The Medical Minute: Clinical trials bring scientific, personal breakthroughs

Bob Parise had run out of options. He was dying of congestive heart failure. When his physician, Dr. John Boehmer, came to him with an idea, Parise was ready to try anything.

Boehmer offered Parise a chance to become a study volunteer for a clinical trial of an experimental drug. That might sound radical to some ears, but Penn State Health and Penn State College of Medicine have for decades helped discover and treat diseases through clinical trials. The trial came with risks, Boehmer explained, but it also came with new and focused care and access to the latest potential therapy.

Parise agreed. His youngest of five daughters, Jewell, says the trial for a drug called Verasonin bought him five more years.

While Parise’s clinical trial allowed him and his family to have more time together, it also benefited the wider world. As a trial participant, Bob Parise, a retired Harrisburg butcher, became a pioneer. His treatment could help determine whether a new medicine could save millions of lives.

Patients who partner with researchers in clinical trials use the treatment of their own diseases to help medical science explore new frontiers. But it’s not just altruism. Clinical trial study volunteers often become healthier, said Dr. Neal Thomas, associate dean for clinical research at Penn State College of Medicine.

“Clinical trials are one of the ways medical problems get solved,” he said. Thomas helps match investigators from the College with clinical research organizations, pharmaceutical companies or federal agencies with devices or drugs they want to test at the clinical research teams at Hershey.

Below, Thomas discusses clinical trials, how they’re invaluable to research and what they can do for patients:

What could you say to someone to put their mind at ease about participating in a clinical trial?

The first thing I tell them is that they’re our partners. They work together with us. We can’t do this without them, whether it’s participants themselves, or if it’s a child, the parents who can agree to their participation.

The other thing people need to know is that it’s all very well regulated. It’s controlled and every study is reviewed for potential risks and benefits. We choose the studies we offer very carefully to make sure that they are important, safe and worthwhile.

Are there risks?

Most clinical trials have some risk. But most of those risks are well-spelled out and described during the informed consent process. Clinical trials also have a safety monitoring board that reviews subjects as they’re enrolled for any new risks that are reported. If new risks are discovered, we find out about them very quickly.

It’s important to understand that every study is different. Generally, the studies that are in an earlier phase or are aiming at diseases where there are not great clinical options outside of the study tend to have higher risks.

Our research participant partners will need to make the best decision for themselves in terms of the risks and benefits. But if it’s not what’s considered an acceptable risk-benefit ratio, then it will not receive approval from our institutional review board.

Are there real benefits to participating in clinical trials for patients?

It’s been shown that people who participate in clinical trials – even if they receive placebo (they’re in a control group that doesn’t receive an active medication) have better outcomes, because they get more detailed and devoted attention. In addition to their clinical providers, they have a study team who is looking at them and following them very closely. So, there is a benefit to a patient just by participating in a clinical trial.

Are the results of the trials always positive?

No. The fact is that is we’re trying to answer questions for all patients with the disease, no only who are participating; also, we are attempting to define treatments for the next generation of patients. If we knew the answer, we wouldn’t have to do the research.

Many times, clinical trials are the only way you can have access to leading-edge therapy. Whether they’re drugs or devices, they need to be tested and shown that they are efficacious, that they have a benefit and that they have a reasonable risk profile. It’s really a balance of benefits and risks.

There are many times – especially when it comes to disease like cancer, advanced heart failure or critical illness – the only way you have access to an unproven therapy that may work would be to participate in a clinical trial.

How are trial participants recruited?

We use Penn State Studyfinder, which is a list of all our clinical research studies that are actively recruiting for research volunteers across the entire the entire university. We also partner with marketing and work with research information technology. For example, if we had a trial that may benefit patients with chronic obstructive pulmonary disease, we can use our electronic medical record database to find all our potential patients, and then have directed marketing materials sent to them about the study or have their physicians reach out to them.

How do clinical trials work? Who initiates them?

Industry sponsored clinical trials come to us when our industry partners have a drug or a device that they want to trial. They need sites with expertise and potential subjects to conduct a trial. Also, there are investigator-initiated trials, or trials that are the innovation of people that work here. They develop an idea, and then submit a grant proposal for funding to conduct the trial. When I say develop a clinical trial, many times it represents years of work before you can get to the point of obtaining funding as a clinical trialist. I know that with clinical trials that I’ve led either funded by federal agencies or industry, it generally takes many years to get the trial from idea to completion.

What’s an example of something the College of Medicine helped solved through clinical trials?

A discovery that started in the 1990s in the College of Medicine led to the human papillomavirus vaccine. The Penn State Heart and Vascular Institute is very active in the development of artificial hearts and has been for many decades. These are two of many examples of discovery at the College of Medicine that are now impacting care around the world.

On a personal note, my youngest son, Kevin, was born in 2002. He was very premature, being born 13 weeks early. At the time I was doing a lot of surfactant research, including some novel clinical trials. Surfactant is a medicine you put into your lungs if you have underdeveloped lungs It was studied in the 80s and 90s, and many of the studies ― including here at the College of Medicine ― were in premature babies. At the time my son was born, I was studying it in older children ― not babies with premature lungs, but children with bad pneumonia.  As I was in the middle of enrolling a patient in a surfactant study, my son was born suddenly. He received surfactant in the delivery room based on the studies that were done in the 80s and 90s, based on the clinical trials that were performed worldwide, including the College of Medicine. And now he’s 21 and completely healthy.

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Bob Parise died in 1995, four months after the birth of Jewell’s first son. The trial had given Bob a chance to meet his grandson.

The drug ultimately was not approved by the Food and Drug Administration. To Jewell’s way of thinking, Bob’s clinical trial was still meaningful. It played a role in proving a drug that wasn’t right for the general public.

And it bought Jewell a chance to connect on a deeper level. Bob had been a drinker. Not abusive ― he had always been a kind man. But now he no longer drank, and Jewell got to know her father. He’d been a reader – could rip through a Robert Ludlum or a Tom Clancy in a day. They watched movies together – Bob loved what he called a “shoot ‘em up” but deep down he was a softy. His two favorites, Jewell discovered, were “Steel Magnolias” and “Terms of Endearment.”

“He was just very wise,” Jewell said. “The conversations we had were very meaningful. He probably would have died a lot sooner. He was just waiting around to die, but this drug – it helped him.”

Years later, Jewell took a job at Penn State Health and Penn State College of Medicine. Today she is a client services specialist in the Office of Marketing and Communications, working with the Penn State Heart and Vascular Institute.

It’s her chance pay back for a breakthrough that for her meant more than anything scientific ever could.

“It gave me a lot,” she said. “I never forgot that.”

Related content:

• The Medical Minute: Healthy volunteers vital to clinical trials
• The Medical Minute: How clinical research studies benefit patients

The Medical Minute is a weekly health news feature produced by Penn State Health. Articles feature the expertise of faculty, physicians and staff, and are designed to offer timely, relevant health information of interest to a broad audience.