In the 12 short years since Stand Up To Cancer (SU2C) was founded, this dynamic group started by journalist Katie Couric and eight other women in the entertainment industry has saved or prolonged countless lives and changed the way we think and talk about cancer. The money raised by SU2C has fueled numerous advances and extended the lifespan of people with a broad range of cancers.
In all, SU2C research has contributed to the Food and Drug Administration’s (FDA) approval of six new cancer therapies, including treatments for breast, ovarian, and pancreatic cancers, and leukemia. Additionally, SU2C-supported research has led to FDA Breakthrough Designation Status for combination treatments in colon and prostate cancers. More than 12,000 patients have participated in SU2C-funded clinical trials and the results of these trials continue to change practice in many cancers, including notoriously hard-to-beat cancers such as pancreatic cancer. These are the 10 most common types of cancer in the U.S.
“I didn’t know any of this would happen,” says Phillip A. Sharp, PhD, Chair of SU2C’s Scientific Advisory Committee (SAC) and a Nobel Prize winner for his research in genetics. “I understood that there was a group of very determined leaders in Hollywood who wanted to accelerate the translation of research into the treatment of cancer and were committed to working to make that happen, but I didn’t realize how many advances there would be in a relatively short period of time.” Dr. Sharp is also an institute professor at the Massachusetts Institute of Technology (MIT) and a member of the department of biology at the Koch Institute for Integrative Cancer Research.
And they’ve really only just begun.
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Teamwork makes the dream work
Collaboration is the cornerstone for all of these efforts, says Los Angeles-based Sung Poblete, PhD, SU2C’s Chief Executive Officer. This whole movement started when the founders joined forces to air the initial SU2C star-studded telethon on several major broadcast networks in more than 170 countries on September 5, 2008.
“There wasn’t funding to support a lot of collaboration, cooperation or communication among large teams when SU2C was started in 2008,” says Poblete. “We started with a Dream Team approach and moved toward other funding models that bring together scientists from different disciplines to better understand and treat cancers.”
Dream Teams, of which there are now 26, bring together multidisciplinary investigators from different institutions and specialties. These teams have budgets of millions of dollars, some as high as $22 million, to support research on new drugs, new combinations of drugs, emerging risk factors, improved diagnostics and more.
Lee J. Helman, MD, section head of basic and translational research within the Cancer and Blood Disease Institute and director of the Cancer and Blood Diseases Research Program of The Saban Research Institute of Children’s Hospital Los Angeles, is a vice-chair of SU2C’s scientific advisory committee. Dr. Helman provides oversight for a Dream Team that helped get a pediatric leukemia therapy approved before it was approved for adults with this cancer, which is a dramatic reversal of how things are usually done.
The pediatric leukemia therapy consists of the drug Kymriah (tisagenlecleucel), a type of immunotherapy drug known as a chimeric antigen receptor (CAR) T-cell. Immunotherapy helps your immune system fight cancer. With CAR T-cells, scientists genetically modify your own immune cells in the lab, then infuse these cells back into your body where they prompt your immune system to attack and kill cancer cells, explains the American Cancer Society.
“In a very short period of time, we not only were able to show the advantages of this drug in treating childhood acute lymphoblastic leukemia (ALL) but, because of collaboration efforts, we were able to define its toxicity and make side effects more manageable,” Dr. Helman says. ALL is the most common type of leukemia diagnosed in children in the US, according to the American Cancer Society, and Kymriah is the standard of care now for patients up to age 25 whose cancer has not responded to other treatments or has returned. “About 90 percent of children with ALL can be cured with available therapies, but thanks to this new drug and protocols, we will be curing even more kids with less toxicity 20-30 years down the road,” Dr. Helman says.
Research funded by SU2C has also changed the way pancreatic cancer is treated. The SU2C Pancreatic Cancer Dream Team’s work led to the FDA approval of a new combination of drugs, Gemcitabine Plus Abraxane, to help people with advanced pancreatic cancer live longer. This was the first approval for a first-line pancreatic cancer treatment in 15 years and it is estimated to now be the first-line treatment for about one-third of all pancreatic cancers. Here are 23 more groundbreaking cancer discoveries that could save your life.
The perfect storm
It was more than just taking down silos between disciplines and institutions that helped SU2C make such tremendous strides in a relatively short period of time, explains SU2C Scientific Advisory Committee vice-chair William Nelson, MD, PhD, the director of the Sidney Kimmel Comprehensive Cancer Research Center at Johns Hopkins in Baltimore.
The group was formed around the time that immunotherapy began to emerge and evolve. This is also around the same time that researchers learned how to test tumors for genetic alterations. This is now standard in the diagnosis of many cancers. Genetic analysis allows doctors to better match treatments to cancers based on genetic profiles. This will help eliminate some of the trial and error involved in choosing therapies.
“Taken together, we were able to put more bets on better drugs,” Dr. Nelson says. In February 2015 the FDA granted accelerated approval to Ibrance (palbociclib) to treat advanced breast cancer after the SU2C Breast Cancer Dream Team reported increased survival in women taking it. This approval is intended for postmenopausal women with estrogen receptor (ER)–positive, human epidermal growth factor receptor 2 (HER2)–negative advanced breast cancer who have not yet received hormone therapy. And in December 2016, the FDA granted accelerated approval to rucaparib (Rubraca) for women with BRCA1/2-mutated ovarian cancer. BRCA 1/2 are often called the breast cancer genes, but they predispose individuals to other cancers as well. Check out these 11 signs of ovarian cancer you might be ignoring. Rubraca has since found a niche in prostate cancer as well. Taken together, it is conceivable that millions of lives have been extended based on the availability of these drugs.
Immunotherapy drugs were first approved for advanced or spreading cancers, but initial data generated from SU2C showed that immunotherapy has benefits outside of stage 4 cancers, explains SU2C-Cancer Research Institute Immunology Dream Team Investigator Padmanee Sharma, MD, an immunologist and oncologist at the University of Texas MD Anderson Cancer Center in Houston. Learn more about metastatic cancer here.
“The data that we have generated have moved the field forward,” Dr. Sharma says. “We now have larger registry trials looking at whether patients with bladder and other cancers can benefit from immunotherapy given earlier in the disease.” She’s hoping to see FDA approval for the use of immunotherapy drugs in earlier disease settings, which would bring advances to many people diagnosed with cancers earlier, when the treatments may a better chance of helping survival.
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The interception approach
SU2C is also championing cancer interception. This results in more people getting diagnosed early when their cancer is in its most treatable and beatable stages, says Dr. Sharp. “With more attention paid to interception where we get the disease earlier with less burden and less damage, we will see a very big benefit in terms of controlling disease,” he says.
Take pancreatic cancer, for example: More than 57,000 Americans will be diagnosed with pancreatic cancer this year, according to The Lustgarten Foundation.
This cancer has an extremely high fatality rate partly due to the fact that doctors can’t diagnose it early. But what if they could? “We are investing in interception to identify patients who are going to progress to malignant pancreatic cancer by looking at medical records five to ten years before diagnosis,” he says. And clear patterns are emerging, especially in regard to genes.
SU2C researchers are exploring the genetics of pancreatic cancer as part of their interception approach. Certain genetic mutations are more common in people with pancreatic cancer, such as breast cancer genes 1 and 2 (BRCA1 and BRCA2), adds Sapna Syngal, MD, MPH, director of research at Dana-Farber’s Center for Cancer Genetics and Prevention in Boston. She is also a principal investigator on the SU2C-Lustgarten Foundation Pancreatic Cancer Interception Dream Team. These 14 women are grateful for their BRCA diagnosis—here’s why.
The goal of the Genetic Education, Risk Assessment, and Testing (GENERATE) study is to improve access to genetic testing and allow for earlier diagnosis among family members of people with pancreatic cancer. As part of the study, high-risk individuals are counseled via video about the pros and cons of pancreatic cancer genetic testing. They are then sent saliva kits to test for genetic mutations. Participants learn about their results from a skilled counselor over video. This shores up gaps in access to skilled genetic counselors, she adds. “We also have the ability to discuss surveillance such as endoscopic ultrasounds or MRIs to help catch pancreatic cancer earlier with individuals who have mutated copies of BRCA1 or BRCA2.” Endoscopic ultrasounds provide images of internal organs such as the pancreas.
Interception has the potential to save millions of lives, including thousands from pancreatic cancer. If you have a first-degree relative with pancreatic cancer, you should consider undergoing genetic testing, Sr. Syngal suggests. “If you have a mutation and undergo surveillance, it is our hope that you will get diagnosed earlier when your chances of survival are much greater,” she says. “We want to get the cancer when it is incipient or really small and can be taken out. This is the springboard we are putting into place.”
Promising new areas in cancer research
It can take years to get grants written, approved and funded, but the SU2C Phillip A. Sharp Awards for Innovation in Collaboration program is changing this. It is akin to speed dating for researchers. These grants bring together two researchers from different teams to collaborate. The grants are written and can be funded within 48 hours.
This helps put SU2C researchers at the frontlines of some of the newest and most exciting areas of research in cancer.
Epigenetics refers to a mechanism in cells that turn genes on and off, and there are now 12 SU2C-funded trials underway looking at drugs that have the potential to impact these processes, explains Peter Jones, PhD, Chief Scientific Officer of Van Andel Research Institute (VARI) in Grand Rapids, Michigan, and the co-leader of the VARI-SU2C Epigenetics Dream Team. The theory here is that if something can be turned on, it can also be turned off, he explains. Proof of concept has been shown in myelodysplastic syndrome (MDS) or cancers that develop when the blood-forming cells in the bone marrow become abnormal. “We want to extend the reach of these therapies into solid tumors and thinking about ways to do this with combination therapies of epigenetic drugs, immunotherapy or standard chemotherapy agents,” he says.
The microbiome or the milieu of good and bad bacteria in our bodies is another important area in all of medicine. SU2C-backed research helped put it on the map for cancer. “This support was critical, and provided the basis for a growing and large body of literature demonstrating that gut microbes can influence therapeutic responses and can even be targeted,” explains Jennifer Wargo, MD, professor of surgical oncology and genomic medicine at The University of Texas MD Anderson Cancer Center in Houston. “Clinical trials targeting the gut and tumor microbiome are currently underway in patients with cancer—with some very promising early results.”