News Archive
April 28, 2008 The North Shore News wrote a great article about Trey and the situation in Canada with rare drugs for rare diseases. To read it, click on the link: http://www.canada.com/northshorenews/news/sundayfocus/story.html?id=7725c2a5-0a7e-4cad-99dc-91516a126569. We hope to see you this Saturday, May 3rd at Lynn Valley Elementary School from 3-6pm for “Tacos for Trey” as it’s going to be tons of fun!! For details, click on “Events & Donation.” April 14, 2008 Lisa Priest wrote an article for today’s Globe & Mail about a boy who has been denied Elaprase in Ontario. Click the link to read it: http://www.theglobeandmail.com/servlet/story/LAC.20080414.ORPHAN14/TPStory/TPNational/Ontario/ April 8, 2008 Bobs and Lolo just confirmed they will be performing at Tacos for Trey 2008 on May 3rd!! The local dynamic duo, Bobs and Lolo, are dedicated to connecting kids to the natural world with music, movement and make-believe. Sharing musical stories that engage, inspire and educate, they teach kids to care about themselves, their neighbours and the planet. For more info about Bobs and Lolo, go to: www.bobsandlolo.com or for more information about our Tacos for Trey fundraiser, click on the “Events & Donations” page. All with Trey is going excellent. We’re just gearing up for our awesome fundraiser!!. March 12, 2008 Our Tacos for Trey invitations are ready and we will be sending them out in a couple of weeks! If you would like either an email or hard copy invitation, please send me an email and I’ll send one off to you. You can view the invitation on our “Home” or “Events and Donations” pages. In other news, Trey celebrated his fourth birthday on March 1, and if you’d like to read the press release issued for Rare Disease Day, click here. February 27, 2008 On Friday February 29 is Canada’s first ever International Rare Disease Day. Help us raise awareness about rare diseases and Canada’s lack of support for rare diseases by writing a letter to your MP, telling someone about MPS II while you’re at Starbucks, the grocery store, wherever! And don’t forget to check out CORD's newspaper insert in the National Post on February 29th!! I’ve also included in my blog, news about an MPS II meeting that happened in Ontario this past weekend to figure out how patients with Hunter Syndrome can secure funding for Elaprase. George Smitherman continues to deny Ontario citizens their rights, so now it is up to individuals to demand them. And last but not least, you’ve got to check out the picture in today’s blog! February 5, 2008 Trey began ERT one year ago today. What a fantastic year it has been. He has not missed one infusion, which is unbelievable. He is growing like a weed and continues to be very tall for his age, his speech is improving quickly and his development is doing great, his range of motion and joints are where they should be at for a kid his age, his xray and othopedics appointment two weeks ago week showed us that his bones are growing and doing well (Trey had and EKG and echocardiogram yesterday at the hospital, but we do not yet have results)... our life is strangely and wonderfully “normal.” The only other news going on right now is that we are gearing up for our second annual “Tacos for Trey” fundraiser on Saturday, May 3 this year. Details are in my blog and on the “Events & Donations” page. January 4, 2008 In disappointing, but expected news, the CDR (aka. Common Drug Review) did NOT recommend Elaprase for funding. As defined by the Canadian Agency for Drugs and Technologies in Health, on January 4, 2008: “The Common Drug Review (CDR) conducts objective, rigorous reviews of the clinical and cost effectiveness of drugs, and provides formulary listing recommendations to the publicly funded drug plans in Canada (except Québec).” Next week, The Canadian MPS Society will be issuing a press release responding to the CDR's decision. This does not mean that funding cannot be secured for people with MPS II, it just means that funding will continue to be a struggle and will continue to be decided on a case by case basis. Attached is a link to the CDR’s negative recommendation: http://www.cadth.ca/media/cdr/complete/cdr_complete_Elaprase_Dec-19-2007.pdf. The CDR has incorrectly concluded that the effectiveness of Elaprase has not been established. In contrast to the CDR’s decision, Health Canada gave Elaprase a Priority Review based on their conclusions of efficacy and the life threatening nature of Hunter syndrome. Coincidentally, global treatment recommendations for Elaprase have just been published. Click on the link to read them: AI - Rare Disorders.pdf. I have also attached (click on the link: Wraith et al 2007 Hunter Syndrome Review and Treatment Recommendations Eur J Peds.pdf) an article reviewing and recommending ERT for Hunter Syndrome. The CDR needs to make changes to the way it deals with rare disease drugs. It is clearly not able to properly value and assess rare disease therapies and has just created another barrier to patients with rare diseases trying to access approved and effective drugs in Canada. Canadian patients with rare diseases are at a serious disadvantage, and as far as I’m concerned, this is a human rights violation. December 21, 2008 I copied the below out of the Winter issue of the Canadian MPS Society’s newsletter, The Connection: Support Patients with Rare Disorders in their Fight for Life-Saving Therapy “Canada is the only developed country without an “Orphan Drug Policy” so Canadians with rare disorders are denied access to therapies available in other countries. There are up to 6,000 rare disorders affecting nearly 10% of the Canadian population. Thanks to advances in science, some disorders can now be treated so patients live longer and have improved quality of life. We need your help to get governments to put in place an “Orphan Drug Policy.” Your support is urgently needed to assure that Canadians with serious and life-threatening rare disorders get the same chance for life as those with more common disorders. A letter of support is available on the website of the Canadian Organization for Rare Disorders (www.raredisorders.ca).” I sent in a letter myself, and it can take as little as a few minutes. All you have to do is click on the link at the top of the home page, fill in your name and address and the site will tell you who your local MP is. It then gives you a generic letter that you can submit as is, or you can edit it as you wish. It is actions like this that make change, so if you can take 2 minutes to send in a letter that will save lives, possibly Trey’s, we would appreciate it. How can an ODP help Trey? If this new Intrathecal Therapy for MPS II gets approved for use, and Trey needs it, or if another treatment becomes available, an ODP means that we will not have to fight tooth and nail to get the treatment for Trey. We will not have to wait months, while Trey’s disease progresses, to see if the treatment will be funded. We will not have to fight for new treatments like we had to fight for Elaprase. We have also started organizing next year’s “Tacos for Trey”(click here for more info) and I have added a new blog for today. December 18, 2007 Happy Holidays! It’s been a while since I’ve updated. Here’s what’s new: another boy started ERT for Hunter Syndrome last Wednesday at BC Children’s Hospital. I consider this big news as kids around Canada are still being denied funding for Elaprase. We are so fortunate to live in BC and so fortunate that our government has agreed to fund Elaprase for us. Other big news is that an Intrathecal (IT) trial for Hunter Syndrome will be starting in the spring of 2008 in North Carolina. With Intrathecal Therapy, the enzyme that kids with Hunter Syndrome are missing will be injected directly into the brain to help the effects of the missing enzyme on the brain. As far as I know, Shire HGT (Human Genetic Therapies) will begin the search for candidates in January. The trial will only be done on kids who have CNS involvement, so fortunately for us, at this point, Trey is not a candidate. If at some point Trey’s development begins to decline (which Dr. Escolar gave us a 99% guarantee it will not), he would then be a candidate for one of the subsequent phases of the trial. It is also great that Trey is not a candidate at this point as the first phase of the trial is a safety phase and not one that I’d be jumping for Trey to be a part of... although if he demonstrated CNS involvement, we’d do anything we could to get him in the trial. We’d also want him to be a part of the trial because of how hard it is to get rare drugs for rare diseases funded in Canada. If we had to wait for a trial to end and then fight for Trey to get the drug in Canada, it would almost certainly be too late for the drug to be effective. This has happened with families fighting to get Elaprase in Ontario. I cannot even imagine. Recently, Trey has been to see his Rheumatologist, his PT & OT, his osteopath, and his ENT. Trey’s joints continue to have good range, the fluids in his head and body are continuing to move much better than before he began ERT, and a check up yesterday with Trey’s ENT told us that Trey’s new tubes are in, doing well and his ears are clear of fluid. Everything is going very well. Trey has a check up with his Orthopedic doctor in early January. November 8, 2007 Trey and Avery’s sister, Sadie, was born October 30 at 7:12pm at home. Ryan caught her and Trey cut the umbilical cord. Everyone is adjusting well and the boys are thrilled. Details about Sadie’s arrival and Trey’s UNC appointment is in my blog. Yesterday, Ryan and Trey flew down to UNC for another developmental test with Dr. Escolar. In a sentence, Trey is doing really well. They return home on Saturday. On October 25, Trey had an appointment with his PT and OT. Ryan went with him, and as Ry is not big on details, all I could get out of him was that Trey was fitted for orthotics. And last but not least, here is an article Simon Ibell sent me about the funding issues for Elaprase in Canada: Medical Post Article - Entering the Enzyme Era (MPS II).doc.pdf It can also be seen on the Media page. October 2, 2007 Trey had a successful tube surgery yesterday morning. Both of his previous tubes were out, so Dr. Moxham replaced them with permanent T-tubes. His left ear had some thin fluid in it, but his right ear was filled with a lot of thick fluid. Dr. Moxham got all of the fluid out, but his right ear required quite a bit of suctioning, so his ear is draining some blood tinged fluid. I’ll give more details in my blog, but all in all, a great and easy surgery...once the anesthetic wore off, he was back to his normal self. We also received awesome news from Trey’s physiotherapist (PT) last week. For about the past year, Trey’s PT has been closely monitoring his joints, as he has been losing range in areas such as his ankles, elbows, wrists, knees, and shoulders. Two appointments ago, Trey’s range had increased a bit, but his PT didn’t want to say too much about it as she had only seen Trey a few times (Trey switched PT’s in June, this one specializes in joints) and he could have just been more comfortable with her. However, Trey’s range has continued to improve. At this point, the range in every joint in Trey’s body has improved as much as 30 degrees and all but his right wrist and elbow are now within “normal” range for a kid his age. With a progressive disease, we are thrilled when things haven’t gotten worse, but to improve...especially when Trey is athletic as he is...we’re ecstatic. And in more good news, the family from Quebec who has been fighting to get Elaprase for their son, Nathaniel, has finally received funding privately through their family’s health plan. This is virtually unheard of in Canada (private funding... well, funding for any rare disease, but especially through private insurance), I don’t actually know anyone who has received funding this way before, but they have done it, and we are so excited for them. The family in Ontario is still fighting. September 19, 2007 In my September 6 blog, I mentioned that I was hoping Trey could have his upcoming MRI done with his tube surgery, so we could avoid another General Anesthetic. Even though I was told it is possible and has been done, I was told it would be a fight to get done and days after I made this request, was denied. So I pushed more. Fortunately, I have a great amount of trust and faith in Dr. Stockler (the doctor who oversees Trey’s care at BCCH), so when she came to Trey’s infusion and said she truly looked into it and this time around it is not possible, I believed her. She said that because the tube surgery was booked so quickly and is urgent, it is just not possible to do an MRI that soon. She could have said it is an emergency MRI, but because it is not, she did not feel comfortable doing so. She also said that because Trey is developing so well and we already know there are changes in his brain due to MPS, there is really no need to do and MRI in December. To avoid yet another anesthetic, we can do the MRI sometime in the new year. She is just great. In Elaprase funding news, our friends the Lanese’s are still fighting hard to get Elaprase for their son, Andrew, in Ontario. Their fight is in the news. The second part of their story (the first part aired last night) airs tonight on Global TV in Toronto. Unfortunately, Ontario has made a preliminary decision to deny funding of Elaprase to all patients living with MPS II in Ontario. More in my blog... And last but definately not least, we have gotten some awesome emails from Stargate Atlantis fans. What led to this is 2 autographed North Face duffle bags being auctioned off on Ebay. Click here to see how the items are doing, or to make a bid. We are forever indebted to the Stargate cast and crew who have done so much for our family and for Trey. More about this also in my blog. September 6, 2007 After a series of audiology and one ENT (Ear, Nose and Throat doctor) appointments, about which I will go into detail in my blog, we have found out that Trey’s right ear tube is out, his left is plugged, and both ears are filled with fluid. This means another surgery to insert ear tubes, this time with Goode (longer lasting) tubes. The pro of Goode or T-tubes is they last longer which means less surgeries. The cons are that because they are longer tubes, they are more likely to get plugged, so we need to keep a close eye on him when he gets a cold, and they have a higher rate (up to 15%, when the other tubes have a 1% chance) of leaving a permanent hole in his ear drum. If this happens, Trey will need surgery down the road to cover up the hole. In the MPS world, Goode tubes are highly recommended. Trey’s tube surgery date is for October 1. In MPS Society news, I have recently been elected to the Canadian MPS Society Board of Directors. There’s not much to report on that front, other than I am really excited to be a part of the Board. The next meeting is in November, so if you have any questions or concerns, I’m all ears. August 21, 2007 Trey had a successful hernia surgery yesterday. The difference in his recovery from his left inguinal and umbilical hernia surgery 3 monthes ago to his recovery from yesterday’s right inguinal surgery is unbelievable. More in my blog... We have also uploaded a copy of the live interview we did for Global television as a part of the BC Children’s Hospital Foundation Miracle Weekend on June 3, 2007. To see it, click here. August 7, 2007 Quite a lot has happened in the past month since my last update. Trey has now been on ERT for six monthes, he has a new hernia so will likely have surgery again within the next month, Trey’s splints are causing blisters so we need to get those adjusted, an Ontario family whose son, Andrew Lanese, has Hunter Syndrome, was just denied a second time funding for ERT, and the list goes on. So as to not bog up the news section, I will write about all of this and more in My Blog. July 11, 2007 On June 27, 2007, the MPS Society issued a press release for the approval of Elaprase by Health Canada. Copies of the print and video press releases are on the Media page, and you can also see the print press release in my blog. In other news, Trey is now wearing his AFO’s (splints to help loosen his achilles tendons) at night. He hasn’t been a huge fan of them, causing over a week of disturbed and restless nights, but last night, for the first time, he slept through the night with them on. Thankfully, as I was wanting to give up. On Monday, while Trey was having infusion #23, Dr. Stockler came for a visit. To read about our conversation, see my blog. June 21, 2007 Elaprase has been approved by Health Canada!! This is a huge step because it means that Elaprase is now accessible to all people in Canada affected by Hunter Syndrome. It was approved on June 14. The catch is that although it means that the drug is approved for use in Canada, it is up to the individual provinces to approve funding. This, as has been demonstrated with many rare disease drugs, is where the struggle (and frustration and despair and and and) comes in and this is why Canada is so badly in need of an Orphan Drug Act. As soon as I have a final copy of the Press Release, I will post it up here. And speaking of Elaprase, on Monday, Trey completed his 20th infusion!! What an absolutely great feeling. We have also received a few more results from Trey’s recent series of tests. His abdominal ultrasound (which was done in May) showed that his spleen (which was enlarged before he began ERT) is now within the limits of what is defined as normal for his age. His liver, which was 1.7 times the “normal” size before ERT, was described as bulky, which from my understanding means that it is still a bit big, but much smaller than it was, and almost within normal range. Trey’s doctors expect that it will continue to shrink. They also said that ultrasound is just a guide and not as accurate as an abdominal MRI, which will be done in December. Trey’s cardiology results. A bit confusing. His electrocardiogram was normal. His echocardiogram report showed that he has trace Mitral Valve Regurgitation and as a result, his left ventricle is a bit larger than normal. I was told one year ago that Trey had Mild Mitral Valve Regurgitation (so trace MR is an improvement), however, the report from last year does not say anything about MR (I would have had no idea what MR was unless someone told me my kid has it, so I know the words were mentioned to me). The cardiologist who works with kids with MPS at BC Children’s is on holidays, so I’ll find out more in a few weeks. With what we are working with so far though, it seems as though Trey’s heart has not gotten any worse, and with a progressive disease, I am pretty thrilled with these results. Lastly, my good friend Tovah has uploaded a copy of the speech I did at the MPS Cup on the media page. To see it, click here. June 10, 2007 On Friday, a dear friend of mine, Amy Donaldson, passed away after a hard fight with cancer. I never met her, but we became close friends after her son, Owen, was diagnosed with Hunter Syndrome last August. Details are in my blog. And last night was the MPS Cup Fantasy Hockey Game and Gala. It was a ton of fun and I look forward to it every year. I already can’t wait for next year!! Details are also in my blog. June 5, 2007 This past weekend, Trey, Ave, Ry and I participated in the BC Children’s Hospital Foundation Miracle Weekend. It was an amazing event with tons of stuff for the kids to do and lots of food. Sunday morning we showed up for a photo shoot with the family and then were interviewed live on Global TV about our experiences at Children’s. They raised in total over $14 million dollars (“Tacos” better step up!?). I’m hoping to get a copy of our interview to put up here. This coming Saturday, June 9, is the MPS Society’s biggest fundraiser, the MPS Cup. The ED, Kirsten Harkins, and her husband Todd put it on. It is a fantastic event where you get to watch friends and family take the ice with famous hockey players (Ryan and some of his friends played last year) and then attend an awesome Gala dinner and auction afterwards. This fundraiser is where pretty much all the Society’s research money comes from. Last year they raised over $60,000. For details, go to: www.mpscup.ca. I will be speaking at the Gala. As far as Trey goes, he’s doing great. He had infusion #18 yesterday along with an ultrasound of his abdomen. The changes we are noticing in Trey as a result of ERT are amazing. He’s growing like a weed (he is so tall!!), his stomach is flat as a board when he wakes up in the morning, his face and facial features are much softer, I notice changes in how he moves, he can hear because his ears aren’t plugged with fluid (which is affecting his speech) and he’s so healthy (although he was healthy for quite some time before ERT as well). We haven’t had to miss a week of ERT yet. Life right now is pretty darned awesome. We are so happy to have gotten Trey on ERT as soon as we did. I will never be able to thank everyone who got us here enough. The only medical result we’ve received recently is his electrocardiogram, which was normal. Oh, and Trey’s hernia sutures are healing up well. And in unrelated news, I am pregnant. We are having a baby girl and she is due on October 29. I have had a CVS and ultrasounds and everything looks good, so we are pretty excited to add a new member to our family, and a baby girl at that. June 1, 2007 Trey had surgery on May 24 for both his inguinal and umbilical hernias. Since a slight temperature and trip to the hospital the next day to make sure everything was okay, everything has been fine. Trey was in quite a bit of pain for the first few days, which was hard to watch, as I’ve never seen him take so long to recover before (although he’s never had a surgery like this before either), but he’s now back to his usual self and everything looks great. In the past 2 weeks, Trey has also seen rheumatology, ENT, opthalmology, and cardiology. Rheumatology didn’t have anything monumental to say, they just referred us to a PT and OT (physiotherapist and occupational therapist) who specialize in joints. We should be seeing them in a few weeks. I will also find out more about splints for Trey’s ankles and elbows at that time. As far as ENT goes, Trey’s ear tubes look great. They’re still fully in and they’re not plugged, which is great because Trey’s last set of tubes did not last nearly this long. Wonder if it’s the ERT or ... Opthalmology also went well. Trey’s corneas and retinas are in good shape. No problems. We’ll see them again in a year, and with regards to cardiology, Trey had a echocardiogram and electrocardiogram, but we have not yet heard about the results. And last but not least, Trey’s weekly infusions are still going well, nothing to report. This past week he had infusion #17. I have also attached a link for an article in the Arizona Republic newspaper on May 22 about our good friend and inspiration, Simon Ibell and his good friend Steve Nash. To read it, click on the link: http://www.azcentral.com/arizonarepublic/sports/articles/0513youngcp0513.html. May 21, 2007 We arrived home last night from our trip to North Carolina to see Dr. Escolar and her team at the UNC Centre for Development and Learning. Dr. Escolar is doing a natural history study of people with Hunter Syndrome. She has a team of specialists who take a history, do development tests, take measurements and in the end give you an idea of where your child is at in terms of development. From my perspective, we went there for one reason: to find out her thoughts on whether or not Trey has neurological involvement. We first saw her last November and she was unable to give us an answer. This time she gave us more of an answer. She said that it is too soon to know for sure, as she knows of one other child whose development didn’t start to drop until closer to age 4, but she said it is her “gut feeling” that Trey will be okay cognitively. For details on our appointment with Dr. Escolar and for information about Trey’s other appointments and upcoming surgery, please see my blog. May 6, 2007 What an amazing event our Tacos for Trey fundraiser was. I can’t even begin to express enough thanks to everyone who supported us in this. Media, hundreds of family and friends and friends of friends came, along with people from Shire, the Canadian MPS Society, Dr. Potter, the researcher from McMaster University in Ontario whose grant we were raising money for, and Dr. Muenzer. Including money donated to the fund leading up to the event and money we know will be coming into the fund in the next weeks, we raised over $30,000 for the MPS II Fund. For details, please see my blog. May 3, 2007 Our fundraiser is on Saturday. We’d love for you to come!! It’s from 3:00-6:00pm at 3657 East 45th Avenue in Vancouver and it is going to be a great event. As my mom and sister were bringing over the raffle and auction items yesterday, I commented that we’re going to have trouble making sure our event next year is as good as this one. Everyone has done such an amazing job getting auction and raffle items donated, as well as food and drinks, I’m excited to eat the tacos, sip margaritas, and bid on items! On the ERT front, this week has been interesting. Infusion #13. Because it wasn’t straightforward, I will give details on my blog. And last, but no least, I have agreed to be a part of the launch of Elaprase in Canada. Elaprase should be approved by Health Canada in mid June. Not only am I completely pleased to help, as Shire and Elaprase and all involved have given Trey a new chance at life, but Elaprase being approved by Health Canada is a big deal because there are still many boys in Canada who have not been able to start Elaprase yet. We need to advocate for them until it is they are hooked up for infusions. The feeling of having Trey on Elaprase and seeing what a difference it is making... I can sleep at night. April 23, 2004 Infusion #12 is done!!! We (read: Trey) has made it through the riskiest time of infusions without reaction! Although serious reactions can happen up until the six month mark (after six monthes, minor reactions can occur, but nothing serious), the highest chance for an allergic reaction to Elaprase is between infusion #4 and infusion #12. What a great way to celebrate Tacos for Trey. There was an excellent article in this past Saturday’s Ottawa Citizen about the lack of Orphan Drug Policy in Canada. It featured family friends of ours who we met at the last Canadian MPS Conference, the Lanese’s. There is also an article about our good friends from Campbellford, Ontario, the McFadyen’s. To read it, click on the link: http://www.rogercollier.com/clipping.feature.20.citizen.observer.orphan.drugs.html. It is also in the media section. In other news, we have planned another trip to North Carolina to see Dr. Escolar for another development test. We will be heading down to UNC in mid to late May. May is full of doctors appointments for us. Trey will see orthopedics, ENT, rheumatology, and Trey will also be having surgery on both his inguinal and umbilical hernias at the end of May. We look forward to May, but I think we will be ready for June when it comes. In Mama’s Blog, I have included information about Tacos for Trey...some of the items that are in the raffle and auction, details about food and drinks, and what else will be going on during the event. To read more, click here: Mama’s Blog. April 2, 2007 We have a grant to fund!! The Canadian MPS Society has agreed to pay half of a $40,000 grant to Dr. Potter at McMaster University who is doing gene therapy research in MPS II, if we can pay the other half. His project has support from well known MPS II researchers around the world like Dr. Muenzer. We’d also like to give a $4,000 summer studentship grant to a student in Dr. Potter’s lab who will be part of the larger gene therapy project as well. One of the reasons for funding summer studentships is to engage students in MPS research in the hopes that they will choose to continue with MPS research once they become doctors. If you have more questions about either of the grants, please feel free to contact myself or the MPS Society, 604-222-2767 (me) or 604-925-5130 (the MPS Society). So we have a goal. We want to raise $24,000 for the MPS II Research Fund between now and “Tacos for Trey.” If you can help us do this, we’d appreciate it! The money is going straight into research that we are very excited about. I am so glad that through our MPS II Research Fund we will be able to fund an MPS II grant this year. Had we not decided to have this fundraiser, it is quite possible that the only grant going out this year would have been to Lorne Clarke from UBC, doing biomarker research in MPS I. His research will eventually apply to all forms of MPS (we had a visit from Dr. Clarke today, so Trey will soon be a part of Dr. Clarke’s biomarker study), and from my understanding it will help diagnose severity as well as help with prenatal diagnosis, but the more grants we can fund, the more research that will be done, and the closer we are to finding a cure. Just in case you’re undecided as to whether or not you should come to the fundraiser or make a donation, I’ve included a picture of our little cutie slinging his baby lion! :) Last week Trey had infusion #8 and today Trey had infusion #9. Everything is going really well and he continues to have no reactions. Trey’s tummy is still getting smaller, his skin feels softer, his joints are loosening and we also notice a difference in his facial features. It’s absolutely great. March 19, 2007 Email invitations are out and mail out invitations are soon to be out for our Tacos for Trey fundraiser on May 5, 2007! If you would like an invitation and have not received one, please email me at: dcehak@telus.net or call me at: 604-222-2767. For more information on Tacos for Trey, please see our Events & Donations page. Trey also had infusion #7 today. Everything went well. No reactions, uneventful. March 5, 2007 One month of infusions! Trey had infusion #5 today. Everything went well. No reactions, only positive outcomes. Trey’s stomach is continuing to shrink and his shoulder range of motion seems completely normal to me. I’m curious to see what Trey’s physio says on Wednesday. It was Trey’s third birthday last Thursday, March 1. We celebrated on the day with a music class, swimming, and getting a kitten, and on Saturday we had a big family and friends birthday party. It was really fun because Trey finally gets it. The singing, the celebration... it was really fun. Trey’s story was also on Shaw cable yesterday and when I get a copy, I will put it up in the media page. February 20, 2007 Two more treatments, and an article in the North Shore News. Trey completed his third infusion of Elaprase yesterday, and we are already noticing changes. His stomach has significantly reduced in size and we also notice a change in his shoulder range of motion. His arms stretch almost all the way up now! It is very exciting and such a good feeling. We were also on the cover of the Live section in the North Shore News this past Sunday. To read the article, click on the following links: Page 1 Page 2 Page 3 February 6, 2007 First Treatment Media Coverage. Trey, Ry and I were on the cover of the Vancouver Sun today, following Trey’s first ERT treatment. Read the article at: http://www.canada.com/vancouversun/news/story.html?id=762964f0-c5e1-4767-81ac-b55451fcdb06&k=26625. We were also interviewed on The Beat 94.5’s morning show. Check out the interview on the media page February 5, 2007 Trey had his first Enzyme Replacement Therapy infusion of Elaprase today. What an amazing experience and feeling. We can now rest easier at night, knowing that the progression of disease has slowed to stopped. When we lie in bed with Trey at night we can stop thinking that every second that passes, more GAGs are storing in his body, causing damage. But enough of that, this is the news section, right? Pamela Fayerman and a photographer from the Vancouver Sun came to BC Children’s Hospital today to visit us at Trey’s first infusion. Trey’s story will be in tomorrow’s Vancouver Sun, so check it out! January 26, 2007 Trey begins ERT on Monday, February 5, 2007. We will be at the Medical Day Unit in BC Children’s Hospital at 7:30 am, where Trey will begin his new journey. For a list of thank you’s, of which there are many, and for details, please see my blog. January 24, 2007 Trey had a successful VAD surgery this morning. We were in and out of the hospital in four hours and there were no complications. Trey was not intubated, he had only a laryngeal mask. And once again, for details, see my blog. January 19, 2007 Trey will have his Vascular Access Device (VAD, aka port) placed this coming Wednesday, January 24. He has his pre-surgery appointments with anesthesiology and blood work on Tuesday. We also found out yesterday, through Trey’s Naturopath, that Trey is allergic to eggs (yikes!) and walnuts. January 17, 2007 Trey will begin ERT in a few weeks. We still do not have a start date, but now we know it’s actually going to happen! Special Access has been approved (as well as funding back in November) and today we went for a tour of the Medical Day Unit (MDU), where Trey will receive his weekly infusion (and where the nurses have already been trained up on Elaprase!!!). Since Trey will be the first child in BC to get Elaprase (possibly the first in Canada who was not on a trial), we know it’s not for anybody else! For details and information on Trey’s other doctors appointments today, please see my blog. January 8, 2007 Check out a recent Silk FM BC Children’s Hospital Foundation Radiothon that we participated in in November. This radiothon helped raise funds for the foundation which contributes to research and facilities at the BC Children’s hospital. Since the hospital is directly contributing to Trey’s receiving ERT, we are especially happy to have helped with this fundraising campaign. Check it out on the media page. December 19, 2006 We have made a decision regarding transplant or ERT. We have decided to go ahead with ERT. The story of how we came to make this decision is long, so for more details, please read our blog. In terms of getting ERT started as soon as possible, we have emailed and told our contacts in government and at BCCH of our decision, so now we have to wait for them to iron out the details (like putting together a treatment protocol, getting the drug up to Canada...). We don’t know how long this process will take, but when we know more, we will let you know. December 15, 2006 The new date for decision making regarding transplant has been moved until February 16. We met with Dr. Schultz at BCCH today and we are waiting until that date for two reasons: one, all involved doctors agree that Trey needs to have another follow-up developmental assessment with Dr. Escolar three months post-ear tube insertion. Trey’s tubes were put in on November 20. Two, on February 7, 2007, a committee will be reviewing an article the Duke transplant doctors are publishing regarding CBT in children with Hunter Syndrome. Dr. Schultz will be there. He wants to learn more about Duke’s findings before agreeing to transplant Trey, since the article’s abstract did not provide him with enough information to make a decision. For more information on this, see today’s blog. On another note, we will be seeing Dr. Lorne Clarke this coming Monday, December 18 to discuss transplant and on December 27, Trey will have a sleep study done. Oh, and preliminary results were correct. Avery is a perfect match for Trey, meaning that decisions do need to be made regarding BMT or CBT, if transplant is the route we choose to take. November 30, 2006 HLA typing was done on our family and preliminary results suggest that Avery is a match for Trey. This means that now a decision has to be made regarding related Bone Marrow Transplant or unrelated Cord Blood Transplant. Finding out that Avery is a match makes things more complicated because historically BMT’s didn’t work on kids with Hunter’s and currently it looks like CBT’s might be working for kids with Hunter’s. Are CBT’s working because transplant procedures have improved, and therefore BMT’s (which are no longer being done) would work also? Or are CBT’s working because CBT’s are superior to BMT for Hunter Syndrome? Or do none of them work and we just don’t know that yet? The schools of thought are divided, which is no help to us and very frustrating considering that transplant, BMT or CBT, is a painful and risky and costly procedure. November 20, 2006 Trey had a successful and uneventful tube surgery this morning with Dr. Moxham. Tubes were put in and a bunch of thick fluid was drained out. We were in and out of the hospital in under two hours and they didn’t even need to give Trey a General Anesthetic, just a little gas, since the procedure was so short (approx. 10 minutes). The only frustrating part was the Recovery Room. Trey has now been put under 3 times: first was an adenoidectomy and tube surgery, then an MRI, and today, a tube surgery. The first time, Trey woke up alone in the Recovery Room and by the time I got to him, he was so hysterical it took 20 minutes, with the help of a high dose of Morphine, to calm him down. After that we began to request being in the Recovery Room when he woke up, and although everyone we have talked to said they’d “do everything they could,” we have yet to be in the Recovery Room when Trey woke up. I’m not sure yet what we’ve got to do to make this happen...When it comes down to it though, we’re just happy to be home, have the procedure done, and be one step closer to making decisions about transplant. November 15, 2006 Trey’s tube surgery has been moved up from November 29 until this Monday, November 20, thanks to our awesome and wonderful Dr. Moxham. We explained to him that the sooner we can get his tubes put in, the sooner we can get another assessment at Duke and the sooner we can make a decision regarding transplant. He was happy to help out and we are relieved that we can get Trey’s surgery done so soon. I never thought that I’d be excited for my kid to get surgery, but here we are. November 10, 2006 I had a meeting with Bob Nakagawa today. THE BC GOVERNMENT HAS AGREED TO FUND TREY’S ERT!!!!!!!! Our government has come through for us. The catch is that the transplant team at Duke has cautioned us against doing ERT before a possible transplant. Nothing has been proven for or against ERT pre-transplant, but theoretically, Trey’s body could have an immunological response to the ERT and cause his body to reject the enzyme in the Cord Blood. So until we make a decision regarding transplant or ERT, we cannot begin ERT. PS. The picture at left is of Trey asleep on the airplane home in his new North Carolina jersey. Ever since he got it, he has refused to take it off. November 3, 2006 Our Cord Blood Transplant Assessment at Duke is done. We have decided to wait 3 monthes before making any decisions regarding transplant. In 3 monthes we will return to Duke for another assessment and appointment with Dr. Szabolcs to make a final decision. In the mean time, we will get a new set of tubes put in Trey’s ears, talk to our geneticist and the transplant doctor at BCCH about transplant, and figure out how the transplant would be funded. For details see my blog. October 30, 2006 Assistant Deputy Minister of Health, Bob Nakagawa, did not give us an answer today regarding the funding of Trey’s ERT through the BC government. We are very disappointed. He had a meeting with Deputy Minister of Health, Gordon Macatee, last Thursday, who asked that Bob give him more information before he makes a decision. From my understanding of our conversation, it sounds like because so many Enzyme Replacement Therapies are becoming available for enzyme deficiency diseases like MPS, they want to be very careful about the decision they make regarding Trey because they don’t want to set any precedents they can’t follow. The issue, Bob said, is financial. He said that they want to find a solution that is national, not just provincial or for Trey. His answer for why this had not been done already, considering all other developed countries in the world have a solution, was: “It’s complicated.” He has agreed to let me know by tomorrow afternoon when his next meeting with Gordon Macatee is. October 27, 2006 MSP has told us that they do not pay for any Stem Cell Transplants (Cord Blood Transplants fit into that category) because they are still experimental. Now we’re onto the search of finding out who pays for these treatments since we know they have been done in BC. October 23, 2006 There is a possibility Trey could have a CBT done here at BC Children’s Hospital (BCCH), if that is the route we choose to take. It is all still just talk, but if it is safe enough and everyone is willing to work together to do this for Trey up here, it could save us a lot of stress. The transplant would be paid for, we have the support of being at home, Ryan can work if he has to, we would be able to come home a lot sooner…the list goes on. It’s not for sure and we won’t know anything until we go to Duke and also talk with the transplant doctor up here, but it’s got us interested. October 2, 2006 We are considering a cord blood transplant for Trey, so here is the low down on the procedure. A Cord Blood Transplant means that Trey would receive a transfusion of Cord Blood (similar to the way a blood transfusion is given) in order to provide him with the missing enzyme. Until now, we have only been working towards Enzyme Replacement Therapy (ERT) - not yet available in Canada. But ERT does not prevent Trey’s brain from being affected. Here’s the catch – we don’t know if Trey’s brain is being affected or if it will be affected down the road, but CBT must be done at a very early age before Hunter’s has progressed, otherwise the risk it too high. We will be having two weeks of intensive testing done at Duke (www.cancer.duke.edu/PDMT/) to determine whether or not Trey is eligible. In order to be a candidate, Trey needs to be healthy and the MPS needs to be not well progressed. Also, they will likely try to predict whether or not Trey’s brain is going to be affected in time. If not, ERT is a much lower risk treatment and equally effective. The cost of the testing is close to US $24,000. If Trey does receive CBT, he would first receive chemotherapy treatment to wipe out his immune system. He would then receive CB transfusions, which would provide him with the enzyme. This would involve two months in hospital. Graft vs Host Disease (GVHD), which refers to the chance of the CB not grafting and is potentially terminal, has the highest chance of occurring during this time. After 2 months, we would move into an apartment on campus and stay for another 4 months. Up to 1 year post-transplant is still a risky time, although. I don't know how risky or if the risk lasts longer than this. The cost of the treatment is approximately US $640,000. For more information about CBT , check out: http://sciam.com/print_version.cfm?articleID=00087759-141E-1CC7-B4A8809EC588EEDF or http://www.bmtinfonet.org/newsletters/issue33/cord.html September 25, 2006 Bob Nakagawa, the Assistant Deputy Minister of Health, had a meeting with our doctors, Dr. Clarke and Dr. Stockler, to discuss Trey and funding ERT. I had a meeting with Dr. Nakagawa this morning to discuss the results. Long story short, Dr. Nakagawa will hopefully have an answer for us the next time we talk, which is on October 25, regarding whether or not the Ministry will pay for Trey’s ERT. Our fingers are crossed. September 21, 2006 BC Children’s Hospital has announced that they want to find solution for funding for ERT for Trey. We don’t know what this means exactly - we’ll keep you posted. For now, it means hope. September 10, 2006 BC Children’s Hospital has declined the request to fund ERT for Trey. They argue that there is not enough evidence that Elaprase (the ERT for Trey) will work, and that evidence is especially lacking for children under 5. Well naturally, there haven’t been any trials on children under 5. That’s the thing with a new treatment and a very rare condition! But since MPS is progressive, and the FDA found enough evidence that it works to approve Elaprase (for all ages), it would follow suit that we should start treatment for children with MPS II as young as possible. Kirsten Harkins, the Executive Director of the Canadian MPS Society and a mother of a child with MPS, has explained that this is the problem with not having an Orphan Drug Policy. Everyone will cite some variation of this response as a reason to not pay for ERT. So now it’s onto the government for funding. Dr. Lorne Clarke and Dr. Sylvia Stockler have a meeting with Bob Nakagawa, Deputy Minister of Health, on Friday to discuss funding and Dr. Clarke says we can expect an answer in 3-5 weeks. So for now, more waiting. At least we’re one down and onto the next. August 28, 2006 Over $60 thousand was raised at the annual MPS Cup Celebrity Hockey Tournament. These funds will be allocated to The Canadian Society for Mucopolysaccharide & Related Diseases, Inc. (MPS) to fund research initiatives for MPS. Trey watched his dad, and three family friends, play in the game with a roster of NHL stars. Our cheering section took an entire bleacher - it was a true moment of unity and support. Thanks to all who attended. Click here to link to the MPS Cup website (http://www.mpscup.ca/) July 24, 2006 Elaprase, the drug that Trey needs has been approved by the FDA. Now the battle really begins to get Trey the treatment here in Canada. Because Health Canada has not approved Elaprase, and Canada does not have an Orphan Drug Policy (a policy regarding treatments that are needed by very very few people), this will be an uphill battle. Our doctor, Dr. Sylvia Stockler, has applied to the government for Special Access for Trey. We should hear about this in the coming weeks. If this is granted, it becomes a matter of finding someone to fund the treatment - which will run in the hundred of thousands per year. Funding could be provided by the hospital, the pharmaceutical company or the government. Once this is in place, Trey will have access to Enzyme Replacement Therapy. Click here to read the press Shire Pharmaceutical press release, Or here to learn about Elaprase (http://www.elaprase.com/) http://www.canada.com/northshorenews/news/sundayfocus/story.html?id=7725c2a5-0a7e-4cad-99dc-91516a126569Events%20%26%20Donations.htmlhttp://www.theglobeandmail.com/servlet/story/LAC.20080414.ORPHAN14/TPStory/TPNational/Ontario/http://www.bobsandlolo.comEvents%20%26%20Donations.htmlHome.htmlEvents%20%26%20Donations.htmlNews Archive_files/RDD%20Feb%2029%20Press.docBlog/Blog.htmlBlog/Blog.htmlBlog/Blog.htmlEvents%20%26%20Donations.htmlhttp://www.cadth.ca/media/cdr/complete/cdr_complete_Elaprase_Dec-19-2007.pdfNews Archive_files/AI%20-%20Rare%20Disorders-1.pdfNews Archive_files/Wraith%20et%20al%202007%20Hunter%20Syndrome%20Review%20and%20Treatment%20Recommendations%20Eur%20J%20Peds.pdfhttp://www.raredisorders.caEvents%20%26%20Donations.htmlBlog/Blog.htmlBlog/Blog.htmlNews Archive_files/Medical%20Post%20Article%20-%20Entering%20the%20Enzyme%20Era%20%28MPS%20II%29.doc.pdfMedia.htmlBlog/Blog.htmlBlog/Blog.htmlhttp://cgi.ebay.com/ws/eBayISAPI.dll?ViewItem&item=290160335842&ssPageName=ADME:B:SS:US:1Blog/Blog.htmlBlog/Blog.htmlBlog/Blog.htmlMedia.htmlBlog/Blog.htmlMedia.htmlBlog/Blog.htmlBlog/Blog.htmlMedia.htmlMedia.htmlBlog/Blog.htmlBlog/Blog.htmlhttp://www.mpscup.cahttp://www.azcentral.com/arizonarepublic/sports/articles/0513youngcp0513.htmlBlog/Blog.htmlBlog/Blog.htmlBlog/Blog.htmlhttp://www.rogercollier.com/clipping.feature.20.citizen.observer.orphan.drugs.htmlMedia.htmlBlog/Blog.htmlBlog/Blog.htmlmailto:dcehak@telus.netEvents%20%26%20Donations.htmlNews Archive_files/17%20NSN%20SUN%20FEB%2018_07.pdfNews Archive_files/18%20NSN%20SUN%20FEB%2018_07.pdfNews Archive_files/19%20NSN%20SUN%20FEB%2018_07.pdfhttp://www.canada.com/vancouversun/news/story.html?id=762964f0-c5e1-4767-81ac-b55451fcdb06&k=26625Media.htmlMedia.htmlPictures.htmlPictures.htmlhttp://www.bcchildrens.caPictures.htmlhttp://www.cancer.duke.edu/PDMT/http://sciam.com/print_version.cfm?articleID=00087759-141E-1CC7-B4A8809EC588EEDFhttp://www.bmtinfonet.org/newsletters/issue33/cord.htmlPictures.htmlhttp://www.mpscup.ca/http://www.mpscup.ca/News Archive_files/Elaprase_Press_Release_APPROVED_240706-2.pdfhttp://www.elaprase.comhttp://www.elaprase.comshapeimage_2_link_0shapeimage_2_link_1shapeimage_2_link_2shapeimage_2_link_3shapeimage_2_link_4shapeimage_2_link_5shapeimage_2_link_6shapeimage_2_link_7shapeimage_2_link_8shapeimage_2_link_9shapeimage_2_link_10shapeimage_2_link_11shapeimage_2_link_12shapeimage_2_link_13shapeimage_2_link_14shapeimage_2_link_15shapeimage_2_link_16shapeimage_2_link_17shapeimage_2_link_18shapeimage_2_link_19shapeimage_2_link_20shapeimage_2_link_21shapeimage_2_link_22shapeimage_2_link_23shapeimage_2_link_24shapeimage_2_link_25shapeimage_2_link_26shapeimage_2_link_27shapeimage_2_link_28shapeimage_2_link_29shapeimage_2_link_30shapeimage_2_link_31shapeimage_2_link_32shapeimage_2_link_33shapeimage_2_link_34shapeimage_2_link_35shapeimage_2_link_36shapeimage_2_link_37shapeimage_2_link_38shapeimage_2_link_39shapeimage_2_link_40shapeimage_2_link_41shapeimage_2_link_42shapeimage_2_link_43shapeimage_2_link_44shapeimage_2_link_45shapeimage_2_link_46shapeimage_2_link_47shapeimage_2_link_48shapeimage_2_link_49shapeimage_2_link_50shapeimage_2_link_51shapeimage_2_link_52shapeimage_2_link_55shapeimage_2_link_57shapeimage_2_link_58shapeimage_2_link_59shapeimage_2_link_61shapeimage_2_link_62shapeimage_2_link_63shapeimage_2_link_64shapeimage_2_link_65
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