Scientists tested whether two common medications could prevent serious damage to the main blood vessel leaving the heart (called the thoracic aorta). These drugs—cilostazol and sildenafil—are already used to treat other blood vessel problems and heart conditions in people. Researchers gave mice a chemical that damages the aorta, then treated some mice with these drugs to see if they would help. Surprisingly, neither drug prevented the aorta from becoming damaged, forming dangerous bulges, or rupturing. While this is early research in mice, it suggests these particular drugs may not be useful for preventing this type of life-threatening heart condition.

The Quick Take

  • What they studied: Whether two medications (cilostazol and sildenafil) could prevent or slow down damage to the thoracic aorta—the large blood vessel that carries blood from the heart to the rest of the body.
  • Who participated: Laboratory mice that were given a chemical substance to damage their aortas. The researchers then treated some mice with the test drugs while others received no treatment.
  • Key finding: Both cilostazol and sildenafil failed to prevent aorta damage, aneurysm formation (dangerous bulges in the vessel), or rupture in the treated mice compared to untreated mice.
  • What it means for you: This research suggests these two drugs may not be effective for preventing thoracic aorta problems, though more research in humans would be needed to confirm this. If you take either of these medications for other conditions, this doesn’t mean you should stop—talk to your doctor about your specific situation.

The Research Details

This was a laboratory study using mice to test whether two existing medications could prevent aorta damage. The researchers first used a technique called RNA sequencing to look at which genes were turned on or off in damaged aortas. They discovered that genes related to both test drugs were activated when the aorta was damaged, suggesting the drugs might help. Then they tested this theory by feeding mice either cilostazol or sildenafil mixed into their food while also exposing them to a damaging chemical. They measured whether the drugs were actually present in the mice’s blood and then observed whether the drugs prevented aorta problems.

The researchers used mass spectrometry (a technique that identifies chemical substances) to confirm the drugs reached the mice’s bloodstreams at adequate levels. They then carefully monitored the mice for signs of aorta damage, including rupture (bursting) and aneurysm formation (bulging). This approach allowed them to test whether the drugs’ presence in the body translated to actual protection against aorta damage.

This type of study is important because it tests drug candidates in living organisms before considering human trials. However, results in mice don’t always translate directly to humans, so negative findings like these need to be confirmed with additional research.

Testing potential treatments in animal models is a crucial step in drug development. When researchers find that genes related to a drug’s action are activated in a disease, it suggests the drug might help. This study is valuable because it shows that even when the genetic signals suggest a drug should work, it may not actually prevent the disease in practice. This helps researchers avoid wasting time and resources pursuing ineffective treatments and guides them toward more promising approaches.

The study has several strengths: the researchers confirmed the drugs actually reached the mice’s bodies through blood testing, they used modern genetic analysis techniques, and they measured multiple outcomes (rupture and aneurysm formation). However, the abstract doesn’t specify how many mice were used, which makes it harder to evaluate the statistical power of the findings. The study was published in PLoS ONE, a peer-reviewed journal, which means other scientists reviewed the work before publication. The main limitation is that this is animal research, and drugs that don’t work in mice sometimes do work in humans, and vice versa.

What the Results Show

The key finding was that neither cilostazol nor sildenafil prevented the chemical-induced damage to the thoracic aorta in mice. Both drugs failed to stop aneurysm formation (the dangerous bulging of the aorta wall) and did not prevent aortic rupture (the vessel bursting). This was surprising because the researchers’ initial genetic analysis showed that genes related to these drugs’ mechanisms were activated in damaged aortas, suggesting the drugs should theoretically help.

The researchers confirmed that both drugs successfully entered the mice’s bloodstreams at measurable levels, so the lack of benefit wasn’t due to the drugs not reaching the target tissue. This means the drugs were present in adequate amounts but still didn’t provide protection against aorta damage.

These results suggest that even though the genetic signals indicated these drugs might work, the actual biological processes causing aorta damage are more complex than the genes alone suggested. The drugs may target only one small part of a larger problem, or they may work through mechanisms that don’t apply to this particular type of aorta disease.

The initial genetic analysis revealed that both cilostazol and sildenafil-related genes (Pde3a and Pde5a) were upregulated in the aorta before visible damage appeared. This suggests these genes play a role in the aorta damage process. However, the fact that blocking these genes’ effects (through the drugs) didn’t help indicates that simply turning off these genes isn’t enough to prevent the disease. This finding highlights that aorta disease involves multiple interconnected biological pathways, not just one simple mechanism.

Previous research had suggested that cilostazol and sildenafil might help prevent abdominal aortic aneurysms (bulges in the lower aorta). This study extends that research to the thoracic aorta (upper aorta) and shows that the drugs’ effectiveness may depend on the type and location of aorta damage. The findings suggest that treatments effective for one type of aorta disease may not work for another, emphasizing that different aorta problems may require different treatment approaches.

The main limitation is that this research was conducted in mice, not humans. Mouse biology doesn’t always match human biology, so these negative results don’t definitively rule out these drugs for human patients. The abstract doesn’t specify the sample size, making it unclear whether the study had enough mice to detect a treatment effect if one existed. The study used a specific chemical (BAPN) to cause aorta damage, which may not perfectly replicate how aorta disease develops naturally in humans. Additionally, the study doesn’t explain why the genetic signals suggested these drugs should work if they ultimately didn’t, leaving questions about the underlying mechanisms unanswered.

The Bottom Line

Based on this research, cilostazol and sildenafil do not appear to be effective for preventing thoracic aorta damage in this mouse model. However, this is preliminary research and should not change current medical practice. If you have risk factors for aorta disease or are concerned about your aortic health, discuss screening and prevention strategies with your doctor. If you currently take either of these medications for other conditions (like peripheral artery disease or erectile dysfunction), continue taking them as prescribed—this research doesn’t suggest they’re harmful, just that they may not help with aorta disease specifically.

This research is most relevant to cardiologists and vascular surgeons who treat aorta disease, as well as pharmaceutical researchers developing new treatments. People with family histories of aorta disease or those with known aorta problems should be aware that these two drugs may not provide the protection researchers hoped for. However, this doesn’t mean other treatments won’t work—it simply means these two specific drugs didn’t show benefit in this animal study.

Since this is early-stage research in mice, it will likely take several years before any human studies could be conducted if researchers decide to pursue other approaches. If you’re concerned about aorta health, focus on proven prevention strategies like managing blood pressure, avoiding smoking, and maintaining a healthy weight—these have clear evidence of benefit.

Want to Apply This Research?

  • If you have risk factors for aorta disease, track your blood pressure daily and log any chest pain, back pain, or shortness of breath. These symptoms warrant immediate medical attention.
  • Use the app to set reminders for blood pressure medication if prescribed, maintain a log of cardiovascular symptoms, and track lifestyle factors like exercise and sodium intake that affect aortic health.
  • Create a long-term health dashboard that monitors blood pressure trends, medication adherence, and cardiovascular symptoms. Share regular reports with your healthcare provider to catch any concerning changes early.

This research is preliminary laboratory work in mice and does not directly apply to human treatment. Thoracic aorta disease is a serious, life-threatening condition that requires professional medical evaluation and care. Do not use this information to make decisions about your medications or health care. If you have symptoms of aorta disease (sudden severe chest or back pain), seek emergency medical care immediately. Always consult with your cardiologist or vascular surgeon before making any changes to your treatment plan. This article is for educational purposes only and should not replace professional medical advice.