Top stories for cancer cell and gene therapy in 2022

Dec 09, 2022
Devin Golden

Top stories for cancer cell and gene therapy in 2022

2022 has been a tremendous year for cancer cell and gene therapy. There have been breakthroughs in developing cell and gene therapy for brain cancer, FDA approvals, a record number of scientists applying to ACGT for grant funding, and major survival milestones for the earliest CAR T-cell therapy patients.

ACGT has listed seven of the top stories for cancer cell and gene therapy in 2022. Sign up for our monthly email newsletter to receive regular updates about advancements in cell and gene therapy.

ACGT funding leads to breakthroughs for pediatric cancers

Crystal Mackall, MD

Crystal Mackall, MD, of Stanford Medicine, became an ACGT Research Fellow in 2016.

Dr. Mackall proposed using ACGT funding to develop a CAR T-cell therapy for pediatric osteosarcoma (bone cancer) and neuroblastoma (nerve tissue cancer).

She and her colleagues at Stanford learned that the protein target (GD2) for these cancers also applied to a type of brain cancer called diffuse intrinsic pontine glioma (DIPG). This cancer primarily affects children.

In 2022, Dr. Mackall’s CAR T-cell therapy moved into a clinical trial for DIPG, and some patients are achieving extraordinary survival results.

Anniversary of first FDA approval for CAR T-cell therapy

Kymriah became the first CAR T-cell therapy approved for cancer. The U.S. Food and Drug Administration (FDA) approved Kymriah in 2017 for acute lymphoblastic leukemia. On August 30, 2022, believers in the potential of cell and gene therapy celebrated the 5-year anniversary of this landmark FDA approval.

Kymriah is now approved for people with large B-cell lymphoma or follicular lymphoma after at least two other therapies have failed. The approval for follicular lymphoma came in 2022.

Since the landmark approval of Kymriah, other CAR T-cell therapies approved for blood cancer are:

  • Yescarta and Breyanzi for non-Hodgkin lymphoma (both were approved as second-line therapies in 2022)
  • Tecartus for mantle cell lymphoma
  • Carvykti (approved in 2022) and Abecma for multiple myeloma

T-cell receptor therapy approved for rare type of melanoma

At the beginning of 2022, the FDA approved a T-cell receptor therapy for a rare type of melanoma in the eye, called uveal melanoma.

The therapy, Kimmtrak, is the first cancer cell and gene therapy approved for a solid tumor. Read more about how T-cell receptor therapy works as a cancer treatment.

Early CAR T-cell patients celebrate survival milestones

Two of the earliest cancer patients to receive CAR T-cell therapy celebrated extraordinary survival milestones. Emily Whitehead (acute lymphoblastic leukemia) and Doug Olson (chronic lymphocytic leukemia) were participants in a clinical trial featuring the work of ACGT Research Fellow Carl June, MD.

In 2022, they celebrated more than a decade of survival from their blood cancers. Some doctors call them “cured” of cancer.

Developing a ‘remote-control CAR T-cell therapy’

A main feature of the recent progress in cell and gene therapy is the use of technology. Dr. Mackall is one of the ACGT scientists making cell and gene therapy safer through technology.

Dr. Mackall is developing a remote-control therapy to activate and deactivate the CAR T cells. The remote-control CAR T-cell therapy is helpful for a few reasons:

  • Turns off CAR T cells that aren’t effective
  • Turns off CAR T cells that cause toxic side effects
  • Gives CAR T cells a rest time to replenish energy

Progress for injectable cell and gene therapy

Matthias Stephan, MD, PhD

ACGT Research Fellow Matthias Stephan, MD, PhD, of Fred Hutchinson Cancer Research Center in Seattle, Wash., is developing an injectable form of cell and gene therapy. In 2018, Dr. Stephan received grant funding from ACGT to create nanoparticles that could reprogram T cells circulating in the body. In 2022, Dr. Stephan reported early success in pre-clinical studies for prostate cancer, liver cancer and leukemia.

Most cell and gene therapies require doctors to remove patients’ cells and send them to a laboratory for reprogramming. Dr Stephan’s approach is to engineer T cells without needing to remove them from the bloodstream. This method may shorten the time to create an effective cell and gene therapy, which currently takes up to six weeks.

Applications accepted for next round of ACGT grant funding

ACGT invited researchers in April to apply for Investigational Awards, which are at the crux of the organization’s mission to advance cell and gene therapy research for cancer. These awards include multi-year grant funding to expedite the research and development of this innovative field of cancer treatment. ACGT’s invitation for applications was for scientists conducting cell and gene therapy research for solid tumors.

As 2022 comes to a close, ACGT intends to issue grants to fund a pediatric brain tumor clinical trial and two new grants for viral therapy for brain cancers. These new grants are in addition to existing grants for cell and gene therapy programs focused on lung cancer, prostate cancer, pancreatic cancer, pediatric sarcomas and ovarian cancer.

These grants are possible thanks to donor support. As ACGT closes 2022, the organization is looking for new donors to strengthen our alliance against cancer. Our goal is to inspire 20 new donors to contribute to ACGT’s mission. Please donate to ACGT now to help us meet this goal before the end of 2022.