Foundation awards two grants to support cell and gene therapy research for childhood tumors.
STAMFORD, Conn., March 29, 2024 — Alliance for Cancer Gene Therapy (ACGT) has invested $3 million to support cross-institution, multidisciplinary collaborations developing promising cancer cell and gene therapies for children with difficult-to-cure tumors.
The ACGT-Barbara Netter Fund Collaboration Challenge Award is a $1.5 million grant awarded to a high-impact, collaborative research project to advance cancer cell and gene therapies for patients with solid tumors. ACGT awarded two grants for research focusing on difficult-to-cure pediatric cancers. Each grant award required matching funds bringing the total invested in the program to $6 million dollars in total.
“Grant proposals were reviewed by a committee made up of members of the ACGT Scientific Advisory Council and ACGT Board of Directors,” says Kevin Honeycutt, CEO and President of ACGT. “Proposals were incredibly competitive, receiving the highest possible scores from the review committee. At the request of the committee and with the support of our donors, Board of Directors and Co-Founder Barbara Netter, we were thrilled to be able to make two awards to two outstanding collaborative teams.”
The first research grant has been awarded to a collaborative effort between Children’s Hospital of Philadelphia (CHOP) and the biotechnology company Hula Therapeutics. Pediatric oncologists John Maris, MD, and Stephan Grupp, MD, PhD, lead the collaboration, titled “Translating peptide-centric CAR T-cell therapies for childhood cancer”.
“While chemoradiotherapy can cure four out of five children with cancer, one of five dying is unacceptable and survivors often have lifelong therapy-related morbidities,” Dr. Maris says. “Our goal is to fundamentally change the landscape for childhood cancer treatment.”
CAR T-cell therapy is a type of cell and gene therapy that modifies the immune system to more effectively attack cancer. Standard CAR T-cell therapy engineers a patient’s own T cells to target antigens on the surface of cancer cells.
In solid tumors, most viable targets exist inside of the cells. Drs. Maris and Grupp are developing CAR T cells to target antigen fragments, called peptides, that rise to the cancer cell surface.
“Unlike many current CAR T-cell strategies, our peptide targets are exquisitely specific and, to date, not prone to downregulation as an immune-escape mechanism,” Dr. Maris says. “By building out a suite of peptide-centric CAR T cells to a variety of peptide targets, we seek to have a CAR T-cell therapy available for any patient.”
Drs. Maris and Grupp will first test the therapy in children with the nerve cell cancer neuroblastoma, which is diagnosed in approximately 700 children in the United States each year. If successful, it could lead to peptide-targeting CAR T-cell therapies for other pediatric cancers.
Dr. Grupp played a key role in developing the first effective CAR T-cell therapy for cancer. He led the first ever phase 1 clinical trial testing the novel therapy in children with leukemia at CHOP. The trial led to the first FDA-approved CAR T-cell therapy for cancer in 2017.
The second grant has been awarded to a collaboration between three leading academic institutions: St. Jude Children’s Research Hospital; Stanford University; and McMaster University Cancer Research Centre. The team leads from each institution are Stephen Gottschalk, MD (St. Jude), Crystal Mackall, MD (Stanford), and Sheila Singh, MD, PhD (McMaster).
The collaboration, titled “Synthetic T-cell therapy against recurrent pediatric brain tumors (STAR),” focuses on the pediatric brain tumors medulloblastoma and ependymoma. Combined, these two diseases are diagnosed in approximately 700 children in the United States each year.
While standard chemotherapy is effective for most patients, children who do not respond to chemotherapy or who relapse following treatment have extremely limited or non-existent therapeutic options. The synthetic T cells in the STAR project are genetically engineered in several ways to achieve higher T-cell persistence against immunosuppressive brain tumors and fewer toxicities for patients.
“Our long-term vision is to develop T cell-based immunotherapies for pediatric brain tumors with the potential to become standards of care,” Dr. Gottschalk says.
The collaboration will combine the leading scientists’ distinct proficiencies. Drs. Gottschalk and Mackall, previous recipients of ACGT research grants, are cellular immunotherapy experts. Dr. Singh, a first-time ACGT grant recipient, is a pediatric neurosurgeon with expertise in developmental neurobiology, brain tumor development and antigen expression.
“We believe that our approach is likely to succeed since investigators in this research collaboration have a long-standing track record of excellence, innovation and translating preclinical research findings into the clinic,” Dr. Gottschalk says. “More importantly, the investigators share a vision, like ACGT, that progress can only be accomplished through multidisciplinary team science and by bringing investigators together with complementary expertise.”
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Alliance for Cancer Gene Therapy (ACGT)
For nearly 25 years, Alliance for Cancer Gene Therapy (ACGT) has funded scientific research to bring innovative treatment options using cancer cell and gene therapy to people living with deadly cancers. These treatments continue to save the lives of many blood cancer patients and offer new hope to all people diagnosed with cancer. ACGT is currently focused on funding talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for solid tumors such as brain, breast, lung, ovarian, pancreatic, pediatric, prostate, sarcoma and skin cancers.
100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit acgtfoundation.org, call (203) 358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube.
