How one patient’s unwavering determination led to heart disease diagnosis and treatment
(BPT) - Imagine living with excruciating chest pain that is a consistent part of your life. Imagine being so fatigued and short of breath that you can’t even do the dishes or walk up the stairs. Imagine seeing multiple different doctors over an 11-year period, and being subjected to test after test, but still not receiving a correct diagnosis. Imagine feeling like it’s all in your head or worse, that your doctor thinks it’s all in your head.
This is exactly what a patient named Vicky Ventura experienced before she was finally diagnosed with coronary microvascular dysfunction (CMD), a difficult-to-detect type of heart disease that is often misdiagnosed and disproportionately afflicts women. CMD affects the heart’s smallest blood vessels, so many CMD patients go undiagnosed or misdiagnosed because traditional medical tests for heart disease, such as an angiogram, are designed to look for blockages in large vessels.1 After 11 years, Ms. Ventura finally received a correct diagnosis after undergoing a procedure known as coronary reactivity testing, which is an invasive procedure that looks at how the small vessels of the heart are functioning. After reviewing all test results, Vicky’s cardiologist diagnosed her with CMD.
In CMD patients, the larger coronary arteries are usually unobstructed. Instead, in CMD patients, the walls of small penetrating coronary arteries are damaged or diseased, causing a decreased flow of oxygenated blood to the heart muscle.1 This decrease in blood flow leads to recurring, debilitating chest pain, fatigue, and shortness of breath.1 Along with the physical symptoms, many patients also report poor quality of life, along with mental and emotional distress.3
“My kids saw how I deteriorated throughout the years, how I changed completely, how I was no longer active,” Ms. Ventura said of her experience living with CMD. “I wasn't spending quality time with them anymore. I couldn't drive anymore. I couldn't walk long distances anymore. Even just getting out of bed was a challenge for me.”
Like Vicky, CMD patients often go years without proper diagnosis, during which time they are untreated or incorrectly treated and at high risk of heart attack and other serious cardiovascular-related events due to the underlying CMD condition.2,4
Limited treatment options
Once patients do finally receive an accurate CMD diagnosis, they are forced to come to terms with the fact that there is no cure for this disease and no approved treatments specifically indicated for CMD. To help relieve symptoms in patients with CMD, doctors generally turn to medications that are used to treat typical angina, including beta blockers, calcium antagonists and nitrates, that provide little relief.2
However, Caladrius Biosciences is evaluating a treatment for CMD called XOWNA® (formerly CLBS16) that it hopes will prove effective through clinical trials to alleviate CMD suffering. It is a type of cell therapy that uses a patient’s own cells to stimulate the growth of new blood vessels in an effort to repair damage associated with the coronary arteries.
While XOWNA® is an investigative therapy that is currently being studied and is not yet approved by the FDA, investigators were encouraged by results from a Phase 2a open label trial that tested XOWNA®, the ESCaPE-CMD study, as reported in 2019 and 2020. Caladrius is currently enrolling new patients in a Phase 2b clinical trial (known as the “FREEDOM Trial”).
Results from the ESCaPE-CMD study showed that, in some cases, a single administration of XOWNA® durably improved heart function and significantly relieved symptoms in study participants for at least 6 months following treatment (the designed follow-up period for the study),5 including Vicky.
“I couldn't take care of me anymore, and that was not the quality of life that I wanted,” Ms. Ventura said, of her decision to participate in the ESCaPE-CMD study.
During the study, doctors collected Vicky’s own cells from her bone marrow, specifically her blood vessel forming cells called CD34+ cells. A few days later, they administered a single concentrated infusion of those same cells back into the vessels of Vicky’s heart.
“My results with this treatment did amazing things for me,” Ms. Ventura said more than two years after participating in the study. “It gave me back my life. I am doing a lot of things that I couldn't do before.”
More hope on the horizon
After the positive outcomes with the ESCaPE-CMD study, Caladrius Biosciences is actively enrolling patients in a new study called the FREEDOM Trial, which aims to further evaluate the effects of XOWNA®.
If you experience chest pain on a daily or weekly basis and have been told by a physician that your coronary arteries appear “normal,” then you may be a candidate for the FREEDOM Trial.
Ms. Ventura said she would highly recommend participating in the trial for anyone like her who has suffered with CMD.
“There’s finally a treatment that could help them feel better like it did with me,” she said. “I wasn't expecting or hoping what I got in return. I am so glad that I did it.”
1 Diagnosis | Stanford Health Care. Retrieved from stanfordhealthcare.org website: https://stanfordhealthcare.org/medical-conditions/blood-heart-circulation/microvascular-dysfunction/diagnosis.html
2 Microvascular angina: angina that predominantly affects women https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4351318/
3 Coronary microvascular dysfunction: an update https://academic.oup.com/eurheartj/article/35/17/1101/2465953
4 Ischemia and No Obstructive Coronary Artery Disease (INOCA): What Is the Risk? https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6201435/
5 Caladrius Biosciences internal database for ESCaPE-CMD Study