MPS II

18 Feb 2016
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LOCAL CHARITY CELEBRATES 10 YEAR ANNIVERSARY

Sticky Post By On 18 February, 2016

THE ISAAC FOUNDATION CONTINUES TO MAKE SIGNIFICANT GAINS IN SEARCH FOR CURE FOR MPS DISEASES WORLDWIDE

On February 21, 2016, The Isaac Foundation will be celebrating its 10th anniversary. The Campbellford, Ontario charity was started to fund research aimed at finding a cure for MPS VI, a rare, progressive, and devastating disease that a local child Isaac McFadyen was diagnosed with in 2005. Since that time, The Isaac Foundation has donated close to $1 million to research projects, with $175,000 being donated in the last year alone. The charity has grown considerably and supports children and families throughout Canada and around the world as they cope with the new reality a diagnosis of MPS brings.

“We’re incredibly proud of the things we’ve been able to accomplish over the past decade,” said Andrew McFadyen, Executive Director of The Isaac Foundation. “Obviously, finding a cure is paramount for us and that drive and focus to help these children will never diminish. But we’re also lucky to have helped children across the country receive the life-saving treatments they desperately need while we search for that cure.”

The Isaac Foundation has funded research projects located in Australia, the United States, and Italy, projects that have led to advances in the treatment and outlook of MPS. “We’ve had some hits and some misses over the years, but we’ve been fortunate to play a role in two exciting projects going to clinical trial. That in itself is incredible, but the fact that these projects will have a direct impact on improving the quality of life for patients suffering from MPS is still unbelievable to us. If you asked us 10 years ago if we thought this is where our organization would be, I don’t think we could have hoped for much more.”

One of the projects The Isaac Foundation helped start is a gene therapy approach to treating MPS VI. The project goes into human clinical trials in 2016 and could prove to be a cure for MPS VI, the form of the disease that young Isaac McFadyen suffers from. “We’ve been very careful not to throw around the word ‘cure’ with respect to this research, but we firmly believe that it will prove to be the answer we’ve been looking for all of these years. We’re lucky to have been involved since the very beginning, and we’ve been directing most of our research funds to this project for some time now. If the trials run as well as we expect it will, it won’t be long until we see the hard work behind this research come to market for our kids and patients. It’s incredibly exciting.”

The organization also has been administering a fund for MPS II research for the past number of years and has 3 gene therapy projects for MPS II underway. Last month, the fund provided another $62,000 grant to one of those gene therapy projects, research that looks very promising and is moving at a rapid pace.

“When we started The Isaac Foundation, we wanted to ensure that money that came into our organization was turned around quickly and sent back out to researchers as soon as possible. We also wanted to ensure we were responsible with the funds people donated and we’re proud to note that almost 99% of all money donated goes directly to our charitable program aimed at finding a cure for MPS. Last year, we hit the 100% mark and we expect to do the same this year. There aren’t many charities in this country that can meet those goals it’s means a lot to us that we can.”

With respect to advocacy and patient support, the organization has helped pave the way for patients across Canada to receive life-prolonging enzyme replacement treatments as the search for a cure continues. “These are incredibly expensive treatments, sometimes upwards of $1 million dollars per year, per patient. But the impact treatment has on the lives of patients is dramatic, and a price should never be placed on the life of a child. The help we’ve been able to provide these families suffering from MPS II, IVA, and VI is probably something that I’m most proud of in my life.”

The organization has garnered the attention and support of many big names throughout its 10-year history, from International music star John Mayer, to retired MLB star Roy Halladay, to Canadian icons The Tragically Hip, Ron Sexsmith, Sarah Harmer, and Danny Michel.

To celebrate their anniversary, Canadian graphic artist Andrew Kolb (www.kolbisneat.com) designed artwork that sought to capture the essence of The Isaac Foundation in one piece. The work features a smiling sun rising over a map tracing a long and winding route. At the end of the route is a large ‘X’ and the tag line “Hope Is Here”. On the horizon, kids and adults rush toward each other to join hands. One of the kids is in a wheelchair.

“It’s beautiful,” says McFadyen. “It sums up perfectly what we’ve been about and where we are going. Hope definitely is here, and I’m glad we’ve arrived there together with all the families we’ve been fortunate to help along the way.”

# # #

The 10th Anniversary artwork can be viewed online at https://www.theisaacfoundation.com/anniversary.jpg

For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com.

10thAnniversaryPoster_Web

14 Jun 2014
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2014 Research Grant Award Recipients

Sticky Post By On 14 June, 2014

Research Logo

RESEARCH GRANTS ANNOUNCED!

We are thrilled to announce that The Isaac Foundation has granted $200,000 in research grants to 3 innovative projects aimed at finding a cure for MPS!

We are proud to award $100,000 to Dr. Alberto Auricchio in Italy for his incredibly exciting work on Gene Therapy. We hope to see this work head to clinical trials in early 2015!

We are also very excited to award 2 $50,000 research grants from the MPS II FUND, under the leadership of Deb Purcell! The recipients of these awards are granted to Dr. Scott McIvor at the University of Minnesota for his work entitled “AAV Mediated IDS Gene Transfer for MPS II” and to Dr. Brian Bigger at the University of Manchester for his work “Evaluating Stem Cell Gene Therapy for Treating the Brain in MPS II”

This brings the total awarded over the past 6 months to $250,000 – all in hopes of finding a cure for our children.We look forward to seeing results from this research and will continue our work raising money to support these projects and more during the course of the next few years.

Thank you for your incredible support. None of this can happen without you.

10 Apr 2014
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Update – Jack Fowler

Sticky Post By On 10 April, 2014

photo-3Hello Everyone,

As many of you have noted, we’ve been quiet of late on news of Jack Fowler.  While we haven’t stopped working on behalf of Jack – work that has been relentless, stressful, and heartbreaking, we went quiet publicly while we worked some back-channel routes to see if Shire would and could be forthcoming and interested in saving this little boys life.

Unfortunately, Shire has continued to rebuff our efforts and continues to put Jack’s life in jeopardy.  Jack faces a steep decline, and his parents are beside themselves with heartache and heartbreak.  It seems unbelievable to me that a drug exists that has the potential to save him, yet it’s being withheld due to reasons that the pharmaceutical company cannot really stand behind.

We will be redoubling our efforts of the coming days and weeks.  Please stay tuned on how you can help #SaveJack.

In the meantime, drop over and read this article.  It’s something that Shire should read as well.  It’s all well and good for the FDA to have a plan.  But if big-pharma doesn’t want anything to do with it, our kids will continue to die needlessly.

I’ll be in touch with you all soon.

With Love,

A.

21 Mar 2014
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Research Grants

Sticky Post By On 21 March, 2014

Research-LogoThe Isaac Foundation is proud to announce that our application for our 2014 Research Grants is now available.  $100,000 is available from our general MPS Research Fund, and another $50,000 grant is available from the MPS II Research Fund.

Application deadline is May 1.  A decision will be reached by June 15, with funds dispersed on or before June 31, 2014.

Our Grant Application Form Can Be Downloaded Here – RFA 2014 – MPS VI and MPS II

If you have any questions regarding the process, please email Ellen Buck-McFadyen – ellen@theisaacfoundation.com

 

05 Mar 2014
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2014 Research Grants

Sticky Post By On 5 March, 2014

research-grantsThe Isaac Foundation is pleased to announce two research grant opportunities with a call for a $100,000 grant through our general MPS Fund and a call for $50,000 from our MPS II Research Fund.

Last month also saw us renew a research project for $50,000, bringing our total research grants from The Isaac Foundation early 2014 to $200,000!

We continue to be proud of the work that we do in the MPS Community. To date, we are well over 1/2 million dollars granted to research projects throughout the world and we will continue to grow and support innovative research until a cure for MPS is found.

Calls for Applications to these grants will be issued soon.

We are also very pleased to announce that Dr. Barbara Burton and Dr. Paul Harmatz, both world-renowned MPS specialist from the United States, have joined our Medical Advisory panel to review and advise on research applications as they come in. Dr. Burton and Dr. Harmatz join Dr. Julian Raiman and Dr. Joe Clarke to round out our 2014 Medical Advisory Panel.

More details to follow soon. Thanks for you continued support as we seek to find a cure for our kids!

15 Feb 2014
Sticky Post By Posted in Advocacy, Jack Fowler Permalink

Family fights to get drug for dying 6-year-old son

Sticky Post By On 15 February, 2014

BY MONIFA THOMAS Staff Reporter February 15, 2014 1:26AM

CHICAGO SUN-TIMES

dt.common.streams.StreamServer.clsShould a dying child receive an unproven experimental drug even if the patient doesn’t fit within a carefully designed clinical trial?

More than 53,000 people who have signed an online petition on behalf of a Mundelein boy say yes, absolutely. But Shire, the Ireland-based pharmaceutical company that owns the drug, says it’s a complicated matter and has refused to make the drug available to the child.

At the heart of this moral and ethical dilemma is 6-year-old Jack Fowler. Jack has a rare disorder called Hunter syndrome, or MPS II, that is expected to kill him because he lacks the enzyme needed to break down cellular waste in his body. Ten to 20 years is the usual life expectancy of someone with the disease, but Jack’s more severe type means he may have less time.

An estimated 2,000 patients — nearly all boys — are affected by the disease worldwide.

The hope is that the drug in question, SHP-609, can for the first time, slow or halt the progression of the disease in the brain. It is going through clinical trials, which means it hasn’t been approved by the Food and Drug Administration yet. The Phase II/III stages are just starting, and FDA approval happens after Phase III.

So Jack’s parents, Jason and Jamie Fowler, have been trying to get a “compassionate use” approval for Jack, which the FDA allows on a case-by-case basis. Clearance for such use means Jack’s case would not influence outcomes in the clinical trial, so an adverse reaction by Jack, for example, would not count against the drug.

For two years, the Fowlers have pleaded with Shire to let their son be part of the clinical trial or get the drug through compassionate use, including during a brief meeting with the company’s CEO, Dr. Flemming Ornskov, in a Chicago airport hotel in January. An advocate for the Fowlers, Andrew McFadyen of The Isaac Foundation, a nonprofit group focused on treatments for rare diseases, started the meeting by stating, “An immoral decision remains immoral if delivered in ones face,” and said that if Shire was still choosing death over saving a life then the meeting was over, according to Jamie Fowler.

Shire “delivered the same grim news, so we upped and walked out,” Jamie Fowler said in an email.

Dr. Barbara K. Burton, Jack’s geneticist at Lurie Children’s Hospital of Chicago and one of the investigators for Shire’s clinical trial, said there’s no evidence one way or another that the drug will save Jack’s life. “It’s conceivable that it would hasten his death,” Burton said, but added, “What I do know is that without [any treatment beyond what he’s getting now], he is definitely going to die.”

For that reason, she supports the Fowlers’ position.

Shire said it has compassion for the Fowler family. Yet “we believe that expanding access to SHP-609 beyond the clinical trial can put the overall development at risk and delay or eliminate the opportunity to make a safe, approved treatment more widely available to the global Hunter syndrome community,” Ornskov said.

An FDA spokeswoman said that of the 940 submitted requests for expanded access for such investigational drugs between October 2011 and September 2012, all but four cases were allowed to proceed.

Unknown adverse events usually do not prevent a compassionate use request from being granted, but that is determined on each case, the FDA added. The agency could not comment specifically on Jack’s case.

Dr. Michael Caplan, pediatrics department chairman at NorthShore University HealthSystem, who is knowledgeable about clinical trials, also did not speak about Jack’s situation, but he said experimental drugs usually aren’t given to patients outside clinical trials until Phase III is complete. Safety is the issue, Caplan said.

Shire didn’t say for sure whether that might be a possibility for Jack after the Phase III is done, but a spokeswoman noted that Shire has done that in the past.

Lewis Smith, an associate vice president of research and a medicine professor at Northwestern University Feinberg School of Medicine, said, “these are moral, ethical dilemmas.”

Companies typically are very concerned about allowing people who are not in the clinical trial access to the drug, because “they’re very concerned about whether that would muddy their results, because this is a rare disease,” Smith said.

Yet, it’s an emotional issue, especially when a child is involved, Smith said.

That’s clear when Jamie Fowler talks about how Jack’s cognitive deterioration, which came with the disease, is slowly taking away his personality. Gone is his ability to say “sis” for his sister, Juliet. Fowler dreads the same fate for “mom.” “It’s heartbreaking,” Fowler said, crying.

They’ve turned to social media, such as Facebook, to try to get Shire to reconsider. An online petition hopes to hit 75,000 signatures by the end of the month; there are more than 50,000 names so far. The family also started doing media interviews to get the word out about their case.

Nothing has worked so far, but the Fowlers said they aren’t stopping until Jack gets the drug to see if it can save his life.

“We won’t sit by and watch our son die,” Jamie said.

Contributing: Chris Fusco

Email: mjthomas@suntimes.com

Twitter: @MonifaThomas1

13 Feb 2014
Sticky Post By Posted in Jack Fowler Permalink

Jack Fowler – Update

Sticky Post By On 13 February, 2014

image-27Hi Everyone,

I thought I would take a moment to update you on our latest attempt, our latest plea, to Shire Pharmaceuticals to open communication and connect with us about Jack Fowler, Expanded Access, and doing what is right for the Hunter Community as a whole.

Yesterday evening, I sent Shire CEO Flemming Ornskov and Head of R and D Phil Vickers an invitation to participate in a teleconference with Jack’s physician (and Internationally renowned MPS expert) Dr. Barbara Burton, and with Dr. Emil Kakkis, an expert in rare diseases who has a wealth of experience in the pharmaceutical world and with expanded access requests to save children’s lives.  The purpose of the call was to ensure that all parties are on the same page moving forward, with the hope that a collaborative discussion can take place to best meet the needs of everyone involved.

I have not had a response from Shire, though I remain hopeful that they will agree to participate.  At some point, someone at that company needs to look at this situation with a different lens.  At some point, someone at Shire will have to look back at their company credo and work to “Be As Brave As The People” they treat.  I’m hoping that some point is soon.

I’ll keep you updated as things progress.  In the meantime, I’ve been busy speaking with numerous media outlets and scheduling in radio appearances.  I can confirm that Jamie and Jason Fowler, Jack’s parents, and The Isaac Foundation will appear on a Boston Call-In Show in late February, from 9 – 10 pm.  This show is carried over 38 states, so chances are we’ll be on the radio somewhere close to you.

We know that our case to #SaveJack is solid.  We have everything in place to save this little boy’s life.  We only need Shire to sponsor our effort, something we all know they should do.

With Thanks,

A.

31 Jan 2014
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Boston Globe – A Few Notes

Sticky Post By On 31 January, 2014

photo-3Hi Everyone,
Jack Fowler’s case was presented in the Boston Globe today. And while it’s nice to get some exposure, I truly feel the editor at the Globe should be held accountable for the biased and single-sided view the article portrayed.

Nowhere in the article was the FDA’s position put forward – a position we have been open and transparent about presenting since day 1. That position is simple – access to investigational medicines outside of the clinical trial setting have NO BEARING on clinical trial results. In fact, there are cases where expanded use have helped to speed up the process. Indeed, the FDA guidelines say the following:

“There are examples in which FDA has made use of adverse events information from expanded access use in the safety assessment of a drug. There are a small number of cases in which an important adverse event was first identified during expanded access use and those adverse events were in clouded in product labelling. This is not a negative from a public health perspective – the sooner important adverse events are identified the better. Even from the sponsor’s viewpoint, early discovery of a rare adverse event is, on the whole, a benefit. Although adverse events first identified during expanded use of a certain drugs have been included in the drugs’ approval product labelling, we are unaware of any cases in which adverse event information obtained from expanded access use has resulted in denial of approval for a product.”

As I have previously noted, the FDA has been in contact with The Isaac Foundation regarding expanded access, and I have assurances that any adverse events that happen outside of the clinical trial setting will not hinder the clinical trial for any reason.  Why the Boston Globe editor did not see fit to ensure that information was presented is beyond me, is unfortunate, and leaves an incomplete picture of the story as a whole.

In addition, no quotes were sought from families who have children currently participating in the trial.  The trial is currently in Phase II/III and has lasted over 4 years.  Families are reporting incredible results in their children – their lives have been saved.  The treatment works, and the reversal of devastating symptoms these children have been experience is proof positive. Moreover, the treatment is safe!  Over 4 years of clinical trial has seen NO ADVERSE EVENTS REPORTED.  None.  Zero.  A few words from families who have experience dealing with this treatment would have shed light on the trial, the effectiveness of the treatment, and the safety of administering that treatment to our kids.

Finally, with all due respect to Dr. Muenzer, I have to  object to his comment that “compassionate use will destroy a trial.”  That statement flies in the face of all evidence the FDA has provided us, and flies in the face of the entire purpose of compassionate use for our kids.  Expanded use guidelines were clarified by the FDA a few short years ago, and they were clarified so that physicians and pharmaceutical companies alike could fully understand that compassionate use does not affect trials.  Their guidelines ensure that expanded access is provided as a treatment option for patients, not a data collection option for principal investigators or pharmaceutical companies.  In short – any expanded use will not impact the trial.  Not by any stretch.  And to further this point, I emailed Janet Woodcock at the FDA for a direct statement on the matter.  She promptly replied and said “…in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs.  In one case, we know the drug development was actually accelerated.”  One would have expected this information would be available to anyone who asked, including Shire Pharmaceuticals.

It is beyond me why the Boston Globe would neglect to present these viewpoints in their story.  Journalism is supposed to present an objective viewpoint on a trending topic.  It’s purview is to educate the public and allow for open discussion.  For that discussion to take place – for any fair and open discussion to take place – all the viewpoints need to be laid out.  Sadly, important information was precluded from the story, and I hope this entry helps clarify the matter once again.

Finally – Phil Vickers – never in this world would it be “ethically wrong” to try to save a child’s life.  EVER.

On another note, I emailed Shire CEO Flemming Ornskov yesterday to ask, once again, for a collaborative solution to this matter.  I am hopeful that Flemming and his team will agree to sit down directly with the FDA, Dr. Muenzer, and Dr. Burton to work through this matter, set a proper policy and guidelines for access to this investigational drug, and work to do what is best for our children.  ALL of our children.

With thanks for your continued support,

The Isaac Foundation

18 Jan 2014
Sticky Post By Posted in Jack Fowler, Uncategorized Permalink

Meeting With Shire – Update

Sticky Post By On 18 January, 2014

flemmingHi Everyone,

It’s with a heavy heart, and with anger and frustration, that I update you on the meeting that took place this morning between the Fowler family, The Isaac Foundation, and Shire Pharmaceuticals.  The meeting was organized after Shire’s decision to deny Jack Fowler the life saving treatment he immediately requires.

This meeting was supposed to take place at the Fowler home, 45 minutes outside of the city of Chicago.  It was set to take place at 1:30 p.m.  However, a few days before the meeting, Shire’s CEO Flemming Ornskov abruptly changed the location of the meeting to the Chicago airport, and changed the time to an unseemly 7:30 am.  The Fowler family struggled to put child care in place.  And when you are dealing with a special-needs child, that is no easy task.

We made it to the meeting on time, and brought Jack in with us to meet with the Shire team.  Present members for Shire were CEO Flemming Ornskov and Head of Research and Development, Phil Vickers.

The Isaac Foundation began the meeting by thanking Shire for taking the time to meet with the Fowler family.  We expressed that the purpose of the meeting from our point of view was to discover how we could work collaboratively with Shire Pharmaceuticals so that we can find the best treatment options for Jack Fowler and in a timely fashion.  We expressed that if Shire was present to simply reiterate their position from December and deny Jack the treatment he needs, then the meeting would need to come to an abrupt end.  I made very clear to Mr. Ornskov one very simple fact – an immoral decision is still immoral, even if it’s delivered while looking us in the eye and said to our face.

Flemming looked at us and said “We are not changing our decision.  I guess this meeting is over.”  With that, the hopes of the Fowler family were dashed, and our hope to work together with Shire to save this little boy was ended.   We left a large print out of the 32,000 signatures that were signed in the online petition, as well as letters of support from a high percentage of the families currently participating in the clinical trial of the drug that Jack desperately needs.

As we were leaving, Jack walked around the table and gave Flemming a hug.  Close to tears, Jack’s mom said “If he could talk, he would be asking you to Be Brave, like your motto says, and save him.”  It was one of the most heartbreaking moments of my life – watching a sweet little boy who doesn’t have any idea what is going on give a hug to the man who just gave him a certain death sentence.  I told Flemming that I had a hard time understanding how he can go home and look his children in the face after that moment.  He just looked at us and said “It was nice to meet the family.”

The facts of this case remain the same, and are very clear.  Jack Fowler needs access to a drug that will save his life.  He easily qualifies for individual use access through the FDA’s Expanded Use guidelines.  It is those guidelines that decide whether any investigational drug is safe for use outside the bounds of a clinical trial.  The question of whether enough safety data exists to proceed or not doesn’t rest with Shire Pharmaceuticals, nor does it rest with any pharmaceutical undergoing the same process.  It rests with the FDA first and foremost, and it rests with the physician in charge of treating the patient.  All Shire has to do is begin the application process on Jack’s behalf and leave the decision to the FDA.  When I stated this very clearly to Mr. Ornskov and asked if he would submit the application, he flatly refused.

There are things in this world that many people would be better off not knowing.  What lurks in the minds of pharmaceutical decision makers should be at the top of everyone’s list.  To have the ability to provide help and support, to be able to save the life of a precious little boy, and then choose not to, shows a callous disregard toward life.  That callous disregard is a painful reminder of what Big Pharma is after – money, product, fortune, and fame.  Don’t ever be fooled that the patient comes first.  As Flemming so plainly stated before I gave my introduction – “We don’t work with patients.  We don’t work with families.  We are in the business of developing product.”

With that, there’s nothing left to be said.

Our press release goes out early this week.  Stay tuned on how you can help us #SaveJack.  We will never quit when the life of a child hangs in the balance.

Thank you for your ongoing and tremendous support.

With Love,

The Isaac Foundation