Pediatric cancer research typically spans decades, with very few treatments being brought to children who urgently need them. In fact, only 3 drugs have been approved for childhood cancer in over 30 years. Project Stella aims to change that and is making rapid progress. Promising advances are being made on several fronts by Dr. Soheil Meshinchi and his team at the Fred Hutchinson Cancer Research Center:
The most encouraging therapy, the FOLR-1 CAR T, is moving along quickly - at a typically unheard of pace in pediatric cancer research. This is our primary focus and is the driver of our plea for additional funds! The plasmid is in hand, and the vector creation began this month. Though the goal of launching a pediatric trial in early 2022 is aggressive, we are currently on track to meet that deadline. The CAR T would provide a potentially curative treatment for various aggressive forms of AML - not just AML-RAM. And it would spare children from a bone marrow transplant in the first place.
STRO-002 is an antibody drug conjugate targeting the unique FOLR-1 receptor, a key target in both AML-RAM and ovarian cancer. The first two pediatric patients treated with STRO-002 showed objective response and significant reduction in disease burden in both their bone marrow and peripheral blood, without any side effects. Three additional patients are either in process of being treated or under evaluation for compassionate use of STRO-002.
Elucida has a promising drug which targets FOLR-1 and FOLR-2, doubling its potency against leukemic blasts. Elucida has just treated their first ovarian cancer patient and expect to have pediatric dose recommendations by mid-2022. They are incredibly engaged and eager to move forward with a pediatric trial, which will likely be run through the TACL (Therapeutic Advances in Childhood Leukemia & Lymphoma) consortium; the fastest path forward.
Dr Meshinchi gives a very frank summary of why Project Stella has achieved major milestones in such a short amount time and why the next phase of CAR T Cell therapy is critical to finding a cure for this rare form of AML.
CAR T cell therapy is a tailored form of immunotherapy that works by modifying the body's own T cells, a type of immune system cell that hunts and destroys abnormal cells. Scientists harvest T cells from patients, genetically alter them, then infuse the resulting CAR T cells into patients to attack cancerous cells. The CAR T cells then act as a "living drug" against cancer cells. When they come in contact with their targeted antigen on a cell, CAR T cells bind to it and become activated, then proliferate and become cytotoxic. In other words, they are cancer-fighting smart missiles.
Creating an effective CAR T is contingent on first identifying a suitable target. For safety, CAR T cells are engineered to be specific to an antigen expressed on a tumor that is not expressed on healthy cells. This is another reason genomic sequencing is so critical. Under the leadership of Dr. Meshinchi, sequencing has become part of pediatric AML protocol - advanced significantly in partnership with the LLS PEDAL initiative. Sequencing helps us understand the mutations underlying the disease, so vulnerabilities can be identified. As a result of Project Stella and the Hutch having the largest repository of pediatric leukemia samples, the Meshinchi Lab has identified several proteins that are highly expressed in AML RAM, like FOLR-1. Because FOLR-1 is found in high expressions on the outside of the cancer cell, it is ideally suited for immunotherapies. Moreover, it is not found on healthy cells.
A New Approach to Treatment: Project Stella works closely with Dr. Soheil Meshinchi, a world renowned pediatric AML expert at The Fred Hutchinson Cancer Research Center to expedite precision medicine research. Dr. Meshinchi runs one of the only labs in the country developing cures for children facing the devastating diagnosis of high-risk AML. His research is tailored to pediatric cancers, aimed specifically at patients under 5 years old. Dr. Meshinchi’s research is focused on finding a treatment protocol that targets the cancer and does not bring the irreversible side effects. Moreover, these therapies will be brought to children on an expedited time frame, bypassing the decades it often takes to bring novel treatments to pediatric cancer patients. Use this space to add more details about your site, a customer quote, or to talk about important news.
Please click on the video to the left to see why Dr Meshinchi was selected to lead Project Stella.
If you know of a child recently diagnosed with AML please go to Curewheel.org for information on expertise in AML.
* Other treatment centers only accepts patients who fit into one of the trials they are focused on. Fred Hutch is often a place of last resort, willing to take on the toughest of cases.
* Fred Hutch is the leading recipient of NIH Funding among all of the 61 NCI designated Cancer Centers- these grants are awarded by an impartial scientific body. This speaks to the quality of the research at The Hutch.
* The Hutch, in partnership with the Global Oncology Group, is the holder of the largest repository of pediatric AML tumor samples in the world. This data is critical to finding cures and doing this research.
* Hutch researchers consult with leading researchers at other facilities – typically on these extremely difficult cases.
Fred Hutch performs research on rare types of pediatric AML, while the majority of AML research is on Adult AML. Pediatric and adult AML are in no way similar.
* Over 1.3M bone marrow transplants have been done worldwide. This process was developed at Fred Hutch who also provided the training required at other locations.
* Drs. E. Donnall Thomas (1990), Lee Hartwell (2001) and Linda Buck (2004). Each received the Nobel Prize in physiology or medicine and further cemented Fred Hutch’s reputation for scientific innovation and excellence.