Journey to Better Health Mobile Exhibit 2023

Journey to Better Health is a mobile exhibit that showcases clinical research information to local communities in a fun, interactive way. In Fall 2023, the exhibit traveled to Philadelphia, PA and Baltimore, MD advocating for the importance of participation in clinical research and the critical need for more diverse representation.

Medical Hero Spotlight: Noa Greenwood, Canavan Disease Clinical Trial Participant

Canavan Disease Diagnosis

Lee and Lori Greenwood welcomed their daughter Noa in August 2020. Born full-term and seemingly healthy, Noa’s first weeks of life passed without issue. However, at around 8 weeks old, Noa began crying much more than the average baby. Concerned she might have colic or a reflux problem, the Greenwoods took her to the pediatrician. Noa’s doctor determined she did not have colic but couldn’t identify the underlying issue.

Soon after, Lee and Lori noticed that Noa wasn’t responding to lights or movements and worried she had vision loss. “She wasn’t looking at us — she was almost looking through us,” Lee recalls. Around the same time, Noa stopped gaining weight and dropped from the 89th percentile to the 26th percentile in her age group.

Referred to an ophthalmologist, they again found no clear answer to Noa’s symptoms, and she continued to miss developmental milestones.

When she began struggling to swallow, the Greenwoods pushed for a consultation with a neurologist, initially meeting via Zoom. “Noa’s neurologist was wonderful,” Lori says. “She originally suspected hydrocephalus and recommended we bring her into the office. However, I could tell from her reaction to Noa that something else was going on.”

Noa was then scheduled for an MRI, which unfortunately took several months of waiting due to the pandemic. In the meantime, she received early intervention care via Zoom calls with different specialists.

In August 2021, a few weeks before her first birthday, Noa’s MRI appointment revealed that she had Canavan disease, a progressive, fatal, genetic disorder affecting the central nervous system.

“We received the news on Zoom,” Lee recalls. “Lori and I joined a video call having no idea that doctors were about to tell us our beautiful little daughter had a fatal disease with a life expectancy of around 10 years.”
Finding Community Support

After Noa was diagnosed, Lee and Lori began researching the disease, seeking information and support online. They found a patient advocacy organization based in Boston that supports children with Canavan and related diseases. This organization helped connect them to relevant Facebook groups and the Canavan community.

“Everyone has been incredibly supportive. They have all experienced the same diagnosis and understand what it’s like. Being involved in this community and patient advocacy has been immensely helpful for us,” Lori says.
Clinical Trial Referral

For most patients, clinical trial participation is only mentioned by doctors after standard treatment options are exhausted. However, there is currently no cure for Canavan disease and the only treatment is palliative care. For such an ultra-rare disease affecting only 1,000 children across the U.S. and the EU, research studies are hard to come by.

The day Noa was diagnosed with Canavan, a new clinical trial became available at Massachusetts General Hospital in Boston, only 30 minutes from the Greenwood’s home. Noa was immediately referred by her neurologist, and within weeks the family was in contact with the trial coordinator.

“Everything came together in a serendipitous way. The timing was right, the location was so close to us, and Noa met all the eligibility criteria to qualify for the trial, which many kids don’t,” Lee says. “It’s funny to think of yourself as fortunate when facing a disease like Canavan, but we really are.”

Since clinical research was one of the only ways Noa might be successfully treated for Canavan disease, Lee and Lori decided that joining a trial for gene therapy was the best choice for their family.

Starting Gene Therapy

Gene therapy corrects genetic information that causes illness by replacing a faulty or missing gene with a genetically engineered ‘vector’. In recent years, gene therapy has been transformative across many diseases.

Before starting treatment, Noa and her family spent a lot of time at doctors’ appointments and in the hospital for the screening process. Noa had multiple MRIs under anesthesia to ensure she would be suitable to receive this type of treatment.

In June of 2022, when Noa was almost 2 years old, she received treatment in the Canavan trial using gene therapy. Noa spent about a week in the hospital afterward and then took steroids for about six months. She continues to be monitored and has regular check-ins with her care team.

Patient Advocacy Work

Before Noa began the trial treatment for Canavan disease, Lee and Lori already understood the complexities of navigating clinical trials and the healthcare system through research and their backgrounds.

“Clinical trials can be complicated and intimidating for patients and their families, especially when there’s a power dynamic between patients and doctors that can make asking questions difficult. Many people, particularly in underserved communities, face additional barriers like language and cultural differences that we don’t,” Lori explains.

Since Noa’s participation in clinical research began, the Greenwoods have made it their mission to raise awareness for Canavan disease and empower other patients to ask questions and advocate for themselves.

“By sharing our experiences, we hope to help others feel more confident in navigating the medical system. We’re fortunate to have a supportive care team and feel a responsibility to share our journey to benefit others, not just Canavan families but anyone involved in the medical field. By sharing our story, we aim to remind those working in research of the real people they are helping,” Lee says.

“It’s important to remind researchers that when they are conducting trials, they are changing the lives of real patients. You’re not just working to treat Canavan disease; you’re working to help kids like Noa.”
Life Today for Noa & Her Family

Today, Noa is currently in the second year of the five-year clinical trial, and for the Greenwood family, the results have been incredible.

“When we found out Noa had Canavan, we never imagined that her life could be the way it is now,” Lori says. “We are so proud of her!”

Today, Noa’s parents describe her as a sweet and affectionate toddler who can walk independently, is learning her ABCs and how to count, and goes to public school every day with other children her age. Just two years ago, Canavan disease would have made these milestones unreachable for Noa.

The Greenwoods share that they are proud of Noa’s participation in the research that may one day cure Canavan’s disease, even if her treatment today is only the first step.

“We have had an amazing experience in the clinical trial with Noa so far, but we understand the nature of this disease. There is so much uncertainty   her progress could reverse unexpectedly at any time,” Lee notes.
“But through this experience, we have gotten so much better at learning to embrace the current moment as parents. Regardless of what happens in the future, no one can take away the joy I’ve had seeing Noa grow and thrive the last  two years, which wouldn’t have been possible without the trial.”

Additional Resources:

https://www.umassmed.edu/news/news-archives/2023/07/family-connects-with-researchers-behind-canavan-gene-therapy/

https://www.canavan.org/

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.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Medical Hero Spotlight: Michael Herman, Multiple Myeloma Clinical Trial Participant & Patient Advocate

Abruptly Diagnosed: Facing Multiple Myeloma

In the Fall of 2012, Mike Herman and his wife Angela were taking their daily walk with their dogs through the neighborhood. When Mike dropped his glasses and bent down to pick them up, he felt a sharp pain, much like walking into the corner of a counter. He assumed it may have been a pulled muscle, and tentatively moved on until it happened again a couple weeks later. This time, Mike was at work and had gone to pick some files up off the floor when the same pain started again. Although he didn’t know it at the time, the pain was caused by multiple micro-fractures.

At the doctor’s office, Mike was referred to a hematologist who believed he had a vitamin B12 deficiency and recommended he go home and take some supplements. “I was so lucky that day to have my wife with me who is a nurse and has a lot of experience navigating the healthcare system,” Mike recalls. “Angela insisted it was not a B12 deficiency and said we would not be leaving with that diagnosis, which was what finally pushed them to order a bone survey. That same afternoon, I was X-rayed from head to toe, which is how the microfractures were found.”

About a week later, Mike noticed an email in his medical portal with test results. He assumed this was good news about his health issues and never imagined he would receive a diagnosis without a phone call.

“I opened the message and must have read it ten times. I thought I must have been missing something, so I called my wife and read it to her,” Mike says. “When she hesitated, I realized that this was real and that I had cancer.”
Finding the Right Doctor

After receiving his diagnosis for multiple myeloma, Mike’s doctors started him on the standard treatment of chemotherapy.

“At that time, the only thought in my head was the fear of dying. I had been told that the life expectancy with this cancer was four years, and only three months prior my first granddaughter had been born. I was devastated that I wouldn’t be there to see her grow up,” Mike says. “Other options for treatment or clinical trials weren’t brought up by my doctors, so I didn’t know it was even possible for me.”

During the early days of treatment, Mike had the opportunity to change health insurance providers, which gave him much more flexibility in finding specialists for his cancer. Through research, the couple found Dr. Bart Barlogie, who had founded the UAMS Myeloma Center in Arkansas. Mike made the difficult decision to temporarily move to Arkansas and receive treatment at the center.

“I was down there for nine months and during that time I had two stem cell transplants and tried a couple different treatments,” Mike says. “My cancer wasn’t going into remission yet, but I was starting to feel a lot better, which is when I started becoming close with my doctor and appreciating his way of treating patients.”

Mike’s doctor believed in an individualized treatment approach, something that had been missing from his care when he was first diagnosed. “I highly value his feedback and we developed a great friendship. He welcomed me to ask questions and get second opinions from other doctors, which is so important when you are dealing with cancer. Any doctor who discourages you from getting another opinion does not have your best interest in mind,” Mike says.

Mike and Dr. Barlogie discussed his treatment, and Dr. Barlogie encouraged Mike to consider clinical trial participation. After Dr. Barlogie retired, Mike, having tried nearly every publicly available myeloma drug, sought new options, and opted for clinical research.

I knew from my wife’s work in clinical research that these studies are essential to developing new medicines. If I could, I wanted give back to other people with myeloma who might need this medicine in the future. For me, clinical trials felt like the right thing to do.”
Clinical Trial Participation & Success

Mike has been in two clinical trials at the University of Pennsylvania, both with incredible results. The first study was in 2020 for a treatment called Teclistamab, sponsored by Johnson & Johnson Innovative Medicine. The trial tested two different ways of administering the treatment, and Mike received his subcutaneously, as a shot into his stomach.

“My doctors said results may take a bit longer this way, so I wasn’t expecting anything immediate. It was only the first mini dose,” Mike explains. However, within 48 hours, Mike was in excruciating pain to the point where he could barely speak. During this, he received a phone call from his doctor who let him know that his cancer levels had dropped 99%. The pain Mike had been in was caused by something called cytokine release syndrome, and while difficult to endure, meant that the drug was effective for him.

“I wish I had known about it before. Maybe anticipating the pain would have made it easier to get through,” Mike reflects.

After finishing up with Teclistamab, Mike’s cancer was undetectable for 18 months. However, because there is currently no cure for multiple myeloma, the cancer tends to have a recurrence. In 2022 when his cancer returned, Mike signed up for another clinical trial run by Genentech for a treatment called Cevostamab. Within two weeks of starting the trial, Mike’s cancer was in total remission and has remained undetectable since.

Patient Advocacy Work

Recently, the drug Teclistamab was approved by the FDA and is now available to multiple myeloma patients as Tecvayli. “It’s incredible knowing that my participation in a study contributed to a new treatment for cancer,” Mike notes. “My experience has driven me to become more involved with patient advocacy, so I can share my story with others and be the impetus for significant change in our broken healthcare system.”

Over the last decade, Mike has spoken at various events and conferences about his experience and the importance of clinical research participation.

In your lifetime, you will likely either be diagnosed with cancer, or have a loved one who is diagnosed. I don’t want people to feel frozen by fear when it happens like I did at first. Having the resources and knowledge you need ahead of time can change that experience and outcome entirely,” Mike says. 

Together, Mike and Angela founded Speaking on Cancer Patient Advocacy (SoCPA), a non-profit with a mission to empower cancer patients and improve their treatment outcomes. The website includes helpful resources for individuals who have just been diagnosed, including guides to reading lab results, and education about different types of cancer.

Looking ahead, Mike is excited about some upcoming projects SoCPA has planned to help promote cancer awareness and education in larger companies and health care systems. His aim is to encourage companies to invest more in employee health and education, ultimately creating an environment where colleagues are comfortable navigating a cancer diagnosis in the workplace and know what their treatment options are.

“Being involved in patient advocacy and meeting such wonderful people has kept me motivated to work even harder.”

Additional Resources:

https://www.socpanow.com/index

https://themmrf.org/multiple-myeloma/

https.myeloma.org  

 

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.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Meeting UK IRB/EC Expectations for Patient review of Research Participant Information

From December 1st 2023, applications to conduct clinical research in the United Kingdom must meet the Health Research Authority’s (HRA) Quality Standards and Design and Review Principles to receive a favorable opinion. Research Ethics Committees (RECs) will check whether the standards have been achieved, including implementation of a sufficient patient review process to ensure that participant information is relevant and understandable for the intended audience.

This webinar reviews key elements of these expectations and provides logistical and practical considerations for meeting them. Speakers include experts in patient engagement and involvement of patient communities in the development of clinical trial related documentation and participant communications, as well as perspectives from stakeholders who have experience in the roles that must fulfil the new requirement.

Featured Speakers

Behtash Bahador

Behtash Bahador

Director, Health Literacy, CISCR

Thomas Rhode Jørgensen

Chief Operating Officer (COO), James Lind Care

 

Results from CISCRP’s 2023 Perception & Insights Study

CISCRP’s Perceptions & Insights Study routinely assesses global trends in attitudes, perceptions, and experiences associated with clinical research among patients and the public. The results from our latest 2023 study are in, with over 12,000 responses from around the world! We discuss the latest findings from this study during the webinar.

Featured Speakers

Annick de Bruin

Chief Research and Insights Officer, Research Services, CISCRP

Rebecca Nordland

Senior Project Manager, Research Services, CISCRP

headshot_Emily Clifford

Emily Clifford

Senior Project Manager, Research Services, CISCRP

Jackie Zimmermann MS Advocate

Jackie Zimmerman

MS & IBD Patient Leader

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Twyla Bode RN BSN MN

Clinical Research Nurse Coordinator

Highlights From the 2023 Study Include:

  • Public understanding of clinical research and perceptions of safety increased post-pandemic
  • Top mentions for increasing trust in pharma companies conducting trials:
    • By the company sharing information about the health risks and benefits of their medicines
    • Knowing that the company actively works with patients, caregivers, and patient communities to make clinical research studies easier to participate in
    • By the company sharing information in patient-friendly language that they can easily understand
  • About half were ‘very willing’ to go to a pharmacy for study visits, however those who were not willing were most concerned:
    • About the facilities (i.e., too public, lack of a private room/area)
    • That their privacy/confidentiality would not be protected, and
    • About the quality of care they would receive

Insights on Developing an Impactful DEI Video

Hear from our panelists who share insights gained while developing CISCRP’s video, The Importance of Diversity in Clinical Trials. The discussion will focus on how to embed key DEI principles and concepts, not only in the product but also in the process, ensuring a balance is struck between nonpromotional education and advocating for participation, logistical tips for planning and executing a successful live-action video project.

Featured Speakers

Lucas Goren

Senior Project Manager, CISCRP

Behtash Bahador

Director, Health Literacy, CISCRP

Tammy Wilkins

Tammy Wilkins

Senior Manager, Applied Innovation and Process Improvement, Otsuka

matt-low

Matt Low

Chief Creative Officer, Praxis Communications

Mel Hardman

Dr. Melissa Hardman, MPH, MS

Faces of Research

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Ashley Nealy

COVID-19 Clinical Trial Participant

Melvin

Melvin Mann

Video Participant

Medical Hero Spotlight: Brittany Foster, Pulmonary Hypertension Patient Advocate

Living with Pulmonary Hypertension

Brittany Foster may not appear to be struggling with a chronic condition upon first impression, however, she has been navigating a series of complex medical diagnoses for decades. At birth, doctors discovered Britt had a blockage in her intestines. She was rushed into emergency surgery, where it was quickly discovered she had also been born with a heart defect when she went into sudden cardiac arrest. At less than a year old, Britt was taking medication for heart failure and had a procedure to repair the hole in the bottom of her heart, called a ventricular septal defect. Soon after, she was diagnosed with pulmonary hypertension, a condition that forces her heart to work overtime to pump blood to her lungs.

Despite this diagnosis early on, Britt enjoyed a relatively normal childhood. She was very active, participating in nearly every sport with no issues. For years, the only evidence of her condition were the scars from her surgery as an infant.

Unfortunately, around the age of thirteen, Britt found herself struggling with shortness of breath while playing sports. Her cardiologist recommended she take a pulmonary function test, believing the symptom to be exercise-induced asthma. She was given inhalers and continued with daily life but soon found the inhalers weren’t fully effective.

“At that age, all I wanted to do was what my friends were doing. I loved playing sports, so I kept at it, despite my continued breathing difficulties,” Britt says.

Upon graduation, Britt began a career path in education as a teacher, a role that exemplified her passion for advocacy and helping others. “I wanted to advocate for the students who need resources, or who in some cases, did not have the ability to speak up for themselves,” Britt says. Four years into her career, Britt was managing an eighth-grade classroom when her condition began to impact her daily life again. “Many people don’t realize what a physically demanding job teaching is,” Britt reflects. “I was on my feet all day walking around the classroom and bending and crouching very often. One day I got incredibly lightheaded and ended up passing out in the classroom.”

Changing Directions: From Teaching to Rare Disease Advocacy
“Unfortunately, a big part of my story has been repeated mistreatment from medical professionals. As a young woman who physically appeared to be in good health, assumptions were made about me by doctors who weren’t interested in my prior history or symptoms,” Britt says.

After being rushed to the ER from her classroom, Britt’s doctors concluded she fainted due to dehydration despite her history of heart disease. They planned to send her home to rest with fluids, but Britt knew something else was wrong.

“I was lucky that right before being discharged, a nurse started asking more probing questions about what I had been doing right before fainting. We decided I should walk down the hallway with a heart monitor and oxygen sensor to evaluate,” Britt recalls. Within minutes, Britt’s oxygen levels had dropped rapidly, and doctors finally decided to admit her into the cardiac unit for further treatment.

Britt was released later from the hospital with an oxygen tank to assist her breathing and was told to adapt her life around her pulmonary hypertension. “I was only twenty-six years old,” Britt says. “The tanks were huge and difficult to get around with. I wasn’t given any resources on how to adapt my life, but I knew I wanted to get back into my classroom and start teaching again.”

Britt went back to teaching for several months and finished the school year but struggled with her oxygen tank throughout. Over the summer, she met with her doctors who strongly advised her to change career paths. Britt made the hardest decision of her life to retire from teaching. “I felt incredibly discouraged and depressed watching other teachers move on with a new school year, while I was sick in bed,” Britt recalls.

Finding Community & Support

During this time, Britt struggled with depression and anxiety. She turned to writing as an outlet, sharing her experience on social media, and eventually going on to write for a nonprofit organization that promotes mental health awareness. Soon after, she was hired as a columnist for BioNews to write about pulmonary hypertension. “From there I was able to start connecting with other patients, caregivers, and medical professionals, which helped build the sense of community and purpose that I had been missing,” Britt says.


In the years since, Britt most recently worked on the People & Culture team, a position that she loved. “I am grateful that BioNews truly values employees’ health and makes sure to accommodate our needs.”


Throughout her medical journey, Britt says her friends and family have been her biggest supporters. She encourages others with rare diseases to not be afraid to speak up about their experiences.

Continued Medical Treatement

Since her initial diagnosis, Britt has continued to undergo medical treatment, as doctors began to better understand her condition. At age twenty-nine, it was discovered the Britt was born with an anatomically misplaced aorta, in addition to the hole in her heart.

“My aorta branches off to the right, so all my arteries are opposite. I was in the hospital, sick and unable to keep food down. Doctors were trying to diagnose me with bulimia, until a GI specialist stepped in and found that because of my aorta, my arteries had formed a tight branch around my esophagus, making it painful to eat or drink,” Britt says.

Because of the long-term compression on her trachea and esophagus, Britt needed surgery to repair the damage. Unfortunately, this condition has caused permanent nerve damage, gastroparesis, and esophageal dysphasia. Since her surgery, Britt has become feeding-tube dependent to ensure she has adequate nutrition.

Advice for Patients

As someone who has navigated the healthcare system with a rare condition, Britt has learned valuable lessons along the way that she shares with others whenever she can.

Find a medical professional you can trust to advocate for your care.

“Rebuilding trust in medical professionals has been a challenge for me, but finding trusted doctors I can rely on has been a big help. I have an excellent relationship with my primary physician who leads my care and truly advocates for me,” Britt says.

Create a medical overview binder to share in emergencies and with new doctors.

Emergency rooms are fast paced and fast moving. When admitted, you may end up speaking with multiple doctors who haven’t spoken directly with each other about your condition. Britt recommends having one provider you trust assist you in building a document that includes all your medical diagnoses with explanations, any medications, or treatments you are currently taking, and the contact information and signature of that doctor. “I bring this folder any time I have to visit the ER and have the file stored on my phone to show staff as well,” Britt says. These documents can be helpful for treatment so that new doctors are aware of your baseline levels and other conditions.

Surround yourself with people who understand your condition and who support you.

“My medical condition has made my life and daily routine very inconsistent,” Britt says. “I never know when something will come up and I’ll need to cancel plans. Socializing takes a lot more planning now, which can be difficult.” If someone in your life is living with a chronic condition or a rare disease, Britt recommends becoming educated about their condition and learning to adjust expectations when it comes to socializing and other daily commitments.

Trust the timing of your life.

“Six years ago, I thought I had no future and I had lost a career I was passionate about because of my rare disease. Today, I have a supportive community of people who understand my experience and for that I am grateful.”

Additional Resources:

https://www.lung.org/lung-health-diseases/lung-disease-lookup/pulmonary-hypertension

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.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Clinical Research for American Indian and Alaskan Native Communities

CISCRP is committed to providing clear, unbiased, and culturally appropriate educational materials to engage and inform communities that have not been well represented in clinical trials.

Purchase Single/Quantity Brochures at our Store

We developed this brochure together with members of American Indian and Alaskan Native communities, as well as subject matter experts who have experience working with these communities. This included receiving feedback from 500 members of the American Indian and Alaskan Native community through an anonymous survey.

They all helped make sure the topics, language, images, and design are appropriate and engaging. We also received feedback on how effective our brochure was for raising awareness about the importance of American Indian and Alaskan Native participation in clinical research.

This process allowed us to confirm that we are addressing the key concerns and barriers that prevent better diversity, equity, and inclusion in clinical research. This brochure was also reviewed by an Institutional Review Board (IRB), which is also known as an independent ethics committee. The IRB review ensures the brochure follows ethical guidelines for providing information about clinical research to patients and the public.

Topics include:

  • Why are Clinical Trials Important?
  • The Need for Diverse Participants in Clinical Trials
  • Why Have American Indian and Alaskan Natives Not Been Represented in Clinical Trials?
  • How American Indian and Alaskan Native Participants are Protected
  • Things to Consider Before Volunteering
  • Different Ways to Get Involved

Medical Hero Spotlight: Katie Hill, Appendix Cancer Clinical Trial Participant

Diagnosed with Appendix Cancer

Like most patients diagnosed with appendix cancer, Katie Hill had no idea her body was battling a deadly disease. Her diagnosis story began in 2021, when she made the decision to undergo a partial hysterectomy after struggling for years with pain caused by fibroids. At the hospital, her care team provided her with consent forms to read through and sign off on before the procedure. These forms gave her doctors permission to make other decisions as medically necessary if needed, a standard practice during surgeries.

“Because of COVID-19, I went in for the procedure by myself, and when I woke up in my recovery room, my surgeon let me know that there was an unexpected discovery they had made while I was under,” Katie recalls.
Her doctors had noticed an unusual spot on her appendix and decided to remove it just to be safe. Katie was informed that the team had also found some excess tissue near the appendix and abdomen, which they thought might be from endometriosis. With her appendix and tissue sent to the pathology department for testing, Katie was released from the hospital to recover at home.

Two days later, Katie noticed an email from her patient portal about her test results.

“Assuming it was nothing, I opened the email and started reading the pathology report from testing my appendix and tissue,” Katie says. “It was from that email that I learned I had Stage 4 metastatic mucinous adenocarcinoma of the appendix.”
“I remember sitting in my living room in complete shock. I didn’t know what else to do but to start Googling my cancer. It felt like a lifetime, but it was only about 5 minutes of reading before my surgeon called, interrupting my research. I could tell immediately by his voice that there hadn’t been a mistake, that this was real. Before he could say anything, I cut him off and said, ‘I know.’”

That afternoon, her surgeon had been desperately trying to prevent the hospital’s patient portal system from releasing the pathology report to Katie so that she would not learn of her diagnosis in such a clinical and shocking way. On their phone call, Katie learned a team at the hospital had been in discussions all day about her rare cancer and were researching treatment options for her.

Finding a Treatment Plan

Some patients might pull back from researching their cancer, but Katie jokes that research has always been a part of her DNA. “I needed to understand everything I could about my cancer.” 

She started by finding online resources and support, through groups like the Appendix Cancer Pseudomyxoma Peritonei Research Foundation (ACPMP), and patient support groups on Facebook. On patient forums, Katie quickly learned that it is critical for appendix cancer patients to select a specialist at a high-volume cancer center, or someone who has treated hundreds of patients with this disease.

“It used to be said that the chances of getting appendix cancer was 1 in a million. Now it’s about 2.5 in a million, so there are still not many doctors who have a lot of experience treating it,” Katie shares.

Katie spent the next week going to consultations in her home state of Pennsylvania and traveling to several appointments in New York. Within 2 weeks, she had selected her cancer specialist at Memorial Sloane Kettering.

“Part of my selection process for a specialist included finding a high-volume cancer center. I wanted a surgeon who has seen at least 100 or more cases of this a year, and I was also interested in finding a cancer center that had clinical trials available,” Katie explains. “If I have a rare cancer and have the opportunity to participate in studies that further care for other patients, I feel a duty to help.” 

Katie began treatment with chemotherapy to shrink her tumors. Her next step involved a procedure called cytoreductive surgery (CRS), which involves scraping the cancer off and, in some instances, removing organs. 

“My cancer is unique because it’s not a solid tumor, which is how most people visualize cancer. My cancer is like a jelly that spreads out across my insides, making it hard to detect on scans and then to remove.”

Joining a Clinical Trial

For Katie, clinical trial participation was a major consideration in her treatment plan, and she was fortunate to qualify for the ICARuS study. “It’s standard practice for most surgeons to use a heated chemotherapy agent as a wash to kill cancer cells. The ICARuS study is a randomized trial comparing two different methods of administering chemotherapy,” Katie explains. The purpose of the trial is to determine if outcomes for patients are different when using different types of chemotherapy.

As a rare disease patient, Katie has also had the opportunity to enroll in an observational study, called the Genetics of Appendix Cancer, or GAP study. “Right now, many appendix cancer patients use the same treatments as colon cancer patients because there is very little awareness or funding for research for this disease,” Katie explains.

“Many surgeons who run into this disease have never seen it outside of medical textbooks and oftentimes will misdiagnose or begin treatment, which could be detrimental to patients. They assume they’re doing good, but don’t realize that this is a different disease that needs to be treated differently.”

Appendix cancer was previously not considered curable, but we now know that some patients are making it for long periods of time with no evidence of disease (NED). Katie is currently 18 months NED! She hopes to be one of the patients who goes for a long time with NED but shares that she will be happy to get a couple of years without recurrence. “Our industry is moving so quickly that there will be new treatment options waiting for me down the line, which I am thankful for.”

Katie the day she arrived home after surgery, completing her treatment journey.

A Career Rooted in Life Science Collaboration

Katie has spent the last 20 years of her career working in different roles at the Drug Information Association (DIA), an organization dedicated to improving outcomes for patients through industry collaboration. Most recently, as Senior Vice President and Managing Director of Learning and Digital Solutions, Katie oversaw the development of DIA’s Patient Engagement eLearning Program, which helps to train professionals on best practices for integrating the patient perspective into the full medicines lifecycle. 

Unlike others fighting cancer, Katie wasn’t hesitant about sharing her diagnosis with her colleagues who were all incredibly supportive during her treatment. “My diagnosis was just another affirmation that the work we do at DIA is important. My colleagues and I come to work every day because of patients like me living with diseases, waiting for breakthrough treatments,” Katie shares.

For others navigating a difficult diagnosis for themselves or a loved one, Katie emphasizes that there is no ‘right’ way to move through your medical journey. “Everyone has different experiences and setbacks when dealing with a disease. I tried to maintain a positive attitude throughout my treatment, but that might not work for everyone,” Katie explains.

To newly diagnosed patients, Katie compares their upcoming medical journey to riding a rollercoaster.

“Everyone who has been on a rollercoaster knows that the scariest part is the slow climb to the top. In healthcare, most patients experience this immediately after their diagnosis, when there is so much uncertainty, fear, and often waiting. Once you’ve established a plan and found your care team, your treatment begins, and things really speed up. I know that for me, it was scary, but I felt secure knowing I had a top-notch medical team by my side and that I was making progress,” Katie says.

Additional Resources:

https://acpmp.org/ 

View an overview of the ICARuS study 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.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Eligibility Criteria for Clinical Trials

Are you, or someone you know, considering joining a clinical trial? We developed this infographic to serve as a resource to better understand eligibility criteria for clinical trials.

This infographic was reviewed by an Institutional Review Board (IRB), which is also known as an independent ethics committee. The IRB ensures the brochure follows ethical guidelines for providing information about clinical research to patients and the public.