital Friday for over two hours of appointments. Before Friday I knew very little about how local dosing was going to go: where in the hospital it would be, what doctors would be involved. Fortunately these appointments answered my questions. The same doctor every month, and also a doctor experienced in lumbar punctures, would be doing the LP’s, instead of other possibilities I had heard. The anesthesiologist would be using fast acting anesthetics similar to UNC (Trey falls asleep and wakes up 15 minutes later like nothing happened) instead of long-lasting sedatives, which was another possibility I had heard about. I found out where the dose was going to happen and where Trey would spend his 4-hour observation period- that’s going to be interesting. I get to spend 4 hours with my 3 kids in a space big enough only to fit a small hospital bed (Avery and Sadie couldn’t even fit into the bed with Trey) with 2 chairs, surrounded by curtains. Literally no space to move and I’m not even sure where I’ll sit! After the first dose, I may be making requests. ☺ Anyways, I grilled the doctors involved (thank goodness I was born into a patient/family centred medical age!), received good news in response to my questions, and left. In any case, Dr. Muenzer would be there if anything arose, and I trust Dr. Muenzer with Trey’s life.
45-minutes after we left the hospital, at Whole Foods to grocery shop for dinner with Dr. Muenzer on Sunday night, I got a call from the hospital. The doctor doing to lumbar puncture had an emergency and could not do the dose Monday.
If Trey doesn’t get his dose this week, he will miss his dose this month. This is Trey’s life-saving drug. It is the only reason he can still ride his bike, speak in full sentences and play baseball. It is not a cardiology or ENT or any other appointment that can just be rescheduled. Rescheduling is not an option.
Why couldn’t someone else do the lumbar puncture (it’s a simple and regular procedure apparently)? If this rescheduling/emergency happened the very first time we were supposed to be dosed locally, who’s to say it won’t happen again? Should we be going to North Carolina until BC Children’s is reliable and set up? I called Dr. Muenzer. I called Dr. Stockler (Trey continued to shop while I did that and picked out all sorts of treats!). Everyone thought I should give BC Children’s another chance. On my child’s life. This time making a political choice, I went against what I know and trust and am giving BC Children’s a chance.
Trey’s dose has been re-scheduled for this Thursday. Dr. Muenzer cannot make it. The doctor doing the lumbar puncture will train two other doctors at that time to do a lumbar puncture aided by fluoroscopy. They will be his back-up in case of another emergency.
My long-time friend called me later just to catch up. She asked how I was doing! Letting go, I said. I am learning that letting go is the only way to move forward. Letting go of today. Letting go of the month (June/July) of Trey’s weekly infusions when his port and/or pump weren’t working and I poked Trey in the chest repeatedly to the point of tears, when I had to end an infusion early, staring at the liquid gold called Elaprase I had to throw in the garbage because a silly machine wouldn’t work, letting go of the vacation I had to cancel due to all these complications. Letting go of past arguments and judgemental comments that lead to resentment.
Holding on bogs us down and gives us baggage. Each IV infusion, each IT dose, each disagreement, each conversation, each day, each moment, is new. Light and fresh. I am learning to let go.
Above is a picture of Avery leading a meditation session. My kids are way ahead of me in this ‘letting go’ business. 😉 The photo cracks me up because I had no idea this was going on in my living room until I walked in and saw this. It’s also classic because Trey supports Dr. Muenzer’s observation that boys with a cognitively affected form of MPS II cannot close their eyes on demand, so here is Trey’s version of closing his eyes to meditate.

The Phase II/III is getting closer! For those families who have been waiting, it couldn’t come soon enough, I know. Here is an update of what I know:
Dose: 10mg every 4 weeks. Trey has been receiving 30mg, so this dose will be different from what Trey is currently receiving.
Criteria: An intracranial pressure of 30 or over will likely remain exclusionary. IQ must be greater than 55, possibly less than 85, the 55 is certain but the upper ceiling is not yet solidified.
Timeline: The Phase II/III trial will be 12 months long. This differs from the Phase I/II as it was 6 months long. This also means that the control (no drug) group will receive 12 months of no drug. I don’t know if there will be re-qualifications after the 12 month control is done, as was done in Phase I/II.
Device: The new device will likely be ready to implant in August on a boy in the Phase I/II trial.
Locations: Definitely Chapel Hill, Chicago, and Oakland. There may be other US locations. There will also be locations in the UK, Argentina, and Spain, possibly a few other Spanish speaking countries, to be determined.
Languages: English & Spanish.
Age: My understanding is that ages 3-18 will be included, with an addition. There will be a sub-study for boys less than 3 years of age whose mutation predicts them to have severe disease.
Natural History Study: The trial has begun a Natural History Study to follow boys with MPS II who may be eligible for the intrathecal trial. Boys who are not in the study will not be excluded from the trial, this study just allows the trial folk to follow the boys and pick them out if they might qualify.
Time to market: The trial is 12 months long, it is estimated it will take 12 months to fully enrol, that’s two years. Then with approval processes, it looks as though this drug is 3-4 years to market.
My word is not set in stone, I am not the boss, I am a mom who is sharing this info so that other moms and dads can do what they need to do in order to get their boys into this trial. If you want to chat, I am here. This is not an easy road and if I can walk side by side with you for a while to make it a little easier, don’t hesitate to ask. My heart is with all of you who are fighting to get this for your boys.

We have been traveling to North Carolina monthly for 22 doses. On Monday, July 22, Trey will receive his 23rd intrathecal dose as part of the MPS II clinical intrathecal trial under Dr. Joseph Muenzer at UNC, at BC Children’s Hospital. This is wonderful news. While everything pales in comparison to how AMAZING this drug is, the strain and stress this trial has put on our family is immeasurable. I do believe we are stronger for it, but we are relieved to no longer have to travel to UNC for monthly doses. We will still travel to UNC every six months for tests and placement of a port that is expected to be approved within the next month or so, but that is peanuts! We’ll also be glad to continue to visit a place that has become a second home and people who have become a second family (see above: Easton and Case are in the intrathecal trial with Trey).
Trey is the only Canadian enrolled in the Phase I/II trial and this will mark the first time this drug has crossed onto Canadian soil. Dr. Muenzer will be flying up for Trey’s first dose. Phase II/III is slated to begin in September of this year.

If only everyone could see Trey the way I see Trey… the kind, uber friendly, lover of sports and life, beautiful blue eyed kid that I love with my entire being. Most, however, don’t. They see hearing aids, scars, ports, just something… ‘different.’ If you’re a doctor you’ll see macrocephaly, hepatomegaly, splenomegaly, macroglossia, aortic regurgitation etc. etc. etc. This is what we heard when Trey was diagnosed (we also heard: ‘Has he always looked like this?’ Ummm… you mean my beautiful perfect almost 2 year old?) and it’s what we hear almost every time we see the doctor- there are a few exceptions of doctors who have gotten to know us and Trey so well that they can see both, and when presenting us with information, do it in a humane, this is a person not a disease kind of way, but they are not in the majority.
In doctors’ defence, they often spend so little time with our kids and family and spend so much more time studying diseases, symptoms, test results and how to treat them that I can see how Trey becomes a leaky heart to the cardiologist, abnormally formed vertibrae to the orthopedic surgeon, joint contractures to the rheumatologist, and a death sentence to the geneticist. BUT, Trey is not that, he is SO much more, and a doctor who can spend enough time with our family to speak to Ryan and I as a parents of a child who is no less perfect than anyone else, to Avery and Sadie as siblings to a brother they get frustrated and fight with but would defend to the death, and to Trey as an awesome kid who will accept you exactly as you are, will hold my heart in her palm.
I’d honestly just gotten used to people describing Trey in this way. He has Hunter Syndrome. I thought it was par for the course. I don’t see Trey this way, but I know most other people do. Rick Guidotti has changed this for me. He is a fashion photographer redefining beauty. I’m going to see if he’ll come to Vancouver and talk to BC Children’s Hospital. His two videos below have changed my entire perspective. I don’t have to accept people seeing Trey as a disease (watch out world!). If Rick can see Trey and not feel pity or fear and instead see a kid and person, first and foremost, so can we all.
The first is a trailer for a documentary Rick is featured in called On Beauty.

[youtube]http://www.youtube.com/watch?v=lDlYOo_tNN4[/youtube]
The second is an interview with NBC. Click here if you want to entertain the idea of redefining beauty, not only for Trey, but for yourself and the rest of the world too.

 
I wrote this post a while ago, it’s not about Mother’s Day, but I felt compelled to post on Mother’s Day, so here you are. This blog post is dedicated to all women who would fight a tiger for those little people (and some now big people) we love to the depths of our souls, to those moms who were called to task and have had to fight lions, tigers, and possibly doctors, drug companies, government agencies, and God, for these beautiful little wonders, and especially to those moms whose children- people we love more than ourselves- are no longer with us. This quote is achingly true: “Making the decision to have a child – it is momentous. It is to decide forever to have your heart go walking around outside your body. ” Today is about honouring the roads you have traveled and all you have done.
This post is called ‘Getting to Know Trey.’
Every time we start a new activity, whether physiotherapy, baseball or art class, I feel compelled to explain ‘our situation.’ At times I have sat back and waited for the looks or the questions (why he uses stamps to print his name, why he can’t play grounders, what those things in his ears are etc.), but the questions only come from toddlers, whose curiosity and lack of social training lead them to ask about any and all questions they have, and the looks, well, they’re heart wrenching. And who knows what kinds of conversation the looks will lead to at home. At least if I introduce the conversation, I can hopefully have some impact on word choice for future private conversations. So, after Trey gets going, I often introduce Hunter Syndrome.
In the past, I have described Trey as ‘harder to get to know’ or ‘harder to understand’ than other typical kids such as Avery and Sadie. However, I have come to the conclusion that this statement is not true. The real and true Avery and Sadie, the deep down you and I, we are just as hard to get to know as Trey; the only difference is that we can hide and choose not to discuss the ‘hard to get to know’ parts of ourselves and Trey cannot.
I have been thinking about this for a while and one day, an idea I was introduced to during my Women’s Studies degree years ago popped into my head. It is referred to as ‘passing.’ I found a definition of ‘ racial passing’ on Wikipedia:
“Racial passing refers to a person classified as a member of one racial group attempting to be accepted as a member of a different racial group. The term was used especially in the U.S. to describe a person of mixed-race heritage assimilating into the white majority during times when legal and social conventions of hypodescent classified the person as a minority, subject to racial segregation and discrimination.”
Although the definition above describes racial passing, I think passing can be used in any context where a person is attempting to be accepted into a group of people (often deemed as having higher status). If you or I have a learning disability, four toes, ADHD, anxiety, a seizure disorder, alcohol addiction, and the list, of course, goes on and on and on, I can ‘pass’ as the same as you and you can ‘pass’ as the same as me. Adults can talk about the weather and other mundane topics, avoid the deep and dirty, and not really get to know each other. Kids can ‘be cool’ and not talk about being dyslexic or their parents’ divorce or their night terrors. With most of our differences and challenges, we can ‘pass’ as whomever our culture (or the group we want to be included into) deems ‘normal’, acceptable and superior. We have the choice to acknowledge our differences and challenges or keep them to ourselves.
Trey does not. His quirks are out in the open. When he is happy and excited and enthusiastic, he beams from ear to ear and he often jumps up and down or does a silly dance (which, by the way, I love. Trey’s silly dance is AWESOME). He hasn’t learned the ‘cool’ way to hide his excitement. When he is disappointed, it’s apparent. He cries. He also has hearing aids, a port, and lots of scars. He doesn’t always understand the games other kids are playing, which is obvious. He is just now learning to print, read and colour in the lines. All of these quirks can lead to exclusion, sadly, while others, with the ability to pass, are trying to fit in.
Nobody is normal. You’re not. I’m sure not. But at least we have to ability to pretend. The only difference between Trey’s differences and your or my differences is that Trey can’t hide his and we can (ours are also likely more socially acceptable). You don’t have the choice of getting to know Trey casually. If you’ve had the honor of meeting Trey, you’re either all in or all out. He cuts out the BS. I happen to like that idea. Of really, truly knowing people. Thanks Trey, you’ve inspired me yet again.

Emily Rapp, mother to a child with Tay-Sachs (a Lysosomal Storage Disease like MPS II) and author of the New York Times article entitled ‘Notes From a Dragon Mom’ (click here to read it) to which I’ve referred more than once, has written a book entitled ‘The Still Point of the Turning World (if you click on the title you can read a Q & A with Rapp about her book).’ Knowledge about this new book quickly filtered through our MPS community. The MPS II moms I know recommended I not read the excerpt of the book, which was about receiving the diagnosis (click here to read the excerpt), because it brought back too many emotions. Of course I read it, I couldn’t not. Rapp is a brilliant writer. Maybe I think she’s brilliant because I feel connected to her as a dragon mom (Emily, can MPS moms be dragon moms too?) and have experienced the urge to go back or run away- Rapp’s urge was to swallow Ronan so they could go back to when life was okay, mine was to buy a cabin in the woods. For weeks after Trey’s diagnosis of MPS II, all I could imagine when I thought about how to go on, was visualizing Trey and I huddled in a cabin isolated in the woods, snuggling and hugging and loving, far away from everyone and everything. More likely though, I think she’s just a gifted writer. I think that anyone who picks up her book will be able to feel her through her writing… if you dare.
Which is one of her points. Most people who aren’t dragon parents won’t want to. Go there. In her NY Times article, she writes:
NOBODY asks dragon parents for advice; we’re too scary. Our grief is primal and unwieldy and embarrassing. The certainties that most parents face are irrelevant to us, and frankly, kind of silly. Our narratives are grisly, the stakes impossibly high. Conversations about which seizure medication is most effective or how to feed children who have trouble swallowing are tantamount to breathing fire at a dinner party or on the playground. Like Dr. Spock suddenly possessed by Al Gore, we offer inconvenient truths and foretell disaster.
And there’s this: parents who, particularly in this country, are expected to be superhuman, to raise children who outpace all their peers, don’t want to see what we see. The long truth about their children, about themselves: that none of it is forever.
She also addresses this in an interview about her book:
Parenting.com: Is there anything you wish parents of healthy children would take away from this book?
Rapp: I wish parents of healthy children wouldn’t be smug about it. There’s something about smug parents that really hacks me off.
Parenting.com: Smug how?
Rapp: I hear this a lot from parents, and it’s like how is this in any way helpful? “Looking at your life makes me feel blessed.” Which is another way of saying “I’m glad I’m not you.”
Parenting.com: What a horrible thing to say.
Rapp: It’s disgusting. And what I want to say but don’t say is “you don’t know what’s going to happen.” I don’t wish anyone ill. But you don’t know what’s going to happen to your children, so you better enjoy them now. They could drown in a pool or get leukemia or shoot themselves in the head when they’re 30.
People don’t want to hear that. But don’t look at me and put that sympathy on me, because you don’t know when chaos will hit you. And it will. I have a great life. It’s a sad, complicated, beautiful, strange life. It’s mine.
I really like her comment: ‘It’s mine.’ I agree. My life with MPS II may not be what we dream about before having kids and you may count your blessings that it’s not yours, but this life is mine. And it’s great. And I LOVE it. I also like her story because I’ve felt so many of the things she’s felt. The excerpt about diagnosis definitely caught me. But you know what stuck with me while I was falling asleep? And what sits with me this morning and what led to this blog? This:
Parenting.com: This book is all about living that knowledge that the end is coming. Now that you’re on the other side, is there something you would tell the you who was living in that?
Rapp: I would tell the me who was living that that when he died I would be relieved. And I was. I don’t know if there’s any right way to grieve. But when Ronan was diagnosed, it was like he died on that day. It was like he got hit by a truck. 
After reading that I asked myself: Will I be relieved? Did Trey die on D-day? Did I feel like Trey got hit by a truck? The answer to the last two questions is no. My journey with Trey and MPS II has been different than Emily’s with Ronan and Tay-Sach’s in that I have had 3x the amount of time so far with Trey than Emily had with Ronan. I also had 4 years of hope that Trey’s brain wouldn’t be affected and then the hope gifted to us through the trial. Trey is still alive and happy and learning. As to the first question, I wonder. I don’t ‘go there’ often. With IT and future treatments, maybe I won’t have to go there. But with Ryan in North Carolina this morning as Trey got his 19^th dose of intrathecal enzyme, I wonder about the first question. UNC and trial trips are so loaded and intense for me, Ryan has taken them on for a couple of months so I can have a break. The night before every IV ERT infusion, I pray the next day will go well. With every check up there is always the ‘what if.’ And then there are multiple surgeries… When it comes to Trey’s life, I have fought for every step, for every service, for appointments, for procedures, for life. I would not have done it any other way, I would do anything for Trey, I love Trey and I love our life more than anything and am grateful for every second I have with him, but when Emily said that when Ronan died she felt relief, it feels heart-wrenching that on some level I understand.

Dear MPS II Research Fund Supporters,
It has been almost a year and a half since our last major fundraiser for the MPS II Research Fund. A year and a half ago, at the time of our Once Upon a Cure Gala in September 2011, our family and our fund were in the midst of major changes, and I have waited until we knew what was going on, to be in touch with you again.
As many of you who read this blog know, in August 2011 Trey qualified for an intrathecal (IT) clinical trial at the University of North Carolina (UNC), where the enzyme he is missing is injected into his central nervous system. Since February 2007, Trey has been receiving a weekly four-hour intravenous (IV) Enzyme Replacement Therapy (ERT) called Elaprase that infuses the enzyme he is missing into his body, however, due to the size of the protein, IV Elaprase cannot cross the blood brain barrier and thus cannot prevent his progressive brain decline.
Trey began monthly IT doses, injected into his central nervous system via lumbar puncture, at UNC in October 2011 and for the first six months of the trial, Trey, Ryan and I spent ten days per month at UNC, while the rest of the family (Avery and Sadie) remained at home. From month seven onwards we have only had to travel four to five days/month. While the shorter trips have been far easier, they remain incredibly time consuming emotionally, medically, and logistically. Fortunately, our family, friends, neighbors, and community have been amazing, joining us on trips to UNC to help, staying days and nights at our house looking after Avery and Sadie, driving them to activities, and more. Also fortunately, this trial has proven to be beyond our wildest dreams, making all the work so incredibly worth it. Trey is stable. Not only is he no longer declining cognitively, but he is also learning again and he is able to continue to participate in activities such as art class, community soccer and baseball, activities that he otherwise would have lost the skills, ability, and attention for. It is our hope that in the near future we will be able to receive these IT doses in Vancouver at BC Children’s Hospital and only travel to UNC every six months for assessments.
A year and a half ago, we were also made aware that The Canadian MPS Society might increase administration fees for the MPS II Research Fund. The Board of Directors has made a final decision regarding the fee, and as of March 31, 2013, administration fees for the MPS II Research Fund will increase from 5-20%. On our own, operating costs and administration fees are not 20%, they are not even 5%, but because when we began the fund we wanted to share in a united front with The MPS Society, we agreed to 5%. The Society does, after all, have costs such as renting office space, paying employees, etcetera, however, with the jump from 5-20%, Ryan and I no longer feel it is in the MPS II Research Fund’s best interests to remain with the Society.
After looking into our options, we have decided to join forces with another registered Canadian charity called The Isaac Foundation for MPS Treatment and Research (charity number: 806 930 079 RR0001).  Isaac is an eight year-old boy, like Trey, who only months before Trey’s diagnosis of MPS II, was diagnosed with MPS VI. Similar to our journey in British Columbia, Isaac’s parents, Andrew and Ellen McFadyen, were forced to fight the Ontario government for the first ever ERT for MPS VI, Naglazyme. Also similar to our story, Andrew and Ellen were driven to fund research for a cure. As a result of this drive, they began The Isaac Foundation. Over the years, we have become close with Andrew and Ellen, not only because of the similarity of our situations, but because we share similar values, beliefs, and we will not rest until we find a cure.
Andrew and Ellen have worked tirelessly for a cure for MPS, holding yearly galas, golf tournaments, and other fundraisers. They have done such good work raising money and finding solid research to fund, that the project they are and have been funding for the past series of years is getting close to entering human clinical trials in MPS.  It is a very exciting time for The Isaac Foundation.
Andrew and Ellen have welcomed us into the arms of The Isaac Foundation. We have opened an MPS II Research Fund within the Isaac Foundation, much like we had an MPS II Research Fund within the Canadian MPS Society. The difference is that instead of donations going to MPS II research within The Canadian MPS Society, donations will go to MPS II research within The Isaac Foundation. Another difference, a GREAT one, is that we will not be charged administration fees aside from the 4% that Canada Helps (the online donation platform) charges upon each online donation, or the 15 cent fee charged to issue your receipt if your donation comes in via cheque or cash.  Your donations are going to fund research, alone.
At this time, while we continue to travel monthly to UNC as part of the clinical trial, we are not organizing any large fundraisers, but your donations are welcome at any time, and we will be in touch as soon as we begin organizing another fundraiser. This is an exciting time in the scientific community, with many treatments on the horizon, so there is no shortage of research to fund.
In terms of logistics, you can donate:
Online at: www.treypurcell.com or www.theisaacfoundation.com where you will be linked to Canada Helps (choose the MPS II Research Fund from the drop down option), or
By mail: You can mail a cheque to The Isaac Foundation MPS II Research Fund, at: 1449 Chamberlain Dr. North Vancouver, BC, V7K 1P8.
A tax receipt will be issued for all donations over $15.
We are grateful to The Canadian MPS Society for supporting our Fund for the past seven years, and we will continue to support them by attending their fundraisers and subscribing to their quarterly publications, but it is time for our fund to move on. Ryan and I are excited about our future with The Isaac Foundation and continuing our work to find a cure for Trey, Isaac, and all kids with MPS. Rest assured that Andrew, Ellen, and The Isaac Foundation are as invested and passionate about finding a cure as we are.
If you have any questions, please do not hesitate to email me at: purcelldeb@gmail.com or call me at: 604-317-3911. This is a big change for us, but a worthwhile one. I look forward to hearing from you and seeing you again soon. For updates on Trey and our MPS II Research Fund, you can visit: www.treypurcell.com and to read more about The Isaac Foundation, you can visit them at: www.theisaacfoundation.com.
With gratitude for your continued support,
Deb and Ryan Purcell
purcelldeb@gmail.com
604-317-3911

Johnson & Johnson has stepped up!! This would not have happened without your voices. We were loud and clear and J&J finally listened. THANK YOU. THANK YOU. THANK YOU. Here is a statement from Andrew McFadyen of The Isaac Foundation:
Numerous times over the weekend, and again late this afternoon, I had the opportunity to have lengthy discussions with Dr. Amrit Ray, the Chief Medical Officer of Pharmaceuticals at Johnson & Johnson. Those discussions proved very fruitful, and I am pleased to report my sincere belief that Janssen Pharmaceuticals is engaged in moving forward in a very real and serious manner with respect to the proper study of Elmiron as it relates to MPS patients.
Janssen has committed to forming a medical advisory panel, and plans to meet with these experts in the very near future. Their aim will be to review current data so they may better understand whether Elmiron might be a candidate for further research in the search for a safe and effective treatment to support children with MPS. This commitment was made and has been followed through on in very short order. I am happy to report that some of the leading experts in the world who specialize in MPS diseases have already signed on to participate in Johnson & Johnson’s medical advisory panel. In short – this is fantastic news for our kids.
The Isaac Foundation has been communicating with Johnson and Johnson (Janssen Pharmaceuticals) for the better part of 8 months in our ongoing effort to ensure that such a review could become a reality for our children. And while these past 8 months have proved frustrating and stressful, the past few days have restored our true hope that the data presented on Elmiron may lead to a clinical trial in our children in the very near future.
Does this mean that a clinical trial is imminent? The answer is no, we aren’t there yet. But this does mean that a true review of Elmiron by Janssen will be thoroughly conducted by the some of the best minds in the field of MPS, from all over the world. We look forward to monitoring the progress of those discussions as we seek to find the best treatment options possible for all of our kids suffering from MPS.
It is important to note that all of your voices have been heard, and we can’t thank you enough for standing up for what you believe in – for standing up for our children. Two short weeks ago, we were at a dead-end with respect to further study of this drug by Johnson and Johnson. Two short weeks ago, hopes were dashed that progress could be made moving forward. But you let your voices be heard, and asked for Johnson & Johnson to live up to it’s Credo Values – values that commit their “first priority is to the doctors, nurses and patients, to the mothers and fathers…”
Working together, your voices united to remind them of those values. After speaking with Dr. Ray late this evening, it is my firm belief that those values will be upheld and that Johnson & Johnson will do everything in its power to put our children first.
Please take a moment to let Johnson & Johnson know how you feel about these latest developments. You can click on the link below to leave them your thoughts, and comment here too, please!
https://www.theisaacfoundation.com/leave-jnj-a-message/
And to leave a message for some of the advocacy groups that spent countless hours working for our kids, please visit their sites below. They did it all – and always had our kids best interest at heart.
http://www.savingcase.com
http://heresjack.com

Deb here again. There is a poem that comes to mind on this evening. Here’s to YOU:

The Low Road

What can they do to you? What ever they want.

They can set you up, they can bust you, they can break your fingers, they can burn your brain with electricity, blur you with drugs till you can’t walk, can’t remember, they can take your child, wall up your lover. They can do anything you can’t stop them from doing.

How can you stop them?

Alone, you can fight, you can refuse, you can take what revenge you can, but they roll over you.

But two people fighting back to back can cut through a mob, a snake-dancing file can break a cordon, an army can meet an army.

Two people can keep each other sane, can give support, conviction, love, massage, hope, sex. Three people are a delegation, a committee, a wedge. With four you can play bridge and start an organization. With six you can rent a whole house, eat pie for dinner with no seconds, and hold a fundraising party. A dozen make a demonstration. A hundred fill a hall. A thousand have solidarity and your own newsletter; ten thousand, power and your own paper; a hundred thousand, your own media; ten million, your own country.

It goes on one at a time,

it starts when you care to act,

it starts when you do it again after they say no,

it starts when you say We and you know who you mean,

and each day you mean one more.

-Marge Piercy