Breaking News:

Accelerated Approval for New Immunotherapy

The FDA has just granted accelerated approval to Tecelra (afamitresgene autoleuecel), a groundbreaking new immunotherapy for synovial sarcoma—a rare and aggressive cancer that primarily affects young adults. Tecelra is the first lab-engineered immune cell therapy to be approved for a solid tumor cancer, offering a new ray of hope for patients who have exhausted other treatment options. In clinical trials, nearly 37% of patients saw their tumors shrink after just one dose!

This approval is a huge step forward in the fight against rare cancers. Your donation to the Jedi Rare Cancer Foundation helps fund more lifesaving advancements like this.

Remembering CANCollaborate Co-founder

Kathleen Mary Harte Wallman

We are deeply saddened by the passing of Kathleen Mary Harte Wallman, a brilliant lawyer, educator, and advocate who co-founded CANCollaborate, a nonprofit devoted to accelerating treatments and cures for rare cancers. Kathy made an indelible mark on the world through her work and spirit as a dedicated advocate for rare cancer research. She passed away peacefully, surrounded by loved ones, after a battle with appendiceal cancer.

Kathy’s visionary work has been instrumental in bringing hope and advancing treatments for those affected by rare cancers. Kathy's passion for life, creativity, and boundless empathy inspired everyone who knew her. Her legacy in the fight against rare cancers and her unwavering kindness will continue to resonate.

September 30 is Rare Cancer Day

Congress passed a bipartisan resolution designating September 30, 2024 as

Rare Cancer Day. This International Day of Awareness led by National Organization for Rare Disorders (NORD) Rare Cancer Coalition is held on September 30 each year to shine a light on the rare cancer community and the unique challenges faced by rare cancer patients, caregivers, families, and researchers.

To address the special needs of those developing treatments for rare diseases, NORD has created a natural histories patient registry platform as part of the IAMRARE Program with extensive input from FDA, NIH, patients, organizations and experts in the field. To learn more about NORD's IAMRARE patient registry platform, click here.

The Rare Cancer Coalition was co-founded by TargetCancer Foundation’s CEO Jim Palma and formalized in 2017 as a program under the National Organization for Rare Diseases (NORD) Membership umbrella. It is comprised of more than 30 rare cancer-focused advocacy organizations to build a unified voice around rare cancer advocacy. Jim remains Co-Chair of the Coalition and TargetCancer Foundation.

This webinar, "How YOU Can Help: Patient-Driven Research in Rare Cancers" was hosted by the National Organization for Rare Disorders (NORD) as part of Rare Cancer Day 2024.

Empowering Patients:

The Rise of Patient-Driven Research in Rare Cancers

In the landscape of cancer research, rare cancers like Sarcoma, Glioblastoma, and Fibrolamellar Carcinoma have often been overshadowed in favor of more prevalent types. Due to their low incidence, these cancers often receive less attention from traditional funding sources, leading to a scarcity of data and research that could inform better patient outcomes. This is where patient-driven research and registries come into play, transforming the field of rare cancer research.

Registries stand as a testament to the power of patient and family involvement in driving research forward​​. These efforts, including the notable Fibrolamellar Registry highlighted in Cancer Today, are filling critical gaps in our understanding of rare cancers and also forging new paths toward effective treatments.

Initiated and managed by those directly impacted by fibrolamellar carcinoma—a rare form of liver cancer—this registry collects and analyzes data that are crucial for understanding the disease’s progression, treatment responses, and potential genetic markers. To learn more, read the complete Jedi Blog Post here.