February is American Heart Month. Implemented speak National Heart, Lung and Blood Institute, American Heart Month is designed to raise awareness of heart issues and encourage everyone to adopt heart-healthy behaviors, such as healthy eating, blood pressure and cholesterol control, and exercise.
Alexander (Sacha) Opotowskyassociate professor at Harvard Medical School and a cardiologist at Boston Children’s Hospital and Brigham and Women’s Hospital took the time to talk with BioSpace on advances in heart health, congenital heart disease (CHD), biopharma and Adult Congenital Heart Association (ACHA). He points out that he has research relationships with several of the companies mentioned in this article.
In the United States, cardiovascular disease, including stroke, is the underlying cause of death in approximately 836,546 people each year, according to the American Heart Association (AHA). That amounts to approximately 2,300 Americans dying of cardiovascular disease every day. And about 92.1 million American adults have some type of cardiovascular disease or the after-effects of a stroke.
Congenital heart disease, on the other hand, refers to a problem with the structure of the heart and is present at birth. It is rarer than cardiovascular disease and is usually defined by a series of birth defects, such as atrial septal defect, tetralogy of Fallot, ventricular septal defect and others.
ACHA is a patient advocacy group. Opotowsky says, “Their mission is to advance care and improve treatment for this population. They do this through a wide range of initiatives aimed at educating patients, advocating for beneficial policy changes, and educating healthcare providers to improve understanding of this diverse and unique group. of patients.
He also works to bring patients, families and providers closer together. “Although the stated goal,” Opotowsky says, “is usually for providers to educate patients, this has a huge effect on providers. Through these initiatives, ACHA has maintained a wonderful and close bond between patients and caregivers.
For example, CDHA has worked to improve the quality and availability of CDHA clinical services across the country. This includes developing criteria for what is expected of an ACHD clinical center as well as a certification process to identify programs that meet these criteria. Opotowski adds, “A second (pathway) is to work with clinical leaders to establish ACHD as a separate Board-certified subspecialty within cardiovascular disease, requiring specific fellowship training and Board review. Of course, this requires medical centers and trainees to devote additional resources and time to ongoing training. ACHA has partnered with philanthropic donors, including within the pharmaceutical industry, to establish funding for such clinical fellowships.
Cardiac care and procedures often fall under the category of medical device companies. Often this is related to specific aspects of adult congenital heart disease (ACHD). “The first effective treatment for severe congenital heart disease involved heart surgery,” says Opotowsky, “and surgical technology, including prosthetic heart valves and grafts, has long been an area of continuous development, with close industry collaboration and academia. Perhaps the most significant recent growth in industry-related development has been catheter-based intervention and device therapy.
An example of advances in this area is a recent story that Abbott‘s Amplatzer Piccolo Occluder was approved by the United States Food and Drug Administration (FDA). The device is smaller than a pea and is used to treat persistent ductus arteriosus (PDA). This is a potentially fatal hole in the hearts of premature babies and newborns. PDAs account for 10% of all congenital heart disease.
“This approval is a potentially life-saving advancement for the smallest preemies that will help us treat these delicate babies who otherwise might not survive,” said Evan Zahndirector of the congenital heart program at Cedars-Sinai‘s Smidt Heart Institute and principal investigator of the Abbott study.
Last year in March, the FDA approved Abbott’s Masters HP 15mm device, the world’s smallest mechanical heart valve. This is used to treat infants and children with mitral or aortic valve problems. It was originally developed by St. Jude Medical before Abbott bought the company.
Opotowski mentions other device developments, saying, “Many of these devices originally developed for specific needs in congenital heart disease were later modified for use in much larger cohorts of patients with common heart conditions and acquired heart disease. An example is atrial septal defect closure devices and their application to patients with patent foramen ovale and cryptogenic stroke.
He goes on to say, “Another is percutaneous valve technology, which was first explored for pulmonary valve replacement in a subset of coronary artery disease patients. There is now an explosion of transcatheter aortic valve disease for acquired aortic stenosis.
Work on the medical front is not as common. However, one notable area is pulmonary arterial hypertension (PAH). PAH is a form of high blood pressure that affects the arteries of the lungs. In January 2017, Johnson & Johnson acquired Based in Switzerland Actelion for $30 billion largely to acquire its Tracleer for HAP. The company also owns Opsumit and Uptravi for PAHs.
Opotowski says, “PAH is a disease that many coronary artery disease patients are predisposed to develop.”
Another company working for heart disease treatments is BioCardia, based in San Carlos, Calif. The company’s lead therapeutic candidate is the CardiAMP cell therapy system. This provides cell therapy derived from autologous bone marrow for the treatment of heart failure after heart attack and chronic myocardial ischemia. The company present positive 12-month data for CardiAMP at the November 2018 American Heart Association meeting in adults with heart failure after a heart attack.
Its second candidate is the CardiALLO cell therapy system, an “off-the-shelf” allogeneic culture cell therapy derived from donor bone marrow cells that meet specific criteria. It has the potential to treat a variety of clinical indications, including heart failure.
And, of course, there is important work on drugs to lower cholesterol. The two biggest advances have been a class of drugs known as PCSK9 inhibitors, such as Regeneron Pharmaceuticals and Sanofi’s Convenient and Amgen’s Repatha.
In September 2018, Amarin society announcement positive data from its REDUCE-IT clinical trial of 8,179 statin-treated adults at increased risk of cardiovascular problems for its drug Vascepa. Vascepa is derived from a purified component of fish oil called eicosapentaenoic acid (EPA) and has been approved for patients with triglyceride levels above 500 milligrams per deciliter, triple normal levels. In the trial, the drug showed a 25% reduction in the relative risk of major adverse cardiovascular events compared to placebo.
And these are just some of the areas that life science companies are working on for heart health. Apotowski also points out that significant work is being done on diagnostic tests for cardiovascular disease. “We recently studied the use of circulating and other biomarkers of cardiovascular disease in ACHD. We have worked with industry partners, including companies that manufacture diagnostic products (e.g. rock Diagnostics) and others interested in supporting understanding of how these tests can help better guide therapy (eg Actelion).