Allison Dsouza was unable to walk a short distance without becoming winded.

The Munhall resident’s trouble breathing led to a diagnosis of pulmonary hypertension, high blood pressure in the blood vessels in the lungs.

Pulmonary hypertension is a rare, incurable lung disease.

She has what’s called “idiopathic” pulmonary hypertension, which means there’s no known cause.

Her current treatment involves Remodulin, a prescription medication, that is delivered 24/7 by a pump under the skin. It’s a drug that works by widening the blood vessels and can help with symptoms of pulmonary hypertension.

But it doesn’t cure the condition.

Courtesy of Allison Dsouza Allison Dsouza of Munhall at the PHenomenal Hope 5K poses for a photo with nurse practitioner Jen Kliner and a former physician at UPMC, Dr. Patty George. The annual event on Pittsburgh’s North Side raises funds for research for pulmonary hypertension.  

Within months of starting treatment, Dsouza was able to walk in The PHenomenal Hope 5K and 1 Mile walk, an annual event to raise funds for research for pulmonary hypertension. She also has started playing polo.

“The treatments are phenomenal, but they’re also a huge burden on life,” said Dsouza, who was diagnosed as a high school senior. “They can be painful.”

They can be and there can be other complications, such as infection from those patients who are on an intravenous treatment, said Dr. Stephen Chan, professor of medicine and director of the Vascular Medicine Institute at the University of Pittsburgh and UPMC.

“The medication is lifesaving, but the side effects can kill you,” Chan said. “And for some people, the therapies don’t work.”

That’s why he is part of a team of researchers at the University of Pittsburgh School of Medicine and Prairie View A&M University in Texas who have been working to identify cancer drugs that show promise for treating pulmonary hypertension.

Pulmonary hypertension and cancer share numerous features so molecular data from cancer studies could be leveraged to predict which cancer drugs may also target pulmonary hypertension, Chan said.

A study published in Science Advances journal identified two candidate cancer drugs. They improved markers of the disease in human cells and rodents, Chan said.

The findings also support broader use of this drug-repurposing platform for other non-cancerous conditions that don’t yet have effective treatments, according to the journal.

The drugs targeted pathways involved in human lung cells. When researchers gave one of the drugs to rodents with pulmonary hypertension disease symptoms were reversed, Chan said.

“In fact when we gave either drug to rodents, pulmonary hypertension was reversed,” Chan said.

Courtesy of UPMC Dr. Stephen Chan, professor of medicine and director of the Vascular Medicine Institute at the University of Pittsburgh and UPMC, is part of a team of researchers looking at using cancer drugs to treat pulmonary hypertension.  

Repurposing drugs can cut downtime and the cost of developing treatments for rare diseases, said Chan.

“Pulmonary hypertension is an example of a rare disease where there is an unmet need for new treatments, given its devastating consequences,” said Chan. “The goal is to get treatments to patients faster.”

Pulmonary hypertension is thought to be triggered by environmental and genetic factors that damage the cells that line blood vessels, leading to inflammation.

Current medications dilate or relax these blood vessels, which can give relief from symptoms and prolong the time it takes for the disease to progress, but they’re not curative, Chan said.

Chan and Seungchan Kim, chief scientist and executive professor of electrical and computer engineering at Prairie View A&M, analyzed gene data from cancer cell lines exposed to cancer therapeutics and assessed rewiring of gene networks associated with drug responses in these cells.

“When we overlay these networks with pulmonary hypertension-specific gene networks, we can predict which drugs may be effective in treating pulmonary hypertension,” Kim said in a statement.

When Chan and Imad Al Ghouleh, Ph.D., assistant professor of medicine at Pitt, gave one of the genes to mice and rats with pulmonary hypertension, disease symptoms were reversed.

Researchers have applied for a provisional patent and plan to move into clinical trials.

According to Chan, the applications of this study go far beyond pulmonary hypertension. They may be able to repurpose existing cancer drugs for the treatment of other rare and emerging diseases.

Dsouza, 24, is a registered nurse in the cardiac Intensive Care Unit at UPMC Children’s Hospital of Pittsburgh in Lawrenceville. Prior, she had cared for pulmonary hypertension patients in the UPMC Lung Transplant Program at UPMC Presbyterian in Oakland.

“Allison is young and vibrant,” said Chan. “She has so much life ahead of her and we want to help patients like Allison.”

Dsouza said she tries to do as much as she can, and her diagnosis is a reason she became a nurse.

“It’s all worth it to me,” she said. “I love working with patients, especially those who have pulmonary hypertension because I know what they are going through. I love being a part of their lives and helping them. Dr. Chan and all the researchers are wonderful. You don’t find many doctors who know about this. Their work is helping people like me breath a little easier.”

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