Research In Gene Therapy: Hope for PAH Patients

Research news and clinical drug trial news often bring hope to our patients. By following late-breaking research news, FLASS keeps you up to the minute concerning new discoveries in lung diseases and treatments.

Mount Sinai announces new research in gene therapy for PAH.
Research News: Gene Therapy Might Aid in Treating Pulmonary Arterial Hypertension. 

Remember, at any given time, a pulmonary condition could affect you or someone you love. However, research is unlocking new discoveries every day. Thus we see hope in the future to balance the practical concerns of fighting chronic disease in the present.Now this is especially true for our Pulmonary Arterial Hypertension patients.

As of July 30, some major research studies have reached some encouraging conclusions. If you have been following our blogs, you know we have been explaining some of the symptoms and treatments for Pulmonary Arterial Hypertension.

The famous Cardiovascular Research Center at Mount Synai broke the news of some very promising research on PHA treatment. We also hope you will take a moment to visit our special informative page on this condition under Our Specialties.

Late Breaking News On a Hot Topic In Pulmonary Medicine:

Roger J. Hajjar, MD, Director of the Arthur & Janet C. Ross Professor of Medicine and Professor of Gene & Cell at Icahn School of Medicine at Mount Sinai and his team recently discovered an amazing application of gene therapy to pulmonary arterial hypertension. On July 30, they announced their that inhalable lung gene therapy could very possibly “restore the function of a crucial enzyme in the lungs.” This would mean the treatment could actually “reverse the damage of deadly PAH.”

Research that Could Help 150,000 Americans per Year

There is no doubt about the killer potency of Pulmonary Arterial Hypertension (PAH), which strikes up to 150,000 Americans each year. At the clinical level doctors such as those here at Florida Lung, Asthma, and Sleep Specialists crave more treatment options for PAH. We also think that reversing a condition like PAH would be a dream come true.Reversing the damage of this disease also would have been beyond the scope science fiction only a few years ago.

However, the science of gene therapy stretches over a vast region of unknown medical potential. Thus, we eagerly await the advances of the 21st century in lung disease research. So let’s take a look at some of the science behind the recent announcement of helpful findings.

1.      The Medication and A New Delivery System of Delivery

In addition to the study utilizing a gene therapy drug, we are impressed that the medicine is delivered in a convenient
manner.  The gene therapy is sprayed directly into airways and lungs by a patient-controlled inhaler.

Research shows new inhalation method for administering gene therapy.
Research shows gene therapy through inhalation could be come common treatment.

Most patients would agree that this would be a very convenient way to control their disease, far preferable to injections, Intravenous Drugs, or even pills. At FLASS our asthma patients are well acquainted with this method of delivering medication right where it is needed.

2.      The Research Study

In their experiments, as reported in the July 30 issue of the journal Circulation, the research scientists actually reversed PAH in rat models of the disease. The next tests will be on larger animal models as well as tissue samples.

3.      A Look Under the Microscope

The inspiration for the research of this new gene therapy began when scientists noticed PAH patient’s lung tissue samples had less than necessary amounts of the enzyme SERCA2A.

By the way, you need this enzyme in order to perfectly pump the exact amount of calcium a cell requires. Plus, SERCA2 directs the calcium precisely into the calcium compartment of each cell.  Read more about heart disease and SERCA2A at the online resource for The Journal of Circulation .

A SERCA2A  Summary:  Gene Therapy With A Purpose

“Put simply, When SERCA2A is down-regulated, calcium stays longer in the cells than it should.” Then, to make matters worse, the calcium facilitates the overgrowth of new and enlarged cells. According to researchers, the delivery of the SERCA2a gene produces some very special enzymes. These enzymes help both heart and lung cells restore their proper use of calcium.  In Dr. Roger J. Hajjar’s words, “The gene therapy could be delivered very easily to patients through simple inhalation, just like the way nebulizers work to treat asthma…”

Today’s Research is Tomorrow’s Miracle

Here at FLASS, our own Dr. Fortune Alabi agreed with the findings, and remarked, “It would be wonderful if this new therapy works in human drug trials, and becomes available to patients with Pulmonary Arterial Hypertension.”  Like the leader of the recent research project at Mount Sinai, FLASS realizes that, for many patients, a deadly clock is ticking like a countdown as they deal with PAH. For this reason, Dr. Hajjar added, “We are excited about testing this therapy in PAH patients who are in critical need of intervention.”

Tried and True Treatment:  SERCA2A Lung Research Echoes Congestive Heart Failure Findings

Not surprisingly, the same SERCA2A dysfunction described above also occurs in congestive heart failure. The new Sinai study utilizes the same gene therapy scientists are testing in some heart patients.  The researchers’ goal is to reverse congestive heart failure. The scientists are already receiving positive feedback in large phase III clinical trials in both the United States and Europe. Results in the heart test studies are very encouraging and the researchers are very optimistic. If this aspect of the SERCA2A studies interests you, and you are ready for some challenging reading, check out the recent findings in the Journal of Phisiology.

The question that only time can now answer is:  Will the inhalable gene therapy restore diseased lungs and stop that deadly count-down on the critical PAH clock?

Gene Therapy Research:  Turning Back The Clock on Damaged Lung Cells

Dr. Hajjar believes it can.
He stated, “We are delighted with these new findings because it suggests that a gene therapy is already showing great benefit in congestive heart failure patients.” This is a strong reason Dr. Hajjar thinks the same type of treatment and delievery technique might be able to help PAH patients.Like the doctors here at FLASS, Dr. Hajjar never loses sight of the patient’s welfare. Maintaining a patient-centered approach can be difficult due to the meticulous detail of scholarly research.

Research in Pulmonary Arterial Hypertension brings us hope for patients of all ages.
Research makes it possible for people with chronic lung disease to breathe easier.

Dr. Hajjar added that today’s PAH patients “…have no good treatment options — and are in critical need of a life sustaining therapy.”

News Flash!  Just as we were publishing this blog article, FLASS discovered that the National Institute of Health has already approved human trials for the SERCA2A inhalable gene therapy for PAH. They will begin in the near future.

For the patients, doctors, and families at FLASS, it cannot happen too soon.

The doctors and staff at FLASS genuinely thank you for reading our blog.  We hope you will return often to see what’s new in pulmonary diseases and treatments. Have you noticed our new look here at the online source for Florida Lung, Asthma and Sleep Specialists?  We’re streamlining our website. Watch for new patient-friendly features and informative pages. We personally invite you to click on our pages and take a good look at the world of pulmonary medicine. FLASS brings you precision medicine that puts patients first.

Leave a Reply