February 2025
Accelerating Biomedical Innovation in Appendiceal Cancer Summary & Report
We are excited to bring you highlights from our October 2024 workshop on Accelerating Biomedical Innovation in Appendiceal Cancer.
We gathered leading experts and researchers from around the world. This groundbreaking event focused on the latest advancements, treatments, and ongoing challenges in appendiceal cancer research. From basic research, to large data sets to innovative therapies, the insights shared are set to make a significant impact on both medical professionals and patients alike. Dive in to explore the key takeaways from this important meeting and stay informed about the evolving landscape of appendiceal cancer research.
2024 WORKSHOP SUMMARY
Speakers included -
Adham Jurdi, Natera - Overview of Signatera data from appendiceal cancers
Haroon Choudry, University of Pittsburgh Medical Center - Mucin targeted small molecule therapeutics and nanotherapeutics.
Jaime Cheah, The Broad Institute - The Drug Repurposing Hub at the Broad Institute
Jason Foster, University of Nebraska Medical Center - EMR informed patient registry designs and plans using our iCARE2 established registry.
Jim Palma, Target Cancer Foundation - Insight from the TRACK program from an appendiceal view
Jorge Barriuso, The Christie - Multiomics in Appendiceal tumours. The CRUK Accelerator Award on Pseudomyxoma Peritonei
JP Shen, MD Anderson Cancer Center - Network & pathway analysis of multi-‘omics data yields new insights regarding heterogeneity and pathophysiology of appendiceal cancer
Kate Mahon, Chris O'Brien Lifehouse - A snapshot of our appendiceal cancer transcriptomic data
Kiran Turage, Yale School of Medicine - Updates on autoimmunity and appendix cancer
Konstantinos Votanopoulos, Wake Forest Health - Adoptive Immunity Organoid Platform- Generating T cells that recognize and kill appendiceal cancer.
Michael Foote, Memorial Sloan Kettering Cancer Center - Challenges and opportunities in immunology-based treatments for appendix cancer.
Mustafa Raoof, City of Hope - Single cell data on appendiceal cancers
Patrick Wagner, Allegheny Health Network - The tumor microenvironment of appendiceal cancer
Rob Kimmerling, Travera - Cell mass measurements for functional assessment of drug response
Susan Horrell, Rare Cancer Research Foundation - RCRF data platform
Tatiana Hurtado De Mendoza, University of California, San Diego - Using epigenetics to identify novel therapeutic targets for mucinous appendiceal neoplasms.
Xu Han, Wake Forest Health - Cryopreservation of appendiceal cancer cells.
June 2024
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News
CANCollaborate Board of Directors - Greg Simon
As a newly formed non-profit we are still in the process of finalizing our entire board, but we are thrilled to announce that Greg Simon has agreed to be our first appointed board member. (see full bio below)
Current Board Members
Mr. Greg Simon
Mr. Steven Wallman, co-founder and chair of the board
Ms. Jane Wilkinson, co-founder and president of CANCollaborate
In addition to creating the board we have a strong team of CANCollaborators that cover every aspect of rare cancer care and research including oncologists, surgeons, technologists, and patient advocates. You can find the full list here - cancollaborate.org/cancollaborators
Mr. Simon has held senior positions in both chambers of Congress, served in two Presidential administrations. He was a senior strategy consultant to a variety of international technology CEO’s, co-founded with Michael Milken, and led, FasterCures, co-founded and led the Melanoma Research Alliance. He was the Senior Vice President at Pfizer for Worldwide Policy and Patient
Engagement, and was the CEO of Poliwogg, a financial services company creating unique capital market opportunities and indexes in healthcare and life sciences. He has developed a reputation as a visionary strategist, a dynamic public speaker and writer, and as an expert analyst of emerging trends in healthcare, information technology, innovative drug research and
development, and patient advocacy. Most recently, Greg was the President of the Biden Cancer Initiative, a nonprofit formed by Vice President Joe Biden and Dr. Jill Biden to continue the work of the White House Cancer Moonshot to double the rate of progress in preventing, detecting, diagnosing, treating, and surviving cancer. He was also instrumental in crafting the regulatory framework that is now the foundation for the biotechnology industry.
Greg Simon, CANCollaborate Board Member & Advisor
Research Updates
Advancing CANCollaborate's Mission: Workshop on CRS - cytoreductive surgery and HIPEC - heated intra-peritoneal chemotherapy
On March 7th and 11th, CANCollaborate hosted a series of virtual discussions with surgical oncologists who perform HIPEC for appendiceal cancer patients. During the discussions, leaders of the field described areas where they see opportunities for advancements that could be made in the field of appendiceal cancer research. A major point shared by everyone in attendance was the need for a clinical trials network, as well as an EMR informed registry to standardize the way that data is collected. With this, there would be the need to use a uniform mechanism to extract data from the EMRs. It was discussed that having a clinical trial network and the infrastructure in place will hopefully attract the interest of the pharmaceutical industry to test new therapies, as well as lower the costs associated with doing the clinical trials that are desperately needed to identify new therapeutic options for patients. Follow up meetings are ongoing.
Collaborations & Partnerships
CANCollaborate & SHEPHERD Health - investigating the transcriptome of appendiceal cancer patients
CANCollaborate and SHEPHERD Health are working together to investigate the transcriptome of appendiceal cancer patients, with the hope of identifying potential new therapeutic opportunities. Working with our CANCollaborators, we are compiling available RNA sequencing data by exploring all potential sources including published data, unpublished studies, and data generated by commercial vendors. SHEPHERD will then deploy their computational platform, DELVE, to identify signatures of drug sensitivity in appendiceal cancer patients with the aim of generating hypotheses regarding new therapeutic options.
Meetings & Conferences
Goodwin’s Rare Disease Day
March 2024 - Goodwin Law’s Life Sciences team hosted its Annual Rare Disease Symposium in Boston on March 13, 2024. CANCollaborate attended the symposium that included engaging fireside chats, inspirational presentations, and networking with the rare disease community.
Goodwin Law - Rare Disease Day
American Association for Cancer Research - Annual Meeting
April 2024 - CANCollaborate attended the American Association for Cancer Research meeting held in San Diego from April 5th through to the 10th. Jane and Jamie presented a poster titled “CANCollaborate: Transforming rare cancer research through cooperation and collaboration”. It was also an opportunity to connect in-person with our network of CANCollaborators with a particular highlight to Dr. Flatmark from Norway and Dr. Mahon from Australia - these in-person international meetings are essential for furthering global collaborations, and for meeting the cancer therapy dogs too.
March 2024
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Research Updates
Advancing CANCollaborate's Mission: Workshop on Organoid Creation of Rare Cancers
In keeping with our mission of creating and facilitating collaboration, CANCollaborate hosted its first virtual workshop following from its origin at MIT’s April Workshop on rare cancers with a focus on appendiceal cancer. We are developing and optimizing collaborative models that bring together leading researchers and innovators. The models, which are designed to consolidate expertise, facilitate data and model sharing – will help ensure a more effective, time-sensitive and comprehensive approach to research.
This workshop was focused on the application and value of organoid models and the challenges of creating organoids for rare cancers, especially appendiceal cancers. We gathered our CANCollaborators from the MIT April Workshop in addition to new collaborators. Our goal was to facilitate the participant’s sharing of their perspectives and expertise, while providing support for and the fostering of new collaborative projects. The workshop included cancer biologists, physician researchers, technologists, industry partners and entrepreneurs.
A particular issue is the challenge in establishing organoids for the multiple types of appendiceal cancer, and especially the rarer forms of goblet cell adenocarcinomas and mucinous appendiceal cancers.
Challenges included -
· Accessing patient samples, a challenge that all rare cancers face as the density of patients at a particular facility can be low.
· Delays in the timely accession of fresh tissue that impacts organoid generation.
· Complexities in generating organoids from low grade tumors and mucinous tumors as the mucin is presumed to be inhibiting the growth of the organoids.
· Difficulties in generating organoids for highly heterogeneous tumors that will contain divergent biological behaviors.
Organoid Models can offer a high value to rare cancer research. They can enable a high-throughput screening of known cancer drugs and therapies, and can be used to identify a combinatorial approach to treatment or the re-purposing of known therapies that have not yet been identified as a potential therapy. In addition, organoids can offer a personalized view of a patient’s tumor allowing for better personalized treatment and the elimination of highly toxic treatments. They can also be used in developing an understanding of the basic biology of the disease.
Committed to finding a path forward, and to forge new collaborations, we identified the following collaborative projects -
· Use organoid models to develop a deeper understanding of clonality and how this impacts tumor progression and therapy resistance.
· Develop a high-throughput platform for new drug screening and drug repurposing, with a special focus on combinatorial therapies.
· Evaluate the generation of personalized organoids, using normal appendiceal tissues with CRISPR editing techniques to create models for difficult tumor types.
· Generate more comprehensive molecular characterization of appendiceal cancers with a commitment to data sharing.
· Direct certain organoid research toward immuno-oncology.
· Commit to the importance of sharing experimental protocols and cross-lab training.
· Share biosamples, especially from collaborators who experience a higher appendiceal caseload than others.
The consensus among participants was clear: collaboration is essential in the face of limited drug trials for appendiceal cancer, and more extensive testing is imperative.
Collaborations & Partnerships
Travera and CANCollaborate create access to Innovative Cancer Treatment
CANCollaborate is collaborating with Travera, a company whose mission is to bring hope to cancer patients who are running out of time and therapeutic options. Through its collaboration with CANCollaborate, Travera is offering its 2-day Rapid Therapy Selection™ test to patients with appendiceal cancers free of charge to help in their efforts to establish more treatment options and outcomes data.
Travera focuses on gastrointestinal and gynecologic cancers, assisting oncologists in selecting treatments for their patients using a revolutionary cancer therapy selection test that predicts which cancer drugs are most likely to be effective for each cancer patient, a potentially valuable tool for patients with rare or advanced diseases. Travera utilizes a disruptive single-cell measurement technology invented at MIT to measure the ex-vivo mass response of live tumor cells to candidate drugs. This novel technology has overcome the problems that have prevented the many previous generations of therapy selection tests from being effective in clinical practice.
CANCollaborate has created a network of appendiceal cancer CANCollaborators – oncologists, surgeons and researchers who will also have access to the Travera technologies through the Travera community of partnerships which include many patient advocacy and research groups.
We invite oncologists, surgeons, patients, and caregivers to hear more about this offering in a recorded webinar. Follow this link to learn more.
News
Joining the ARPA-H health innovation network
We are thrilled to announce that we’ve been selected as a spoke of the ARPA-H Investor Catalyst Hub network, a regional hub of ARPANET-H, a nationwide health innovation network launched by the Advanced Research Projects Agency for Health (ARPA-H).
Based in the Greater Boston area and managed by VentureWell, the Investor Catalyst Hub seeks to accelerate the commercialization of groundbreaking and accessible biomedical solutions. It utilizes an innovative hub-and-spoke model designed to reach a wide range of nonprofit organizations and Minority-Serving Institutions, with the ultimate aim of delivering scalable healthcare outcomes for all Americans.
As an Investor Catalyst Hub spoke, CANCollaborate gains access to potential funding and flexible contracting for faster award execution compared to traditional government contracts. Spoke membership also offers opportunities to provide input on ARPA-H challenge areas and priorities, along with access to valuable networking opportunities and a robust resource library.
Meetings & Conferences
Target Cancer Foundation - Think Tank on Advancing Precision Medicine in Rare Cancers
November 2023 - The Target Cancer Foundation Think Tank on Advancing Precision Medicine in Rare Cancers was held in Boston. Bringing together patients, patient advocates, foundations, clinicians, researchers and industry partners to highlight the challenges in rare cancers. Talks included a focus on tumor agnostic clinical trials and how these may offer important breakthroughs in rare cancers.
Advanced Cancer Therapies Meeting
February 2024 - CANCollaborate attended the Society for Surgical Oncology’s 2024 Advanced Cancer Therapies meeting held in San Juan Puerto Rico. We met many of our CANCollaborators in person. There were sessions detailing the current state of treatment for appendiceal cancer patients with presentations on optimizing CRS/HIPEC and the role of circulating tumor DNA (ctDNA), as well as research talks including studies looking at the single cell transcriptomics of appendiceal cancer as well as the metabolomics of appendiceal cancer. We saw the progress being made in identifying a biomarker for treatment response in other peritoneal diseases with work looking at the role of exosomes from the peritoneal space. The message was clear that there is a team of experts who are excited to work together to improve upon the care and treatment of patients with appendiceal cancer.
Rare Disease Day at the Broad Institute
March 2024 - The Broad Institute of MIT and Harvard in collaboration with The Termeer Foundation hosted the 9th annual Rare Disease Day event, “Climbing Ladders to Cures in Rare Disease Research''. Rare disease and rare cancer share many challenges that often stifle our progress in research - business models, regulatory requirements, traditional funding structures, and research that is often siloed. Like many of our rare disease colleagues we hope to change these research norms. You can view the event talks here.
New Appendiceal and Rare Cancer Publications
The future of precision cancer therapy might be to try everything Elie Dolgan 2024 featuring Alice Soragni
Cancer Is Striking More Young People, and Doctors Are Alarmed and Baffled Brianna Abbot 2024
Serum Tumor Markers and Outcomes in Patients With Appendiceal Adenocarcinoma JP Shen 2024
Doublet vs. Triplet Systemic Chemotherapy for High Grade Appendiceal Adenocarcinoma with Peritoneal Metastases Oliver Eng and Kiran Turaga 2023 (online 2024)
The Growing Problem of Early-Onset GI Cancers - ASCO GI Keynote Lecture with Kimmie Ng, MD, MPH 2024
Team Updates
We are hiring - summer internship
Firstly - a big thank you to Kyra Itskowitz for joining us this winter as our first intern and we’re looking forward to continuing our work with Kyra this summer.
If you’re interested in joining CANCollaborate for a remote summer internship please drop us a line here.
