Posts Tagged ‘Orphan Drugs’

PAH Spotlight: Small Species with Dual Pressure Telemetry

Posted by CorDynamics on July 23rd, 2013

by Dr. Michael Gralinski, CorDynamics CEO

We continue to expand our capabilities in pulmonary arterial hypertension, and one thing is for certain: surgically instrumenting rats to measure systemic blood pressure AND pulmonary artery pressure—simultaneously—is an effective model for clients researching both prevention AND intervention of PAH.

To obtain continuous recording of pulmonary artery pressure, a telemetry catheter is placed into the pulmonary artery via the right ventricle. Post-recovery, the subjects are then exposed to classic PAH initiation agents such as monocrotaline or newer methods like hypoxia/semaxanib.

Measuring multiple pressures simultaneously allows our experts to differentiate test article effects on the pulmonary vasculature from those on the systemic circulation.

Up until recently, this level of in vivo telemetry instrumentation was only available in large animal models.

View Data Set #1

 View Data Set #2

Efficiency All-Around

At the risk of being obvious, the ability to measure multiple pressures clearly saves time and effort all around.

In addition, our low technical failure rate and clinical observations consistent with the development of robust pulmonary hypertension makes this an efficient option in terms of technician time as well as overall study deliverables.

As always, our models are designed with the 3R’s in mind. Collecting multiple variables from the same experimental subject reduces the need for redundant groups when separate pressures are interrogated.

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

New Model for Investigating Orphan Drug PAH

Posted by Michael Gralinski, Chief Executive Officer at CorDynamics on May 09th, 2012

There is still a lot to learn and discover about Pulmonary Arterial Hypertension (PAH).

It’s something CorDynamics has been developing in our laboratories. We were pleased to share the resulting PAH model with fellow researchers in a poster presented at last month’s Experimental Biology 2012 conference.

Our poster: Bosentan and Sildenafil Reduce Pulmonary Arterial Hypertension in Rats Induced by Semaxanib and a Low Oxygen Environment.

What did we show?

Oral administration of bosentan and sildenafil reduce PAH induced by semaxanib and a low oxygen environment. We demonstrated that this is a clinically relevant model to evaluate efficacy of test articles.

How did we do it?

It occurred to us that a commercially available hypoxic tent could be turned into a low oxygen chamber that mimics PAH. It worked. From there, we were the first to use bosentan and sildenafil (two approved clinical treatments for pulmonary hypertension) to validate the reduction of PAH in this model.

The result is a new model that is predictive of the human PAH condition.

Continually moving toward finding treatments for PAH—one of the 6,000 orphan diseases—is the wave of the future.

Our belief was underscored this week with GBI Research predicting orphan drugs will represent a significant revenue increase over the next five years—increasing its market share from $2.3 billion in 2010 to $6 billion in 2018.

More importantly, the millions affected by these diseases will have hope for life-improving treatments.


Filed under: Pulmonary Arterial Hypertension | No 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

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