National Cancer Research Month: Getting closer to a cancer-free future.

May is National Cancer Research Month. There have been remarkable advancements in the treatment of cancer and this progress is only possible thanks to innovative scientific research.

Since 2001, Alliance for Cancer Gene Therapy (ACGT) has awarded 67 research grants to scientists developing novel cell and gene therapies for cancer. Our alliance’s funding has helped scientists work to develop CAR T cells, oncolytic viruses, vaccines, engineered T-cell receptors and other therapies capable of harnessing the power of the immune system to defeat cancer. ACGT grants have led to FDA-approved therapies for resistant and recurrent blood cancers, clinical trials for difficult-to-treat solid tumors, and patients living cancer-free for more than 10 years.

Visit ACGT’s website to read more about five novel research projects currently being funded by the alliance. To learn more about ACGT’s mission during National Cancer Research Month, share this newsletter with your friends and family and ask them to sign up for ACGT’s e-communications.

The more people know about and support the potential of cancer cell and gene therapy, the more we can fund crucial research leading to scientific breakthroughs.

Experts explore the horizons of cancer cell and gene therapy at ACGT Summit 2024.

On March 29, ACGT presented its annual scientific gathering — ACGT Summit 2024 — an invitation-only event held in New York City bringing together top scientists and thought-leaders in cancer cell and gene therapy.

Read the full recap of ACGT Summit 2024 — including highlights from each panel — on our website.

Attendees talk during break (left to right): ACGT Research Fellow Hideho Okada, MD, PhD (University of California, San Francisco); Caroline Corner, PhD (patient advocate); and ACGT Chief Program Officer Barbara Lavery.

Guests from Biotech: ACGT Board of Directors member Chau Khuong (CJNV Bioventures, Inc.); Lachlan MacKinnon, MD, PhD (Replay Bio); Andy Scharenberg, MD (Umoja Biopharma); ACGT Board of Directors member Maria Fardis, PhD, MBA (Lassen Therapeutics); and Barbra Sasu, PhD (Dispatch Biotherapeutics).

In-vivo cell and gene therapy panel (left to right): ACGT Scientific Advisory Council member Mitchell H. Finer, PhD (ElevateBio); Andrew Scharenberg (Umoja Biopharma); ACGT Scientific Advisory Council member Stephen Russell, MD, PhD (Vyriad); ACGT Research Fellow and Scientific Advisory Council member Carl June, MD (University of Pennsylvania).

Barbara Netter, ACGT Co-Founder and Honorary Chair; and Ira Harkavy, PhD, Barbara and Edward Netter Director at the Netter Center for Community Partnerships (University of Pennsylvania).

Caroline Corner, PhD (patient advocate), telling the audience about her son’s experience with CAR T-cell therapy.

ACGT Summit Attendees: ACGT Board of Directors member Bruce Levine, PhD (University of Pennsylvania); Marcela Maus, MD (Massachusetts General Hospital); ACGT Research Fellow and Scientific Advisory Council member Crystal Mackall, MD (Stanford University); ACGT Research Fellow and Scientific Advisory Council member Carl H. June, MD (University of Pennsylvania)

#FightForTheBrain with ACGT during Brain Cancer Awareness Month.

More than 24,000 people in the U.S. are diagnosed every year with brain cancer, which can be one of the most difficult tumors to treat. The location of the tumor, the immunosuppressive environment, and other factors can prevent surgery, chemotherapy and radiation from providing long-term solutions to the disease.

This May, for Brain Cancer Awareness Month, ACGT continues to #FightForTheBrain by raising awareness of how cell and gene therapy is being studied to treat brain tumors and the progress that scientists are making. Visit ACGT′s website to learn more about the latest brain cancer research ACGT is funding.

Donor highlight: Lori Lowe.

Brain Cancer Awareness Month serves as a reminder of the profound impact glioblastoma and other forms of brain cancer have on individuals, families and communities worldwide – like Ming Lowe and his family. 

Ming′s wife, Lori Lowe, witnessed firsthand the effect glioblastoma had on her husband’s life. In honor of Ming′s beautiful spirit and his dedication to helping others, Lori and her family chose to support ACGT.

“Glioblastoma is a formidable adversary,” Lori explains. “By supporting ACGT, we are investing in hope for the brain tumor community.”

This month also sheds light on the critical funding need to turn bright ideas into promising treatments. ACGT is at the forefront of this effort, driving progress in understanding the underlying mechanisms of brain cancer and developing novel cell and gene therapies that potentially transform patient outcomes.

By making a gift to ACGT during Brain Cancer Awareness Month, you can play a vital role in supporting groundbreaking research initiatives and fostering hope for those impacted by brain cancer. 

Every donation, no matter the size, has the power to make a meaningful difference in the lives of patients and families confronting this disease. 

Lori′s decision to support ACGT reflects a shared commitment to transforming cancer care and honoring the memories of those lost to this disease, while also recognizing our opportunity to shape a brighter future for those impacted by brain cancer. 

Make your gift today.

Multiple myeloma: FDA updates.

The U.S. Food and Drug Administration (FDA) updated the approvals for the two CAR T-cell therapies used to treat people with the blood cancer multiple myeloma, which is diagnosed in approximately 36,000 people in the United States each year.  

Ciltacabtagene autoleucel (brand name Carvykti), which is manufactured by Johnson & Johnson, is now approved for adults with refractory or relapsed multiple myeloma who have received one prior line of therapy. Idecabtagene vicleucel (brand name Abecma), manufactured by Bristol Myers Squibb, is approved for adults with refractory or relapsed multiple myeloma who have received two prior lines of therapy. Both were originally approved for use after at least four prior lines of therapy.  

CAR T-cell therapies use genetically modified T cells to target cancer cells expressing a specific protein. Funding from ACGT has helped propel CAR T-cell therapy forward as an effective treatment for blood cancers such as lymphoma, leukemia and multiple myeloma. The first two FDA-approved CAR T-cell therapies for cancer in 2017 were the direct result of ACGT-funded research.

Request for Applications

Now accepting proposals for the:

ACGT – Edward Netter Memorial Investigator Award in Cell and Gene Therapy Research for Pancreatic Cancer (2024).

Application Timeline

• Abstract deadline:
  June 1, 2024 at 11 a.m. (EST)

• Invitation to submit application:
  July 15, 2024

• Deadline for completed application:
  Sept. 15, 2024 at 11 a.m. (EST)

• Finalists selected:
  Nov. 15, 2024

• Award:
  December, 2024

Details may be found at:
acgtfoundation.org/pancreatic-cancer-rfa/

ACGT scientists honored with ‘Oscars of Science.’

Last month, two ACGT Research Fellows and Scientific Advisory Council members – Carl H. June, MD (University of Pennsylvania), and Michel Sadelain, MD, PhD (Memorial Sloan Kettering Cancer Center) – each received the 2024 Breakthrough Prize in Life Sciences, more popularly known as the “Oscars of Science.” They were recognized for pioneering the development of the first effective CAR T-cell therapies for cancer.

Drs. June and Sadelain each received ACGT Research Grants in 2004. Their brilliance combined with ACGT’s funding led to the first two FDA-approved CAR T-cell therapies for certain types of blood cancer: Kymriah and Yescarta.

“I think the most important thing is for people to learn about this new treatment,” Dr. June said after the ceremony. “It came out of research done right here in the United States. There have been 34,000 people treated with CAR T-cell therapy, and it just started 10 years ago. I want people to understand the value of research in making these new breakthrough cancer therapies.”

ACGT congratulates Drs. June and Sadelain for this honor. Watch the full ceremony on the Breakthrough Prize YouTube channel.

May is Melanoma and Skin Cancer Awareness Month.

May is Melanoma and Skin Cancer Awareness Month, and we recognize the recent FDA approval of the cell therapy lifileucel for advanced melanoma.

Every year in the United States, approximately 100,000 people are diagnosed with melanoma. The long-term survival rates are poor for people with advanced melanoma.

Lifileucel (brand name Amtagvi), which the FDA approved in March, is the first cell therapy approved to treat a solid tumor. The therapy multiplies T cells which have shown the ability to infiltrate and attack melanoma tumors. This approval will give people with metastatic or recurrent melanoma a new treatment option.

ACGT Research Fellow receives major grant to continue gene therapy research.

ACGT Research Fellow and Scientific Advisory Council member Noriyuki Kasahara, MD, PhD (University of California, San Francisco), received an $11.8 million grant from the California Institute for Regenerative Medicine (CIRM) to support a clinical trial targeting newly diagnosed high-grade gliomas, which are fast-growing cancers that form in the brain or spinal cord and are difficult to treat.

The CIRM award will allow Dr. Kasahara to explore the potential of DB107 (formerly Toca 511), which uses a retroviral-replicating vector expressing the cytosine deaminase suicide gene (RRV-CD). This self-replicating gene therapy platform selectively targets and penetrates glioma tumor cells, delivering a unique enzyme that transforms a harmless compound into a cancer-fighting drug right at the core of the cancer cells.

The first-in-human clinical trial will evaluate the safety and efficacy of this novel gene therapy technology in newly diagnosed high-grade glioma patients.

In 2017, ACGT awarded a research grant to Dr. Kasahara to develop an RRV expressing a different suicide gene, Nitroreductase (RRV-NTR), which generates an alkylating drug that cross-links DNA.

“Through our ACGT-funded project, we were able to show synergistic effects with RRV-NTR (generating DNA alkylating drug) and RRV-CD on killing cancer cells,” Dr. Kasahara said.

He added that the success of the ACGT-funded project plus the upcoming CIRM-funded clinical trial may lead to evaluating RRV-CD together with RRV-NTR for a combination suicide gene therapy approach in the clinic.

Dr. Kasahara states that the 2017 ACGT-funded project set the stage for developing the next generation of RRV suicide gene therapies to be tested in the clinic.

In the news

The latest from around the cancer cell and gene therapy research world.

A new CAR T-cell therapy clinical trial for acute myeloid leukemia is the first of its kind. The clinical trial is based on research from ACGT Scientific Advisor Council member Michel Sadelain, MD, PhD (Memorial Sloan Kettering Cancer Center).

ACGT Research Fellow Hideho Okada, MD, PhD (University of California, San Francisco) received a grant of up to $11 million from the California Institute for Regenerative Medicine to fund a glioblastoma clinical trial at UCSF that uses a smarter CAR T-cell therapy guided by precision technology.

Replay, a biotech company headquartered in San Diego, has announced that its first patient has been dosed with an engineered T-cell receptor natural killer (TCR-NK) cell therapy for relapsed or refractory multiple myeloma. ACGT Scientific Advisory Council member Katy Rezvani, MD, PhD (The University of Texas MD Anderson Cancer Center) developed the cell therapy.

Penn Medicine’s E. John Wherry, PhD, and Children’s Hospital of Philadelphia’s John M. Maris, MD, were named 2024 Fellows of the AACR Academy. Dr. Maris is an ACGT Research Fellow.

A CAR T-cell therapy targeting the HER2 antigen and developed by two ACGT Research Fellows – Drs. Meenakshi Hegde and Nabil Ahmed – shows promise against advanced sarcoma in phase I trial at Baylor College of Medicine.