Medical Hero Spotlight: Rev. Donna J. Matlach & Eosinophilic Asthma

“My sister Roseanne and and I love the movie, ‘The Wizard of Oz’. I told Roseanne I felt like I was going to see the Wizard. There was so much that happened before I got to see her, just like a lot happened to Dorothy in the movie, recounts Reverend Donna J. Matlach, about meeting Dr. Sally Wenzel of the University of Pittsburg Medical Center to consult about Donna’s severe eosinophilic asthma. My granddaughter, Phoebe, even said ‘Nana is going to see the wizard!'” Donna’s medical journey has been arduous and at times, terrifying, (Donna has had severe eosinophilic asthma for more than a decade) but her upbeat nature shines through during our conversation about her experience with clinical research participation.

“It can’t be controlled with the typical medications that are used with other types of asthma. It’s another level of asthma – it goes up in levels. Mine is severe, which is the highest level. It took many years to figure out why it couldn’t be controlled,” explains Donna. “I was in and out of the hospital, 3 to 4 times a year, and in the doctor’s office 3 to 4 times per month. I was on mass quantities of corticosteroids taken orally and by inhalation.” The steroids impacted her overall health, including weight gain and brittle bones leading to several fractures.

The severity of her symptoms sapped Donna of her physical strength, but not her inner fortitude. Taking matters decidedly into her own hands, Donna went on a cross-country journey in order to find medical advice and effective treatment. Conducting a lot of research on her own, Donna visited 28 doctors, the majority being pulmonary specialists, in 12 hospitals, nationwide. In a frustrating turn of events, they all provided different diagnoses and advice. To make matters worse, the nebulizer and cortiscosteroid medications Donna was taking were not adequately controlling her illness.

“My asthma was so misdiagnosed that at one point, doctors from a major medical facility told me I should see a psychiatrist. I was on a nebulizer every hour to stop the coughing and wheezing,” says Donna. “I’ve had over 600 allergy tests. I’m not allergic to anything.” Donna continued on her quest for relief from her symptoms. Donna consulted with a physician in Colorado who recommended she meet with Dr. Wenzel.

“Dr. Wenzel is the guru. She founded the University of Pittsburgh Asthma Institute,” says Donna.

During one of her many visits to Pittsburghwith Dr Wenzel, Donna had video assisted thoracic surgery to take biopsies and view her lungs with a camera. “I woke up with a tube in my chest to prevent my lungs from collapsing. The pain was so bad I ended up having a full-blown asthma attack, even with the pain medication pump. I’ve had a lot of surgeries in my life, including two C-Sections, and this was the worst!” says Donna.

Donna has had two 6-hour surgeries on her sinuses in the past several years. Both times, her sinuses were evaluated to be 98% blocked. “I had no taste or smell for 3 years because the sinuses and asthma were impacting each other. Everything is connected,” says Donna. When Donna arrived for the first surgery, she was told her lungs were too weak to undergo the procedure. Dr. Wenzel was concerned she would not survive the anesthesia. When Donna was able to have the first sinus surgery, several months later, Dr. Wenzel advised Donna that the positive results she was experiencing would last about three years.

“Wouldn’t you know it, it was just about 3 years to the month and I needed to have another surgery. Dr. Wenzel was right – that’s why I call her The Wizard,” says Donna. The second sinus surgery removed a bone from the left and right side of Donna’s frontal sinuses.

Dr. Wenzel advised Donna to investigate participating in clinical research to find other ways to manage her asthma. Dr. Wenzel assisted her with this process, but Donna was told she was not sick enough to participate in the first clinical trial to which she applied. This was perplexing to Donna because, she recounts that “I have been catching pneumonia twice year!”.

Dr. Wenzel located another clinical trial and Donna qualified as a participant. The clinical trial site was in California.

“I flew from my home in Arizona to California, once a month, at my own cost, for three years. I ended up being a poster child for the sponsoring company. I now speak at lectures for them about my experience, in New York City and other places,” says Donna.

The medication she received in the clinical trial in California was not immediately effective. “It took about 6 months to get into your system. At first it was an IV medication, and then it became an injectable,” says Donna. “My asthma is like a firecracker. It will either fizzle out or it will explode.” A couple of years later, that treatment stopped working.

During this time, Donna’s health was also being monitored in her then-home-state of Arizona. “I was at the Mayo Clinic in Scottsdale, waiting for a lung function test, and while I was there, I had a severe asthma attack. It came out of the clear blue sky. I ended up in the ER. God put me in a place where I could be helped and I wasn’t alone. I’ve had three near fatal episodes because of the asthma,” Donna recalls.

After the clinical trial that Donna participated in, Dr. Wenzel prescribed a biologic that had recently come to market. “It took 4 months for it to start working and I took it for a year. But I was still back and forth with ER visits that always turned into a hospital stay, unfortunately,” says Donna.

During her hospital stays, Donna continued to work on her studies towards her Masters of Divinity. Even though she was very ill, Donna says that “God told me… You’re not done.” She subsequently studied several more years and received her Doctorate in Ministry and Master’s in life coaching.

Dr. Wenzel then prescribed a third biologic medication, which was not part of a clinical trial.

“I go to Pittsburgh twice a year to see Dr. Wenzel and we’ve become close friends. I love her. My heart told me, when I met her, that this was going to be the best doctor I could possibly see,” says Donna, smiling.

Donna’s family was supportive of her decision to join a clinical trial. She looked for patient advocacy organizations for severe asthma, and there were none established at that time. In response, Donna co-founded the Severe Asthma Foundation with Dr. Wenzel and Brenda, another severe asthma patient of Dr Wenzel’s.  Donna served as president until they were subsequently invited to merge with the Allergy and Asthma Network where Donna serves on the Board of Directors as an asthma advocate.

Donna has experienced, first-hand, the serious impacts of severe asthma. “It’s not just physical. It effects your emotional well-being. It effects your family, your day-to-day life, your finances. No one ever asked me about those things, in doctor’s appointments – not until I met Dr. Wenzel,” says Donna.

When asked if she would recommend clinical research participation to others, Donna enthusiastically responds “Absolutely. It can be life saving. Why would you not? You could have side effects, but for me, how could it be any worse, since I was having near fatal episodes? You have to keep searching. Clinical trials can be the answer. Why would you not want to try it? This is why I am a patient advocate. I love sharing information about clinical trials with people. That’s why I went into the ministry. I know God has a purpose for my life to reach out to people with similar physical issues as a friend, minister, and counselor. ”

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

 

AWARE for All – Midwest Event Overview

On July 22nd, attendees logged on to view the AWARE for All – Midwest virtual event. The third event in a sequence of five running throughout 2021, AWARE for All – Midwest aimed to break down the clinical trial process, share a variety of perspectives from trial participants and healthcare professionals, and answer common questions and misconceptions many have about clinical trials.

The event started with an overview presentation about how clinical trials work and the important role trial participants play in the process. The presentation was led by Steve Satek, Founder & President at Great Lakes Clinical Trials, who explained the importance of informed consent, how to decide if a clinical trial is right for you, and why clinical research is so important to the advancement of medicine.

As Steve noted, “It could have been five, ten, or even twenty years ago that people chose to participate in clinical trials that paved the way for the medication you take today.”

The next segment of the webinar featured a panel discussion, led by a group of clinical trial participants and healthcare professionals who shared their stories and perspectives. Among the clinical trial participants were Nia Grant, a Type 1 Diabetes trial participant, Lynne Jordan, a talented performer and COVID-19 vaccine trial participant, and Dorie Rivera, a mother, caregiver, and rare disease advocate.

There are many different reasons someone might choose to join a clinical trial. For Nia, participating in clinical research gave her a chance to represent her community and ensure diverse populations were included. Nia said, “Representation in research is important. If there are no trial participants who look like me, how can a doctor be sure a treatment is effective?”

For Lynne, joining a clinical trial gave her the chance to be a part of the effort to tackle the COVID-19 public health crisis, making a difference for her community and around the world. As Lynne recalled, “As a member of a community disproportionately affected by COVID-19, I wanted to be a part of the solution to this crisis.”

For Dorie, enrolling her daughter in a clinical trial gave her access to new treatments and gave her family the hope they needed. After being referred by other parents, Dorie decided to give the trial a try, stating “It was either joining a trial or facing complete hopelessness.”

The AWARE – Midwest panel discussion also featured several healthcare professionals who shared their experiences, including Dr. Anna Lok, Holly Milaeger, MPH, and Dr. Manish Jain. Together, the panelists tackled issues related to clinical trials such as the importance of diversity, how barriers to participation can be overcome, and the best ways to educate and inform about clinical trials, starting at a community level.

After the webinar concluded, viewers were able to navigate to the Informational Exhibit Center, a virtual exhibit hall that offers resources and information from over 40 health and wellness organizations in the Midwest region and across the country. A quick ‘click’ connects visitors with local advocacy organizations like Latino Union of Chicago, Indigenous Peoples Task Force, and The Chrysalis Initiative. The Informational Exhibit Center also features a Health and Wellness Pavilion where visitors can watch health exercises and tips, and a theater with short educational videos about clinical trials.

If you missed the AWARE for All – Midwest event or would like to tune in again, the recorded webinar and Informational Exhibit Center remain accessible here.

View more 2021 AWARE for All events here.

Women in Clinical Trials

From “The Gift of Participation” by Ken Getz, Founder & Board Chair, CISCRP

Gender mix in clinical trials, overall, appears to be relatively balanced. Research conducted by the Center for the Study of Drug Development at the Tufts University School of Medicine found that, in clinical trials conducted in support of drugs submitted to the FDA, 52% of all patients who participated were men and 48% were women.

There is no question, however, that protocol designs have historically addressed disease as it manifests in adult males. Beginning in the early 1990s, public pressures fueled stricter government requirements for the presentation of data by gender in market applications to the FDA and valid analysis by gender at the NIH. In 2000, the FDA further specified that a clinical trial excluding persons having reproductive potential could be placed “on hold,” preventing further product development. This requirement helped ensure that women of childbearing potential were included in studies.

Pharmaceutical and biotechnology companies have also sought ways to increase the market potential for new and existing drugs by gathering clinical data to make specific claims about drug safety and effectiveness among women. As a result, clinical trials are increasingly being designed to assess the safety and efficacy of gender-specific medical treatment, and medical treatments are being “personalized” for gender differences in response.

Many diseases behave differently in women than in men. Risk factors, symptoms, the clinical course, and response to treatment can all be gender-specific. Among a long list of differences, men and women vary by:

  • body size, composition, and metabolism
  • the ways their bodies change during the aging process, e.g., puberty and midlife
  • endogenous hormones
  • exogenous hormones

Due to these differences and to other factors researchers have discovered that:

  • Lung cancer kills more women than any other cancer.
  • Alzheimer’s disease is twice as prevalent in women.
  • Men and women experience pain differently.
  • Women are two to three times more likely to experience depression, due to less serotonin uptake in the brain.
  • About 75% of autoimmune diseases occur in women, most frequently during childbearing years.
  • Urinary incontinence and dysfunction are more common in women and often have an entirely different cause than the same conditions in men.
  • Cardiovascular disease kills approximately 250,000 more women each year than all forms of cancer combined, accounting for 58% of all deaths. Within a year of the first myocardial infarction, 44% of women die, compared to 27% of men. Hormone-replacement therapy does not prevent heart disease, as was previously assumed.
  • The initial HIV viral load may be significantly lower in women, who represent an estimated 30% of new infections, but both sexes develop AIDS at the same rate

Although the FDA recommended in 1993 that clinical studies include enough women to understand the unique ways in which their bodies respond to drugs, women are still underrepresented in small, phase I trials. And when eligibility is restricted by age, older women are disproportionately excluded from studies of diseases that are more common in women at older ages. Although regulations prohibit the explicit exclusion of women of childbearing potential, the possibility of becoming pregnant can result in women in their childbearing years not being included in studies.

Generally, a woman capable of conceiving a child won’t be considered for a clinical trial unless she’s not pregnant and agrees to use birth control. Some studies require that women of childbearing age use two forms of contraception to participate in a study. Pharmaceutical companies don’t want their drugs tested among women who are—or might get—pregnant, mostly because the risk of exposure or a lawsuit by the mother is too high. Even in normal pregnancies, 1% to 2% end with an abnormal birth. Many parents are quick to blame poor birth outcomes on drugs. Some doctors erroneously believe that certain drugs cause fetal abnormalities. But genes and chromosomes are the primary culprits, according to Marilynn C. Frederiksen, M.D., associate professor of obstetrics and gynecology at Northwestern University Medical School.

“All of this presents a major barrier to clinical trial participation by women who don’t want, can’t afford, or are religiously opposed to contraception,” says Frederiksen.

Things aren’t bound to change unless the NIH comes up with the funds to conduct special dosing studies in pregnant women. And that probably won’t happen quickly or easily.

The NIH has an Office of Research on Women’s Health to help strengthen policies requiring inclusion of women in clinical research and to help translate new knowledge into clinical practice, but it doesn’t have any institutes that devote research dollars specifically to female health issues. As a direct result of the 1993 NIH Revitalization Act, NIH-sponsored clinical research now routinely includes sufficient numbers of non-pregnant women. In 2001, additional protections were given to pregnant women (as well as human fetuses and neonates) that spell out the conditions under which they can be involved in federally funded research— if earlier studies provide data on the potential risks, for example, and the risk to the fetus is caused solely by interventions that could directly benefit either the women or the fetus. Participation of women in NIH-funded studies, overall, is proportional to the percentage of women in the general population when sex-specific studies are excluded.

The participation of women in clinical trials is essential. The exclusion of women from early-phase studies, in particular, delays the discovery of sex-specific dosing requirements and the identification of gender-specific side effects, limiting the identification of drugs that are useful just for women. The problem is compounded by the fact that animal studies, when scientists learn about many of a drug’s potential adverse reactions, also tend to exclude females. Limiting studies to a single gender requires fewer study subjects (animal or human) and, thus, shorter and less costly studies.

There are many hopeful signs of change. Pharmaceutical companies are devoting a tremendous amount of money to trials focusing on diseases and conditions that only affect women.

For more information on clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.

You can find the book here.

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Clinical Trials: Improving Patient & Physician Education

CISCRP Perceptions & Insight Study Data Cited in Clinical Leader

A large and diverse pool of clinical trial participants scales the successful development of medications, therapies and treatments. Making trustworthy information about clinical research readily available to the public and physicians can serve to reduce mistrust of clinical studies. Clinical Leader addresses this topic in an article titled “3 Key Ways to Improve Patient & Physician Education on Clinical Trials”, citing data from CISCRP’s Perceptions & Insights Study. You can learn more about clinical research here.

CISCRP & Partners’ PLSP on Breast Cancer Study Published in Future Oncology

CISCRP (Center for Information and Study on Clinical Research Participation) and Oxford PharmaGenesis worked together with Daiichi Sankyo, AstraZeneca and Dr. Shanu Modi of Memorial Sloan Kettering Cancer Center in New York to write a plain language summary publication (PLSP) of the results of the DESTINY-Breast01 clinical study.

The participants in the study received a treatment called trastuzumab deruxtecan, also known as T-DXd. T-DXd consists of a chemotherapy drug linked to a manmade antibody. The antibody in T-DXd is a protein that specifically targets and attaches to the HER2 protein on tumor cells.

The PLSP was recently published in Future Oncology with the title “Trastuzumab Deruxtecan in Previously Treated HER2-Positive Metastatic Breast Cancer: Plain Language Summary of the DESTINY-Breast01 Study”. View the article here.

Why Clinical Trials Are Conducted

From “The Gift of Participation” by Ken Getz, Founder & Board Chair, CISCRP

Close up of African American physician listening to heart and lungs of patient

People want and expect their doctors to use treatments that work well and to stop using those that do not. Long ago, trial and error was the primary way that physicians and medical care providers learned how to recognize treatment alternatives. Later, through rigorous approaches that use clinical trials, physicians and researchers were able to gather far more meaningful information about diseases and how best to treat them.

For thousands of years, healers, shamans, and medical care providers have been administering treatments and remedies. One of the earliest known medical treatments dates back more than 3,500 years to ancient Egypt. Some ancient remedies, such as those used for simple fractures and minor injuries, are effective even today. However, many ancient medical treatments did not work and were actually harmful and even fatal. Two hundred years ago, cutting open a vein to drain a pint or more of blood and giving toxic substances to force vomiting or diarrhea were common remedies. And only a century ago, along with mention of some useful drugs such as aspirin and digitalis, the Merck Manual— one of the most respected sources for information on medical treatments then as well as now mentioned cocaine as a treatment for alcoholism; arsenic and tobacco smoke as treatments for asthma; and sulfuric acid nasal spray as a treatment for the common cold. Today these approaches are known to be very dangerous.

There are many reasons that doctors recommended ineffective and harmful treatments and that people accepted them. In many cases there were no alternatives. Doctors and patients usually prefer doing something to doing nothing. Patients also find comfort in sharing their problems and ailments with an authority figure. And doctors feel compelled to provide attention, support, and reassurance.

The primary reason doctors recommended ineffective and harmful treatments is that doctors couldn’t tell what worked from what didn’t. Doctors relied on cause-and-effect to identify potential treatments. For example, if an ill person’s fever broke after the doctor drained a pint of blood or after the shaman chanted a certain spell, then people naturally assumed those actions must have been what caused the fever to break. To the person desperately seeking relief, getting better was all the proof necessary. Unfortunately, these apparent causal relationships observed in early medicine were rarely correct. Still, they were enough to promulgate centuries of ineffective remedies. Of course, people had to be getting better in order to reassure doctors that a given treatment was working. Indeed, this is exactly what often happens. People do get better spontaneously. Sick people often get well on their own—and despite their doctor’s care—when the body heals itself or the disease runs its course. Colds are gone in a week; stomach flu passes within hours; migraine headaches typically last a day or two; and food poisoning symptoms may end in 12 hours. Many people even recover from life-threatening disorders, such as a heart attack or pneumonia, without treatment. Symptoms of chronic diseases (such as asthma or sickle-cell disease) come and go. Many treatments may seem to be effective if given enough time. And any treatment given near the time of spontaneous recovery may seem dramatically effective.

Belief in the power of a treatment or remedy is often enough to make people feel better. Belief cannot cause an underlying disorder—such as a broken bone, heart disease, or diabetes—to disappear. But people who believe they are receiving a strong, effective treatment very often feel better. Pain, nausea, fatigue, and many other symptoms can diminish. This happens even when the drug contains no active ingredients and can be of no possible benefit, such as a sugar pill or an inactive substance called a placebo. An ineffective (or even harmful) treatment prescribed by a confident doctor to a trusting, hopeful person often results in remarkable improvement of symptoms. This improvement is termed the placebo effect. People may see an actual (not simply misperceived) benefit from a treatment that has no real effect on the disease itself.

Some people argue that the only matter of importance is whether a treatment or remedy makes people feel better. Whether it works or not is of little consequence. This argument may be reasonable when the symptom is the problem, such as in many day-to-day aches and pains, or in illnesses such as colds, which always go away on their own. In such cases, doctors do sometimes prescribe treatments for their placebo effect. However, in any dangerous or potentially serious disorder, or when the treatment itself may cause side effects, it is critically important for doctors not to miss an opportunity to prescribe a treatment that really does work.

For more information on clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.

You can find the book here.

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Medical Hero Spotlight: Desiree De Luca-Johnson & Breast Cancer

“I am a lawyer, my husband is a pathologist, and I had no idea that there were hospitals that just treated cancer,” says Desiree De Luca-Johnson, an attorney, breast cancer survivor and patient advocate. At the age of 40, just when she was ready to go back to work after staying home with her young children, Desiree was diagnosed with breast cancer. Desiree’s road to being a clinical trial participant is reflective of her legal training. “I made a plan. I had a white board. I decided that if ‘A’ happens, then we would move on to ‘B’. I’m passionate about clinical trials.”

Desiree was diagnosed while living in a military community in Virginia, and her husband was deployed. “I was exhausted, and literally crawling upstairs to put my kids to bed,” Desiree recalls. Her oncologist at the time was a military physician and the treatment plan recommended for Desiree was conservative in nature. She was not told about clinical research as a healthcare option.

“I got 200 pages of where I could get free makeup and wigs, and not one page on clinical trials,” says Desiree. “I discovered that oncologists have different attitudes about treating cancer and I wish someone had told me about this. My oncologist was not aggressive about different treatment options.” Desiree recalls she was willing to deal with a higher level of side effects when her doctor told her she had an 80%/20% chance of survival.

“I was suicidal because of the anxiety. I found out (in addition to the breast cancer), I had a benign brain tumor,” says Desiree.

When she posed a question about clinical trials, Desiree’s doctor told her she was not sick enough to be in a clinical trial.

Desiree’s background as an attorney had honed her research and investigative skills. She decided to take definitive action.

“I made lists of cancer hospitals. I investigated their websites. I learned about ClinicalTrials.gov, which is a hard website to navigate. My husband was opposed to me doing a clinical trial, so I was doing this research all alone. But it was keeping me alive,” says Desiree. (Desiree’s husband ultimately embraced her decision to participate in clinical trials). “After 40 or 50 hours of work, I found BreastCancerTrials.org, where I found 6 clinical trials I might be eligible for. We have a saying in the law. When you’re repeating your results, you have done your research. I was ready to apply to clinical trials,” says Desiree.

Her oncologist in Virginia was not supportive of Desiree’s decision, and voiced a level of concern about clinical trial participation. Undeterred, Desiree pursued clinical research options and other avenues for her health and wellness.

“The most important thing about choosing to enroll in a clinical trial, for me, were childcare and the affiliated travel costs,” says Desiree. “I became my own doctor, in a sense. I read that doing yoga was good when you have cancer, so I did yoga. I read that exercise was good, so I hired a health coach,” says Desiree.

And, she participated in clinical trials. “I did an early-stage trial,” says Desiree. “The big barrier, again, was childcare. The navy has a program so my husband didn’t have to deploy during my treatment, so he could take care of our children.”

Desiree’s sister, who lives in Boston, recommended looking at Massachusetts General Hospital for relevant clinical trials to join. Desiree enrolled in a one-year treatment clinical trial. At the end of this clinical study, it was deemed a negative trial, meaning that the results demonstrated that the new treatment was inferior to standard treatment. (1) Overall, the experience was still a positive one for Desiree.

“I felt safer in a clinical trial because I had a better relationship with my research oncologist and oncology nurse in Boston. We were all up to date on the same research,” says Desiree. “I was grateful to be at a top research hospital. I was able to feel safe for a year – it gave me some time to breathe and research my options.”

“Through my clinical trial participation, I got access to other treatments for other conditions, such as bone density, and I was able to access other clinical trials,” says Desiree. Her research oncologist became her primary oncologist.

“Now I am in a trial to monitor for re-occurrence and I can make an action plan for quality of life,” says Desiree.

“Before participating in clinical research, every single day my first thought was, how will I know if my cancer has returned? Now I wake up and feel free,” says Desiree.

Desiree and her children have relocated to her home state of Vermont, in order to be closer to Massachusetts General Hospital, the clinical trial and family. “The relationships really matter,” says Desiree.

Desirae’s clinical research journey has brought her full circle, professionally. She currently works full-time at BreastCancerTrials.org as Outreach and Operations Manager.

Desiree advises people considering clinical research that, “When it comes to your oncologist, it’s a fiduciary relationship. It was my job to know the consequences of the decisions I was making to help them reach their goals. You have to be your own best advocate. No one is going to care as much about your life as you do.”

To search for medical conditions in a specific location visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Sources:

  1. https://www.rxlist.com/negative_clinical_trial/definition.htm)

Pros & Cons of DCTs & Virtual Clinical Trials

From "The Gift of Participation" by Ken Getz, Founder & Board Chair, CISCRP

They are known by different names: DCTs (decentralized clinical trials), remote, direct-to-patient, virtual, digital, site-less or simply patient centric clinical trials. All of these approaches share the common goal of making it easier to participate in research by reducing—or eliminating altogether—the number of study visits patients must make to conventional investigative sites or labs and allowing for more flexibility in carrying out study-related activities. Many of these approaches use smartphones, mobile devices, and wearable sensors to collect and evaluate patient data during the study.

Study volunteers often need to travel long distances to medical facilities, many need to stay overnight in a hotel, and take time off work to participate in a conventional clinical trial. Research from CISCRP shows that about one-fifth of study volunteers find clinical trial participation stressful and report the investigative site location and time-consuming study visits are among the least-liked aspects of the experience. Half of volunteers also feel that participation causes disruption to their daily routine. New, more convenient approaches are especially valuable for patients who may be too sick to travel or for those who rely on caregivers for support. Study volunteers who find it difficult to fit additional medical appointments into an already busy schedule or those who live far from the investigative site and wouldn’t otherwise be able to participate in the trial also benefit.

Not every clinical trial currently offers study volunteers an in-home or remote option, and it will take quite some time for a large number of trials to be done this way, but use of these approaches is expected to increase as pharmaceutical and biotechnology companies invest more widely in efforts to improve the clinical trial experience for patients. Research sponsors and regulators are working on initiatives that better take patient needs into account and could eventually allow patients to participate in clinical research wherever and whenever they want, whether it be their own primary-care doctor’s office, home, workplace, school, or anywhere else.

You shouldn’t feel forced to participate in a remote or at home study if you live near an investigative site and would prefer to have a face-to-face relationship with the study staff.

Pros:

  • You won’t need to travel and make frequent visits to an investigative site.
  • You’ll spend less time in a medical office.
  • You can participate in telemedicine visits at a time convenient for you, perhaps in the evening or on weekends.
  • You can contact someone on the research team 24 hours a day.
  • You may feel empowered by being able to participate whenever it is convenient to do so.

Cons:

  • You won’t have the same number of face-to-face interactions with study staff.
  • If you have a technical problem, you have to reach someone on your own to resolve the issue.
  • You’ll need to make sure you’re home to sign for clinical trial-related deliveries.
  • You may be asked to take your own vital signs or perform tests several times a day.
  • You may need to travel to a lab or medical facility for lab work or exams.
  • You may be asked to collect samples and arrange for them to be picked up.
  • You’ll likely need to send back all of the loaned devices and monitors at the end of the trial.
  • Not all wearable technologies have been validated, so you may need to repeat tests or travel to the research center for a special assessment.

When deciding whether a home-based or remote clinical trial is right for you, after you’ve learned as much as you can about the study visits and what activities you’ll need to perform on your own, discuss the pros and cons with your family, friends and primary care physician. It’s best to ask for input from people you know and trust and to involve your support network in your decision-making process.

For more information on decentralized clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.

You can find the book here.

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Out of the Dark: A Journey Through Postpartum Depression, Part 2

Authored by Melissa E. Daley, Communications & Marketing Manager, CISCRP

& Emma Kane, Senior Clinical Research Coordinator, Clinical Operations & Development, Sage Therapeutics

NOTE: CISCRP hosted a 2-part webinar series on postpartum depression (PPD), in collaboration with Sage Therapeutics, Inc. This is Part 2 of a 2-part article series, providing an overview of the second webinar, which focused on PPD, clinical research, and shared perspectives from a PPD survivor/patient ambassador, medical professionals, and a patient advocate. You can access Part 1 of this article series here (link to be created).

“The best-planned life can turn as quickly as a rainstorm on a summer’s day,” shares Chelsie, a PPD (postpartum depression) awareness ambassador and moreover, a survivor. Married to her high school sweetheart, she had accomplished many goals on her way to starting her family: she loved her job as an elementary school teacher, had purchased a home and was financially stable. “Everything fell into place according to plan. Teaching students was my passion, but motherhood was my calling,” explains Chelsie. Three years into their marriage, she and her husband were delighted to learn that Chelsie was expecting. Her pregnancy was typical until her 36th week, when she was diagnosed with pre-eclampsia and put on bedrest until her delivery.

Chelsie gave birth during a storm, “…amid tornado sirens and pounding rain,” she recalls. “Even more miraculous, the sun broke through and created a glorious rainbow just minutes after our son Weston was born.”

But almost immediately, Chelsie felt that something was wrong.

She did not feel an instant connection to Weston and she realized her maternal instinct was not taking over. Instead, Chelsie’s mind was swirling with questions about her baby’s welfare, as well as her own. Her anxiety escalated when her first attempts to nurse Weston were unsuccessful.

Chelsie did not know it yet, but she was beginning her journey with PPD.

A few days later Chelsie returned home with Weston. “I was physically weak and emotionally drained.” Everyday tasks were overwhelming. She was nervous, and her baby could feel her tension. “I was wracked with anxiety. I didn’t feel like I could even hold him,” says Chelsie.

A week after returning home, Chelsie was rushed to the emergency room with a racing heart and unresolved pre-eclampsia. Recognizing that Chelsie needed sleep, her OB/GYN prescribed a sleeping pill. Chelsie and her mother hoped rest would help. Still, she felt that her mind would not rest. “The next night would prove to be the start of the worst time of my entire life. I cried until I couldn’t cry anymore, and what came next was an utter lack of emotion. I knew I wasn’t myself, and I spoke up,” Chelsie shares.

“With my mom, dad, and husband in the living room, I explained I felt no connection to Weston. I felt like a terrible person and mother,” says Chelsie. Her family consoled her as best they could and suggested she speak with a cousin who had suffered with PPD. Her cousin advised Chelsie to call her OB/GYN. Chelsie’s OB/GYN had never treated anyone with PPD, and recommended a psychiatric facility.

“I felt nothing,” Chelsie says, as she left her six-day old son in her mother’s care. After an evaluation at the hospital, she voluntarily checked herself into a psychiatric hospital where she was officially diagnosed with PPD. She was prescribed medications to help with anxiety, depression, and insomnia.

“The longer I stayed (in the hospital), the worse I got,” says Chelsie. She was transferred to a second hospital.

In the span of 30 days, Chelsie had given birth, been diagnosed with PPD and had been a patient in two psychiatric hospitals. But there were glimmers of hope. “Even when PPD broke me down, I was gently reminded of a higher purpose for my life,” recalls Chelsie. Her parents found the right help for Chelsie, and eventually, with an adjustment of medication and the assistance of a mental health professional, Chelsie began to recover.

“I opened up to my doctors and therapists and we built a relationship where I could tell them anything. They helped me find solutions for whatever I was dealing with,” says Chelsie. Her recovery was challenging, but with assistance from her family, she began to focus on the victories she was starting to have as a mother. Returning to teaching scaled Chelsie’s confidence as well. She joined a local PPD support group and met other mothers navigating the same issues.

“Power in community is so precious. The key to my recovery was not a simple one. I needed the right therapy, medication, and family and friends to support me. Recovery takes time. I love my son more than I can put into words. PPD is nothing to be ashamed of or embarrassed about,” says Chelsie.

Chelsie is active in a non-profit organization focused on serving and building community with moms along with providing them with resources for PPD, regardless of their ability to pay.

There is an unmet need in treating PPD. Dr. Bassem Maximos, OB/GYN, explains that “A lot of physicians don’t recognize it (PPD) and even if we do recognize it, it’s difficult to find resources to point our patients to get the treatment they need.”

 Standard-of-care options for the treatment of PPD can be outlined in three categories.

“It takes a long time to find that therapist that you can trust, work with, and open your heart and mind to allow them to help you in your journey,” says Dr. Maximos. “It’s an ongoing treatment,” he adds, “and also sometimes you need other supportive therapies with it.” He also explained that unfortunately, it can be challenging to find a therapist, depending upon the community where you live and the resources that are available. In some regions, there are not many mental health professionals, and the few that are in the area may have so many patients that they are not able to assist new ones.

While it may be challenging to find these groups, Dr. Maximos stresses the importance of supportive psychotherapy and mother-infant therapy groups. “We’ve found in our practice if we get moms to see other moms that have gone through the same experience she’s going through, or have experienced PPD, they are more willing to open up and talk about their experiences – it takes away the fear or stigma.”

Peer support can also be found through groups active on social media when it’s difficult to find professional support. The COVID-19 pandemic has scaled the importance of this channel, with the need for social distancing measures to limit the spread of the virus.

“We’re still not screening our moms enough,” says Dr. Maximos. “The American College of Obstetricians and Gynecologists has been putting a lot of effort into encouraging obstetricians and gynecologists to screen for postpartum depression during the pregnancy and afterwards by giving them screening tools.” But there is still progress to be made.

There are organizations working to escalate access to support mothers suffering with PPD.

Tonya Fulwider, Associate Director for Mental Health America of Ohio and Program Director for POEM (Perinatal Outreach & Encouragement for Moms), says “We work with frontline providers, OB/GYNs, pediatricians and home health professionals. We have a team of certified peer support specialists who work as care navigators. We serve as that single-entry point of care for any birthing person who is struggling with a mental or emotional health complication.” The healthcare provider can reach out to the POEM team and directly connect the patient to receive assistance from a care navigator or peer

support specialist. They can offer a menu of options to address their needs, “…recognizing that she is the expert in her life,” says Tonya.

Sage Therapeutics has conducted multiple clinical trials in PPD over the past several years, and is currently conducting a trial studying an investigational oral tablet to treat PPD. A full listing of clinical trials for women with PPD is available at clinicaltrials.gov.

If you’re considering participating in a clinical trial, Dr. Gus Alva, a psychiatrist focusing on neuropsychiatric conditions with over 27 years of clinical trial experience, advises “Always be informed.” He explains that in his practice, they provide patients with a consent form, a standard clinical trial document which explains the trial in detail and confirms a patient’s willingness to take part in the study, and have them  take it home with them. They encourage patients to discuss it with their primary care doctor or other professionals in the medical field. “The consenting process,” he adds, “entails pros, cons, alternatives, and being well-educated.”

There is a regulated process that takes place in not just PPD clinical research, but in all clinical trials.

“There are several inclusion and exclusion criteria that are tied to all studies,” explains Dr. Alva. Inclusion and exclusion criteria are requirements that must be met in order to take part in the study. “Going in and getting screened would certainly be a good option.” He adds, “the nice thing about being involved in a clinical trial is that it’s not binding. There’s no penalty to withdrawing consent at any point. It’s a matter of trying to figure out whether this is a good fit or not, but not necessarily feeling that you are now bound to it.”

Individuals considering clinical research should consult the medical professionals involved in their care.

Dr. Maximos explains that before signing up for a clinical trial, “patients should speak to their physician, whether it’s their OB or psychiatrist.” Clinical research sites also conduct outreach efforts to community healthcare providers to raise awareness about clinical research participation opportunities.

“Be a question-asker,” advises Tonya Fulwider, saying that the care navigators she works with often encourage patients to write questions down as they come up so they can address them with their health care providers, whether it’s their OB/GYN, mental health professional or someone on the clinical trial team.

COVID-19 has impacted clinical trials across the board. Very strict guidelines have been put in place at all clinical trial sites, such as taking temperature readings, requiring masks, social distancing, and disinfecting of surfaces continually.

“Individuals that do come in know that we’ve placed into play a lot of different modalities to ensure that there is appropriate disinfecting, minimal touching of door handles or other objects, and that if they’re coming in, there’s a very specific purpose,” Dr. Alva explains. “We take every precaution possible to keep patients safe,” Dr. Maximos adds, including that his entire staff is now vaccinated in addition to the other precautions they’ve put into place over the course of the pandemic.

“Of course, COVID-19 has impacted everything about everyone’s lives, and it certainly has impacted mothers,” says Tonya. “Being able to continue to converse with them and talk about their concerns –   that’s really the most important thing.”

To learn more about postpartum depression, access the webinar series, “Postpartum Depression & Being Informed” here. You can also learn more about the Skylark study for PPD here, or access resources from Sage Therapeutics here.

ABOUT SAGE THERAPEUTICS

Sage Therapeutics is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the brain. We are pursuing new pathways with the goal of improving brain health, and our depression, neurology and neuropsychiatry franchise programs aim to change how brain disorders are thought about and treated. Our mission is to make medicines that matter so people can get better, sooner.

ABOUT CISCRP

CISCRP (Center for Information and Study on Clinical Research Participation) is a Boston-based, globally focused, non-profit 501(c)(3) organization providing public and patient education and advocacy.  CISCRP’s mission is to inform patients and the public about clinical research and the important role that it plays in advancing public health and to help stakeholders in drug development engage with patients and the public as clinical research partners (www.CISCRP.org)

Out of the Dark: A Journey Through Postpartum Depression, Part 1

Authored by Melissa E. Daley, Communications & Marketing Manager, CISCRP

& Emma Kane, Senior Clinical Research Coordinator, Clinical Operations & Development, Sage Therapeutics

NOTE: CISCRP hosted a 2-part webinar series on postpartum depression (PPD), in collaboration with Sage Therapeutics, Inc. This is Part 1 of a 2-part article series, providing an overview of the first webinar, which focused on PPD, and shared perspectives from a PPD survivor, medical professionals, and a patient advocate. You can access Part 2 of this article series here (link to be created).

“I did not want to move. I did not want to breathe. And I did not want anyone to know,” explained Alexis, about her experience with postpartum depression (PPD). PPD is one of the most common medical complications during and after pregnancy[i]. PPD is more severe in scope than the “baby blues” that many mothers experience, which generally occur within the first few days of delivery and resolve without treatment in 2 weeks[ii].

Symptoms of PPD may vary, but can include:

  • Changes in mood, including sadness, emptiness, hopelessness, irritability, persistent doubt, feeling overly anxious and thoughts of suicide
  • Changes in the body including fatigue, difficulty sleeping or sleeping too much, aches and pains, and changes in appetite
  • Changes such as trouble concentrating, remembering details or making decisions
  • Social differences, such as withdrawing from family and friends, and distress or impaired ability to functional in social or work settings[iii]

“Clinically, we basically say that if someone has had an episode of depression during pregnancy or the postpartum year, we’re going to call it postpartum depression,” explains Dr. Constance Guille, Associate Professor, Department of Psychiatry & Behavior Sciences; Director of the Women’s Reproductive & Behavioral Health Program, MUSC. “Postpartum depression and baby blues are two very distinct entities.”

It’s estimated that about 50-80% of woman are affected by the “baby blues” after childbirth[iv].

“The symptoms are not severe, and they do not impact activities of functioning in any way,” explains Dr. Guille about the baby blues.

Alexis had an unexpectedly difficult delivery, but her daughter was healthy. From the outset, she had problems bonding with her baby. “I looked at her like I was holding an alien from another planet,” Alexis recalls. Initially, she blamed it on exhaustion, but it quickly became apparent to her that there was something more serious happening. With the support of her husband and parents, Alexis sought medical assistance. She ended up switching psychiatrists and medications many times, and nothing seemed to be working. Alexis voluntarily admitted herself to a psychiatric hospital where she remained for a month.

“At one point, I was sitting and waiting to see the doctor, when I overheard the social worker telling the counselor that I was faking it,” says Alexis. “I slid even further into the abyss. If they did not believe me, who would?”

After she left the hospital, her depression deepened.

“I struggled with thoughts of not wanting to be alive anymore,” says Alexis.

The stigma around experiencing PPD is real. However, there are healthcare providers who are working to change attitudes and perceptions around postpartum depression and individuals suffering with it. One approach adopted by medical professionals is to routinely ask about a patient’s mental wellness, both before and after delivery.

“You see a patient during her pregnancy about 20 times,” says Dr. Jason James, OBGYN, Private Practice in Miami. “For me to ask how she’s doing, how is she coping, is she feeling down or depressed, is she feeling anxious or guilty, all the kind of non-judgmental questions – if I ask it all the time and get lots of “No’s”, it normalizes the situation.” Routinely checking in with a patient about her mental health can reinforce her comfort level about transparently sharing any changes she may be experiencing.

“The more that people can share their stories, the more that other people can look at that person and say, ‘Oh gosh, I’m just like them!’, the better off we’ll be,” says Dr. Guille.

Providing a robust familial and social support system to postpartum mothers is key to assisting them in their recovery.  “Having a baby is emotionally, psychologically, physically and financially depleting,” says Dr. Guille.  Finding time to relax, getting enough sleep, and ensuring proper nutrition is essential.

Heather Dopp is a patient advocate and Mom Ambassador with 2020 Mom, a national organization with a mission to close gaps in maternal mental health care. She is a mother to two children. Her advocacy work is spurred by her own experience as a survivor of perinatal anxiety, depression, and suicidal ideation during her second pregnancy. Heather’s advocacy has taken her from the halls of the Utah State Capitol to the United States Capitol, with the purpose of improving awareness and increasing resources available to mothers facing maternal mental illness within the United States.

“From a non-clinical point of view, I have an acronym we like to use with maternal mental health. It’s SUNSHINE, and it’s a great way to remember the basic things you can help with for the mother struggling, so she can manage everything she is going through and hopefully try to thrive,” says Heather.

“For many women that I have talked to across the United States, we don’t know about postpartum and perinatal mental disorders in the first place. When it happens to us, we’re already experiencing ‘mom guilt’, and we don’t want to own up to it, because maybe we’re the only ones… we haven’t heard our friends talk about it, and we haven’t heard our providers mention anything about it.” says Heather.

Other patient barriers to discussing symptoms include:

  • Mistrust and fear of being judged
  • Lack of insurance coverage
  • Time constraints
  • Access to childcare during postpartum visits
  • Lack of awareness regarding impact on own health and infant health
  • Lack of information regarding where to seek treatmentv

Alexis changed physicians and treatments many times and took the time to do research and learn everything she could about PPD given there were very few medical professionals specializing in PPD in her location. Eventually, Alexis found a counselor and psychiatrist who supported her. They developed a treatment plan to best address her symptoms and over the next year, she was able to begin the healing process.

“I began to truly laugh and smile again. Life became brighter, more colorful and the darkness began to drift away,” Alexis recounts. Her daughter has no recollection of Alexis’ struggle with PPD, and they are very close.

Alexis says that PPD has taught her five important lessons:

  1. Never give up; postpartum depression doesn’t last forever.
  2. Don’t be embarrassed. Reach out and find a support system.
  3. Self-care is essential and it doesn’t have to cost money. It can be as simple as reading a book or taking a bubble bath.
  4. Do your best not to feel guilty.
  5. Tell your story, when the time is right, because it can help you heal, and also help others.

“I did not give up. I made it out of the darkness. And through the pain, I found a strength and courage that was always there, but needed some prompting to come out,” says Alexis.

To learn more about postpartum depression, access the webinar series, “Postpartum Depression & Being Informed” here. You can also access resources from Sage Therapeutics here and from 2020Mom here. Your healthcare provider should always be your primary source of information about diseases or disorders and treatment options. Please contact your healthcare provider with any questions pertaining to a medical condition.

ABOUT SAGE THERAPEUTICS

Sage Therapeutics is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the brain. We are pursuing new pathways with the goal of improving brain health, and our depression, neurology and neuropsychiatry franchise programs aim to change how brain disorders are thought about and treated. Our mission is to make medicines that matter so people can get better, sooner.

ABOUT CISCRP

CISCRP (Center for Information and Study on Clinical Research Participation) is a Boston-based, globally focused, non-profit 501(c)(3) organization providing public and patient education and advocacy.  CISCRP’s mission is to inform patients and the public about clinical research and the important role that it plays in advancing public health and to help stakeholders in drug development engage with patients and the public as clinical research partners (www.CISCRP.org)

Sources:

[1] Ko JY et al. MMWR Morb Mortal Wkly Rep. 2017;66(6):153-158.;  Martin JA et al. National Vital Statistics Reports; vol 68 no 13. Hyattsville, MD: National Center for Health Statistics. 2019.;  DeSisto CL et al. Prev Chronic Dis. 2014;11:E10.; Centers for Disease Control and Prevention. Updated June 12, 2018. Accessed May 5, 2021 https://www.cdc.gov/reproductivehealth/maternalinfanthealth/diabetes-during-pregnancy.htm; Centers for Disease Control and Prevention. Updated February 28, 2019. Accessed May 5, 2021 https://www.cdc.gov/reproductivehealth/maternalinfanthealth/pregnancy-complications-data.htm; Roberts JM et al. American College of Obstetricians and Gynecologists Task Force on Hypertension in Pregnancy. Obstet Gynecol. 2013;122(5):1122-1131.; Callaghan WM et al. Am J Obstet Gynecol. 2010;202(4):353.e1-e6.

[1] 1. National Institute of Mental Health. Accessed May 5, 2021. https://www.nimh.nih.gov/health/publications/postpartum-depression-facts/index.shtml; American College of Obstetricians and Gynecologists website. Frequently Asked Questions: Postpartum Depression. Accessed May 5, 2021. https://www.acog.org/patient-resources/faqs/labor-delivery-and-postpartum-care/postpartum-depression; Thurgood S et al. Am J Clin Med. 2009;6(2):17-22)

[1] American Psychiatric Association: Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition. Arlington, VA: American Psychiatric Association, 2013.; National Institute of Mental Health. Accessed May 5, 2021 https://www.nimh.nih.gov/health/publications/postpartum-depression-facts/index.shtml)

[1] Thurgood S et al. Am J Clin Med. 2009;6(2):17-22)

v Prevatt BS et al. Matern Child Health J. 2018;22(1):120-129.; Byatt N et al. Arch Womens Ment Health. 2013;16:429-432.; Santora K et al. NIHCM Issue Brief. 2010. Accessed May 5, 2021 https://www.nihcm.org/pdf/FINAL_MaternalDepression6-7.pdf; Goodman JH. Birth. 2009;36(1):60-69.