Normalcy is hard to achieve when the Kennedys go out. Brielle and Brooke turn heads, and not just because of their wheelchairs or leg braces. The little girls with curly blonde hair have a certain presence about them. They are polite, courteous, happy and sweet. Brielle, despite her own challenges dealt by SMA, takes care of her younger sister.

"When we go out, people just want to talk to us," Eric said. "The girls allow us to meet so many more people than we ever would have otherwise."

That, in turn, becomes an opportunity for Eric and Sarah to educate others about SMA.

The Kennedys and other advocates for SMA research and treatment say it is a disease that with proper funding could yield a cure. But because SMA affects far fewer lives than many other diseases, it is not a prime candidate for funding, they say, and because pharmaceutical companies won't make a lot of money off of it, drug development falls by the wayside.

While the future remains uncertain for the Kennedys, there is hope. Physical therapy has allowed Brooke and Brielle to make improvements to their physical health, despite their degenerative disease.

For these golden-haired little girls who love Disney princesses more than just about anything, all that their parents ask is a chance at a happily-ever-after ending to the story.

-Written by Josh Mauser for The Kalamazoo Gazette, find the entire article here.

 

A dream is a wish your heart makes.
— Cinderella


House overwhelmingly passes bill to speed FDA drug approvals

By Carolyn Johnson July 10  

A bipartisan bill that would make significant changes to the process for developing new drugs and medical devices overwhelmingly passed the House in a 344-77 vote Friday morning.

The bill, called 21st Century Cures, was cheered by rare across-the-aisle support from politicians, with 230 co-sponsors nearly evenly split between Democrats and Republicans. The pharmaceutical industry, patient advocacy groups, and medical organizations also support the bill, which calls for an additional $8.75 billion for the National Institutes of Health.

"This bill is about making sure our laws, regulations, and resources keep pace with scientific advances," House Energy and Commerce Committee chairman Fred Upton (R-Mich.) said in a statement.

The bill tries to address the impatience that stems from a major societal problem: despite billions of dollars of research into diseases that range from common cancers to the rarest genetic diseases, we still lack treatments for thousands of conditions.  Many of its provisions seek to make the drug approval process less burdensome.

But its laundry list of provisions that tweak the process for approving new drugs or devices have raised significant concern from industry watchdogs and physicians who say the legislation is aimed more at helping drug and device companies than patients. Critics say the bill's regulatory alterations do not address the real problem with the development of new therapies and could lead to the approval of treatments that don't work and could even harm vulnerable sick people.

"The bill unfortunately offers a horse trade," said Vijay Das, a healthcare policy advocate at Public Citizen, a patient advocacy organization. "It increases funding for the world-renowned NIH in exchange for providing perks for the pharmaceutical and medical device industries."

The White House issued a statement that welcomed the additional support for biomedical research, but flagged the part of the bill that would extend a company's exclusive right to market brand name drugs if they are repurposed to treat a rare disease. The Congressional Budget Office estimated that this could increase spending on prescription drugs by $869 million from 2016 to 2025.

"The Administration remains concerned about extending drug exclusivity... and how this provision will affect drug costs," the statement said.


Brielle (left) and Brooke Kennedy of Mattawan, Mich. have Spinal Muscular Atrophy and helped trigger the 21st Century Cures legislation. (Courtesy of Sarah Kennedy)

The passage of the bill brings hope, however, to many people who have watched desperately ill family members struggle with devastating diseases that may have no effective treatments or cures.

Sarah Kennedy was glued to the television in Mattawan, Mich. as the House took its vote. Her experience as a parent illustrates the powerful emotional pull of a bill that promises to increase the development of new treatments for diseases that today have little hope. For years, Kennedy has navigated the frustration and uncertainty of a terrifying disease with no known cure. Her daughters, Brielle, 7, and Brooke, 6, both suffer from Spinal Muscular Atrophy -- a genetic disease that attacks the nerve cells behind voluntary movements. The disease has no cure and eventually robs children of the ability to walk, eat, and breathe.

In many ways, Kennedy's daughters have a life like other kids. Brielle and Brooke are happy and joyful. They love everything Disney, navigate the world in their wheelchairs, take the bus to school, and have tons of friends.

But they are also taking a drug originally developed and approved for seizures that comes with serious potential liver side effects. Although the drug, valproic acid, hasn't been proven and approved for their disease, it seems to help both girls retain their strength and ability to control their movements.

Brielle "had trouble even lifting arm above her head and after we started her on the valproic acid, she could lift her hand above her head," Kennedy said. "We felt right then, 'OK. We can see a difference. Let’s get on it, let’s try it.'"

For Kennedy, as for any parent, it's hard to give children drugs that carry major risks or come with side effects. As a baby, Brooke was on a clinical trial for an experimental drug that tasted so bad that she stopped eating. Eventually, a feeding tube need to be placed. But in their quest to find ways to give their daughters extra strength and more time, the Kennedys are willing to accept far more risk than if they were simply trying to treat an ordinary disease of childhood, like an earache or sore throat.

"When you’re given life or death, you want to do anything you can to give them life," Kennedy said.

While these types of patient experiences are powerful, policy experts worry that such arguments will be used to erode a carefully-wrought standard of evidence that today helps to avoid the tragedies of the past, such as the birth defects caused by thalidomide in the 1960s and infertility caused by the Dalkon intrauterine device in the 1970s.

"We share Congress’ desire to increase funding for NIH, but there are dangerous parts of this bill that many members of Congress did not fully understand," Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank, said in a statement. "As often happens, well-funded pharma lobbying was more effective than experts’ concerns about patient safety."

One part of the bill that has generated discussion among physicians are changes intended to speed the development of new antibiotics. These allow evidence that would be considered preliminary data today to be considered in the approval of new drugs for a limited population of patients. It also includes a provision that would incentivize the use of new antibiotics by increasing the payments hospitals receive from Medicare for the drugs. To many scientists, this was seen as a problem because overuse of antibiotics has created a crisis of infections that aren't vulnerable to any treatments.

Elizabeth Jungman, director of public health programs at the Pew Charitable Trusts, said that allowing drug approvals for narrower groups of people -- for example people with drug-resistant pneumonia instead of all pneumonia patients -- could help solve that problem. She added that specific language in the bill ensures that the safety and efficacy standards are not lowered for antibiotics.

An amendment that passed Thursday, proposed by Rep. Louise Slaughter (D), called for a study on whether those extra payments resulted in the development of pathogens resistant to those new drugs.

"It's foolish to pay someone to create more antibiotics if they get frittered away the same way," Slaughter said in an interview.

The Senate is working on a parallel track and the Health, Education, Labor and Pensions Committee headed by Sen. Lamar Alexander has held four hearings on the initiative. The hope is to have legislation through the committee by the end of the year, a committee aide said.


Carolyn Johnson is a reporter covering the business of health. She previously wrote about science at The Boston Globe.

http://www.washingtonpost.com/blogs/wonkblog/wp/2015/07/10/house-overwhelmingly-passes-bill-to-speed-fda-drug-approvals/


Vote for U.S. Congressman Fred Upton!

I Can and I Will!  

WATCH this 60 second commercial for U.S. Congressman Fred Upton featuring Brooke and Brielle. 


August is Spinal Muscular Awareness Month in the State of Michigan!

Aug SMA 2014

Thank you Senator Tonya Schuitmaker for delivering Senate Resolution No. 166 to our house today! This resolution declares the entire month of August as SMA Awareness month in the state of Michigan!! It was adopted by the Senate on July 16, 2014.
The Resolution was offered by Senators Schuitmaker, Anderson, Bieda, Hildenbrand, Kowall and Proos. Thank you!! 


A Family's Love: Mattawan family looks to defy odds in fighting rare muscle disease afflicting 2 young sisters.

Kalamazoo Gazette Article: Written by Josh Mauser  December 31, 2012

I can and I will.

This is the mantra that has driven the Kennedy family to find normalcy in an unforeseeable future.

Eric, Sarah and their two daughters, Brooke, 4, and Brielle, 5, are the epitome of an American family.

Eric Kennedy is a health care provider for Bronson Family Practice in Mattawan, a recently activated flight surgeon for the Michigan National Guard, an outdoors enthusiast and a true family man. Sarah is a stay-at-home mom who cares for Brooke and Brielle.

But life is a lot more complicated for the Kennedys than first appears.

When Brielle was around 12 months old, Eric and Sarah noticed some things thatdidn't seem quite right.

"We thought that she was just slow to walk or slow developing, physically," Sarah said.

Sarah, Eric and Brielle's pediatrician weren't particularly alarmed at first. But then, when she was about 16 months old, came the devastating diagnosis that Brielle had type II spinal muscular atrophy.

"SMA is the leading genetic killer of infants and young children. It is a terminal, degenerative progressive disease that results in the loss of nerves in the spinal cord and the weakness of the muscles connected with those nerves. SMA impacts the ability to walk, stand, sit, eat, breathe and even swallow," according to brookeandbrielle.com, a website set up by Sarah Kennedy.

The family learned of Brielle's diagnosis soon after Sarah had given birth in December 2008 to their second daughter, Brooke, and less than a week before Eric was to be deployed with the National Guard to Afghanistan.

Eric was unable to get out of his tour of duty and was off to Afghanistan five days later, reluctantly leaving Sarah alone with Brielle and their newborn daughter, Brooke.

"It was really tough for us when they told us the diagnosis," Eric said.

One in 40 people carries the gene responsible for SMA, and there is often no family history of the disease. Eric and Sarah were unknowing carriers, giving their second daughter a 25 percent chance that SMA would also affect her.

An early diagnosis for Brooke was key for her development, and the family was eager to have her tested. Sarah broke the news to Eric over the phone -- Brooke, too, had tested positive for SMA. 

Read the entire article here: http://www.mlive.com/news/kalamazoo/index.ssf/2012/12/a_families_love.html


A 60 second commercial done by the Kennedy Family - raising SMA awareness.

Rep. Fred Upton has always been open and willing to meet with us and listen to our needs.

Congressman Upton is the US House of Representatives Chairman of the Energy and Commerce Committee. In September of 2012, he spoke on the House floor in support of getting the National Pediatric Research Network Act (NPRNA) passed and talked about meeting with our family and how much our story of SMA has touched him. His leadership was critical to its passage in the House. NPRNA is a bipartisan pediatric research consortia measure that would benefit multiple rare diseases including SMA. It will help us put in place urgently needed clinical trial infrastructure so we can move several promising therapies to FDA for approval and increase federal funds for SMA research.

Congressman Upton asked our family if we would help with a commercial spot for him. So, of course, we were willing to help out!  

We spent a few hours filming and I just spoke candidly from the heart. I almost wish I had cue cards because I was so nervous!   

From that interview, they formed an amazing 60 second commercial that I just saw on tv for the first time this morning!! It just brought tears to my eyes. It’s huge awareness for SMA! Watch for us on on the local channels!


    Cheff Therapeutic Riding Center Newsletter- Discovering Diagnoses

fall2011-1.jpg

Thursday, December 8, 2011

As you probably already know, Brooke and Brielle both take Hippotherapy lessons at the Cheff Therapeutic Riding Center.  They were selected for the first “Discovering Diagnoses” article for the front page of Cheff’s newsletter!  The article explains what SMA is and how the Cheff Center has helped us in our journey.  We hope the newsletter spreads awareness and ecourages people to take action to help find a cure for SMA! 

Read the entire newsletter here

We helped the Cheff Center with a video for one of their fundraisers.  You can see how much Brooke and Brielle enjoy riding.


Our family was invited to the Michigan Capitol Building by our Representative Aric Nesbitt last week to hear him introduce House Resolution No. 118 on the house floor!  It was such an amazing experience!  Rep. Nesbitt gave us a personal tour of the historical building and his desk on the House Floor.  We were also able to view the Senate Floor, Supreme Court room and the House Appropriations room. After our tour was over, Rep. Nesbitt took us up to the viewing gallery in the House of Representatives room.  He told us to wait there as he left to check in for attendance and the session began.  He introduced House Resolution No. 118 - A Resolution To Declare August 2011 As Spinal Muscular Atrophy Awareness Month In The State Of Michigan.  After he read the resolution, he introduced our family and my mom, Liz.  Then the Representatives on the floor stood up, turned around, clapped and waved to us!  Brooke and Brielle loved this part and excitedly gave their best Princess wave and said, “Hi” right back.  What an awesome moment! 

It was such an honor to be introduced on the house floor for H.R. No. 118 and we are so happy about spreading the awareness about Spinal Muscular Atrophy.  We would like to say a huge thank you to Representative Aric Nesbitt and his Aide Ryan for inviting us to come out for the day and give us this amazing opportunity!