AACR report details improvement in treatment of blood cancers; rise in early-onset cancer


AACR report details improvement in treatment of blood cancers; rise in early-onset cancer

Early-onset cancer incidence has increased for multiple malignancy types.

Treatment for blood cancers has significantly improved over the past 3-plus decades, with mortality rates for non-Hodgkin lymphoma and multiple myeloma declining by more than 40% and 30%, respectively.

The progress, which includes 50 combined FDA-approved molecularly targeted therapies and immunotherapies in the past 10 years alone, highlighted the 15th annual American Association for Cancer Research's Cancer Progress Report.

"Progress against blood cancer has led to a revolution in precision cancer medicine and has been a trailblazer in the field of immunotherapy," AACR President Lillian L. Siu, MD, FAACR, FRCPC, senior medical oncologist and director of the phase 1 clinical trials program at University Health Network's Princess Margaret Cancer Centre in Toronto, said during a media briefing.

The report also detailed the importance of targeted interventions against modifiable risk factors, such as obesity; the increased incidence and mortality of certain early-onset malignancies, which Siu described as an "urgent" priority; the need for more funding; and the uncertainty of national health care policy.

"Instability at our federal health agencies has slowed research, delayed clinical trials and shaken the confidence of investigators across generations," Margaret Foti, PhD, MD (hc), AACR's CEO, said during the briefing. "The progress we celebrate is real but is also fragile, and it must not be taken for granted."

Leukemia, lymphoma and multiple myeloma will account for an estimated 192,070 new cancer diagnoses and 56,110 deaths in the U.S. in 2025, and 1,393,600 new cases and 745,000 deaths globally.

However, mortality rates have substantially improved over the years.

Between 1991 and 2023, the mortality rate decreased 71% for chronic myeloid leukemia, 43% for non-Hodgkin lymphoma and 31% for multiple myeloma.

In all, 1.67 million people in the U.S. are survivors of blood cancer.

The FDA has approved 29 molecularly targeted therapies, including 4 antibody-drug conjugates, and 21 immunotherapies, including seven chimeric antigen receptor T-cell therapies, during the past decade for hematologic malignancies.

In the past year, the FDA approved revumenib (Revuforj, Syndax Pharmaceuticals) as the first menin inhibitor for acute myeloid leukemia; denileukin diftitox-cxdl (Lymphir, Citius Oncology), a recombinant fusion protein, for certain patients with relapsed or refractory cutaneous T-cell lymphoma; and obecabtagene autoleucel (Aucatyzl, Autolus Therapeutics), a new CAR-T for acute lymphocytic leukemia.

Siu called revumenib a "game-changer" for the patient population.

The AACR report also highlighted the myeloMATCH network, which is enrolling patients with acute myeloid leukemia and myelodysplastic syndromes into clinical trials based on their genomic profiles to personalize care.

"Patients who participate in clinical studies pave the way for countless other patients to benefit from these lifesaving therapeutics," Siu said. "[Clinical trials] provide the framework to test and refine investigational therapies for the benefit of our patients, and they are central to outstanding progress we have made against cancer."

The cancer mortality rate decreased 34% in the U.S. between 1991 and 2023, equating to approximately 4.5 million lives saved.

Rates declined 1.54% on average per year, with a greater decrease in men (1.81%) than women (1.33%).

Researchers attributed the reduction in deaths to lower smoking rates, which substantially improved survival from lung cancer. Also, colorectal cancer mortality decreased 49% between 1990 and 2023, and breast cancer deaths dropped 44% between 1989 and 2022.

The number of cancer survivors in the U.S. has increased from 3 million (1.4% of the population) in 1971 to 18.6 million (5.5%) in 2025. That number could exceed 22 million in 2035.

"This is a powerful testament to the progress we are celebrating today, and a reminder of how much more work needs to be done," Foti said.

Additionally, the number of survivors expected to live at least 15 years after diagnosis is projected to increase from 6.2 million in 2025 to 10.4 million in 2040.

The FDA approved 15 new anti-cancer therapies between July 1, 2024, and June 30, 2025.

These include afamitresgene autoleucel (Tecelra, Adaptimmune), a melanoma-associated antigen A4-directed genetically modified autologous T-cell immunotherapy for unresectable or metastatic synovial sarcoma; and vorasidenib (Voranigo, Servier Pharmaceuticals), the first oral IDH1 and IDH2 inhibitor for individuals aged 12 years or older with grade 2 astrocytoma or oligodendroglioma and a susceptible IDH1 or IDH2 mutation after surgery.

Siu described afamitresgene autoleucel as a "cutting-edge immunotherapy," and said vorasidenib "renewed hopes" for that patient population.

She also highlighted the rise of antibody-drug conjugates like datopotamab deruxtecan-dlnk (Datroway; AstraZeneca, Daiichi Sankyo), which received FDA approval earlier this year for certain lung and breast cancers.

The FDA also approved Optune Lua (Novocure), a tumor-treating fields device for certain patients with metastatic non-small cell lung cancer, as well.

Despite this progress, 2 million people are expected to be diagnosed with cancer in 2025.

Interventions aimed at tobacco control, screening uptake and vaccinations helped prevent about 5.94 million cancer deaths between 1975 and 2020.

In all, 100% of cervical, 79% of colorectal, 56% of prostate and 25% of breast cancer deaths averted can be attributed to screening and removal of precancerous lesions.

Interventions targeting other modifiable risk factors, such as obesity, could have a similar effect.

Approximately 40% of all cancer cases in the U.S. could be attributed to modifiable risk factors, including cigarette smoking (19.3%), excess body weight (7.6%), alcohol (5.4%), UV exposure (4.6%), poor diet (4.2%), pathogenic infections (3.8%) and inactivity (3.1%).

Between 2021 and 2023, more than 40% of adults in the U.S. had obesity. Some studies have shown bariatric surgery and GLP-1 agents could reduce incidence of obesity-related cancers.

Healio previously reported GLP-1 agents significantly reduced risk for overall cancer among patients with overweight and obesity. They had the strongest associations with endometrial and ovarian cancer, and meningioma.

Investigations into early-onset cancer also are needed.

Diagnoses of 14 different cancer types -- including melanoma and colon, breast, pancreas and stomach cancers -- increased from 2010 to 2019 among individuals aged younger than 50 years. In this population, colorectal cancer incidence rose an average of 5% annually and mortality increased 1.1% during those 10 years.

"Understanding the driver of this trend -- whether obesity; environmental exposures such as PFAS, a class of synthetic forever chemicals that persist in the environment and accumulate in the human body, and microplastics; or changes in our microbiome -- really is an urgent research priority for the AACR and the entire medical research community," Siu said.

She added the AACR would convene a conference in December to discuss causes and mechanisms that lead to early-onset cancers.

Racial disparities in cancer care persist, too.

For example, Black men had a 16% higher cancer-mortality rate than white men between 2018 and 2022. Black women had a 10% higher rate than white women over the same period.

"Racial and ethnic minority groups and medically underserved populations continue to shoulder a disproportionate cancer burden," Siu said. "Although precision medicine is delivering remarkable benefits for our patients, equitable and affordable access to cutting edge therapies remains a major challenge."

Federal funding has had a dramatic impact on cancer therapeutics for decades.

This support boosted screening and preventative measures that have saved 4.75 million lives from breast, cervical, lung, colorectal and prostate cancers.

Additionally, federally funded clinical trials have saved 14 million years of additional life over the past 4 decades.

Of 356 FDA-approved drugs between 2010 and 2019, 354 received funding through NIH.

However, the Big Beautiful Bill proposed nearly $18 billion in cuts to NIH.

AACR reported each dollar of NIH funding resulted in $2.56 of economic activity.

A national poll of 1,001 registered voters commissioned in August by AACR showed 89% of people favored federal funding for medical research, and 83% supported increased federal funding for cancer research.

"The [latest AACR Cancer Progress Report] is not only a record of how far we have come but also a reminder of how much remains at stake," Siu said in closing. "It demonstrates what sustained investments in cancer research have made possible and why continued commitment is essential to advance discovery, improve outcomes and save lives around the world.

"Cancer touches every family, every community and every generation," she continued. "At this defining moment, Congress owes it to every patient, every survivor and every family to protect the progress we have made and deliver the promise of a future without cancer."

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