Clinical studies offer hope for participants, future generations

This past November, Dale Fishburn’s 20-year-old daughter Melissa was lying in a Springfield, Mo., hospital bed in a coma. Doctors told him they were out of options: None of the medications or procedures available would stop Melissa’s brain from continuous seizing.

Melissa, diagnosed with epilepsy at the age of 15, routinely had 25 to 30 seizures a day, including a couple that would make her lose consciousness briefly. But the length and severity of the seizures this time had sent her into a potentially fatal phase of the disease.

Melissa’s older sister got online to research experts and information about epilepsy treatment. She found an active clinical trial taking place at several locations across the U.S., including Wichita.

“We told our doctors about it, and within 12 hours, Melissa had been airlifted and was in a hospital bed at Via Christi in Wichita,” Fishburn said.

“I honestly didn’t know if anything could save her life at that point. But I thought maybe there’s a chance, and even if she doesn’t come out of this, maybe it’ll help someone else’s daughter down the road,” he said.

Melissa was Patient 17 in a clinical trial taking place across about 15 U.S. sites to try an investigational five-day course of medication developed by Sage Therapeutics. The seizures stopped, Melissa suffered no permanent damage, and she was able to return home.

“The trial ended in early December, and not only did the continuous seizures stop and allow her to come out of the coma, this is the best she’s been since she was diagnosed at age 15,” Fishburn said in late February. “She’s having very few seizures, and she’s even had a few days in a row with no seizures at all, and that’s really something for her. Without this medication, my daughter would not be alive. It was a last-ditch effort, and I’m thankful for the medical professionals who are working to make drugs like this available.”

There are hundreds taking place in Wichita at any given time through hospitals, medical schools and independent research organizations.

Epilepsy is one of three diseases that saw major breakthroughs in 2014 in clinical studies at Via Christi and nationally, said Michael Good, director of Clinical Research Operations for Via Christi Hospitals Wichita. Along with the Sage trial, Via Christi saw good results with a device implanted in the brain that acts as a defibrillator to stop epileptic seizures.

Patients suffering from cystic fibrosis, a life-threatening genetic disease that primarily affects the lungs and digestive system, who were once expected to not survive past their 20s now can live near-normal life expectancies with inhaled medications that help their bodies break down and absorb food.

And the first two FDA-approved treatments for idiopathic pulmonary fibrosis, a deadly lung disease also known as IPF, went on the market in 2014 after years of clinical trials, including many in Wichita by principal investigator Mark Wencel.

“You do the research year in and year out, and eventually the dam breaks and you see some real advancements,” Good said. “That’s why we push to the public that participating in clinical studies are not just helping patients today but generations to come.”

Clinical studies involve human volunteers who help medical professionals and researchers “improve patient care and better the science that we perform under,” Good said.

Via Christi Research has been performing clinical trials for 27 years and conducts the only in-patient clinical trials in Wichita, Good said. It currently has 59 studies open and is enrolling in 18 therapeutic areas such as cardiology, infectious disease, pulmonology, neurology and psychiatry.

There are hundreds taking place in Wichita at any given time through hospitals, medical schools and independent research organizations.

KU School of Medicine

A local and national leader in psychiatry clinical studies is the University of Kansas School of Medicine-Wichita.

“Every antidepressant, antipsychotic and antidementia approved in the U.S. in a quarter of a century has been tested in Wichita at KUSM-W before being marketed,” said K. James Kallail, associate dean for research. “We have a leading researcher in Dr. Sheldon Preskorn, who has the reputation and respect to draw major trials into the university and Wichita.”

Preskorn has had continuous funding for more than 30 years from sponsors including private foundations, the pharmaceuticals industry and the National Institute of Mental Health. Preskorn and Matthew Macaluso, director of research, psychiatry and behavioral sciences, have 10 active studies at the school, ranging from areas of depression, Alzheimer’s and schizophrenia and involving investigational medication, repurposing approved medication and processes, such as whether genetic testing is helpful in guiding clinicians’ selection of medications for depression.

Cancer trials

Another research entity looking not only at investigational medications is Wichita NCI Community Oncology Research Program. NCORP is sponsored and funded by the National Cancer Institute and managed locally by the Via Christi Cancer Institute.

“We’re not only looking at new ways to treat the disease portion of cancer but also our cancer care delivery, meaning how we do things. For example, does additional follow-up make a difference,” said Keisha Humphries, oncology service line administrator for Via Christi, who oversees operations for NCORP. “Sometimes we are so used to looking at the science, we forget to look at the everyday aspects of how we are delivering the care.”

Based at Via Christi Hospital St. Francis, Wichita NCORP is one of 34 community sites selected in 2014 by the National Cancer Institute to receive a five-year federal grant for $1.7 million each year. The program works with MD Anderson in Texas, the Mayo Clinic in Minnesota and Johns Hopkins hospital in Maryland to bring clinical trials to patients in the Midwest.

Previously known as Wichita CCOP, the local cancer clinical trials started in 1983. More than 16,000 patients have participated, and NCORP currently is running 84 clinical trials studying new ways to treat, prevent, detect, diagnose and control many types of cancers at various stages.

Heartland Research Associates

One of the largest research groups is Heartland Research Associates, a local independent organization founded in 1991 that conducts roughly 100 trials each year at five locations: east Wichita, west Wichita, Augusta, Park City and Newton. The group specializes primarily in family practice medicine such as diabetes, cardiovascular diseases, female and male health, musculoskeletal, gastrointestinal and preventive medicine. Heartland also has specialty investigators for immunology, dermatology and psychiatry trials.

“We find that our patients are happy to be involved, some simply because they are doing it to help improve medicine for mankind and others because they personally can benefit greatly by participating,” said Terry Poling, medical director and founder of Heartland.

Several recent popular trials include diabetes, baldness and acne, where patients get one-on-one care that usually improves compliance, free meds in some cases and compensation for their time. Heartland was honored in 2013 by the World Vaccine Congress for its contributions to vaccine research, which includes flu vaccinations. Poling said Heartland has many annual participants because they get the vaccination for free and it’s a newer, improved vaccine than they would get elsewhere.

Two of the most exciting areas of study at Heartland, Poling said, are an innovative procedure that removes a patient’s own fat to harvest stem cells, then injects the cells into their knees to restore degenerated tissue, and Alzheimer’s investigational medications.

Alzheimer’s is one therapeutic area that nearly every research organization is working to accelerate treatment and prevention. According to the Alzheimer’s Association, it is the sixth-leading cause of death in the U.S.

“I hope people will look at clinical trials as an opportunity to help the greater good. The more volunteers, the quicker we can advance the science,” said Good of Via Christi. “We’ve got an open and enrolling Alzheimer’s study right now. This is a tremendously debilitating disease that we don’t have a lot of good answers for. The only way we can get those answers is through good, solid research, and the way to get good, solid research is clinical trials.

“Through clinical trials, we’ll start to see the medical breakthroughs in Alzheimer’s in six or seven years that this year we saw in epilepsy, cystic fibrosis and idiopathic pulmonary fibrosis.”

This story was originally published March 1, 2015 at 1:43 PM with the headline "Clinical studies offer hope for participants, future generations."