Pediatric Rare Disease Group Works To Advance Clinical Research Bill

August 25, 2012

FDA Week | August 23, 2012 | By Nanci Bompey

Bipartisan legislation aimed at expanding the National Institutes of Health’s investments in pediatric research through the creation of pediatric research consortia could work with newly enacted provisions in the FDA Safety and Innovation Act aimed at accelerating drug approvals, according to an advocacy group spearheading the effort. FightSMA, the group backing the legislation, is pushing for the House Energy and Commerce Committee to take up the bill when it returns from August recess.

The National Pediatric Research Network Act, introduced by Reps. Cathy McMorris Rodgers (R-WA) and Lois Capps (D-CA), directs NIH to establish a National Pediatric Research Network consisting of up to 20 pediatric research consortia over a five year period that would be eligible for awards, including grants, for basic, clinical, behavioral or translational research to meet unmet pediatric population needs, including rare diseases.

The legislation is aimed at improving clinical trials to quickly move promising drugs to approval, said Steven Eichenauer, a partner at Public Strategies Washington, who is working with FightSMA to pass the legislation.

Eichenauer added the bill would bolster research that could utilize the recently expanded accelerated approval pathway, a provision in FDASIA. He said the National Pediatric Research Network Act would help put drugs in the pipeline that can then be sent to FDA for approval, providing “a supply of newly minted therapies that have shown promise in clinical trials and are now before FDA.”

The bill would ensure these consortia focus on conducting or coordinating multisite clinical trials for pediatric rare diseases, and would establish a data coordinating center to distribute the findings, including to FDA. The consortia would be modeled after the National Cancer Institute centers and would ensure funds are dedicated exclusively towards basic and translational pediatric research and would enable inter-institutional networking, according to FightSMA. The group works to accelerate the search for a treatment and cure for spinal muscular atrophy (SMA), the leading inherited cause of infant death, according to the group.

Martha Slay, co-founder of FightSMA, said the group decided to take a broader approach with the currently introduced legislation than it has in past efforts, which focused specifically on spinal muscular atrophy.

“I consider this a really historic moment for SMA and other pediatric rare diseases, going from obscurity to a place where something significant and material could be done to make clinical trials available to children who desperately need it,” she said.

Reps. Diana DeGette (D-CO), Gregg Harper (R-MS) and Peter King (R-NY) are cosponsors and a companion bill in the Senate was introduced by Sens. Sherrod Brown (D-OH) and Roger Wicker (R-MS), and is co-sponsored by Sens. Sheldon Whitehouse (D-RI), John Kerry (D-MA), Richard Blumenthal (D-CT) and Mark Begich (D-AK).

Eichenauer said the group is pressing for consideration of the bill by the House Energy and Commerce Committee when lawmakers return to Washington in September as several sponsors sit on the panel. He said committee staff have been involved in writing the legislation and it has been vetted by the committee. “The committee knows the bill well,” he said. Further, FightSMA said House Majority Leader Eric Cantor (R-VA) is a longtime supporter of their cause.

Eichenauer said the group hopes the House could approve the legislation on the suspension calendar and then the bill could come before the Senate. Some cosponsors of the bill also sit on the Senate health committee, which FightSMA has also been working with on the legislation. Eichenauer added the group doesn’t know if the bill will move on its own or will be attached to a bigger package. Sources have said moving any legislation before the elections could be difficult.

Eichenauer said sponsors of the measure are exploring the possibility of a score from the Congressional Budget Office, but said it would not affect FightSMA’s ability to move forward with the bill. The legislation could also face difficulty with the looming “fiscal cliff.”

“There is no question that federal funding is going to be a challenge now and in the coming fiscal year,” Eichenauer said. “One of the chief advantages (of the bill), is that it does use federal dollars in a more highly-leveraged way. It takes infrastructure that is already there and expands it to leverage those dollars smartly.”

FightSMA is working over the recess to gather support in Congress and among other groups, with the Coalition for Pediatric Research, the National Down Syndrome Society and the Parent Project Muscular Dystrophy endorsing the bill. Eichenauer said sponsors have discussed reaching out to other groups, including the National Organization for Rare Disorders.

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FightSMA Announces Introduction in the House of Representatives of the National Pediatric Research Network Act (NPRNA)

July 21, 2012

FightSMA Announces Introduction in the House of Representatives of the National Pediatric Research Network Act (NPRNA)

Press Release | July 20, 2012

ALEXANDRIA, VA. July 20, 2012—A bill to authorize the National Institutes of Health (NIH) to support the creation of up to 20 pediatric research consortia focused on diseases such as spinal muscular atrophy (SMA) was introduced yesterday in the U.S. House of Representatives.

The bipartisan bill, called the National Pediatric Research Network Act (NPRNA) is being introduced by Representatives Cathy McMorris Rodgers (R-WA) and Lois Capps (D-CA). It is designed in part to help achieve the goal of securing additional federal resources to accelerate clinical trials for spinal muscular atrophy (SMA).

“We are thrilled about this bill which is the result of the dedication of so many in the SMA community,” said Martha Slay, founder and immediate past president of FightSMA. “Legislation to benefit SMA is at the heart of the FightSMA mission.”

“Over the past year, FightSMA has worked with our champions in the Congress, Representatives McMorris Rodgers and Lois Capps, on this important legislation. With the support of longtime FightSMA ally House Majority Leader Eric Cantor (R-VA), these two key members of the House Energy and Commerce Committee’s Subcommittee on Health have collaborated to craft bipartisan legislation that will advance translational research and clinical trials for a variety of rare pediatric disorders, with SMA serving as a model disease,” said Ms. Slay.

“I am proud to join Rep. Capps in introducing the very important National Pediatric Research Network Act. She has been an instrumental leader in the fight to promote pediatric research for genetic disorders, especially spinal muscular atrophy and Down syndrome,” said Rep. McMorris Rodgers. “As the mother of a son with Down syndrome, I am confident this legislation will go a long way to improving the lives of those with genetic disorders. I look forward to working in a bipartisan way to make sure this bill is signed into law.”

“I am proud to co-author this legislation with my friend (Rep.) Cathy McMorris Rodgers, which would go a long way to increasing and improving research on children’s illnesses–especially rare and complex diseases–and developing new treatments to fight them,” said Congresswoman Capps. “Every parent’s worst fear is that their child becomes sick, and we owe it to all parents to do what we can to fight childhood illnesses. I would also like to thank my constituents, Bill and Victoria Strong, for their tireless work on behalf of their daughter, Gwendolyn, and all children with Spinal Muscular Atrophy and other rare diseases. Their dedication to fighting this terrible disease has been an inspiration for me and a driving force behind this bill,” said Congresswoman Capps.

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FightSMA Names New Executive Director; Relocates Headquarters to Nation’s Capital

January 23, 2012

FightSMA Names New Executive Director; Relocates Headquarters to Nation’s Capital

Press Release | January 18, 2012

FightSMA, an international nonprofit organization created to strategically accelerate the search for a treatment and cure for spinal muscular atrophy (SMA), has named Ron Imbach as its new Executive Director, effective Dec. 1, 2011.

Ron Imbach is replacing the current CEO and founder of FightSMA, Martha Slay, who guided the organization from its inception in 1991 until the present. Under Martha’s leadership, FightSMA has grown from an initial crusade to bring higher levels of awareness and understanding of spinal muscular atrophy (SMA) to the public to a leading advocate and research-centered organization focusing on the treatment and cure of the disease and support for the families of children with SMA.

“For the past 20 years, Martha Slay has led FightSMA with great passion, intelligence, and dedication; the SMA community owes her a great deal of gratitude for her accomplishments,” said FightSMA Chair Mike Calise. “She has been a great inspiration to us all personally and professionally.”

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theGSF and FightSMA Award $100,000 Spinal Muscular Atrophy Research Grant

December 16, 2011

theGSF.org | December 15, 2011

The Gwendolyn Strong Foundation (theGSF) and FightSMA are excited to announce a $100,000 award to Dr. Monique A. Lorson and University of Missouri in support of Dr. Lorson’s research focused on developing a large animal model of Spinal Muscular Atrophy (SMA).

Currently, no large animal model of SMA exists and the goal of Dr. Lorson’s research is to develop the first ever pig based model of SMA to be used by the broad, global SMA research community and other research groups to allow them to more efficiently and effectively move promising research from the bench to bedside. In short, this is a very exciting program that has the potential to have a material, positive impact on the future of SMA research in almost every category (e.g. gene therapy, compounds, antisense oligonucleotides).

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Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

July 25, 2011

Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

Press Release | July 25, 2011

‘Biggest increase anyone has seen’ in the production of essential compound for deadly childhood disease

OTTAWA – July 25, 2011 – Researchers in Ottawa report new hope for the treatment of infants born with serious genetic disorder.

Over 1000 children in Canada are affected with Spinal Muscular Atrophy (SMA), a genetic disorder that causes muscle weakness and loss of motor control. In its most severe form survival of children with SMA beyond 5 years is rare. Although the disorder is caused by the loss of a specific gene, all infants and children with SMA have an untouched highly similar gene within their genetic make up. Activation of this copy gene has the potential to treat SMA, and thus has been a goal of researchers around the world. Now, researchers at the Children’s Hospital of Eastern Ontario Research Institute in Ottawa report the strongest such activation yet observed with attendant benefit on mice genetically engineered to have SMA.

PhD student Faraz Farooq working in the laboratory of University of Ottawa professor, Alex MacKenzie has discovered that the pregnancy hormone Prolactin, a Canadian discovery in itself, not only activates the copy gene but if given over time extends the lifespan of SMA mice by up to 60%. The research report is published today in the Journal of Clinical Investigation.

“Prolactin causes a dramatic regulation of copy gene SMN2 which results in high production of SMN protein, resulting in the extension in the lifespan of mice with SMA,” said Mr. Faraz Farooq. “Labs around the world have been trying to produce more protein from copy gene SMN2 but with Prolactin (an insulin like protein) we’re seeing up-regulation that’s more than tenfold. It’s the biggest increase anyone has yet seen in the SMA Field with any potential therapeutic compound. This represents a significant advance in search for a therapy for this disease.”

The laboratory testing of Prolactin on SMA not only shows an extended lifespan but also improved motor control. Prolactin has been used in clinical trials for unrelated studies, so it is expected that the path between pre-clinical validation and actual clinic trials of Prolactin with SMA patients will be reasonably short.

“News of prolactin’s role and effectiveness in SMN regulation breathes fresh hope into all of the SMA community,” said Martha Slay, president and co-founder of FightSMA. “FightSMA congratulates Dr. MacKenzie and his colleagues on this exciting breakthrough in SMA research.”

“We believe we’re moving in the direction of an effective pre-symptomatic treatment of kids with SMA,” said Dr. Alex MacKenzie, principal investigator, CHEO Research Institute. “We want to somehow stop the progress of this disorder in its tracks, and let our tiniest patients build strength. Today’s findings are not curative, but we think this is a breakthrough discovery. Hopefully by using different approaches to increase SMN protein we can develop a combination therapy for the treatment of SMA.”

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About the CHEO Research Institute: Established in 1984, the CHEO Research Institute coordinates the research activities of the Children’s Hospital of Eastern Ontario (CHEO) and is one of the institutes associated with the University of Ottawa Teaching Hospitals. The Research Institute brings together health professionals from within CHEO to share their efforts in solving paediatric health problems. It also promotes collaborative research outside the hospital with partners from the immediate community, industry and the international scientific world.

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FightSMA to host panel discussions on spinal muscular atrophy

April 30, 2011

FightSMA to host panel discussions on spinal muscular atrophy

News Medical | April 30, 2011

International non-profit organization FightSMA will be hosting highly respected experts from across the country on Thursday, May 5, 2011 for a series of panel discussions, Thriving with SMA: LIVE! This panel is a unique, one-of-a-kind series of presentations from influential leaders in the fields of pulmonary issues, orthopedics, nutrition, neurology and clinical trial.

FightSMA will be joined by families, friends, healthcare professionals and more for this historic presentation. In addition, the panel will be offered online with free and unlimited access via the web broadcasting service, UStream. The panel will be live broadcast from 9:00am EDT to 2:00pm EDT on the FightSMA website (www.fightsma.org). Attendees can register for the webcast, as well as submit questions for the panelists by visiting the FightSMA website and pointing towards the “Celebrating 20 Years” block.

“There are hundreds of families across the U.S. and across the globe who simply cannot physically attend the conference for various reasons,” said FightSMA President and founder, Martha Slay. “So, on May 5, we’re bringing the conference to them! Families will be able to ask questions of SMA experts in the areas of nutrition, exercise, pulmonary issues, and clinical trials. This is unprecedented and it’s free, and can be attended from your own computer. Go to www.fightsma.org for more details.”

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FightSMA Celebrates 20 Years Fighting Spinal Muscular Atrophy; Gears Up for Annual Conference in DC

April 29, 2011

FightSMA Celebrates 20 Years Fighting Spinal Muscular Atrophy; Gears Up for Annual Conference in DC

PR Newswire | April 28, 2011

FightSMA will be welcoming elite researchers and scientific professionals, as well as families and friends in the SMA community, to the L’Enfant Plaza Hotel May 3-6, 2011 in Washington, D.C. The group, which was founded in 1991, is celebrating its 20th anniversary this year. To date, FightSMA has raised more than $7 million for SMA research and boasts 19 chapters in the U.S. and Canada.

The centerpiece of the Conference will be the Thriving with SMA: LIVE! panel discussions on Thursday, May 5, a unique, one-of-a-kind series of presentations from influential leaders in the fields of pulmonology, orthopedics, nutrition, neurology and clinical trial. In addition, FightSMA will be offering free and unlimited access to the Thriving with SMA panel via the web broadcasting service, UStream. The panel will be live broadcast from 9:00am EDT to 2:00pm EDT on the FightSMA website (www.fightsma.org). Attendees can register for the webcast, as well as submit questions, for the panelists by visiting the FightSMA website and pointing towards the “Celebrating 20 Years” block.

Click HERE or on the image below to read more…