At the 2018 LA Party for a Cure fundraising event, Mission: Cure had the honor of awarding the Courage Award to four remarkable young individuals living with chronic pancreatitis. While the event as a whole was a verifiable success, raising over $150,000 to support the search for a cure, it was the stories of Matthew Musto (aka blackbear), Kayla-Ann Keagy, Diana Miranda, and Diego Miranda that made the biggest impact of the night. We are now sharing their stories of how chronic pancreatitis has affected their lives and the hopes they hold for the future, as well as updates from our scientific research team who attended the event and are working on cutting edge research. Here is the story of Kayla-Ann Keagy, who was diagnosed with chronic pancreatitis as a young child, in her own words:
At the 2018 LA Party for a Cure fundraising event, Mission: Cure had the honor of awarding the Courage Award to four remarkable young individuals living with chronic pancreatitis. While the event as a whole was a verifiable success, raising over $150,000 to support the search for a cure, it was the stories of Matthew Musto (aka blackbear), Kayla-Ann Keagy, Diana Miranda, and Diego Miranda that made the biggest impact of the night. Over the next few days, we will be sharing their stories of how chronic pancreatitis has affected their lives and the hopes they hold for the future, as well as updates from our scientific research team who attended the event and are working on cutting edge research. Today we are starting with the stories of Diana and Diego Miranda, two siblings who were both diagnosed with chronic pancreatitis before the age of seven, in their own words:
Chronic pancreatitis (CP) is a poorly understood disease, to the immense frustration of patients, clinicians, and researchers. There is good news: consensus guidelines on chronic pancreatitis pain have been developed by an international team of experts. The task now is to make clinicians and patients aware of them.
The challenge of managing chronic pancreatitis pain
Abdominal pain is the most prevalent symptom of chronic pancreatitis, yet chronic pain is notoriously difficult to assess and treat. Doctors must often rely on patients’ descriptions of their pain, but they tend to distrust these reports. Doctors, and sometimes family and community members, may dismiss patients’ complaints of chronic pain as exaggerated, or view the patients as overly sensitive.
When chronic pain comes from pancreatitis, additional factors come into play. Very often, the cause of the pain isn’t clear, making it hard for clinicians to address it. Sometimes, the reasons for pain are found outside of the pancreas (such as a peptic ulcer), which can be easier to treat once identified. Adding to identification and treatment complexity is that some patients have multiple sources of pain that can flare up at the same time or at different times. Read More
Interview with Chronic Pancreatitis Patient, Christine Cataldo
Earlier this summer, Mission: Cure’s own intern, Gabriela Gil, sat down with Christine Cataldo, a patient volunteer with Mission: Cure, to learn more about Christine’s experiences living with chronic pancreatitis. Diagnosed with pancreatitis linked to the PRSS1 genetic mutation in 2002, Christine shared stories how her condition was identified, discussed her family’s relationship with the disease, and emphasized the importance of patient advocacy in pancreatitis research:
As many of you may know, Mission: Cure recently launched its first patient survey to gain real-world patient perspectives on living with the disease. Although the survey is ongoing, and we ask that patients who have not completed the survey take a moment to do so, we are also excited to share some preliminary insights that we have gathered from the first 145 responses. We are excited to present a white paper report on the results of these first responses that was shared with researchers, clinicians, and FDA authorities at the PancreasFest conference in Pittsburgh, PA earlier this week. The report is in tune with an FDA draft guidance issued just last month on “Patient-Focused Drug Development” and came at a pivotal time in a transforming US healthcare system in which patients are beginning to take the driver’s seat in directing care.
The survey, phrased as an open dialogue with the FDA, asks patients to shed light on their symptoms and current treatment plan and to describe the aspects of the disease they find most difficult to live with. Initial analysis reveals four recurrent themes in patient experiences: a huge lack of understanding of pancreatitis from medical professionals, a lack of available treatments, the need for continuing research, and burdensome symptoms that place a large toll on daily life. We invite everyone to read through and download the report found by following this link to glean more detailed insights into our results.
Putting patients at the forefront of decision making has the potential to unify researchers, drug developers, and government officials with a streamlined goal of improving patient outcomes. In the words of co-director Linda Martin, “This survey is just the beginning. As we continue to connect with and understand the patient perspective at a deeper level, we will be able to guide the industry to make meaningful choices that will have a direct impact on patients.”
We would like to thank Annie Kennedy and Ryan Fischer from Parent Project Muscular Dystrophy for kindly providing advice and suggestions for our patient survey and helping make this project possible.
For any further questions regarding our report or our patient survey project as a whole, please reach out to email@example.com.
An official press release regarding our patient survey can be found here.
Here at Mission: Cure, we strive to bring the patients’ voices to the forefront of our work. Our mission is to cure chronic and recurrent acute pancreatitis by using an innovative financing model to incentivize research and development. We are asking for you, patients and caregivers, to take a couple of minutes to fill out our patient survey. We believe that the needs and perspectives of you – someone who lives with pancreatitis – should be placed at the center of finding cures. We want researchers to know what improvements you most want to see so that they can identify the most appropriate treatment targets. We want drug developers to know what has not yet been addressed so they can prioritize future decision making. We want the Food and Drug Administration (FDA) to know how you evaluate risks and benefits so drug regulation and approval can be aligned with your preferences. We want to bring your input into every aspect of the drug development and evaluation process, so drugs that will address your needs can be brought to you faster. Our patient survey aims to provide us with a better understanding of the challenges that you want us to focus on and to lay the groundwork for us to better connect and mobilize the patient community.
Mission: Cure is starting this effort with our first patient survey, today. This short survey should only take about 10 minutes to complete and will provide valuable primary information for our future efforts to collect comprehensive patient experience data. We would really appreciate it if you could take a few moments to complete the survey, and share it with others who can also contribute.
Mission: Cure’s first pancreatitis patient survey is here: https://goo.gl/forms/ZSqdXdBjGmlrUwcL2
I’ve been researching and treating chronic pancreatitis for decades, so I know firsthand what a complicated disease it is and how many questions and unknowns remain. Answers are finally beginning to emerge, but the research is still very preliminary, underscoring how much more we need to learn. It has become increasingly clear that chronic pancreatitis (CP) is caused by multiple factors. Those factors vary from patient to patient and in how they interact with each other.
The focus on alcohol harms patients and delays progress on chronic pancreatitis
As a scientist, I’m worried by the continued misunderstanding of the etiology of chronic pancreatitis. As a clinician, I’m troubled by the often-tragic effects this has on CP sufferers.
The focus on drinking and alcoholism stigmatizes pancreatitis patients and prevents them from getting the best care. Patients are counseled to “just quit drinking,” advice that is as inappropriate as it is insensitive. Parents of pediatric patients are accused of giving their children alcohol. Conversely, light drinkers or nondrinkers receive incorrect or delayed diagnoses. The beliefs about alcohol’s role have also gotten in the way of developing effective treatments.
If you’re a CP patient, you probably know all this. I’ve seen it with my own patients over the years: the suffering of the disease is compounded by frustration at the lack of treatment options. It’s a terrible mix that leaves many patients debilitated, discouraged, and depressed.
Unfortunately, most people—including most doctors—cling to an outdated, one-dimensional view: that CP is caused by alcohol. This belief is constantly reinforced by the media. We’ve seen this recently in coverage of the suicide of Swedish DJ Avicii, which often links his pancreatitis to heavy drinking.
New research is giving us a fuller picture of chronic pancreatitis
Fortunately, a small group of determined researchers has begun to untangle the causes of CP and put the role of alcohol in better context. Advances in genetics have played a huge role. Precision medicine—tailored to particular combinations of genetic, environmental, and lifestyle conditions—is an exploding area of research, and chronic pancreatitis is a natural target. Because CP is so complex, we needed these developments to get a better handle on it.
New science has also shifted our approach. For a long time, research focused on the fully diseased pancreas in long-term chronic pancreatitis. But now we’re looking at the process of reaching that stage of disease. This not only helps us better understand CP’s progression; it will also help us find therapies for earlier stages of the disease.
Here’s what the science is now telling us about alcohol and CP:
- In general, drinking must be very heavy to show an association with chronic pancreatitis: Only very heavy drinking—four or five drinks a day over a prolonged period (five years)—appears to be associated with the onset of CP. Even among people who drink that amount, only about 3 percent develop pancreatitis.
- Genetic factors appear to be linked to alcohol-induced chronic pancreatitis: In that small group of heavy drinkers who develop CP, genetic alterations appear to be involved. The mechanisms of alcohol-induced pancreatitis are complex and involve disorders of both the acinar cell and ductal cells of the exocrine pancreas.
- Moderate drinking appears to be associated with lower rates of chronic pancreatitis: Moderate drinking is defined for women as less than one alcoholic drink per day and for men, less than two alcoholic drinks per day. (But there is a crucial caveat: once a person has CP, any amount of alcohol may contribute to progression of the disease.)
- Tobacco use is an even greater risk factor than alcohol use: Evidence increasingly points to tobacco as a risk factor; it is also associated with progression of the disease. Tobacco appears to inhibit a protective pathway in acinar cells.
Next steps in CP research and treatment
These are important findings, but the new wave of research is still in its early stages. And the group of scientists exploring these issues remains relatively small.
We need more attention to this disease, and more collaboration. We need to collect and analyze more information on alcohol, genetics, and other factors that influence the symptoms and progression of CP.
Some needed steps include
- Determining the potential role of genetics in the formation of the disease. Patients should encourage their doctors to obtain genetic testing as treatments are being developed.
- Analyzing a wide range of information (including genetics) from pancreatitis patients to understand the many factors involved in this complex disease, which will lead us to treatments that might not otherwise have been considered.
- Conducting pilot clinical trials based on already approved drugs that attack the disease mechanisms that drive or contribute to chronic pancreatitis.
- Creating, developing, and testing the next generation of therapeutics directed to specific molecular pathways.
I’m working with researchers on all of these fronts. We are fortunate to have Mission: Cure behind us. Its innovative financing model will jumpstart these early advances and move us more quickly toward the goal we all share: finally delivering effective treatments and better outcomes to chronic pancreatitis patients.