Campaign for the Cure - C4C22

Dear Friends,

We are excited to launch Campaign for the Cure 2022 (C4C22) this week, with the goal of raising $100,000 during the month of May in support of the Cholangiocarcinoma Foundation's (CCF) mission. Throughout the campaign, you will learn about how the Foundation plays a pivotal role in advancing one crucial aspect of our mission—championing research that leads to the development of new treatment options and, ultimately, a cure.

Our research philosophy is based on a single objective: to find a cure as quickly as possible. However, we understand that achieving this goal is not so straightforward. The Foundation is building the path to a cure through a combination of strategic activities designed to overcome barriers to progress and better leverage existing resources so that they can have a greater impact.

When determining how to invest our time and resources into research activities, we prioritize the following:

• Projects that provide value to the entire CCA research community. This includes research that increases our understanding of the genetic factors and biological mechanisms that contribute to disease progression or remission.
• Projects that foster collaboration and/or improve research efficiency. Examples of this include research consortiums or activities that benefit multiple researchers through the use of model systems, shared patient data (de-identified), or shared access to patient bio-specimens.
• Projects that have the greatest chance of delivering new, effective treatment options to patients as soon as possible. We focus on innovative research designed to open new pathways for drug discovery, enhance diagnostic capabilities, improve patient outcomes, or address the unmet needs of specific patient populations.
• Projects that contribute to the overall vitality and growth of the CCA research community. For instance, efforts to invest in the career development of young CCA investigators, projects that are less likely to get approved for traditional funding, or initiatives that have a long-term (but important) payoff.

We hope you will be inspired by what you learn during C4C22. From fostering collaboration between researchers all over the globe, funding high-priority research, and ensuring the patient voice is included in every phase of the drug development process, the Foundation is making a tremendous impact on the field of CCA research… but we need your support to continue our momentum.

Please help us reach our goal of raising $100,000 through C4C22 by donating now.

Thanks to the generosity of the Scott Family, your donation to this year's campaign will be matched dollar-for-dollar up to $20,000—meaning the impact of your donation will be doubled!

Together, we can give patients the support they need today while making great strides towards a future free from this terrible disease.

Dear Friends,

The Cholangiocarcinoma Foundation realizes that the fastest way to advance research is by working together. As the leading advocate for patients and the hub of the cholangiocarcinoma (CCA) community, the Foundation is uniquely positioned to bring together world-renowned clinicians, researchers, institutions, and industry partners in a collaborative effort to achieve a common goal: finding a cure.

Collaboration accelerates the pace of research for many reasons, including the following:

• It helps identify the most promising areas of investigation within the field of CCA research by harnessing the collective knowledge of a diverse group of experts. Building consensus around scientific priorities helps guide researchers’ activities, allowing the field to be more effective.
• Sharing resources (including expertise, data, etc.) enables researchers to invest more energy into the most impactful areas of their work and operate more efficiently.
• Finding a cure for a disease as rare and complex as CCA demands more creative solutions. When clinicians and researchers collaborate, their distinct experiences, common challenges, and unique perspectives can contribute to a greater understanding of CCA. In turn, these shared learning opportunities foster innovation (and at a much faster pace).

Below are highlights of a few collaborative research efforts led by the Foundation:

• Launched by CCF in 2015, the International Cholangiocarcinoma Research Network (ICRN) is composed of highly accomplished researchers from around the globe that represent a full spectrum of disciplines, perspectives, and research methods. The purpose of the network is to rapidly translate scientific discoveries into new treatment options or diagnostic tools that improve the outcomes of CCA patients. You can learn about the ICRN and their work HERE.
• Our International Cholangiocarcinoma Patient Registry (ICPR), launched in 2014, is the only database of its kind, offering direct access to de-identified CCA patient data to approved researchers for use in their studies. Patient data is critical to the success of research projects; but because the population of CCA patients is relatively small, it can be difficult for many researchers to access the volume of information they need on their own. The ICPR helps remove this barrier and allows researchers to complete studies more rapidly. Patients can learn more about contributing their data to the ICPR HERE.
• Later this week, the Foundation is hosting the Cholangiocarcinoma Think Tank (known as C2T2) meeting in Las Vegas. This first-of-its-kind meeting will bring together a select, multidisciplinary group of researchers to determine research parameters for projects seeking to advance two specific areas of study: 1.) early diagnosis and prevention, and 2.) precision immuno-oncology therapeutics. We look forward to sharing the outcomes of this meeting soon!

The Foundation is committed to advancing treatment options for CCA patients as rapidly as possible, but we know that time is not on our side when fighting this terrible disease. We need your help.

Please help the Foundation continue to drive research innovation and give patients access to the support they need by donating to C4C22.

Sincerely,

Stacie

P.S. Remember, all donations to C4C22 are eligible for a dollar-for-dollar match—meaning the impact of your gift will be doubled!

Dear Friends,

The Cholangiocarcinoma Foundation (CCF) knows that ensuring the long-term vitality and growth of the cholangiocarcinoma (CCA) research community must include investing in promising young investigators, which is why we offer the Research Fellowship Program and two mentoring programs.

Did you know that receiving early-career funding is a key factor influencing both research advancements and the career paths of researchers?

• The probability of scientists who receive career development awards obtaining a full professorship is 2x greater than that of rejected applicants. The likelihood of general career advancement is also about 9% higher for grant recipients.
• Grants from the National Institutes of Health (NIH) are among the most common sources of seed funding and career development awards, but the process is incredibly competitive. There is a direct correlation between the amount of NIH funding available and the number of medical students who choose to pursue research careers in specific fields.
• In 2020, the NIH awarded $130M for 291 projects investigating various types of cancer impacting the liver. Only 12 of these awards were specific to cholangiocarcinoma research (3.4% of these funds).

Scarcity of early-career CCA research funding → fewer scientists entering the CCA research field → stifled innovation → fewer CCA research breakthroughs!The Foundation helps overcome this hurdle by providing seed funding for young investigators through our Research Fellowship Program and an opportunity for them to enhance their skills through two mentoring programs. These programs empower early career researchers to become more competitive in seeking funding from the NIH and other sources. You can read more about the Career Mentoring Program and Scientific Mentoring Program programs HERE.

Since 2015, the Cholangiocarcinoma Foundation has awarded 39 fellowships totaling over $2M in seed funding through our Research Fellowship Program. These awards aim to support early-career investigators who are conducting innovative, high-quality cholangiocarcinoma research. The benefit of our Research Fellowship Program is two-fold—in addition to the value of the research conducted under the grant itself, this funding plays an important role in developing a robust pipeline of new CCA scientists who can contribute to the long-term advancement of CCA research. You can learn more about each scientist and their research by clicking HERE.

Dr. Lipika Goyal is a perfect example of how the Foundation's initial investments into the career of a young investigator can have a ripple effect that continues to grow and shape the face of CCA research today. Dr. Goyal recently shared that the initial Research Fellowship Award she received from us in 2016 inspired her to become more involved in CCA research and eventually drove her to apply for additional funds from the NIH and other large grant sources. As a result, the $50,000 award we originally gave her enabled her to receive several million dollars in grant funds for CCA research!

There are several stories like Dr. Goyal's that illustrate the importance of supporting early-career CCA researchers. The transformational work they are conducting will pave the way for finding better treatments for cholangiocarcinoma, but this is only made possible through your willingness to entrust the Foundation with your generous support.

Please partner with us by supporting this year's Campaign for the Cure, enabling us to continue down the path towards a cure.

P.S. IMPORTANT C4C22 UPDATE: Allard's Art has generously offered an additional C4C22 match of $5,000 in honor of CCA warrior Lisa Heard—bringing the total dollar-for-dollar match available to $25,000!

Dear Friends,

The Cholangiocarcinoma Foundation (CCF) believes that an important part of nurturing a robust scientific ecosystem where research innovation can flourish must include building strong, patient-centered relationships with industry partners. To this end, the Foundation actively engages with companies that share our commitment to cholangiocarcinoma (CCA) patients and our common goal of providing them with better therapeutic options and improved quality of life.

Some of the key reasons CCF engages with industry to benefit CCA patients include the following:

• CCF ensures that the patient experience and voice are considered throughout the drug development process, which often helps shape clinical trial protocols and highlight the treatment considerations that matter most to patients.
• CCF advocates for the development of products that help address unmet patient needs.
• CCF is able to share timely, accurate information about clinical trials and their outcomes with the CCA community, empowering patients and caregivers to better understand treatments that are in development and trials that may be available to them.
• CCF helps bridge the divide that traditionally exists between research being conducted in academic settings versus an industry setting to ensure that the most promising scientific leads are pursued.
• CCF uses grants and sponsorships from industry partners to help expand upon important mission activities, such as funding research projects and providing new education and support services to patients and caregivers.
• You can learn more about how CCF collaborates with industry and hear a few examples of our successful partnerships by watching the webinar located HERE.

CCF first began efforts to proactively connect with industry in 2015. Since this time, the number and breadth of companies working in the CCA space have substantially grown to include those that develop drugs, biologics, diagnostics, devices, and patient data collection platforms. The value of CCF helping these companies to consider and understand the patient perspective in their work is undeniable. Here are two recent examples of feedback that industry partners have shared about the impact of our collaboration:

• "Engagement with patients and healthcare providers is incredibly valuable as we explore clinical trial design in CCA. As scientists, we do not often get a chance to directly hear the patient's perspective on current and emerging therapeutic options. Meeting patients and better understanding their journeys provided a powerful impetus to us as researchers and profoundly impacted and energized our entire team..."
• "We believe that an industry-advocacy relationship not only supports our company goals, but also benefits the patient and caregiver community by being valued stakeholders with important voices in our drug development programs, helping us ensure that study designs, endpoints, and materials meet the needs of patients and caregivers, and hopefully improve adherence and patient outcomes, as well as accelerating research for new CCA therapies."

Moving towards better treatments and finding a cure requires the support of the entire CCA community—including donors like you. As CCF enters the fourth week of Campaign for the Cure 2022 (C4C22), I'd like to thank you for the generosity that many of you have already shown. If you haven't been able to contribute to C4C22 yet, I hope that you will consider helping us reach our $100k goal by donating HERE.

Thank you for being an important partner in our journey towards a cure.

Stacie Lindsey

Dear Friends,

Since our founding in 2006, the Cholangiocarcinoma Foundation (CCF) has grown to become the leading global resource for cholangiocarcinoma (CCA) research, education, and public awareness. We are proud to say that, over the years, we've seen the impact of our efforts manifest in numerous ways, including the advancement of new treatment options for CCA patients.

As Campaign for the Cure 2022 (C4C22) draws to a close today, we'd like to highlight a few recent CCA treatment advancements as well as some of the encouraging, new research projects on the horizon.

• Over the last two years, we have seen the approval of three new drugs to treat CCA by the Food and Drug Administration (FDA): TRUSELTIQ TM, Pemazyre™, and TIBSOVO®. These drugs have offered new hope for patients and serve as evidence of the importance of CCF's collaborative efforts with industry partners.
• Between February 2019 and February 2021 and in the middle of COVID, researchers successfully completed the accrual of 441 patients with advanced biliary tract cancer in a phase III trial to evaluate the impact of adding Nab-paclitaxel to the standard Gemcitabine and Cisplatin chemotherapy. An amazing accomplishment in such a short time for a rare cancer illustrating how the CCA Community is unique.
• In January 2022, we shared the promising results from the TOPAZ-1 Phase III trial; which pending the final study report, could ultimately result in future changes in CCA treatment guidelines.
• In February 2022, a session of the CCF Annual Conference allowed researchers to share how the gut microbiome is related to CCA patient health. Future research on this topic could be beneficial in many ways, including determining if microbiome biomarkers could be used as key indicators of treatment response or toxicity in CCA patients.
• In March 2022, the FDA approved priority review for the new drug application of futibatinib in the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma. If successful, futibatinib could represent a new targeted therapy option for patients who have failed to respond to first-line treatments.
• May of 2022, CCF shared information about the phase II, single-arm clinical trial to evaluate the use of Ramucirumab in the treatment of advanced biliary tract cancer patients. The trial analysis showed that Ramucirumab is well tolerated and may result in clinical benefits for previously treated patients, with future trials planned for further assessment.

Advancing treatment options and offering hope to patients and their families remain core to the Foundation's mission. With 24 hours remaining in C4C22, we hope that we can count on you to partner with us in this endeavor. We are humbled by the generosity many of you have already shown to the campaign, but we have yet to reach our $100,000 goal. We hope you will consider helping C4C22 be successful by donating now.