Cell and gene therapy highlights from 2023.

Dec 04, 2023
Devin Golden

ACGT Research Fellow Daniel Powell (middle), PhD, of the University of Pennsylvania, talks with ACGT Scientific Advisory Council Chair Michael Lotze, MD, of the University of Pittsburgh, during the ACGT Summit 2023 held in March.

Critical research in 2023 took another step forward in progressing the science of cancer cell and gene therapy. Throughout the year, Alliance for Cancer Gene Therapy (ACGT) awarded important grants for promising research, celebrated survival milestones for people with cancer, and was part of a multitude of notable achievements.

Here are some highlights in cell and gene therapy from 2023.

ACGT awards grants for brain cancer studies.

ACGT’s two newest Research Fellows are: Hideho Okada, MD, PhD, of the University of California, San Francisco; and Juan Fueyo, MD, of the University of Texas MD Anderson Cancer Center. Each scientist received a grant to run a brain cancer study using cell and gene therapy.

Dr. Okada is leading a clinical trial to test an engineered T-cell receptor therapy for children with a rare and aggressive brain tumor called diffuse intrinsic pontine glioma. Dr. Fueyo is developing an oncolytic virus to fight glioblastoma, which is the most common brain tumor.

These grants will help the scientific community learn more about the potential of cell and gene therapies to defeat brain tumors.

Scientists gather for successful Summit 2023.

In March, ACGT hosted Summit 2023, an invitation-only symposium that brought together top cancer scientists. Attendees included ACGT Research Fellows, ACGT Scientific Advisory Council members, biotech leaders, and other thought-leaders.

Scientists shared ideas to help advance the field of cell and gene therapy, including: how to combine different therapeutic approaches using cell and gene therapies; opportunities to advance cell and gene therapies for pediatric cancers; and presentations from current ACGT Research Fellows about their ongoing research.

ACGT honors Dr. Crystal Mackall with prestigious award.

ACGT Research Fellow Crystal Mackall, MD, of Stanford University, talks about cell and gene therapy to treat cancer.

ACGT awarded The Edward Netter Leadership Award to Crystal Mackall, MD, of Stanford University, at the ACGT Awards Luncheon in March. The event celebrated the nonprofit’s 20th anniversary and ushered in a new decade.

Dr. Mackall, an ACGT Research Fellow and a member of ACGT’s Scientific Advisory Council, has advanced understanding of fundamental immunology and translated this understanding for treatment of human disease with a major focus on children’s cancers.

She has led numerous first-in-human and first-in-child clinical trials spanning dendritic cell vaccines, cytokines, and adoptive immunotherapy using NK cells and genetically modified T cells.

Named for ACGT co-founder Edward Netter, the award recognizes a researcher who has made unparalleled and groundbreaking contributions to the field of cell and gene therapy.

Swim Across America Fairfield County raises money to benefit ACGT.

This year, Swim Across America Fairfield County (SAA-FC) held the 17th-annual Open Water Swim. As of Dec. 1, this year’s event has raised more than $400,000, which benefits ACGT’s mission to fund scientists working to develop new treatments for cancer. SAA-FC’s total contribution to ACGT’s efforts has now surpassed $5 million.

The 2024 Open Water Swim will be held Saturday, June 29.

Updates on research funded by ACGT.

Three ACGT Research Fellows completed their 3-year funded research in 2023:

All of this important research will help the scientific community learn more about how cell and gene therapies can be developed to fight difficult-to-treat tumors in children and adults.

ACGT funding linked to exciting brain cancer study.

ACGT Research Fellow E. Antonio Chiocca, MD, PhD, of Brigham and Women’s Hospital, talks about how ACGT funding helped him research and develop an oncolytic virus that led to a clinical trial for brain cancer.

ACGT Research Fellow E. Antonio Chiocca, MD, PhD, of Brigham and Women’s Hospital, led a promising clinical trial using an oncolytic virus for people with an aggressive brain tumor. This clinical trial was the result of grant funding Dr. Chiocca received from ACGT.

According to a press release from Brigham and Women’s Hospital, the therapy extended survival for people with recurrent glioblastoma, meaning the cancer returned after previous treatment. The treatment was especially beneficial for people whose immune systems had pre-existing antibodies for the virus used in the trial: an oncolytic herpes simplex virus. Approximately 66% of the study participants had pre-existing antibodies.

Two childhood cancer survivors reach milestones.

Nearly 20 years ago, ACGT funded the research of Carl June, MD, of the University of Pennsylvania. His research resulted in a clinical trial to test a CAR T-cell therapy for children with leukemia beginning in 2012.

Austin Schuetz and Tori Lee, two of the children who participated in this study, both reached 10 years cancer-free in 2023. This survival milestone is when scientists begin saying people are “cured” of cancer. Austin is in high school and Tori is a sophomore at the University of North Carolina.

Their stories are a testament to how far we’ve come in advancing cell and gene therapies – and why we must continue to fund novel research in 2024 and beyond. Sign up for our email list to get updates every month on advancements in cell and gene therapy research and how ACGT is helping to drive this science forward.