Posts Tagged ‘Rare Disease’

Pulmonary Arterial Hypertension Model Targets Disease Reversal

Posted by CorDynamics on February 13th, 2013

Finding Treatments for an Orphan Drug

We have added a powerful capability to our most requested small animal pulmonary arterial hypertension model – measuring pulmonary artery pressure via single or dual channel telemetry.

The hypoxia-semaxanib rat assessment has rapidly become the most requested assay among our Pulmonary Arterial Hypertension offerings. PAH — an orphan disease, with unmet medical treatment—is characterized by increased blood pressure in the arteries of lungs, causing dizziness, shortness of breath and can lead to heart failure.

CorDynamics was the first laboratory to report the protective effects of bosentan and sildenafil in this model.

In an effort to refine the data, we have now added the option to include daily readings of pulmonary artery pressures.

 View Data

Why is this important in the discovery of new therapeutics for PAH?

While a terminal assessment of pulmonary pressure provides end-stage confirmation of efficacy, interim measurements of the PAH temporal course can be critical when timing the initiation of reversal therapy.

Our daily data demonstrate that the variability associated with the model is small, even over six weeks of hypoxia. Animals progressed through development of PAH in a similar linear fashion.  After six weeks in hypoxia, systolic pulmonary pressure increased greater than 400% versus baseline. In addition, right ventricular hypertrophy was robust. The low variability helps in the assessment of test article efficacy – and allows for better distribution of dose levels without having to add inordinate experimental numbers.

As we approach another celebration of World Rare Disease Day on February 28th it’s gratifying to have something new to report in the campaign to develop an orphan drug for PAH.

 

Filed under: Drug Discovery Services, Pulmonary Arterial Hypertension, Telemetry | No Comments

American College of Cardiology Heads to Chicago

Posted by Theresa Gralinski, Marketing Director at CorDynamics on March 26th, 2012

Big City, Big Advantages for Drug Discovery and Development as American College of Cardiology heads to Chicago.

The heart of our business is in the heart of the city of Chicago.

A contract research organization specializing in cardiovascular drug discovery, safety and efficacy in Chicago? Why Chicago?

We believe the city of Big Shoulders gives us big advantages.

Proximity to world-class universities, thought leaders and talent—The University of Chicago, University of Illinois at Chicago, Northwestern University, Loyola University and DePaul.

Access to O’Hare International Airport and daily flights to almost anywhere in the world.

Centrally located in the Midwest for access to clients on both Coasts and worldwide.

Deep roots in the community, where we are proud to attend and sponsor events— a runner in the recent Shamrock Shuffle as well as in the 15th Chicago Half Marathon held in September; Dollar Denim Days in celebration of World Rare Disease Day; the Rock for Rare disease Jesse Malin benefit concert.

Not to mention, Chicago is a great place to welcome guests and colleagues.

If you’ll be in town for the American College of Cardiology Annual Conference, or any other time, let us know. We’d love to meet with you, give you a tour of our facilities and show you some of our favorite Chicago hot spots.

 

Filed under: About Us, Drug Discovery Services, Drug Safety Services, Events, Facility | No Comments

World Rare Disease Day

Posted by Michael Gralinski, Chief Executive Officer at CorDynamics on February 29th, 2012

The Future Looks Bright

It’s World Rare Disease Day and supporters of all ages are coming together to raise awareness. From my point of view, the next generation makes the future look bright.

Today, I find myself back at the University of Michigan Medical School talking with pharmacology graduate students. Many of these future researchers will dedicate their careers to developing the very drugs and therapies needed to help those struggling with unmet medical needs.

My seminar is entitled World Rare Disease Day: Current Pharmacological Targets and Animal Models of Pulmonary Arterial Hypertension.”

I talk about my Michigan history and the career path that took me from grad student, to post doc, to industry researcher and ultimately entrepreneur. From the CorDynamics point of view, I discuss the extensive preclinical and discovery studies done for clients researching treatments for pulmonary arterial hypertension—an orphan disease.

Meanwhile, back in Illinois, kids at Spencer Loomis Elementary School are helping other kids in the hopes of eradicating some of these diseases in their lifetimes. For Dollar Denim Days, the students are purchasing denim ribbons as well as wearing their favorite pair of jeans in a nod to the Global Genes Project. The proceeds will go toward research on Rett Syndrome, a rare genetic disorder of the nervous system and the diagnosis given to the 3-year-old daughter of a former teacher at the school.

Inspired, CorDynamics pledged to match the funds raised by the Spencer Loomis Elementary students.

In the interest of full disclosure, the teacher was my oldest son’s kindergarten teacher and my youngest son still attends the school. Not to mention, Rett Syndrome is often associated with a dangerous heart condition that we regularly research—long QT syndrome.

Odds are, most of us are connected to one of the nearly 30 million Americans affected by a rare disease. From one generation to the next, I believe these connections will lead to solutions and cures.

Filed under: Press Releases, Pulmonary Arterial Hypertension | No Comments

The Rare Disease Connection

Posted by Theresa Gralinski, Marketing Director at CorDynamics on February 24th, 2012

In honor of RARE Disease Day on February 29th, CorDynamics was looking for a way to contribute, participate and make a difference.

Little did I know the ideal opportunity was right in my backyard. I would learn that I was connected to it in more ways than one.

Connection 1: My youngest son came home from school excited about “Dollar Denim Days” in honor of RARE Disease Day and in support of the Global Genes Project. The Spencer Loomis Elementary students would make and purchase denim ribbons as well as wear their favorite pair of jeans to school.

Connection 2: Two Hearts Rock, the non-profit organization we collaborated with in July for The Beat Batten & Rock Rare Disease concert given by Jesse Malin, was orchestrating the Dollar Denim Days event.

Connection 3: The proceeds would go toward research on Rett Syndrome, a rare genetic disorder of the nervous system and the devastating diagnosis given to Amelia “Emmy” Foster, the 3-year-old daughter of Colleen Foster, who was a former teacher kindergarten teacher at the school. My oldest son had been one of Mrs. Foster’s students.

Connection 4: Upon further investigation, I learned Rett Syndrome robs children of their verbal and motor skills and is often associated with a dangerous heart condition that CorDynamics regularly researches—long QT syndrome.

Connection 5: CorDynamics jumped on board and pledged to match the funds raised by Spencer Loomis Elementary students in their Dollar Denim Days fundraiser next week.

With millions of Americans suffering from rare diseases, these connections are everywhere—perhaps in your backyard too.

Make the most of February’s bonus day on the 29th…get connected to Rare Disease Day.

 

Filed under: Press Releases | 2 Comments

FDA Orphan Drug Designation

Posted by Michael Gralinski, Chief Executive Officer at CorDynamics on January 30th, 2012

There are currently about 7,000 orphans out there waiting to be adopted, to be supported, to be cured. Many of us don’t even know their names: Kawasaki. Porphyria. Pulmonary Arterial Disease.

Orphan Status is a designation given by the FDA to identify rare diseases with unmet medical needs but with potentially promising compounds currently being researched. Orphan drugs receive expedited FDA reviews and fast tracks to clinical trials, providing hope for cures, treatments or comfort.

>>>>>  Nearly 2,400 experimental compounds have orphan drug designations.

>>>>>  So far, only 367 of these orphan drugs have been approved by the FDA.

Experts agree, coordinated collaboration and resources on all fronts will be the key to finding a potential drug therapy for these orphan drugs. Collaboration is also essential for the rare diseases still searching for enough answers to even begin the orphan drug development stage.

You may ask, what can I do to help? What can my organization do?

Adopt An Orphan

  • Pick an orphan disease with a compound in the works or a rare disease to adopt.
  • Show your support and learn more about the disease, follow related organizations on Facebook or in the news.  One that we follow on Facebook is Two Hearts Rock and Global Genes Project.
  • Donate your time, participate in a sponsored event, or fundraiser.
  • “Wear That You Care” on World Rare Disease Day, February 29th. Wear “jeans” for “genes” and encourage your workplace, schools, and sport teams to do so too to raise awareness for the Global Genes Project.

At CorDynamics, we sponsored a fundraising event for Hope4Bridget and Batten Disease (a rare disease) as well as continue to work with our clients on promising compounds for the potential treatment of Pulmonary Arterial Hypertension (an orphan designated rare disease).

Working together, we will continue to adopt more of these orphan diseases and meet more unmet medical needs. To learn more about this issue and how you can help, jump into today’s blog hop sponsored by the R.A.R.E Project.

 

Filed under: Drug Discovery Services, Drug Safety Services, Events, Pulmonary Arterial Hypertension | 4 Comments

Producing PAH Results: Elaborating on Collaborating

Posted by Theresa Gralinski, Marketing Director at CorDynamics on December 02nd, 2011

Drug development researchers have been putting a lot of emphasis on collaborating in the effort to produce safe and effective drugs as quickly as possible. This is especially true in the area of rare disease research.

The CorDynamics team collaborates day in and day out—with each other, with colleagues, with our clients and their project teams. But collaborating is kind of like parenting, you think and hope you are doing it well but there isn’t a definitive gauge to tell you if you are making progress.

Well lo and behold, a tangible affirmation of collaboration was recently celebrated in our lab. CorDynamics Senior Scientist Dr. Liomar Neves, with our colleagues at Corridor Pharmaceuticals, demonstrated the novel serotonin receptor antagonist C-122 prevents monocrotaline induced pulmonary arterial hypertension (PAH) in rats in a paper published in the European Journal of Pharmacology.

PAH—an orphan disease, with unmet medical treatment—is characterized by increased blood pressure in the arteries of lungs, causing dizziness, shortness of breath and can lead to heart failure. Current therapies for pulmonary artery hypertension (PAH) improve longevity and performance of daily activities in the lives of PAH patients, but do not notably affect the disease processes that lead to morbidity and mortality. Since serotonin has a definitive role in the development of PAH, the authors hypothesized that interfering with serotonin function may reduce the vascular remodeling and hemodynamic changes that occur in this preclinical model.

With the industry continuing to embrace a collaborative environment, I look forward to more research results that will ultimately provide more treatment options for patients in the future. As far as my kids and my parenting skills go, I guess I’ll have to assume no coal in their stockings means I’m doing something right and continue to believe.

 

Filed under: Drug Discovery Services, Publications, Pulmonary Arterial Hypertension, Uncategorized | No Comments

Adopting Orphan Diseases and Drugs

Posted by Michael Gralinski, Chief Executive Officer at CorDynamics on August 29th, 2011

What Does it Mean to Drug Developers?

Orphan Status is a designation given by the FDA to identify diseases with unmet medical needs and promising compounds in these areas with the potential of providing cures, treatments and comfort.

From a business standpoint, the numbers shouldn’t be ignored. From the patient perspective, the need is paramount.

  • More than 7,000 diseases have orphan status.
  • An estimated 25 million Americans are afflicted with an orphan disease.
  • Nearly 2,400 experimental compounds have orphan designations.
  • So far, only 367 of these drugs have been approved.

For those of us involved in drug development, an orphan drug designation is a game changer and for patients it’s a cause for hope. Orphan Drugs receive expedited FDA reviews and fast tracks to clinical trials. It’s a process that’s been in place since the 80′s and has been used often in cancer trials. With an emphasis on collaboration, researchers are pooling resources and findings on other types of rare diseases as well.

At CorDynamics, we’ve done extensive preclinical and discovery studies for clients researching treatments for pulmonary arterial hypertension—an orphan disease. PAH is characterized by increased blood pressure in the arteries of lungs, causing dizziness, shortness of breath and can lead to heart failure.

On the charitable front, CorDynamics is sponsoring an event this month in the hopes of raising funds for Batten Disease— another devastating orphan condition.

Working together, we continue to strive to help our clients adopt more of these orphan diseases and meet more unmet medical needs.

Filed under: Drug Discovery Services, Pulmonary Arterial Hypertension | No Comments

New Drug Therapy Would Give Hope4Bridget and Batten Disease

Posted by Theresa Gralinski, Marketing Director at CorDynamics on May 01st, 2011

A rock concert for rare diseases? What’s to sing about?

Hope.

The Beat Batten & Rock Rare Disease concert given by Jesse Malin and the St. Marks Social in Chicago on July 23 will celebrate hope for awareness, new drug discoveries and specifically Hope4Bridget.

Bridget Kennicott was a spirited, rambunctious three-year old with delayed speech. Nothing rare about that. However her mother, an elementary school teacher and a breast cancer survivor, sensed there was something more. Then the seizures hit. After a marathon of doctors Bridget’s final diagnosis was devastating—Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) often referred to as Batten Disease. The children slowly deteriorate; the disease is fatal; there is no cure. Research is ongoing and some experimental drugs are currently in a limited clinical trial.

Through friends of friends, we heard about Bridget’s story and have followed her family’s journey to find treatments, causes, comfort and when all else fails—hope.

Developing lifesaving drug therapies for diseases of all magnitudes is what drives us day in and day out at CorDynamics. So why not carry that tune into our nightlife and back the band—so to speak?

Join us. If not that night, please join us in spirit, dedication and most of all—hope. Because where there is hope, there is always a glimmer of joy.

Filed under: Drug Discovery Services, Events | No Comments