Wednesday, January 20, 2016

A STATE OF DISGUSTING IN FLINT MICHIGAN'S POISONED WATER

Water towers in America usually boast the names of the communities they serve, often with a mascot or slogan; totems of civic pride. Not in Flint Michigan. This one merely proclaims: FLINT WATER PLANT. And this one stands for betrayal, mismanagement corruption and scandal.



Flint Michigan, U.S.A., is a town of 100,000 human beings located about 70 miles from the shores of the largest group of fresh water bodies in the world: the Great Lakes. 
Yet its residents can't get clean water from their home faucets.

What is the dollar value of knowingly using poisoned water on innocent victims? 

In Flint Michigan, the answer is zero.

Nearly two years ago, the State of Michigan decided to save money by switching Flint's water supply from Lake Huron (which they were paying the city of Detroit for), to the Flint River, a notorious polluted tributary known to locals for its filth.

The pollutants in the Flint River, a result of decades of unchecked chemical dumping by General Motors and other companies, have made the river water highly toxic.

In 2011, Flint was declared to be in a financial state of emergency, and the State took budgetary control. Therefore, all the decisions made during the water crisis were at the State level, which State officials confirmed, not by the City Council or the Mayor.
The switch was made during this financial emergency in April 2014 after Darnell Earley, an unelected emergency manager appointed by Michigan Governor Rick Snyder, switched Flint’s water source to the long-polluted and corrosive Flint River in a bid to save money.  

It was supposed to be temporary while a new State run supply line to Lake Huron was to be constructed for connection. The project was estimated to take about two years.

Soon after the switch, the water started to look, smell and taste funny. Residents said it often looked dirty and brown in color.
But what residents couldn't see was far worse. About half of the service lines to homes in Flint are made of lead and because the water wasn't properly treated, lead  and iron began leaching into the water supply.

Instead of immediately addressing the problem, the Flint emergency manager Darnell Earley paid the Veolia Corp. $40,000 to conduct a study which recommended reducing other unsafe pollutants with chemicals and using filters. 

Incredibly, this Veolia Corp. study through incompetence, or possibly deliberate reasons, failed to address the lead pipe corrosion issue by completely ignoring it. 

Bob Bowcock, an associate of environmental activist Erin Brockovich, after studying the situation for no charge, recommended the obvious: reconnect to Detroit until the new project is finished.

Nothing was done.

This has been the status quo for nearly two years, and until September 2015, City and State officials told worried residents that everything was fine. Former Flint Mayor Dayne Walling even drank it on local TV to make the point.
No one believed State and local officials when they said that this disgusting looking brown water was safe.
The Flint River is highly corrosive: 19 times more so than the Lake Huron supply, according to researchers from Virginia Tech.


The Flint River is highly corrosive: 19 times more so than the Lake Huron supply, according to researchers from Virginia Tech.



According to a class-action lawsuit, the State Department of Environmental Quality (DEQ) wasn't treating the Flint River water with an anti-corrosive agent, in violation of Federal law. Therefore, the water was eroding the iron water mains, turning water brown.
In August 2015, it took an independent effort by community activists, aided by University of Virginia professor and water safety specialist Marc Edwards, pediatrician Dr. Mona Hanna-Attisha, and Michigan ACLU investigative journalist Curt Guyette, to expose both the toxic lead levels in the water, and the lead poisoning already impacting the children of Flint Michigan.
This group of researchers from Virginia Tech came up to Flint and did in home testing and found elevated levels of lead in the drinking water and made those findings public. 

State officials insisted their own research was more accurate and attempted tp discredit the above independent study.
The Town residents did not trust the public officials and finally realized that their water supply was poisoned and the State had been lying to them in a cover-up about the truth.

Residents are now part of the above class-action lawsuit that alleges not only lead poisoning but several medical conditions resulting from contaminated water after the switch. 

Despite enormous outrage from residents, officials repeatedly insisted that the water was safe to drink. 


The crisis only came to national attention in mid-December 2015, after Mayor Karen Weaver issued an official declaration of a state of emergency over the city's drinking water. In the statement, she called the situation a "man-made disaster" and the damage irreversible. 

Further Testing has found that the drinking water is extremely contaminated with lead, which can cause serious brain damage, especially in children.

Ten people there have also now died from Legionnaire's disease, a form of pneumonia likely linked to bacteria in the city’s water. Lead levels perilously above federal limits have put 100,000 people lives at risk.

Who's to blame?
A state appointed task force found that fault lies with the State, and on December 29, 2015, the Director of DEP,  Dan Wyant resigned because of the situation.

Former emergency manager Darnell Earley (who now has been appointed by Gov. Snyder as the emergency manager of Detroit's Public schools) says he's not to blame for the decision to use the Flint River as the city's source of drinking water.

Earley, who served as Flint's emergency manager from September 2013 until January 2015, said in an email to The Flint Journal-MLive on Tuesday, Oct. 13, 2015 that the water source decision was made months before he was appointed to run the city by Gov. Rick Snyder.
Earley said he had no reason at the time to second guess what appeared to have been a consensus decision.
"The decision to separate from (the Detroit Water and Sewerage Department) and go with the Karegnondi Water Authority, including the decision to pump Flint River water in the interim, were both a part of a long-term plan that was approved by Flint's mayor, and confirmed by a City Council vote of 7-1 in March of 2013, a full seven months before I began my term as emergency manager," Earley's email says.
Although the Flint City Council voted in March 2013 in support of moving to the KWA pipeline, a new pipeline that would serve the region with Lake Huron water, there is no record that the council voted to use the Flint River as a short-term drinking water source.
Earley, who toasted the switch to the river with city leaders in a ceremony in April 2014, said it was his "responsibility to implement the previously accepted and approved plan" since the city's contract with the Detroit water system expired during his term as emergency manager.
"It did not fall to me to second guess or to invalidate the actions that were taken prior to my appointment," his statement says.
While Earley oversaw the switch, the decision to switch was signed by Flint's previous State appointed emergency manager, Ed Kurtz.
Sara Wurfel, a spokeswoman for Snyder, said in a statement on Tuesday, Oct. 13, 2015 that the decision to use the Flint River "was pushed or supported by the city and community" and said the city had no choice but to find another source of water after the Detroit Water and Sewerage Department (DWSD) terminated Flint's contract to continue purchasing water under the terms of its expired contract.
Former Flint Mayor Dayne Walling has responded that the vote to become a partner in the KWA, a new regional water authority, should not be confused with a vote to use the Flint River as a temporary source of drinking water, a vote that never happened according to him.
In contrast to using the river, the city also could have negotiated a short-term contract to continue buying water from DWSD, for example.
"No resolution was ever brought before myself or the City Council by any emergency manager to use the Flint River as the temporary source," Walling said.
Kurtz signed an order March 29, 2013, for Flint to eventually purchase water from the KWA pipeline after it was finished, but that order says nothing about the city's use of the river.
So in following the bouncing ball of the blame "game" between Republicans and Democrats who were involved, it is clear that no-one is willing to take accountability for poisoning the people of Flint, and that there never was anything done that remotely involved what was in the best interests of the citizens of Flint Michigan.
Yet, after nearly two years of living with poisonous water and astonishing government negligence, Flint residents still pay between $100 and $200 a month for their water bills.
Amazingly, to add insult to literal injury, the city has started to send overdue payment notices that threaten to cut off water service. 

Six months before Michigan’s governor declared a state of emergency over high lead levels in the water in Flint, his top aide wrote in an email that worried residents were “basically getting blown off by us.”
“I’m frustrated by the water issue in Flint,” Dennis Muchmore, then chief of staff to Gov. Rick Snyder, wrote in the email to a top health department staffer obtained by NBC News.
“I really don’t think people are getting the benefit of the doubt. Now they are concerned and rightfully so about the lead level studies they are receiving,” Muchmore said.
Why those DEQ officials took the actions they did is a question at the center of a tragedy that has left an unknown number of children and other Flint residents poisoned by lead, has led to a federal lawsuit and calls for a U.S. Justice Department investigation. 
And:
Lead levels in Flint’s drinking water would have spurred action months sooner if the results of city testing that wrapped up in June 2015 had not been revised by the Michigan Department of Environmental Quality to wrongly indicate the water was safe to drink, e-mails show.
The records obtained by the Michigan ACLU and by Marc Edwards, the Virginia Tech researcher who helped raise concerns about Flint’s water show how State officials first appear to have encouraged the City of Flint to find water samples with low lead levels and later told Flint officials to disqualify two samples with high readings. The move changed the overall lead level results to acceptable from unacceptable.
The Michigan Department of Environmental Quality manipulated the samples tested for lead to eliminate the samples with the highest concentration and thereby produce the result that it wanted: The appearance that the water was safe. 
Even using the Flint River water, the City of Flint could have prevented the corrosion of its copper and lead pipes relatively inexpensively:
Marc Edwards,  the professor from Virginia Tech who has been testing Flint water, says treatment could have corrected much of the problem early on,for as little as $100 a day, but officials didn’t take action because "they considered the cost of $100 per day as too expensive".
“There is no question that if the city had followed the minimum requirements under federal law that none of this would have happened,” said Edwards, who obtained the Muchmore email through a Michigan Freedom of Information Act request.
 As discussed above, this leaching could have been prevented by adding anti-corrosive chemicals to the water at a cost of roughly $100 per day and experts say 90% of the problems with Flint's water would have been avoided. Now, the corrosion of the lead pipes has destroyed the entire infrastructure. 
One hundred dollars a day would equal a mere $36,500 a year, a pittance in a budget of millions. To save $36,500 a year or maybe a little more, the city failed to treat the Flint River water, leaving it corrosive and able to leach lead and copper from the aging pipes used to transport it.
"When the governor appointed an emergency financial manager (in 2011), that person came here  to simply do one thing and one thing only, and that's cut the budget, at any cost," said Michigan Congressman Dan Kildee.
Kildee said the water crisis is indicative of an attitude about industrial towns such as Flint that have seen hard times in the past 30 years. They're often just forgotten, he said.
"This case shows that you can't treat cities the way you treat some Corporation that you might just sort of sell off," Kildee said.
Lead poisoning is irreversible. Pediatricians  fear the Flint children who tested with elevated levels will suffer lifelong consequences.
"If you were to put something in a population to keep them down for generation and generations to come, it would be lead,"  Dr. Hanna-Attisha said. "It's a well-known, potent neurotoxin. There's tons of evidence on what lead does to a child, and it is one of the most damning things that you can do to a population. It drops your IQ, it affects your behavior, it's been linked to criminality, it has multigenerational impacts. There is no safe level of lead in a child."
On January 5, 2016, three months after high lead levels were detected in Flint children, Michigan Gov. Rick Snyder declared a state of emergency over the issue.

He then apologized for the State's actions, but residents and officials remain furious because the State had previously strongly denied the problem existed for months and failed to use anti-corrosive treatment on the river water, which might have reduced the iron. 

Too little, too late from the Governor, as the facts are that the State government officials knew that the water supply was poisoning the people of Flint, did nothing to fix the problem, instead adopting a policy of  knowingly lying to hide the truth of their actions, and did not admit they caused it until this scandal was exposed to the public.  

The U.S. Attorney in Michigan and the federal Environmental Protection Agency are also investigating why the state chose to ignore federal law and go without the anti-corrosive agent, as the lawsuit contends

Dayne Walling, the former mayor who so confidently went on TV and drank Flint River water to try to quell the early protests, lost his recent re-election bid in a campaign centered around the issue.
"In retrospect, I regret all of it," Walling said this weekend.
"All the way back to seeing the city move to a different drinking water source. You can't put a dollar amount of the devastation to our community, our kids, and it was completely avoidable."
Michigan Governor Rick Snyder ran for re-election and won as a numbers man.
"I'm an accountant. I've found I can help more people when the numbers add up," he explained in a political ad.
Not easy in a State still dealing with the collapse of the auto industry.
Flint was one of nine Michigan cities put under the control of State appointed emergency managers whose job it was to cut pay and cut services to make those numbers add up. 

And Flint's emergency managers found that switching the city's water system to a cheaper toxic source would save $5 million over two years.
Mind boggling, but it worked. The State of Michigan expects to show a budget surplus this year of $575 million. 

Which is good, because the cost of saving that $5 million in Flint will be very expensive.

It's already cost $6 million to switch the water back, plus fixing the damaged pipes could increase the total expenses to $1.5 billion. 

And according to one doctor, those thousands of children with lead poisoning: "These children will be injured for life."
There are many people who were involved in these decisions, others who are part of the cover-ups of the real situation by deliberately falsifying information, all are sworn to be doing their jobs of protecting the citizens they supposedly serve.

They should be arrested for their criminal acts.

There is a petition being circulated to have those public officials responsible for this tragedy to be held accountable, and if found guilty by a Federal investigation, sent to jail for what they have done.
But I know that won't happen, no-one will go to jail.
State officials were just doing their jobs, making sure the numbers add up. 
And jail time is too expensive.

















Wednesday, December 23, 2015

FESTIVUS FOR THE REST OF US IN 2015

The Winter Holiday season, in particular Christmas  are portrayed in our society as the happiest time of the year, an opportunity to be joyful and grateful with family, friends and colleagues.

It has always seemed to me that this time of the year highlights the hypocrisy of Society by designating the period for everyone to be joyful, compassionate toward those less fortunate, being friendly, courteous, and doing "all the right" choreographed things in their interactions with others.

Suddenly sick children in hospitals are showered with free gifts by strangers, magical Santa Claus appears to provide needy children with toys, the poor are given free warm clothes, food, and treated as human beings.

What about the rest of the year, when most of these very same people who are still suffering, become invisible, ignored by most others, where does the kindness they received during the Holiday Season go? It simply disappears, these humans no longer are worthy of being recognized.

According to the National Institute of Health Christmas/Holiday Season is the time of year that people experience a high incidence of depression.

Hospitals and police forces report high incidences of suicide and attempted suicide.

Psychiatrists, Psychologists, Social Workers and other mental health professionals report a significant increase in patients complaining about depression.

One North American survey reported that 45% of respondents dreaded the season.

It appears to have more to do with unrealistic expectations and excessive self-reflection for many people.

For some people, they get depressed at this time of the year and even angry because of the extreme commercialization with the focus on gifts and the emphasis on "perfect" social activities.

Others get depressed because it appears to be a trigger to engage in excessive self-reflection and rumination about the inadequacies of life (and creates a "victim" mentality) in comparison with other people who seem to have more and do more.

Still others become anxious because of the pressure (both commercial and self-induced) to spend a lot of money on gifts and incur increasing debt.

Some report that they dread Christmas because of the expectations for social gatherings with family, friends and acquaintances that they'd rather not spend time with.

And finally, many people feel very lonely at Christmas, because they have suffered the loss of loved ones, are distant from family members, or live alone.

To counter all of this, I suggest that we all look back to the truly positive holiday spirit of "Happy Festivus" which is the traditional greeting of Festivus, a holiday featured in "The Strike" episode of The Seinfeld Televison show aired on December 18, 1997.

According to the Seinfeld model, Festivus is celebrated each year on December 23rd. 

However many people celebrate it other times in December, and even at other times throughout the year.

The slogan of Festivus is "A Festivus for the rest of us!"

The usual holiday tradition of a tree is replaced by an unadorned aluminum pole, which is in direct contrast to normal holiday materialism.

Those attending Festivus may also participate in the "Airing of Grievances" which is an opportunity to tell others how they have disappointed you in the past year, followed by a Festivus dinner, and then completed by the "Feats of Strength" where the head of the household must be pinned.

The Festivus celebration includes four main components:

The Festivus PoleThe tradition begins with an aluminum pole, which is used for its "very high strength-to-weight ratio."

During Festivus, the unadorned Festivus Pole is displayed.

The pole was chosen apparently in opposition to the commercialization of highly decorated Christmas trees, because it is "very low-maintenance," and also because the holiday's patron, Frank Costanza, "finds tinsel distracting."

The Airing of Grievances: At the beginning of the Festivus dinner, each participant tells friends and family of all the instances where they disappointed him or her that year.

As quoted from Frank Costanza: "I've got a lot of problems with you people, and now you're going to hear about it!"

Festivus Dinner: A celebratory dinner is shown on the evening of Festivus prior to the Feats of Strength. In the episode the meal appeared to be meat loaf or spaghetti in a red sauce.

The Feats of Strength: After the dinner, the head of the household tests his or her strength against one participant of their choosing.

Festivus is not considered over until the head of the family has been pinned. However, a participant may be allowed to decline to attempt to pin the head of the household only if they have something better to do instead.

Set aside today,  the day of December 23rd or another day this month and break out the meat loaf, because Festivus is once again upon us! 

Tuesday, December 22, 2015

THERE IS NO WORD FOR A PARENT THAT LOSES A CHILD. A NIGHTMARE OF STEVEN AND MYSELF.




A wife who loses a husband is called a widow. 

A husband who loses a wife is called a widower. 

A child who loses his parents is called an orphan. 

There is no word for a parent who loses a child. That’s how awful the loss is.   
(Jay Neugeboren  "An Orphan’s Tale",1976).

The death of a child is every parent’s worst fear and has been rendered to be “a catastrophic stressor unlike any other” by the "Psychiatric Diagnostic and Statistical Manual". 

Grief is not an illness to be treated or cured.


Grief is a response to a painful reality that one’s world is forever altered, and will never be the same. 

Absorbing this loss, and trying to adapt to the changes it unleashes, has its own unique course for every person, and will not be stilled or stopped by quick fixes or simple solutions.
And so, it was that kind of night again.

A nightmare of Steven and myself .

He was a teenager and cheerfully explaining to me how his grades in School were so excellent.

I could barely see his face, but Steven's voice, his mannerisms, all of  him were clear, talking to me, vividly, truly it appeared to be in real life.

I remember thinking, "how can he have grades in school if he is dead, how do I tell him that he is dead,  how did he get to be back in school again", and my feeling completely confused about what was happening.

I struggled, agonized to see more, hear more of Steven, but what I was experiencing became slower and slower, crawling, almost frozen in time.

I tried to see if I was awake or deep within some nightmare, it all had seemed so life like, so real, but nothing moved for what seemed like a long time.

Suddenly I awoke, within a nano second, a painful burning sense of reality struck me full force.

Steven is dead, and dead means forever gone.


Thursday, December 10, 2015

HAPPINESS CAN'T BE BOUGHT. IT"S ELUSIVE, IT"S AWESOME LOOKING, INSIDE AND OUTSIDE YOUR BRAIN.

This is what happiness really looks like: Molecules of the motor protein myosin drag a ball of endorphins along an actin filament into the inner part of the brain's parietal cortex, which produces feelings of happiness.  They are "strutting' as they "walk", seemingly aware of their impressive powers.


                      
Below is also myosin walking along an actin filament. This is a bit harder to see because these are real electron microscope images pasted together into a sequence.

This single molecule of myosin is "walking" because of an oscillating chemical reaction causing the molecule to rotate, attach, detach its other "foot", rotate again, attach, repeat. 

This is one little protein doing one little tiny thing, inside one tiny little cell, somewhere in your body. 

This is only one kind of protein performing one kind of action in one cell in your body. 

There are things like this, there are enzymes, there are all kinds of other mechanisms doing little "jobs" like this within each cell in your body. 

We're talking large numbers of things like this happening all the time in every cell, billions or trillions of actions. That's a lot of shit happening.

Perhaps, due to an error made by one of these little proteins at conception, or a change in your metabolism, your body doesn't "know" how to produce this or other molecules. 

Perhaps your body makes a little bit more or less of this myosin than another person
  
When you begin to realize the utter complexity of what's going on in the body, you can understand it isn't that easy to feel happiness, let alone actually finding happiness.

Happiness cannot be traveled to, owned, earned, worn or consumed.

You can buy an education, but happiness comes separately. 


You can buy a new car or you can buy a new house, but happiness comes separately. 


You can go shopping and buy everything you want, but happiness comes separately. 


You can get married or you can get divorced, but happiness comes separately. 


You can make a lot of money, but happiness comes separately. 


You can win the Lottery, but happiness comes separately.


You think you will be happy when you buy the new car or the larger house. And you will, but not for very long. 


Money can buy you a new car or a larger house. 


Money can buy you many things, but money can't buy you happiness.


Happiness is sold separately and it cannot be bought with money.


Money buys things, and happiness is not a "thing." Happiness is an inner state of mind.


We all want happiness. People want to be happy. This is the basic reason we do everything that we do.


We may think we are trying to get things, so we can feel more secure, be more loved or maybe we feel more important. 


The truth is that we do everything we do to derive happiness. 


If we learned that the main reason we desire the things we want are to experience happiness, we would value happiness more than just "things."


Happiness is probably the most important feeling that a human being can attain in their life. It is also sometimes the most elusive of all emotions.


We know the pleasurable feelings inside when we are happy but now we know what happiness actually look like in our brain.


Endorphins are chemicals that are able to cross through the gaps between neurons in order to pass along a message from one to the next. There are many different kinds, and much remains to be learned about their different purposes and functions.

One thing is known for certain about endorphins: their ability to make you feel oh-so-good. When your body is subjected to certain stimuli like sex, food or pain, your hypothalamus calls for endorphins, and the cells throughout your body that contain them heed the call. 
When endorphins lock into special receptor cells, called opioid receptors, because opiates also fit them, they block the transmission of pain signals and also produce a euphoric feeling, exactly like opiates.
Endorphins act as both a painkiller and as the pay off for your body's reward system. When you hurt yourself you may get a big dose of endorphins to ease the pain. 
You may also get an endorphin surge from talking to a stranger, eating chocolate, eating a satisfying meal, especially a sweet dessert, or being exposed to ultraviolet light. 
Everyone has different amounts of endorphins, and what may trigger an endorphin rush for one person could very well produce a dud for someone else.

Are you less happy than your parents were at the same age? 

It may not be all in your head. Researchers led by San Diego State University professor Jean M. Twenge found adults over age 30 are not as happy as they used to be, but teens and young adults are happier than ever.

Researchers,including Ryne A. Sherman of Florida Atlantic University and Sonja Lyubomirsky of University of California, Riverside, analyzed data from four nationally representative samples of 1.3 million Americans ages 13 to 96 taken from 1972 to 2014.

They found that after 2010, the age advantage for happiness found in prior research vanished. There is no longer a positive correlation between age and happiness among adults, and adults older than 30 are no longer significantly happier than those ages 18 to 29.

"Our current culture of pervasive technology, attention-seeking, and fleeting relationships is exciting and stimulating for teens and young adults, but may not provide the stability and sense of community that mature adults require," said Twenge, who is also the author of "Generation Me."

Data showed that 38 percent of adults older than 30 said they were "very happy" in the early 1970s, which shrunk to 32 percent in the 2010s. Twenty-eight percent of adults ages 18 to 29 said they were "very happy" in the early 1970s, versus 30 percent in the 2010s.

Over the same time, teens' happiness increased: 19 percent of 12th graders said they were "very happy" in the late 1970s, versus 23 percent in the 2010s.

"American culture has increasingly emphasized high expectations and following your dreams,things that feel good when you're young," Twenge said. 

"However, the average mature adult has realized that their dreams might not be fulfilled, and less happiness is the inevitable result. Mature adults in previous eras might not have expected so much, but expectations are now so high they can't be met."

That drop in happiness occurred for both men and women, said Twenge.

 "A previous study in 2008 got quite a bit of attention when it found that women's happiness had declined relative to men's. 

We now find declines in both men's and women's happiness, especially after 2010."

Sure we can feed ourselves sweets, ice cream, good food, drugs, alcohol, or exercise and they will temporarily create happiness by pushing the myosin onto stimulating the endorphins.

If you can find a way to bottle consistent happiness, you will be the "richest" person in the world.

For now, eat your chocolate, choose your favorite flavors of ice cream, find someone you can love, do whatever it takes, and if you are lucky enough, you will feel happy some of the time.





Sunday, December 6, 2015

UPDATED DECEMBER 6, 2015-JENNIFER MCNARY-DUCHENNE'S MUSCULAR DYSTROPHY-MOTHER OF MAX AND AUSTIN


                                     UPDATED DECEMBER 6, 2015


They call themselves the “fierce moms:” women who formed an alliance out of desperation and need. All have children with Duchenne muscular dystrophy, a rare and fatal disease. And for nearly four years they have single-mindedly lobbied politicians and federal regulators to speed up approval of experimental medicines.
“I don’t think we’ve seen anything like this since the days of AIDS and HIV activism,” said Ira Loss, who tracks the pharmaceutical industry for Washington Analysis, a consulting firm.
The fierce moms and other parents have notched some key victories: A Food and Drug Administration panel of experts will hold a pair of widely anticipated meetings to review two drugs designed to the slow the muscle wasting associated with DMD. The first occurs next Tuesday when a treatment from BioMarin Pharmaceutical will be considered. The other takes place in January to examine a drug developed by Sarepta Therapeutics.
But their victories have also raised uncomfortable questions.
Patients suffering from an array of rare conditions are now putting huge pressure on the FDA, hoping to shape deliberations about the risks and benefits of new medicines. That worries some experts who fear that emotional pleas from patients — or their mothers — could compromise scientific judgment.
Austin Leclaire, 16, makes his way through his apartment complex to the dog park, in Pembroke, Mass. Austin and his brother Max have Duchenne muscular dystrophy, and are taking an experimental drug that has had a positive impact on their quality of life.
KAYANA SZYMCZAK FOR STAT
Austin Leclaire, 16, makes his way through his apartment complex to the dog park, in Pembroke, Mass.
“The days of a controlled, experts-only system are fast ending,” said Daniel Sarewitz, a professor of science and society at Arizona State University. “On the whole, this is not a bad thing. But one can imagine things might get rushed through the regulatory process without adequate time to see all of the risks.”
Parents whose children have DMD, however, will tell you that there is no time to waste.

A DEVASTATING DISEASE

DMD is caused by an error in a gene that produces a protein called dystrophin. The gene is found on the X chromosome, so the disease primarily affects boys — up to one in every 3,500 inherit the mutation, causing about 20,000 new cases each year worldwide.
Without dystrophin, muscle fibers degenerate and are gradually replaced by fat and connective tissue until voluntary movement becomes impossible. By their teens, boys with DMD are usually confined to wheelchairs. Later, they are unable to breathe independently, and their life expectancy generally doesn’t run past 25.
The disease has no cure, and there are no drugs available to even slow the loss of muscle strength. For decades, there was little focus on drug development, as pharmaceutical companies focused primarily on chasing blockbusters for treating common ailments, such as diabetes, while ignoring rare disorders. But that all changed thanks to a confluence of recent events.
Notably, scientific advances recently identified ways to thwart the progression of DMD. The drugs up for review — drisapersen from BioMarin and eteplirsen from Sarepta — both rely on a technique known as exon skipping. The treatments coax the cell’s internal machinery to skip over sections of faulty genetic code. This in turn helps create a partially functional dystrophin protein. The goal is to create enough dystrophin so boys can regain mobility, or at least to slow the pace of their decline. (These two drugs, however, target only one of many coding segments of the dystrophin gene, and thus may help just the 13 percent of the DMD patient population who have mutations in this region).
But perhaps the biggest boost to drug development for DMD came on the regulatory front, when in 2012 President Obama signed into law the Food and Drug Administration Safety and Innovation Act. This allowed the FDA to accelerate approval when a drug satisfies an unmet medical need for a serious condition, and it empowered agency officials to rely on surrogate markers to green-light these kinds of drugs, rather than requiring evidence of actual clinical improvement. For Duchenne, a proxy could be increased dystrophin production.
“By doing that, Congress opened the door for patient involvement,” said Pat Furlong, chief executive of Parent Project Muscular Dystrophy, the largest DMD-focused nonprofit organization in the United States.
Founded in 1994, the PPMD more closely resembles a well-oiled advocacy machine than a grassroots collective. Thanks to its fundraising prowess, the organization has doled out grants totaling more than $45 million for DMD research to scientists and physicians. And its $6 million annual budget allows it to hire lobbyists and policy experts to woo lawmakers.
So it was not surprising that Furlong and her organization capably jumped through the door opened by Congress. With help from a bevy of scientific experts and industry representatives, PPMD crafted a set of guidelines in 2014 for developing drugs for DMD. The FDA even used the group’s regulatory guidance — the first-ever from a rare-disease patient organization — as a blueprint for its own draft recommendations, which were published this past June.
“We’ve met with numerous groups representing many different diseases, but they did something unique,” said Dr. Janet Woodcock, the FDA official who oversees drug approvals. “They were able to bring a whole community together.”
Still, even patient advocacy couldn’t always overcome the realities of drug development.

NO CAKEWALK

Consider eteplirsen, the drug being evaluated by the FDA in January. In 2011, the drug’s sponsor, Sarepta Therapeutics of Cambridge, Mass., was running out of money, and the company gambled it all on a very small study of just 12 young boys with DMD. The hope was that the boys given eteplirsen would show improvement in a six-minute walking test, a standard measure of physical function. Positive results could then be used to seek FDA approval and to raise funds for further drug development.
Jenn McNary, a “fierce mom” with two teenage boys with DMD, enrolled her younger son, Max, in the Sarepta trial. His mobility had been declining. Within a year of starting treatment, he was running with other children. During a recent afternoon in the family’s home on the south shore of Boston, Max, now 13, darted about the living room as he searched for a game to play.
Max Leclaire, 13, relaxes at home in his apartment.
KAYANA SZYMCZAK FOR STAT
Max Leclaire, 13, relaxes at home in his apartment.
After Max visibly improved, McNary also wanted to give the drug to her older son, Austin, but he hadn’t qualified for the same study because he was no longer ambulatory, which meant he couldn’t take a walking test. McNary figured her best chance of getting the drug for Austin, now 16, was to push the FDA to accept the 12-person trial as valid evidence for an accelerated approval.
“Austin was declining at a typical rate,” said McNary, who now works for the Jett Foundation, a nonprofit devoted to DMD awareness and research. “Meanwhile, I’m watching his brother get better on this drug and I just felt utterly hopeless.”
McNary teamed up with Sarepta officials, who announced in 2012 that the drug displayed two benefits in the 12-person trial. The first was that boys given eteplirsen for about a year could travel, on average, 220 feet further during the six-minute walking test than other boys who were given a placebo for 5.5 months before being switched to the Sarepta drug.
The other benefit, according to the published study data, was that eteplirsen increased the amount of muscle fibers containing the dystrophin protein by about 50 percent after a year of treatment. Sarepta has always argued this change in the surrogate marker should be seen as a welcome sign that its treatment can eventually restore mobility, although it has yet to prove this link.
But convincing the FDA to accept this data, or even the walking test results from such a small study, as grounds for market approval is easier said than done. “Any time you’re the first with FDA to see how it interprets legislation, they get nervous,” said Chris Garabedian, who led Sarepta until earlier this year. “We knew this would be a challenge.”
Initially, the FDA encouraged Sarepta to submit its limited trial data for review. But in late 2013, a bomb dropped: A 186-person trial that was testing drisapersen, the competing drug then being developed by Prosensa Therapeutics and its partner GlaxoSmithKline, failed. The drug did not produce a meaningful difference in a six-minute walking test compared with boys who were on a placebo for close to a year.

Listen to the Signal podcast: For boys with Duchenne, and two drug companies, a moment of shared hope

Glaxo ended its partnership with Prosensa, which was left to fund further development on its own. And FDA officials reversed course and told Sarepta its small trial was insufficient for regulatory submission. The chain of events was a one-two punch to families affected by DMD. Suddenly, two promising drugs held less potential.
“It makes me feel kind of mad,” said Austin Leclaire, McNary’s son, as he sat in his wheelchair and held one of his dogs in his lap. Austin eventually managed to get eteplirsen in a safety trial started last year for boys with advanced-stage DMD, but the frustration his family experienced at the time is still palpable. “The reason the FDA is there is to help people and to make sure they’re safe. But they got in the way.”

THE $840 MILLION GAMBLE

For months, the mood in the DMD patient community was grim. But in November 2014, BioMarin, a drug company in California that specializes in rare diseases, bought Prosensa for $680 million upfront, with the possibility of an additional $160 million if drisapersen gains approval.
Clearly, this was a gambit. But after Glaxo walked away, Prosensa mined its study data and found its drug appeared to be effective in younger patients if given over a longer period of time. BioMarin, drisapersen’s new owner, believed it could convince the FDA to review the drug on the basis of this subgroup analysis. (The company declined to comment for this story).
By then, the FDA was more willing to consider formal reviews for both DMD drugs. Agency officials recognized an opportunity to use a platform technology — in this case, exon skipping — to develop a new category of drugs. And patient groups had made their mark by pulling heartstrings, lobbying aggressively, and working with the agency on ways to advance drug development.
“There’s been an evolution in the four years I’ve been working on this,” said Ed Kaye, who had been chief medical officer at Sarepta and succeeded Garabedian as interim CEO in April of this year. “You had what was a fairly conservative FDA going head on with a very aggressive and liberal patient group. But there’s been a tempering on both sides. Patient voices were heard and patients learned about the regulatory view.”
Norah McNary, 4, James McNary, 8, Austin Leclaire, 16, Jenn McNary, and Max Leclaire, 13, look through a box of toys in their apartment. Austin and Max have Duchenne muscular dystrophy, and are taking an experimental drug that has had a positive impact on their quality of life.
KAYANA SZYMCZAK FOR STAT
Jenn McNary and her children look through a box of toys in their apartment. Austin and Max have Duchenne muscular dystrophy, Norah and James do not.
Neither eteplirsen or drisapersen is a shoe-in for approval, though. “Given the disease and the pressure FDA is under, they want to do a panel and show the world there is transparency,” said Simos Simeonidis, an analyst at RBC Capital Markets. “But this is not a slam dunk one way or the other.”
A key to agency thinking is likely to emerge in briefing documents that will be released prior to the BioMarin panel meeting next week. And a wild card in the approval process may turn out to be the patient groups.
Many parents are planning to attend the meetings next week and in January. Some are concerned that the FDA may eventually approve one drug but not the other. This could prove problematic for some families, because each drug maker is already studying other experimental agents that target different exons. If an FDA panel favors one medicine over another, parents fear this would cause setbacks for entire pipelines. So a very vocal outpouring is expected.
Patient groups are well-aware that the FDA is susceptible to patient outcries. In August, for instance, the agency approved Addyi, a drug to combat female sexual dysfunction, despite side effect issues and limited effectiveness. The approval followed sustained public pressure on the FDA. So the sight of youngsters with limited life spans in wheelchairs may tip agency officials and advisers toward approval.
At least that is what the parent groups are hoping.
“Ultimately, we want approval for all drugs,” McNary said. “I don’t just represent my kids. There are other kids waiting for one drug or the other._______________________________________________________________






I I I Received the following email below 
today from Jennifer Mcnary (See Below). 

previous story on this Blog dated
January 5, 2013 that told of her battle 
on behalf of her two sons. 

Some potentially positive events 
have subsequently taken place and 
Jennifer as explained in her e-mail. 
I wish her children all of the best in 
receiving the life sustaining care 
that they deserve.

Only one of my sons had access to a 

life-changing drug while his brother 

with the same disease got sicker. 

Now the drug could have FDA approval 

as soon as next year!


Jerry --
I wrote to you last December telling you
about my two sons who were both born
with life-threatening Duchenne's Muscular Dystrophy.

But only one of my sons, Max,
had access to an experimental treatment
that was drastically improving his life
while his brother Austin got sicker. 

I could not be happier to 
announce that just last week 
the drug company decided to 
file for approval of the
"miracle drug" Eteplirsen 
after promising talks with 
the FDA! 

This means that the drug 
could be available as soon 
as next year, and both my sons 
will soon have a chance at living 
a better and longer life.

The petition you signed helped the
drug garner unprecedented attention
from the FDA. I thought I would have
to wait for years for both of my sons
to have access to this life-changing
drugand stand by helplessly as one
of my sons thrived while the other
suffered silently.

But thanks to you, all children 
with Duchenne's will be more likely 
to have access to this breakthrough treatment 
as early as next year.

Austin wants supporters like you to
know that our work isn't done yet and
that he'll be happy when he is sitting
down at the doctor's office to receive
his first treatment. Max hopes that his
brother Austin will soon be stronger
like he is. 

I'll be keeping up the positive 
pressureon the FDA knowing 
that I have over 180,000 people 
standing behind me. 

Thank you so much for your help, this
couldn't have happened without you. 

Jennifer McNary
Saxtons River, Vermont
 -----------------------------------------------------------------------------------

SATURDAY, JANUARY 5, 2013


HELP SIGN PETITION FOR JENNIFER MCNARY MOTHER OF MAX AND AUSTIN

I received an e-mail below from Jennifer McNary Mother of Max and Austin. Please take a minute of your time and sign her worthy petition to the FDA. These are the "small" things that each of us can do to make a difference in the lives of others who need our help, in this case a simple signature in their support to keep her kids alive.


Both of my sons have the same debilitating disease  -- Duchenne Muscular Dystrophy -- that's kept them dependent on wheelchairs to get around.But now only one of my sons has access to a "miracle drug" that is saving his life.
Max was fortunate enough to take part in a study of a breakthrough medication, and now he can walk on his own for longer than he ever could. But Austin wasn't as lucky. 
Without access to this miracle drug, I watch Austin suffer silently as his brother thrives. The FDA has the power to make this drug available to kids like Austin by putting it through the "accelerated approval" program. It could otherwise take years for this important drug to be available to kids like Austin, denying him the same chance as his brother at a better and longer life.
Duchenne's is a disease that causes loss of muscle, to the point where children stop walking and eventually cannot breathe on their own. It is a slow death sentence with no effective treatment available. Watching Max make progress with this medication has been nothing short of a miracle, but bittersweet -- Austin grows steadily weaker with each passing day. 
Eteplirsen has helped one of my sons accomplish what I never believed possible. And this year, a law was passed that allows the Food and Drug Administration (FDA) to expedite the approval of experimental medications that have been proven to work.
The company that produces Eteplirsen is going to officially ask the FDA soon for accelerated approval because of its miraculous trial results. I am doing everything I can to make sure the FDA knows how crucial this drug is to the survival of my sons. But they need to know that the public supports an accelerated approval process too -- and since they have the power to act, your signature will add the pressure they need to move quickly.
Thank you for your help.
Jennifer McNary
Mother of Max and Austin
Saxtons River, Vermont


3 comments:

  1. Hi I do have a relative overseas suffering from the same and is deteriarating day by day.
    If you know any updates on this drug and the current status of these 2 kids of Jennifer, greatly appreciated.
    I would like to see if that clinical trials already ended or are they still recruiting candidates?

    Thanks, Ind
    ReplyDelete
  2. As of October 2015- The medication Eteplirsen mentioned in my post above is still being tested and has continued to show significant improvements in slowing down the debilitating effects DMD. The Company that makes this medication is Sarepta Therapeutics, Inc in Cambridge Mass. They are expecting FDA approval to make available this drug in early 2016. The latest update on Eteplirsen can be found at this link below:

    https://www.mda.org/media/press-releases/sarepta-announces-data-phase-2-trial-eteplirsen-treatment-dmd

    There is also another medication called KyndrisaTM (drisapersen) that is currently being reviewd for approval by the FDA- Made by a Company called BioMarin Pharma-The link to this drug update is below:
    http://investors.bmrn.com/releasedetail.cfm?ReleaseID=944400

    Other current DMD treatment options and the latest news on DMD reasearch can be found at the CureDuchenne website at this link below:

    http://www.cureduchenne.org/?referrer=https://www.google.com/

    Clinical Trial for DMD treatments are being done in many places, including at the link below for the Gene Institute Center at:

    http://www.nationwidechildrens.org/gene-therapy-clinical-studies--1

    Current Clinical Trials still recruiting patients for DMD Treatment are listed at below link:

    https://clinicaltrials.gov/ct2/results?term=+Duchenne+Muscular+Dystrophy+&Search=Search

    Jennifer Mcnary and her 2 children are alive and still fighting for accelerated research to treat DMD-For an update on them you can read my newest news on their fight that I have posted today in this blog post. I hope this helps. Oh, by the way, many of these medications for DMD are also being reviewed by the European Unions equivalent of the FDA, if your relative live in Europe, there is info related specifically to there in these links above. Good luck!

    ReplyDelete
  3. As of October 2015- The medication Eteplirsen mentioned in my post above is still being tested and has continued to show significant improvements in slowing down the debilitating effects DMD. The Company that makes this medication is Sarepta Therapeutics, Inc in Cambridge Mass. They are expecting FDA approval to make available this drug in early 2016. The latest update on Eteplirsen can be found at this link below:

    https://www.mda.org/media/press-releases/sarepta-announces-data-phase-2-trial-eteplirsen-treatment-dmd

    There is also another medication called KyndrisaTM (drisapersen) that is currently being reviewd for approval by the FDA- Made by a Company called BioMarin Pharma-The link to this drug update is below:
    http://investors.bmrn.com/releasedetail.cfm?ReleaseID=944400

    Other current DMD treatment options and the latest news on DMD reasearch can be found at the CureDuchenne website at this link below:

    http://www.cureduchenne.org/?referrer=https://www.google.com/

    Clinical Trial for DMD treatments are being done in many places, including at the link below for the Gene Institute Center at:

    http://www.nationwidechildrens.org/gene-therapy-clinical-studies--1

    Current Clinical Trials still recruiting patients for DMD Treatment are listed at below link:

    https://clinicaltrials.gov/ct2/results?term=+Duchenne+Muscular+Dystrophy+&Search=Search

    Jennifer Mcnary and her 2 children are alive and still fighting for accelerated research to treat DMD-For an update on them you can read my newest news on their fight that I have posted today in this blog post. I hope this helps. Oh, by the way, many of these medications for DMD are also being reviewed by the European Unions equivalent of the FDA, if your relative live in Europe, there is info related specifically to there in these links above. Good luck!