Advocacy

11 Apr 2013
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JnJ Update

Sticky Post By On 11 April, 2013

johnson_and_johnson_noUpdate – JNJ

Hi Everyone,
I just wanted to drop in to provide a very quick update about recent progress made with JnJ and their commitment to do a full and proper study of Elmiron and it’s uses within the MPS Community. I will provide a broader update as soon as possible, but wanted to give a briefing while I had the chance.

Representatives of The Isaac Foundation met with Dr. Amrit Ray, Johnson and Johnson’s Chief Medical Officer, this afternoon in Toronto. Dr. Ray’s commitment to seeing this project move forward was very refreshing, and we trust him to ensure the speedy movement of this file moving forward.

In the coming weeks, the medical advisory panel assembled by the JnJ brass will gather to look at the data currently available and plot possible paths forward for the full study this drug needs as we seek to provide the best treatment options available for our children suffering from MPS.

While we are heartened with the level of dedication JnJ seems to have regarding Elmiron and MPS, The Isaac Foundation will continue to tirelessly advocate for the advancement of this process throughout it’s various stages and be on hand to ensure a thorough study is done for our kids. We know you will be with us along the way, ready to fight for what we all believe in. Our kids deserve the best treatment modern science can provide, and it’s our job to help them win the battles they are fighting.

Thank you for your continued support.

With Love,
The Isaac Foundation

14 Mar 2013
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Drug Company’s Decision Gives Trent Hills Father Hope

Sticky Post By On 14 March, 2013

Experts will review research data on rare disease

Northumberland NewsTRENT HILLS – Months of lobbying capped by an intense advocacy campaign conducted online have paid off for Andrew McFadyen: Johnson and Johnson has agreed to form a medical advisory panel to determine whether one of its medications could help his son, Isaac, and others who suffer from a rare disorder.

“That’s incredible news for our families and our kids,” Mr. McFadyen said. “We’re pretty pleased.”

The elementary school teacher has been relentless in trying to persuade the pharmaceutical company to undertake a clinical trial of an anti-inflammatory drug it makes to treat a bladder condition.

Research involving rats funded by Mr. McFadyen’s Isaac Foundation indicates the medication could also benefit people with MPS, a group of disorders caused by an enzyme deficiency. Its symptoms vary but in the case of his eight-year-old son, MPS IV has stunted his growth, restricted his mobility and affected his breathing.

Mr. McFadyen received word of Johnson and Johnson’s decision from the company’s chief medical officer for its pharmaceuticals division, Dr. Amrit Ray, last Saturday. They talked several times over the phone and are to meet in person in a couple of weeks.

Isaac “was pretty excited to hear the news,” he said.

Mr. McFadyen said “almost every MPS specialist in the world … the who’s who” of the profession, will sit on the panel. They will review the research data and recommend if a clinical trial is warranted.

He’s “very confident” they will but should they decide against it, “that’s also in the best interests of our kids. I would never want to move forward with something that’s unsafe or not going to help.”

Mr. McFadyen said a public advocacy campaign he mounted recently resulted in “hundreds and hundreds of people sending e-mails” to Johnson and Johnson in support of his efforts.

14 Mar 2013
Sticky Post By Posted in Advocacy, Media, Newspaper Permalink

Drug Company's Decision Gives Trent Hills Father Hope

Sticky Post By On 14 March, 2013

Experts will review research data on rare disease

Northumberland NewsTRENT HILLS – Months of lobbying capped by an intense advocacy campaign conducted online have paid off for Andrew McFadyen: Johnson and Johnson has agreed to form a medical advisory panel to determine whether one of its medications could help his son, Isaac, and others who suffer from a rare disorder.

“That’s incredible news for our families and our kids,” Mr. McFadyen said. “We’re pretty pleased.”

The elementary school teacher has been relentless in trying to persuade the pharmaceutical company to undertake a clinical trial of an anti-inflammatory drug it makes to treat a bladder condition.

Research involving rats funded by Mr. McFadyen’s Isaac Foundation indicates the medication could also benefit people with MPS, a group of disorders caused by an enzyme deficiency. Its symptoms vary but in the case of his eight-year-old son, MPS IV has stunted his growth, restricted his mobility and affected his breathing.

Mr. McFadyen received word of Johnson and Johnson’s decision from the company’s chief medical officer for its pharmaceuticals division, Dr. Amrit Ray, last Saturday. They talked several times over the phone and are to meet in person in a couple of weeks.

Isaac “was pretty excited to hear the news,” he said.

Mr. McFadyen said “almost every MPS specialist in the world … the who’s who” of the profession, will sit on the panel. They will review the research data and recommend if a clinical trial is warranted.

He’s “very confident” they will but should they decide against it, “that’s also in the best interests of our kids. I would never want to move forward with something that’s unsafe or not going to help.”

Mr. McFadyen said a public advocacy campaign he mounted recently resulted in “hundreds and hundreds of people sending e-mails” to Johnson and Johnson in support of his efforts.

08 Mar 2013
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Virtual Zeal Rare Disease Caregivers Carry A Powerful Voice In Social Media

Sticky Post By On 8 March, 2013
Globe and Mail Photo

 

isaac10lf4-2Figure 2: Isaac McFadyen (front) who suffers from MPS VI, flanked by his family including his father and advocate Andrew McFadyen (right). Image courtesy of The ISAAC Foundation.

According to Mr. McFadyen, his efforts were rebuffed several times until finally in February 2012, he began a social media campaign, including a website not so subtly titled “ShameonJNJ.com”, Twitter and e-mail barrage, and Facebook community to encourage Janssen / Johnson &, Johnson to reconsider its decision. The campaign culminated around February 28, Rare Disease Day, with several Janssen executives receiving hundreds of e-mails from impassioned parents and supporters of MPS families.

 

This article highlights how rare disease patients and caregivers can harness the power of social media to support or challenge pharmaceutical companies. The equivalent of a modern day megaphone, telephone, and printing press combined, social media showcases its power in no more passionate…

Melissa Hogan – SavingCase

This article explores the voice of rare disease patients and caregivers in social media and the power they wield to support or challenge the pharmaceutical companies that serve them.

The equivalent of a modern day megaphone, telephone, and printing press combined, social media showcases its power in no more passionate an arena than in rare disease.

While some think of Facebook as a fun pastime, in private rooms or sometimes publicly, rare disease sufferers and their caregivers are offering diagnoses and treatment advice, discussing side effects, and advising on every other aspect of life with a chronic or rare condition. While some follow celebrities on Twitter, those affected by rare disease might use Twitter to make sure their views are heard by pharma and their governments alike.

On the heels of Rare Disease Day, you’d be remiss not to know that rare disease advocates are often proud of their status as ‘zebras’. “If you hear hoofbeats, think horses, not zebras” is the oft-quoted tenet of medical diagnosis, the assumption being that often the simplest explanation, rather than a rare or exotic disease, is usually the best.

Zeal is not just a pack of zebras

It is that perspective that has made rare disease groups stand up and proudly claim the title of zebras, often displaying stuffed zebras at their events. But when zebras become a pack, as they often do on social media, they become a zeal, a title that is not only categorically appropriate, but descriptively appropriate as well.

In addition to describing a pack of zebras, zeal is defined as “great energy or enthusiasm in pursuit of a cause or an objective.” More than almost any other online group, those affected by rare disease approach their cause of support, education, medical care, and advocacy with great energy and enthusiasm. In describing them and others like them as the “alpha geeks” of health care, internet geologist Susannah Fox notes: “They are in the crucible. They ‘roll their own’ by creating communities of health information exchange where none had existed.”

“On the heels of Rare Disease Day, you’d be remiss not to know that rare disease advocates are often proud of their status as ‘zebras’.”

Feeling the zeal

Not only can rare disease patients utilize social media in their diagnosis, support, and treatment efforts within their population, one should be cognizant of both the benefits patients can confer in raising awareness and getting expensive treatments reimbursed, but also the damage they can inflict via the democratizing medium of social media. An example of both can be found in the efforts of Canadian-based The ISAAC Foundation and its founder, Andrew McFadyen, a parent of a child with the rare disease Mucopolysaccharidosis (MPS) VI.

To date, Mr. McFadyen’s social media efforts have succeeded in obtaining reimbursement of high priced enzyme replacement therapies for several patients, including his own son Isaac, by their Canadian provinces after the provinces first declined or delayed reimbursement. While pharma companies are surely lobbying for reimbursement of their treatments, sometimes only the efforts of the patients they serve are viewed with collective sympathy.

In 2012, for example, the family of a young girl with MPS VI, Violet Revet, had been awaiting for approximately six months for word on reimbursement for the drug Naglazyme® by the Saskatchewan, Canada health ministry. Without an answer, and watching the disease progress in their daughter, the parents went to Mr. McFadyen for help. A Twitter campaign caught the attention of Premier Brad Wall who responded and the treatment was approved within days. The effect was as clear as the Premier’s statement: Twitter “democratizes things.”

“While pharma companies are surely lobbying for reimbursement of their treatments, sometimes only the efforts of the patients they serve are viewed with collective sympathy.”

Amplifying the voice of reason

While pharma can benefit from the efforts of patient advocates, they can be the target of such efforts as well, such as Mr. McFadyen’s latest endeavor, which I was able to view from the inside out.

Over the last several years, scientists began studying the use of a Janssen FDA-approved drug for interstitial cystitis called Elmiron® for the bone and joint problems that plague children with MPS. The ISAAC Foundation was one financial supporter of those studies. Data from small animal studies were completed in 2012 and presented at several conferences. This precipitated Mr. McFadyen’s conversations with Janssen itself to explore the research further and possibly support human trials.

“…it is clear that their behaviors and efforts cannot be ignored.”

On March 1, Johnson &, Johnson changed its Twitter handles that had been receiving some of the barrage, from @JNJComm and @JNJStories to the new @JNJCares, @JNJParents, and @JNJNews. Janssen also quickly began organizing a medical advisory board to both bring Janssen up to speed on a disease with which it was unfamiliar (Mucopolysaccharidosis) and to consider the scientific evidence and what next steps to take.

Some might disagree with the public pressure of social media tactics like those employed by Mr. McFadyen and his supporters, calling it public bullying. When asked what he thinks of those who might say that using such tactics make him a bully, Mr. McFadyen replied:

This is not being a bully. I’m just one man with a firm belief in the rights of those with rare disease to have treatments just like those with cancer or heart disease. One might instead call a large pharmaceutical company or a government a bully when they make decisions without considering who they affect. With social media, we simply help amplify the voice of reason.

Whether studying, interacting, advertising to, benefitting from, or even suffering from the behaviors of rare disease sufferers on social media, it is clear that their behaviors and efforts cannot be ignored.

Related articles

Angry Parents Force J&,J To Do Damage Control – pharmalot

When Rare Just Isn’t Enough: The Case of Elmiron – SavingCase.com

References

1. http://susannahfox.com/2011/07/06/alpha-geeks-in-health-care/.

2. http://www.newstalk980.com/story/saskatchewan-girl-waiting-help-rare-disease/67994

3. http://www2.canada.com/saskatoonstarphoenix/news/story.html?id=6e79808f-1adf-45de-92ba-589a8517e2d1

About the author:

Melissa Hogan is a lawyer and strategic consultant by profession, but since her youngest son Case’s diagnosis with MPS II in 2009, she has also applied her experience to become an advocate, author, and speaker on behalf of rare disease families. She writes about advocacy, medical research, pharma, clinical trials, therapies, social media and special education on SavingCase.com, a blog that is now read in over 100 countries. She also uses other social media strategies such as Twitter, Facebook, Pinterest, YouTube, and LinkedIn and serves on the Advisory Board for the Mayo Clinic Center for Social Media.

She is also the author of the e-book Calmer: Medical Events with Cognitively Impaired Children (2012) which seeks to share strategies for preventing medical trauma in children with chronic medical conditions.

For more information, visit www.savingcase.com. Melissa can be reached via Twitter @savingcase or by e-mail at melissa@savingcase.com.

 

 

06 Mar 2013
Sticky Post By Posted in Advocacy, Media, Newspaper Permalink

Angry Parents Force J&J To Do Damage Control

Sticky Post By On 6 March, 2013
http://www.pharmalive.com/angry-parents-force-jj-do-damage-control
There is nothing like a bit of pressure from an angry mom or dad to generate heat. And thanks to the modern wonders of the Internet, an Ontario schoolteacher has succeeded in forcing Johnson & Johnson to scramble to contain a mushrooming controversy. At issue: a months-long refusal by the health care giant to support further research involving one of its own drugs for a debilitating disease.

The uproar emerged two weeks ago. Andrew McFadyen, whose eight-year-old son suffers from MPS, a group of rare genetic disorders caused by the absence or malfunctioning of lysosomal enzymes, grew frustrated with J&J and turned to the Internet to publicize his quest. For more than a year, he had hoped J&J would agree to work with researchers at the Mt. Sinai School of Medicine who found that a J&J drug called Elmiron may offer some hope.

His primary contact at Mt. Sinai is Calogera Simonaro, an associate professor in the Department of Genetics and Genomic Sciences, who recently co-authored a paper showing various improvements in rats given Elmiron, a J&J drug that is approved for treating interstitial cystitis, which is also known as painful bladder syndrome. The two met a few years ago when Simonaro applied for a grant from the foundation that McFadyen created to further MPS research and help his son, Isaac (see photo above).

“She met with (J&J representatives) last spring to present her data to show how the drug worked and she reported back to me that they weren’t interested,” says McFadyen, who hoped J&J would back additional studies, such as testing in larger animals. “Essentially, we were put off. The discussions were not going anywhere. Our researchers and their medical team finally held a teleconference last November, but then, there was no follow up.”

“We spent nearly a year trying to convince J&J that they should (support the MPS research at Mt. Sinai and clinical research using Elmiron). It could save the healthcare industry lots and lots of money. Right now, it can cost $500,000 to $1 million a year for enzyme replacement therapy, which is an imperfect situation,” he says. “But most important, it might increase the quality of life for the children and save a lot of lives.”

Simonaro declined to comment, other than to offer a statement in which she said “we are in discussions with potential partners who have an interest in testing (Elmiron) for MPS in a formal clinical trial setting so that the therapy can be approved and available for use by all patients.” Mt. Sinai, by the way, is obtaining intellectual property rights to use the drug for MPS treatment, according to sources.

There are actually several forms of MPS, which can cause a variety of symptoms, including mental retardation, cloudy corneas, short stature, stiff joints, incontinence, speech and hearing impairment, chronic runny nose, hernia, heart disease, hyperactivity, depression, pain and a shortened life span. The disease occurs in about one in every 25,000 births, according to theInternational MPS Network.

In other words, this is a rare disease and Fadyen has openly expressed concerns that J&J was not interested in making an investment that called for pursuing years worth of costly research for a treatment for a small patient population, even though orphan drugs are increasingly commanding price tags of $250,000 or more per year for each patient (read this and this).

So last month, McFadyen reached out once again to J&J and received a reply from Steve Silber of the R&D team at Janssen, the J&J unit that sells Elmiron. Silber offered to make the drug available to McFadyen’s son on a compassionate use basis and to work with physicians on a so-called investigator-initiated study (read his letter here). McFadyen responded two ways – he wrote a harsh response that accused J&J of stalling tactics and he created a web site calledshameonjnj.com.

The web site quickly attracted attention not only among those with MPS and their family members, but people who were upset that J&J appeared unwilling to extend its largesse to others. As McFadyen viewed it, J&J was offering only his son compassionate access, which he declined to accept because he believed the health care giant should make Elmiron available to anyone who might benefit. And since J&J released its letter publicly, he placed that and his own reply on the new web site.

“From the very beginning, we have approached Johnson & Johnson about working toward such a study and, time and time again, we were ignored, rebuffed and put off,” he responded to Silber. “Families dealing with this disease are incredibly vulnerable and being caught in the middle of weighing the risks vs. rewards of putting our children on this treatment off label without adequate safety and efficacy data.”

By last week, this very public exchange, which prompted angry parents to Tweet links to the web site and post on a Facebook page as well, was on the verge of giving J&J (JNJ) yet another image headache. The health care giant, you may recall, has suffered a series of embarrassing gaffes over the past three years – manufacturing problems that led to the recall of countless over-the-counter items such as Tylenol and Motrin; courtroom setbacks over Risperdal marketing (seethis and this) and a scandal over the safety of hip implant replacements.

And so, J&J late last week began a counterattack. In response to the sudden burst of negative publicity, Jannsen had its chief medical officer, Amrit Ray, respond to McFadyen in yet another letter. And this time, he made a point of writing that compassionate access would be available to any child and reiterated the offer to support an investigator-initiated study with any physician would be interested in doing so. Ray also maintained that J&J was convening a group of experts to explore the possiblities for supporting MPS research with Elmiron.

“We’re trying to get in the right place to where we can get the right data. It’s not one of the areas where we have a lot of experience,” Ray told us. “There has never been any data to indicate it would help patients.. but we’re eager to understand the data… and we’re certainly open to hearing a proposal. In this case, we would like to get some additional expertise to assess any proposal” from a physician willing to administer the drug. However, he disputed some of the chronology that McFadyen offered about miscues last year for substantive discussions.

Initially, McFadyen responded with skepticism and continued to express concern that experimental usage of the drug poses risks and that J&J should be willing to commit to supporting a regular clincical trial. By the start of this week, though, he had held several telephone conversations with Ray and now tells us he is willing to give J&J a chance to work with families. In fact, he is considering pulling down the shameonjnj web site. “I think the web site achieved its goal,” he says.

“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,” McFadyen wrote on the Isaac Foundation web site (read here). “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.”

Whether J&J follows through, of course, remains to be seen. The drugmaker is not under any obligation to fund additional research, although the resources needed to explore the possibilities – with the help of scientific experts who can ballpark the odds of success – are relatively modest. For now, though, J&J has scored a win. In a hurly burly world where just about everything can – and often does – go viral quickly, the health care giant has managed to diffuse a potential crisis. And unless J&J commits yet another blunder and angers MPS families again, this is a rare accomplishment when considering the recent spate of scandals, gaffes and setbacks on so many fronts.

06 Mar 2013
Sticky Post By Posted in Advocacy, Media, Newspaper Permalink

Angry Parents Force J&J To Do Damage Control

Sticky Post By On 6 March, 2013
http://www.pharmalive.com/angry-parents-force-jj-do-damage-control
There is nothing like a bit of pressure from an angry mom or dad to generate heat. And thanks to the modern wonders of the Internet, an Ontario schoolteacher has succeeded in forcing Johnson & Johnson to scramble to contain a mushrooming controversy. At issue: a months-long refusal by the health care giant to support further research involving one of its own drugs for a debilitating disease.

The uproar emerged two weeks ago. Andrew McFadyen, whose eight-year-old son suffers from MPS, a group of rare genetic disorders caused by the absence or malfunctioning of lysosomal enzymes, grew frustrated with J&J and turned to the Internet to publicize his quest. For more than a year, he had hoped J&J would agree to work with researchers at the Mt. Sinai School of Medicine who found that a J&J drug called Elmiron may offer some hope.

His primary contact at Mt. Sinai is Calogera Simonaro, an associate professor in the Department of Genetics and Genomic Sciences, who recently co-authored a paper showing various improvements in rats given Elmiron, a J&J drug that is approved for treating interstitial cystitis, which is also known as painful bladder syndrome. The two met a few years ago when Simonaro applied for a grant from the foundation that McFadyen created to further MPS research and help his son, Isaac (see photo above).

“She met with (J&J representatives) last spring to present her data to show how the drug worked and she reported back to me that they weren’t interested,” says McFadyen, who hoped J&J would back additional studies, such as testing in larger animals. “Essentially, we were put off. The discussions were not going anywhere. Our researchers and their medical team finally held a teleconference last November, but then, there was no follow up.”

“We spent nearly a year trying to convince J&J that they should (support the MPS research at Mt. Sinai and clinical research using Elmiron). It could save the healthcare industry lots and lots of money. Right now, it can cost $500,000 to $1 million a year for enzyme replacement therapy, which is an imperfect situation,” he says. “But most important, it might increase the quality of life for the children and save a lot of lives.”

Simonaro declined to comment, other than to offer a statement in which she said “we are in discussions with potential partners who have an interest in testing (Elmiron) for MPS in a formal clinical trial setting so that the therapy can be approved and available for use by all patients.” Mt. Sinai, by the way, is obtaining intellectual property rights to use the drug for MPS treatment, according to sources.

There are actually several forms of MPS, which can cause a variety of symptoms, including mental retardation, cloudy corneas, short stature, stiff joints, incontinence, speech and hearing impairment, chronic runny nose, hernia, heart disease, hyperactivity, depression, pain and a shortened life span. The disease occurs in about one in every 25,000 births, according to theInternational MPS Network.

In other words, this is a rare disease and Fadyen has openly expressed concerns that J&J was not interested in making an investment that called for pursuing years worth of costly research for a treatment for a small patient population, even though orphan drugs are increasingly commanding price tags of $250,000 or more per year for each patient (read this and this).

So last month, McFadyen reached out once again to J&J and received a reply from Steve Silber of the R&D team at Janssen, the J&J unit that sells Elmiron. Silber offered to make the drug available to McFadyen’s son on a compassionate use basis and to work with physicians on a so-called investigator-initiated study (read his letter here). McFadyen responded two ways – he wrote a harsh response that accused J&J of stalling tactics and he created a web site calledshameonjnj.com.

The web site quickly attracted attention not only among those with MPS and their family members, but people who were upset that J&J appeared unwilling to extend its largesse to others. As McFadyen viewed it, J&J was offering only his son compassionate access, which he declined to accept because he believed the health care giant should make Elmiron available to anyone who might benefit. And since J&J released its letter publicly, he placed that and his own reply on the new web site.

“From the very beginning, we have approached Johnson & Johnson about working toward such a study and, time and time again, we were ignored, rebuffed and put off,” he responded to Silber. “Families dealing with this disease are incredibly vulnerable and being caught in the middle of weighing the risks vs. rewards of putting our children on this treatment off label without adequate safety and efficacy data.”

By last week, this very public exchange, which prompted angry parents to Tweet links to the web site and post on a Facebook page as well, was on the verge of giving J&J (JNJ) yet another image headache. The health care giant, you may recall, has suffered a series of embarrassing gaffes over the past three years – manufacturing problems that led to the recall of countless over-the-counter items such as Tylenol and Motrin; courtroom setbacks over Risperdal marketing (seethis and this) and a scandal over the safety of hip implant replacements.

And so, J&J late last week began a counterattack. In response to the sudden burst of negative publicity, Jannsen had its chief medical officer, Amrit Ray, respond to McFadyen in yet another letter. And this time, he made a point of writing that compassionate access would be available to any child and reiterated the offer to support an investigator-initiated study with any physician would be interested in doing so. Ray also maintained that J&J was convening a group of experts to explore the possiblities for supporting MPS research with Elmiron.

“We’re trying to get in the right place to where we can get the right data. It’s not one of the areas where we have a lot of experience,” Ray told us. “There has never been any data to indicate it would help patients.. but we’re eager to understand the data… and we’re certainly open to hearing a proposal. In this case, we would like to get some additional expertise to assess any proposal” from a physician willing to administer the drug. However, he disputed some of the chronology that McFadyen offered about miscues last year for substantive discussions.

Initially, McFadyen responded with skepticism and continued to express concern that experimental usage of the drug poses risks and that J&J should be willing to commit to supporting a regular clincical trial. By the start of this week, though, he had held several telephone conversations with Ray and now tells us he is willing to give J&J a chance to work with families. In fact, he is considering pulling down the shameonjnj web site. “I think the web site achieved its goal,” he says.

“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,” McFadyen wrote on the Isaac Foundation web site (read here). “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.”

Whether J&J follows through, of course, remains to be seen. The drugmaker is not under any obligation to fund additional research, although the resources needed to explore the possibilities – with the help of scientific experts who can ballpark the odds of success – are relatively modest. For now, though, J&J has scored a win. In a hurly burly world where just about everything can – and often does – go viral quickly, the health care giant has managed to diffuse a potential crisis. And unless J&J commits yet another blunder and angers MPS families again, this is a rare accomplishment when considering the recent spate of scandals, gaffes and setbacks on so many fronts.

04 Mar 2013
Sticky Post By Posted in Blog Postings Permalink

Johnson and Johnson Update

Sticky Post By On 4 March, 2013

Red-Foundation-HopeIMPORTANT UPDATE – PLEASE SHARE

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 to 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
http://www.treypurcell.com/blog/

17 Feb 2013
Sticky Post By Posted in Advocacy, Media, Newspaper Permalink

Trent Hills Father Pushing For Cure For His Son

Sticky Post By On 17 February, 2013
Feb 17, 2013

Million dollars needed to fund clinical trial

Trent Hills father pushing for a cure for his son

John Campbell / The Independent

TRENT HILLS — Andrew McFadyen and his wife Ellen established the Isaac Foundation to raise money for research to find a cure for their son, who has MPS VI. They believe one has been found, but it will need a clinical trial to prove it’s safe and effective. Project One Million is a campaign to raise the necessary money. January 2013
Northumberland NewsTRENT HILLS — Andrew McFadyen is convinced research shows a drug exists that will help his son Isaac get better.

Now he has to convince the drug manufacturer that makes it what he says is true.

Isaac suffers from MPS VI, a rare disease caused by an enzyme deficiency. Research with rats indicates a medication used to treat bladder inflammation could reverse its symptoms, which include stunted growth, stiff joints, heart and eye problems.

A human clinical trial is required to prove the anti-inflammatory oral drug is safe and effective when used to treat MPS VI.

Mr. McFadyen has been urging its manufacturer, Johnson and Johnson, to conduct the trial. He was encouraged when the company said it would make a decision soon, following a conference call last November involving the researcher who discovered the groundbreaking treatment and medical researchers at the pharmaceutical giant.

When he hadn’t received word by mid-January, he sent Johnson and Johnson a note “letting them know time is a luxury our kids can’t afford,” referring to the thousands of children around the world who have MPS in various forms.

Johnson and Johnson said it is looking at how it “can be helpful.”

In a statement issued through spokeswoman Suzanne Frost, the company said: “We empathize with the McFadyen family and all families who face rare diseases.

“A senior staff member in our research and development organization has assembled a team to fully evaluate this situation and determine if and how we can be helpful. He is a physician with extensive experience in drug development for a variety of diseases.”

The company said it gives “careful consideration to many requests for assistance each year. Unfortunately, we are not able to help in every situation.”

Isaac can’t close his hands very well anymore or lift his arms above his head, and his bones are starting to put pressure on his lungs and internal organs.

He recently asked his father about “the new pill” and told him, “‘I just really want to know what it’s like to be like everybody else,'” Mr. McFadyen said. “That was heartbreaking.'”

It was “the push” Mr. McFadyen said he needed to redouble his efforts to raise $1 million for the clinical trial and to get Johnson and Johnson behind Project One Million.

“I can’t live every day just with hope, I need to move forward and see if we can’t get them onboard by any means that we have,” he said. “I’m hopeful they’ll come through.”

11 Feb 2013
Sticky Post By Posted in Media, Newspaper Permalink

Family Day Concert To Combat Bullying

Sticky Post By On 11 February, 2013

1297375499796_ORIGINALCAMPBELLFORD – Children’s entertainer Andrew “Too Tall” Queen is getting ready for his fourth annual Family Day concert. The first fundraisers were in support of the Kennedy Park Revitalization and the last two years they have been raising funds for the Isaac Foundation.

Andrew McFadyen from Campbellford is the father of eight-year old Isaac, who has a rare enzyme deficiency disease called MPS VI (Maroteaux Lamy Syndrome). He started the Isaac Foundation to support research into ground-breaking treatments, potential cures and also to increase public awareness. There are approximately 10 cases in Canada and 1,100 worldwide. Symptoms of the disease include: stiffening of joints, spinal cord compression, stunted growth, heart and airway disease and a shortened life span. There is no cure at the moment but there is treatment called Naglazyme. It is an Enzyme Replacement Therapy that is designed to provide patients with a synthetic version of the enzyme they are lacking. It is a very expensive and is only available to Canadian patients through the federal government’s Special Access Program.

McFadyen said “We’re touched that Andrew and Karen (his wife) have decided to support our organization again this year,” said McFadyen. “For us to find a cure for Isaac, it’s going to take help from many different people. To have the support of our community really goes a long way to helping us reach our dream of finding a cure.”

Last year’s concert raised around $1,000, a number Queen hopes to surpass this year. The theme for this year’s concert is “Celebrate Friendship and Stand Tall.” The anti-bullying themed performance is really close to Queen’s heart as he remembers being teased as a child. Now as a parent, he can share his own experiences about bullying. Queen recently met fellow teacher and author Heather Rankin who wrote the book All It Takes is One Friend, which is illustrated by students at Earl Prentice Public School in Marmora. “Heather’s book really resonated with me and I shared it with my wife (Karen Stille). A couple of weeks later she had a beautiful new song in the works,” said Queen.

The songwriting duo was very happy with the song and sucessfully submitted it for a recording grant with FACTOR (Foundation Assisting Canadian Talent On Recordings). The song, a duet, is called It Just Takes One. Local singer Janet Jeffery rounds out the recording with her sweet and soulful vocals. Queen and Stille plan to release the single in the spring to coincide with the International Day of Pink on April 10.

“The message of the song is that each of us has the power to stop bullying by standing up and being a friend to someone in trouble,” said Queen. “When bystanders get involved, bullying usually stops within seconds.”

Family Day is Monday, Feb. 18 and the show will begin at 2 p.m. at the Aron Theatre in Campbellford. Queen and Stille will be joined on stage by Luke Mercier on fiddle and Tim Hadley on double bass. Special guests will include Janet Jeffery and, for the first time, a children’s chorus singing backup vocals. Everyone is encouraged to wear pink and/or purple for the event.

Tickets are now available at the Aron Theatre, Kerr’s Corner Books and the Grindhouse Café. Advance tickets are recommended and cost $5 per person or $7 at the door. All proceeds will go to the Isaac Foundation in support of Project One Million. For more information, call 705-632-1616 or visit www.andrewqueen.ca  and www.theisaacfoundation.com

03 Oct 2012
Sticky Post By Posted in Advocacy, Media, Newspaper Permalink

Trent Hills Couple Set $1 Million Goal To Find Cure For Disease

Sticky Post By On 3 October, 2012

Son Isaac has extremely rare disease

Northumberland News

TRENT HILLS — Andrew and Ellen McFadyen are prepared to take on big pharma to raise $1 million for a clinical trial of a drug that could prove life-changing for their oldest son.

Isaac, eight, has an extremely rare disease, MPS VI, which requires that he receive treatment for an enzyme deficiency one day a week at Toronto’s SickKids hospital.

The enzyme replacement therapy he began five years ago has slowed down the progress of the disease, which stunts growth, stiffens joints, affects the heart and airways, and shortens lifespan.

There is no cure, but the McFadyens believe one is close at hand. Proving it, however, will take $1 million. Their faith is based on “positive results” from ground-breaking research done in the U.S. that was funded by The Isaac Foundation.

The Meyersburg couple established the foundation in 2005 to raise money for research into innovative treatments and potential cures for MPS VI and other MPS diseases, and to increase public awareness about the disorders.

“One of the projects we’ve been funding for the last three years out of New York City is reversing all of the symptoms of this disease in lab animals; it’s as close to a cure as we’ve ever been,” Mr. McFadyen said. “Now we just need to get it into human clinical trial and ensure the safety is there for our children, which we’re very, very confident it is.”

The drug under study is an oral anti-inflammatory that is already used to treat bladder inflammation and has been approved by Health Canada and the Food and Drug Administration in the U.S.

“We’re incredibly hopeful that this is a turning point in the lives of all kids suffering from MPS,” Mr. McFadyen said. “It’s not a huge trial that needs to be done; probably it would take nine months to have good data.”

To raise the capital required, he conceived Project One Million, which is “very ambitious” but it’s a goal he’s confident can be achieved.

“We’ve set our sights high and we’re going to get it,” he said.

He’s pinning his hopes on getting the American pharmaceutical company that manufactures the anti-inflammatory medication to underwrite the cost of the clinical trial. So far it has refused, but that’s not stopping Mr. McFadyen, whose extensive lobbying persuaded the provincial government several years ago to agree to pay for Isaac’s enzyme replacement therapy, which costs about $400,000 annually for the medication.

In the last two years he was also successful in helping to persuade the Ontario and Saskatchewan governments to extend the same benefit to two other children with MPS VI.

“This product has potential to save thousands and thousands of children’s lives (around the world) and I’m not going to rest until they ensure that product gets effectively used for those kids,” Mr. McFadyen said.

“They know we’re fighting against time,” he said. “I will not sit idly by.”

A teleconference involving researchers at the pharmaceutical company and those Mr. McFadyen has been working with is scheduled for Oct. 11.

“They’ve made no promises, but it’s a start,” he said.

 

SIDEBAR

Cobourg gala to raise money to find a cure

TRENT HILLS – Project One Million is more than about raising a huge amount of capital, “it’s a million conversations, it’s a million people talking about rare diseases,” and making them aware “they can make a difference,” Andrew McFadyen said.One way they can show their support is to attend the third annual Gala for a Cure, which includes dinner, wine tasting, a silent auction, and entertainment by Juno Award-winning musician Ron Sexsmith. The fundraiser will take place Oct. 13 at the Best Western Convention Centre in Cobourg.

Eight-year-old Isaac McFadyen, who has been taking piano lessons for a year, will play a number and present a Lifetime Impact Achievement award to Dr. Lilla Simonaro, whose research has fuelled optimism a cure has been found.

Tickets are $100 per person (a charitable tax receipt will be written for $60). They can be purchased online atwww.theisaacfoundation.com or by phoning 613-328-9136.

23 Aug 2012
Sticky Post By Posted in Media, Newspaper Permalink

Project One Million Launches

Sticky Post By On 23 August, 2012

1344538301760_ORIGINALThe following is the third and final part of an interview with Andrew McFadyen, the father of eight-year-old Isaac who has the rare disease MPS VI. Wednesday’s story featured Isaac’s continued battle with the disease. Thursday’s story focuses on the McFadyen family’s advocacy for other children diagnosed with the disease. Today’s story announces their lofty new fundraising project.

CAMPBELLFORD — Today is the launch of a million-dollar project for The Isaac Foundation.

That is what this story is supposed to be about.

First, though, it’s worth updating Thursday’s story which talked about the McFadyen family from Campbellford and their advocacy for provincial funding for life-sustaining treatment for children who suffer from the rare disease MPS VI.

Andrew and Ellen McFadyen’s eight-year-old son Isaac has the disease, which is caused by an enzyme deficiency. As previously reported, there is a treatment — Enzyme Replacement Therapy — available through the country’s Special Access Program, but it can cost up to a million dollars per year and the respective provincial governments must agree to pay for it.

When Isaac was first diagnosed years ago, the McFadyens were initially turned down, but the decision was reversed after a successful lobby effort. Last year, the Darren and Pam More from Palmerston had the same problem with their two-year-old son Jasper. With help from the McFadyens and their Isaac Foundation the Mores also eventually got funding.

This past week, the McFadyens have been advocating for Violet Revet, a three-year-old girl from Saskatchewan who was diagnosed with MPS VI seven months ago. Her parents — Jennifer Yanke and Larry Revet — had been waiting all those months to hear whether their daughter would be approved, or denied, for funding. They turned to The Isaac Foundation for help and McFayden advocated behind the scenes while public pressure through social media to the Saskatchewan government also aided the lobby effort.

Wednesday night it was announced that Violet has been approved for the funding and she will get the treatment she needs.

“I was unbelievably overcome with emotion when I got that notification that Violet was going to get covered,” Andrew McFadyen said yesterday. “It’s bittersweet to have to battle, but then to win that battle again on behalf of a family, on behalf of a child is really special to us, probably because we know how important it is for these kids to have this treatment. Being down this road for six years, I know what it means for Violet and her family.”

McFadyen expressed gratitude to those who made their voices heard by the Saskatchewan government.

“It’s one thing for one person or one family or one tiny organization to be calling governments into account. Governments can weather that storm, they can weather the storm of me calling them every day and advocating and telling them all of the different things that I want them to do on behalf of children,” McFadyen said. “They can get through that, but once there’s momentum behind it, once people are sharing the story, once people are talking about it and tweeting about it and calling into the minister’s office to express their concern…then that prompts action. We’ve been incredibly blessed that there are a lot of people who are interested in The Isaac Foundation and always looking for ways that they can give back and help. For the second straight year, this is a way they’ve been able to do that.”

Violet, like Isaac and like Jasper, will now get Enzyme Replacement Therapy.

While ERT treatment has proven to be essential, McFadyen has repeatedly stated that it’s not a cure.

Today, The Isaac Foundation is launching Project One Million. Andrew and Ellen McFadyen established the foundation in 2006 to not only raise awareness for MPS VI, but to also help fund innovative research projects.

Currently, they are ecstatic about a research project in New York City related to bone and joint disease in children affected by MPS VI.

“We’re starting to discover that the disease progression is the buildup of these complex sugars in the body and attacking the organs, but it’s also the inflammation that’s taking place in the bones and joints,” McFadyen said in an interview earlier this week. “It’s that inflammation that is leading to many of the devastating physical symptoms that our kids are seeing; the stiffening of joints, the spinal cord compression, in my son his hands are starting to become very ineffective — he has no strength and can’t make a fist. He can’t even do up the buttons of his shirt anymore.”

McFadyen said through The Isaac Foundation they focus on research projects that aim to find a cure or offer a short-term solution. The researcher in New York City has discovered oral anti-inflammatory medication that’s already on the market, already FDA approved, already approved by Health Canada.

This anti-inflammatory has already been tested on lab rats that are affected by MPS VI.

“Rats and cats develop MPS VI naturally and so they’re a really good indictor for what medications work and which medications don’t work for humans,” McFadyen said. “The progression of the disease is almost identical in these animals as it is in humans. What she’s found is this anti-inflammatory is reversing almost all of the bone and joint disease in these animals.”

A video was sent to the McFadyens showing two different rats. The untreated rat had difficulty walking, coarse facial features, matted fur, its eyes were clouded over and had still joints, “actually, very depressing to see,” McFadyen said.

As for the rat treated with the anti-inflammatory, “you would never know there’s a problem. The animal is trying to jump out of its box in the lab, it’s running around, the facial features aren’t as coarse.

“For us, we naturally looked at that and said let’s start a human clinical trial. We’re ready,” he added. “(The Isaac Foundation) funded a trial in lab animals using the anti-inflammatory in conjunction with ERT just so we know that it’s safe and those early results show that it’s very safe and no change in the blood chemistry.”

Of course, it’s not as simple as just starting the human trial.

“We need a million dollars,” McFadyen said. “This number continues to come up in my life. A million dollars a year to fund to fund my child’s treatment. Now we need a million dollars to do a human clinical trial.”

Pharmaceutical companies have said they not interested in funding the trials, McFadyen said. So once again, the McFadyens are taking it upon themselves to make something happen.

The result: Project One Million.

“Our hope is that we can start a million conversations about rare diseases across the world,” McFadyen said. “We want to have a million people view our video, we want a million likes and shares (on Facebook), a million tweets (on Twitter), and perhaps we can inspire a million people to donate $1 each so that we can fund this clinical trial.”

McFadyen said he felt the world of MPS shift a bit last month when the results of this study were presented.

“It will only enhance the quality of life for these kids,” he said, while expanding using Isaac as an example.

“We have every hope that his hands will begin to work again, and that he’ll be able to raise his arms up above his head, that his stamina will increase because the tightness in his hips will decrease, that there won’t be any more danger of spinal cord compression in him. The hope for this drug is off the charts.”

One million dollars is a lot of money to raise and that fact hasn’t been lost on the McFadyens.

“We know we have lofty goals, but if we just sit around and wait for something to happen, we’re never going to accomplish anything,” he said.

They will get some help, including from international music star John Mayer, who is a big supporter of Isaac’s. He’s met with the McFadyens several times prior to concerts in Toronto.

“We’re lucky that musician John Mayer is going to help get this video out,” McFadyen said. “He’s going to share it on his blog online and ask people to go and view it, and that’s a big boost. He’s got a lot of followers online.”

For instance, if you go to Mayer’s Facebook page, he has well over 5,300,000 ‘likes’.

They are also taking a ‘Share It Forward’ approach. They would like everyone to share the video and information about Project One Million, but they’ve also specifically identified 16 of the most influential social media celebrities, like Ellen DeGeneres, Johnny Depp and Anderson Cooper.

“The idea is the first celebrity that shares it, in this case John Mayer, will not only share it with the people that follow him online, but he’ll also share it forward to the next person in our chain (of celebrities) ask to send it forward,” McFadyen said. “We don’t know how far we can have this chain go, perhaps it stops at one, but maybe it goes all the way through that chain.”

McFadyen knows the million-dollar goal is a lofty one.

“We’re not naïve,” he said. “We may launch this and have 100 views and that’s it, but our business is hope and sometimes that’s all you have left.”

The website for Project One Million is www.projectonemillion.ca. The link for their video, which they hope people will watch and pass on, is http://www.youtube.com/watch?v=iPhisB8_-wM.

jeff.gard@sunmedia.ca

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