Westley has been diagnosed with Duchenne Muscular Dystrophy.

Archive for July, 2013

Hot Off the Presses

Last week I announce that their was rumor of a new clinical trial coming soon that would help Westley. I wanted to share the 1st article I have read concerning this trial.

Its real, its happening and its happening in Westley’s lifetime!!!

Jessica

 

http://quest.mda.org/news/dmd-sarepta-submit-new-drug-application-fda-eteplirsen-2014

DMD: Sarepta to Submit New Drug Application to FDA for Eteplirsen in 2014

Drug may be eligible for the agency’s accelerated approval pathway if dystrophin levels are accepted as an endpoint; confirmatory trial also planned for 2014

Article Highlights:
  • Sarepta Therapeutics, developer of eteplirsen to treat Duchenne muscular dystrophy (DMD), was told by the U.S. Food and Drug Administration on July 23, 2013, that the agency would accept a new drug application from Sarepta for eteplirsen, but that it could not commit to reviewing the drug under the agency’s accelerated approval program at this time.
  • The accelerated approval program allows a surrogate endpoint, such as dystrophin levels in muscle biopsy samples, to substitute for an endpoint that reflects true clinical benefit, such as increased life span or improved function.
  • Sarepta says it will conduct a large-scale, confirmatory (phase 3) trial of etepliren in 2014 even if the FDA does not require one.
  • Eteplirsen is designed to treat DMD that is amenable to treatment by skipping exon 51 of the dystrophin gene. Sarepta plans to target exons 44, 45, 53 and 55 next.

 

by Margaret Wahl on July 24, 2013 – 8:48am

In a July 24, 2013, press release and conference call, biotechnology company Sarepta Therapeutics provided an update on the regulatory pathway for eteplirsen, the company’s experimental drug for Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene.
DMD: Exon-Skipping Timeline Laboratory development of exon skipping began in the 1990s with significant funding from MDA.

Read more
DNA

Sarepta CEO Chris Garabedian said the company will submit a new drug application (NDA) for eteplirsen to the U.S. Food and Drug Administration (FDA) in the first half of 2014, based on a July 23 meeting the company had with the agency.

However, Sarepta does not yet know whether eteplirsen will be eligible for the FDA’s accelerated approval program.

Accelerated approval allows a drug to be conditionally approved, based on a surrogate endpoint and pending results of a large-scale, confirmatory trial. A surrogate endpoint is a measurement that “stands in” for a clinical benefit and usually can be arrived at faster than a clinical benefit endpoint, such as an increase in life expectancy or improvements in function.

A possible surrogate endpoint for eteplirsen’s benefit is the level of the dystrophin protein present in the muscle biopsy samples of trial participants. (An absence of dystrophin protein is the underlying cause of DMD.)

Sarepta reports that the FDA would not commit to declaring dystrophin an acceptable surrogate endpoint prior to the company’s submission of an NDA for eteplirsen.

“We are very excited about this announcement,” said Jane Larkindale, MDA vice president of research. “By the end of 2014, we could have the first drug specifically for DMD approved by the FDA! This is an important step forward toward that goal. Importantly, although eteplirsen would only treat about 15 percent of boys with DMD, if approved it may open a pathway for more rapid approval of other drugs for DMD, both additional exon-skipping drugs and potentially other types of drugs.”

She added, “MDA’s advocacy team has supported the development of accelerated approval policy through the years, and we will continue to support its use in the development of drugs for our diseases.”

Possible next steps by FDA

After the FDA receives the new drug application from Sarepta, it may:

  • allow the company to market eteplirsen prior to conducting a confirmatory trial, with the stipulation that full approval will be contingent on the outcome of that trial (i.e., allow the company to use the accelerated approval pathway, with dystrophin level as a surrogate endpoint);
  • allow the company to market eteplirsen without conducting any additional trials (i.e., grant full approval for the drug); or
  • require additional information from current and future trials before allowing Sarepta to do any marketing of etepliren.

Phase 3 trial planned for 2014

During the July 24 conference call, Garabedian said Sarepta plans to conduct a large-scale, confirmatory (phase 3) trial of eteplirsen in 2014, regardless of whether the FDA requires such a trial for approval of the drug.

He said the company will need the data from this larger trial as it moves forward with its program of exon-skipping drugs for DMD that target additional dystrophin gene exons, including 44, 45, 53 and 55.

He also said Sarepta is considering enrolling patients with DMD who may benefit from skipping these other exons but would not be expected to benefit from skipping exon 51, to serve as a control group in the confirmatory eteplirsen trial. Normally, the control group would consist of participants who could benefit from skipping exon 51 but would be treated with a placebo.

Garabedian explained that enrolling participants who could not benefit from skipping exon 51 but who could potentially benefit from skipping other exons would:

  • provide a control group for the eteplirsen trial, since these participants would, in effect, be “untreated”;
  • allow all patients with dystrophin mutations amenable to exon-51 skipping to receive eteplirsen, rather than requiring some to receive a placebo; and
  • provide information about patients with mutations amenable to skipping exons 44, 45, 53 or 55, which are future targets for Sarepta’s DMD drug development program.

About eteplirsen

Etepirsen is an exon-skipping drug that targets a part of the dystrophin gene known as exon 51. In about 15 percent of boys with DMD, skipping exon 51 with a drug like eteplirsen theoretically should result in production of functional dystrophin.

For more information

The July 24, 2013, conference call will be available for replay at Events and Presentations on the Sarepta site.

To learn more, also read Exon Skipping in DMD: What Is It and Whom Can It Help? and A Snapshot View: Drisapersen and Eteplirsen, which provides information about the two exon-skipping drugs farthest along in development for Duchenne muscular dystrophy.


Halleluiah!!!

Halleluiah!!! I just got an “insider tip” that a drug trial that covers Westley’s exon mutation SHOULD be approved within 6-12 months!! This is exciting times!!! Tearing up with JOY!!!

This drug trial will tell Westley’s body to skip the mutated exon allowing the DNA strand to start making the dystrophin his body needs. In some way you have all made this possible! All your prayers and fund raising is allowing for more and more effective drug trials to come our way. THANK YOU for being a Warrior!

Please continue to pray for this trial to move forward, for the scientists and all involved and if it is His Will for Westley to participate in this study.

Also keep Dallas in your prayers as he raises money for his trip to Honduras next month and for the safety of his team.

http://www.gofundme.com/3kk5j0

 

God is Good! Today is a day to rejoice!

 

God Speed,

Jessica


Honduras or Bust!

Hello All!

Dallas has been presented with an opportunity to travel to Honduras to aid the sick. With no cure for Westley he has been feeling helpless and I believe God has placed this in front of him to help him fill a void in his heart.

I took a mission trip to Uganda at 17 and it was an experience like no other. I too want Dallas to experience this life changing event to help heal his heart.

We all know trips like this can be expensive but I know God will provide. If you feel it in your heart to give please use GoFundMe below.

http://www.gofundme.com/3kk5j0

 

FROM DALLAS

To my friends and family,

Recently I was invited to join a small group of people for the chance of a lifetime trip.  I have the opportunity to travel to Honduras and assist a Mountain Medical Missions team with treatment and care of children in the area.  The group I am traveling with is called the Friends of Barnabas Foundation Inc. ( http://www.fobf.org) Each year, the Friends of Barnabas Foundation sends eleven Mountain Medical Mission Teams to Honduras.  FOBF staff members select communities in need and travel to clinic sites weeks ahead of the team to notify community leaders of the team’s arrival and their services.  The teams then travel throughout the impoverished areas of central Honduras and serve between 1,500 and 2,000 people in their traveling clinics.
FOBF Mountain Medical Mission Teams provide primary and preventative healthcare.  They set up anti-parasite & Vitamin A stations as well as general medicine, eye, and dental clinics in small schools and churches.  The majority of the patients have never seen a healthcare provider.
Each team is comprised of 4-5 medical professionals, 2-3 Spanish speakers, and the remaining team members have a heart for serving their fellow man.  Teams work side by side with FOBF staff members in Honduras and are a key component to the model of healthcare FOBF strives to promote.
It is my plan to travel with them and assist where I can.  I do not speak Spanish and I have no medical training to provide.  What I do have is a loving heart and strong back.  As you already know, my son Westley suffers from a disease in which there is no cure.  As a father, this leaves me feeling helpless at times.  This presents a chance for me to help others in need.  While this will be a very rewarding experience for the children of Honduras and me, there is a great cost associated with this trip and I’m asking for your help.  The FOBF is a non-profit organization that works solely on volunteers and donations.  In order to make this trip a reality for me, I need to raise just under $2000.  This will pay for my flight, land transportation while there, lodging (Tents), and food.  Some of the money goes to pay for medical treatments and medicine for the children.
It would mean the world to me if you can find it in your heart to help me fund this opportunity of a lifetime.

Sincerely,
Dallas