Research Details
Dr. Rudloff's Research Update
Donations for Running-for-Rachel continue to support cancer research including areas of high medical need and prior accomplishments sponsored by the amazing $450,000 fundraised by us so far. Foremost, our fundraising and our support has led to the discovery and development of a novel form of cancer therapy. Metarrestin (ML-246) is novel treatment against cancer metastasis, the most common cause of cancer-related death in our country.
Since last year, patients with advanced solid organ cancers are treated within a first-in-human clinical study at NIH’s Center for Cancer Research (CCR) with metarrestin administered as oral capsules. Metarrestin is fundamentally different from chemotherapy or other standard-of-care treatment option. It is an oral treatment with the goal of being taken at home avoiding frequent trips to the patients’ oncologists. While the study is open for patients with any solid organ cancer, prior work has also focused on the analysis of duodenal cancer tissues which has shown that the marker, which is targeted by metarrestin, is present in duodenal cancer; thus, patients with duodenal cancer who have completed standard-of-care chemotherapy are encouraged to explore whether enrollment is aligned with their goals and best interests. Current focus in the early stage of clinical development is safety, with a later evaluation of metarrestin for efficacy. Recognizing the unmet need of pediatric and young adults afflicted by solid tumors with metastasis, including duodenal cancer, the study will include adolescents and young adults after establishing the maximum tolerated dose in adults. This will allow for much earlier evaluation of a potentially active therapy in adolescents and young adults than is typically seen in conventional drug development.
We are very proud to have achieved this milestone and that through our support a new treatment may bring hope and may make a tangible difference in the lives of cancer patients. But we don’t rest on these accomplishments: Dr. Rudloff and his collaborators have learned so much over the last years about metarrestin’s novel anti-cancer mechanisms that donations from Running-for-Rachel are already supporting work to make this treatment better like work on a second-generation molecule with improved safety and efficacy features.
And we don’t rest on one . . .: Donations for Running for Rachel also continue to support anti-cancer drug development in the arena of cancer immunotherapy, one of the currently most promising fields. Harnessing the patients’ own immune response to effectively fight cancer has long been a ‘dream’ of cancer researchers and physician scientists. With donations from Running-for-Rachel a drug candidate was identified which is able to reprogram immune cells in patients’ tumors; cancers frequently attract and surround themselves with immune cells which shield them from the natural immune system and allows tumors to grow and spread; the novel agent targets exactly these initially tumor-supporting cells and reprograms them towards effective killer immune cells. The identified drug candidate different from current immunotherapies is currently undergoing studies on safety and determination of dosing levels in various animal models. It is also an oral form of treatment primarily taken at home. While there are still a number of critical development stages ahead, Running-for-Rachel is committed to see this second new development reach patients in the next 2 to 3 years as well.
Courage - Strength - Hope - Faith,
Your Running for Rachel Team
March 2021
Metarrestin Capsules
Image of 1mg metarrestin capsules. Investigational agent administered within NCT04222413; courtesty of IriSys LLC.
This many people -- to make this much drug!
This image shows how much expertise is needed and how much collaborative and team work goes into drug development.
January 2020