WASHINGTON—At a news briefing at the National Press Club here featuring cancer researchers and cancer survivors, the American Association for Cancer Research released its most recent Cancer Progress Report. This third such report from the association highlights progress in immunotherapy, noting that the U.S. Food and Drug Administration has approved two immunotherapies, sipuleucel-T and ipilimumab, and that there are many others in clinical testing.
While hailing the approval of 11 new drugs from Sept. 1, 2012 to July 31, 2013 to treat a variety of cancers, along with three new uses for previously approved anti-cancer drugs and three new imaging technologies, AACR CEO Margaret Foti, PhD, sounded a note of alarm about whether the strong pace of progress can be maintained. The sobering reality, she said, is that the nation's ability to “continue making life-saving progress is in peril due to the most serious funding crisis in decades.”
The AACR also held its Rally for Medical Research Hill Day on Capitol Hill a day after the release of the report, with more than 170 other organizations participating, with the aim of calling the attention of Congress to the urgent need for strong funding for cancer research.
DREW M. PARDOLL, MD, PHD
Among the sponsoring organizations was the American Society of Clinical Oncology. In a statement, ASCO President Clifford A. Hudis, MD, Chief of the Breast Cancer Medicine Service at Memorial Sloan-Kettering Cancer Center, said that with the rapid pace of scientific progress against cancer, “it is a tragedy that we are even considering cuts to our nation's investment in biomedical research.”
Foti noted that since 2003 the budgets for the National Institutes of Health and the National Cancer Institute have been steadily shrinking: In addition to this monetary erosion, she said, “sequestration forced the NIH to absorb a direct budget cut of $1.6 billion in March 2013. The current course is simply unacceptable.”
Not only will cancer patients' lives be lost if policymakers don't act to boost funding, Foti warned, but “the crucial investments in research that we have already made will be jeopardized.” She said she hopes the new report will encourage Americans to get behind cancer research and urge policymakers to make the NIH and NCI “national priorities.”
AACR President Charles L. Sawyers, MD, who chaired the committee that wrote the report, said, “Decades of basic research have taught us much about how cancers develop, grow, and threaten the lives of millions.” He decried the “most serious funding crisis in decades,” and said, “This could not have happened at a worse time, as we are beginning to lose the scientific momentum that is enabling us to develop more effective interventions and save more lives from cancer.”
It is now known that gene mutations alter specific protein components of the cell, driving cancer initiation, development, and spread, and that targeted therapies are often beneficial to patients while being less toxic than more traditional therapies, said Sawyers, who is Chairman of the Human Oncology and Pathogenesis Program at Memorial Sloan-Kettering Cancer Center and a Howard Hughes Medical Institute investigator.
AACR President CHARLES L. SAWYERS, MD
He referred to a patient featured in the report, Hans Loland, 45, noted as having chronic myeloid leukemia kept in check by ponatinib, which was approved by the FDA in December. “Ponatinib specifically targets the defective protein driving the disease, keeping him in remission while allowing him to live a full and productive life,” Sawyers said.
“It is not just cancer genomics that is driving this field forward,” he said of targeted therapies. “Every cancer research discipline is contributing to our ever-increasing knowledge of the biology of cancer. This, in turn, is driving the development of a rising number of drugs that target specific molecules involved in different stages of the cancer process.”
He noted that there are now 39 FDA-approved targeted cancer therapies, compared with 17 five years ago and five such therapies 10 years ago. “This is a return on our investment,” he said.
‘Dream Now Becoming a Reality’
Sawyers called immunotherapy for treating cancer “a dream that is now becoming a reality” and is “beginning to revolutionize the treatment of certain cancers, yielding both remarkable and durable responses.”
Asked if he is concerned about the ever-escalating cost of new cancer therapies, Sawyers said he is: “I think about this a lot. The pricing system of cancer drugs is illogical... it's a system that has to change.”
Sawyers said he believes that “incremental benefit should not be rewarded with premium pricing”—that is, a month or so of extended life should not come at an unaffordable price. “We're going to need combinations of these drugs. The pricing has to accommodate that.”
Also speaking at the event announcing the AACR report, another of the report authors, Drew M. Pardoll, MD, PhD, Professor of Oncology and Director of Cancer Immunology at the Sidney Kimmel Comprehensive Cancer at Johns Hopkins University, said that when he trained as an oncology fellow, there were three accepted pillars of cancer treatment. “A number of us felt unsatisfied with that.”
When targeted therapies such as trastuzumab came into use in the late 1990s, they became the fourth pillar, he said. And now, as he wrote in the report, “I have such confidence in the potential of immunotherapy that I think the years from 2010 to 2015 will be looked at historically as the time that immunotherapy became the fifth pillar of cancer treatment. Immunotherapy gives us more weapons in the armamentarium, but we are just scratching the surface. We need the help of the advocacy community; we need the help of government.”
The section on immunotherapy in the report highlights therapeutic cancer vaccines such as sipuleucel-T for advanced prostate cancer and immunotherapies such as ipilimumab, which was approved by the FDA for metastatic melanoma in 2011. Pardoll discussed early-stage clinical trial testing of immunotherapies that disable an immune system “brake,” PD1. These investigational studies, he said, have shown clinical responses not only for patients with metastatic melanoma but also for those with kidney or lung cancers.
Asked if he is concerned that some immunotherapies are very individualistic in patient response and may not have a broad benefit, he said, “Cancers are a very diverse set of diseases. They vary in their immune disease mechanisms.”
But, he noted, the common denominator is the genetic mutation a given immunotherapy targets: “When we look in diverse cancers, we always find these inhibitory molecules,” he noted. “There are so many targets we can go after. That's very exciting.”
Information in the AACR report also highlights molecularly targeted therapies; technology advances; genomic medicine; and the steps required for continued progress against cancer, such as the importance of having a highly skilled and diverse cancer research workforce.
The report's section on therapies on the horizon discusses combinations of molecularly targeted therapies, which it says are likely to become the standard of care in the near future, and small, synthetic noncoding nucleotides (DNA or RNA), which are being studied for their potential to precisely eliminate the effects of disease-causing genetic mutations.
In addition to strong research funding, the AACR calls for:
- Advancing regulatory science and policy to develop improved approaches to identifying, qualifying, and validating biomarkers;
- Increasing patient participation in cancer clinical trials;
- Enhancing the cycle of research through the adoption of electronic health records;
- Improving the quality and consistency of biospecimen resources; and
- Cultivating a cancer research workforce skilled enough to continue the swift pace of progress.
AACR President MARGARET FOTI, PHD
“Investing in research is investing in America,” Foti summed up, “and we call on all Americans to urge their policymakers to make every possible effort to help eradicate cancer.”