In late June I wrote about a 13-year-old boy who was waiting for a double lung transplant. Today I am happy to say that on Monday July, 28^th Nick LeGrand got his wish, and received a new pair of lungs! Nick and his mother have been living in Houston away from the rest of there family awaiting a transplant since late December.

Nick underwent the long 13 hour procedure at Texas Children’s Hospital in Houston late Monday into Tuesday. His father Mark said, “Things went as good as could be expected.” His parents are undoubtedly excited, but remain cautious as the next 24 to 72 hours are crucial. Doctors are keeping Nick sedated for up to two days in order to allow his body to slowly become accustomed to the lungs. Nick will remain hospitalized for three to six months post transplant and his father hopes to join his son and wife in a couple of weeks. Once Nick has recovered enough to travel, he will finally be able to return home to his family.

This is Nick’s second transplant due to the rare lung disease called primary pulmonary hypertension. Doctor’s had hopes the first transplant would grow with Nick but they were unfortunately compromised by chronic rejection, and his condition gradually worsened. His quality of life was substantially restricted preventing Nick from attending Eleanor Roosevelt Middle School, and also confining him to a motorized scooter. Nick’s body will hopefully accept the transplant this time around allowing him to return home a far healthier 13-year-old boy.

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According to a recent French study, patients with pulmonary arterial hypertension that have germline mutations in the gene encoding bone morphogenetic protein receptor 2 (BMPR2) present at an earlier age and with more severe hemodynamic compromise.

The University of Paris study conducted by Dr. Marc Humbert and his colleagues screened 223 consecutive patients with PAH for point mutations and large size rearrangements of BMPR2. They focused on the clinical, functional and hemodynamic characteristics between carriers and non-carriers. The investigators identified 68 carriers of the BMPR2 mutation. Forty had idiopathic PAH and 28 had familial PAH. The 155 non-carriers all had idiopathic PAH.

The team reported in the June 15 issue of the American Journal of Respiratory and Critical Care Medicine, that carriers were nearly 10 years younger at diagnosis than non-carriers, with mean ages of 36.5 years and 46.0 years, respectively. Also carriers had a shorter time to death or lung transplantation and a younger age at death than non-carriers.

“Despite major advances in the treatment of PAH, the pathogenesis of this condition remains obscure,” Dr. Lewis J. Rubin of the University of California at San Diego writes in an accompanying editorial. He adds, “Patients and physicians will be best served by referral to expert centers, where collaborative and translational efforts will further our understanding of this condition and lead to more effective treatments in the future.”

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One of the world’s largest research and advisory firms focusing on pharmaceutical and healthcare issues, Decision Resources, finds that a therapy that improves exercise capacity by a greater percentage than Tracleer at sixteen weeks, would earn a forty-eight percent patient share in the treatment of pulmonary arterial hypertension. According to pulmonologists, improving exercise capacity remains an opportunity that is clinically relevant and that has also been incrementally improved by recently launched therapies.

The drug Novartis’s Gleevec will earn Decision Resources’ clinical gold standard status in the treatment of pulmonary arterial hypertension. Gleevec has competitive advantages in the amount of safety and delivery over the current clinical gold standard. Gleevec’s efficiency will be similar to that of Flonan but not greater.

Pulmonologists hope that Gleevec will at least emulate Flolan’s ability to prolong survival and improve the patients’ functional capacity. Unfortunately, there is no clinical data on this drug at this time, yet the drug has markedly improved six-minute walking distance and functional case reports of severe pulmonary arterial hypertension when other therapeutic regimens failed.

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When his wife was diagnosed with pulmonary arterial hypertension in 2006, Joe Haan never questioned whether he’d be able to take care of her. “We’re a team. When you get sick in marriage, it’s through sickness and health,” Joe Haan said. “I just thought, things will be OK. We’ll make the best of it.” Cindy and Joe have been married for many years and have three beautiful children – Samantha age 8, Matthew age 6, and Jade age 4.

In late June, Joe was named the Pulmonary Hypertension Association’s Outstanding Caregiver, which is an award presented to someone whose efforts are focused on the needs of a loved one with the condition. With the help of family and friends, Joe has been able to care for his wife from the beginning of her diagnosis – whether it is providing medication to helping her complete everyday tasks to being there for emotional support. Cindy’s original prognosis has improved, which demonstrates that love and support can help people through their toughest times.

Joe has recently spoken up about the benefits of the Pulmonary Hypertension Association conference. He and Cindy began attending when she was newly diagnosed. He recalls, “[i]t’s a great opportunity to meet the ones still living with the condition. It gave us both a lot of hope… You hear and see a lot of positive things instead of maybe learning the negative ones from the internet.” The conference allows people to hear inspirational stories from others going through similar experiences with PAH, rather than just focusing on all of the negatives surrounding the disease.

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In June 2007, the FDA approved ambrisentan for the once-daily treatment of pulmonary arterial hypertension to improve exercise capacity and delay clinical worsening.

Ambrisentan is a valuable addition to the treatment alternatives for this disease. It includes prostacylin, inhaled nitric oxide, iloprost, sildenafil, and conventional Ca+ + channel blockers. It is taken orally and is similar to the existing drug, bosentan. Both of these drugs are competitive inhibitors of endothelin receptors; however bosentan lacks the receptor selectivity of ambrisentan.

Ambrisentan exhibits one-hundred times greater selectivity for endothelin receptor A (ETA) as compared to endothelin receptor B (ETB). The moderate selectivity for the ET receptor may offer therapeutic advantages that bonsentan lacks.

This drug is not recommended for use in patients with moderate or severe hepatic impairment, because it may cause additional liver damage. Ambrisentan, “is an FDA category X pregnancy drug and is contraindicated in women who are or who may become pregnant.” In addition women who are breast feeding should avoid ambrisentan.

This drug is relatively new and does carry the chance of side effects, yet serves as a helpful addition to the list of drugs that help treat PAH.

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Researchers have been making headway in finding new ways to treat Pulmonary Arterial Hypertension. They currently are testing the affects Adenosine has on PAH. Adenosine is a building block of DNA and a nucleoside composed of a molecule of adenine attached to a ribose sugar molecule.

One of the most useful affects Adenosine has is that it may prevent or inhibit body tissue damage. Regarding PAH, researchers believe it can possibly prevent further damage from occurring in the pulmonary articles. In some animal studies, it has shown the ability to repair injured body tissue. Ongoing trials are studying the usages of adenosine in regulating vascular tone in cases where pulmonary arterial hypertension has already taken place.

Adenosine is not the only treatment being studied in the treatment of PAH; researchers are also studying the effects of calcium channel blockers on PAH, both by itself and in combination with adenosine.

Researchers continue to find new and different ways to help battle this devastating disease. I hope sometime soon they will find a definitive way to cure this disease.

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