The LLS Blog

Highlights from AACR 2021

By Lee Greenberger, Ph.D., LLS Chief Scientific Officer | April, 2021

The first big oncology meeting of the year, American Association for Cancer Research (AACR) kicked off with week one of the meeting held from April 10-15, 2021. Key topics from the virtual meeting include concerns for blood cancer patients such as building COVID-19 vaccine confidence and addressing cancer health disparities, as well as research into ways to improve CAR T-cell immunotherapy.

Read on for a closer look at the top news from AACR:

Building Vaccine Confidence

I was invited to speak on a panel called “Best Practices to Combat Misinformation and Vaccine Hesitancy in COVID-19 Vaccines,” with esteemed speakers including Francis Collins, director of the National Institutes of Health (NIH), and co-moderators Dr. Gil Omenn, chair of the AACR Health Policy Subcommittee and Dr. Antoni Ribas, president of AACR.

Experts have predicted that we are about to reach the point where vaccine supply will start exceeding demand, and in fact that’s already happening in some places. Building vaccine confidence and uptake among the blood cancer patient community is of particular importance to The Leukemia & Lymphoma Society (LLS). Blood cancer patients are much more likely than the general population to die from COVID-19. 

During the panel, I discussed recently published survey results  from LLS, Boston University Questrom School of Business and The Behaviouralist which found that one in five blood cancer patients and survivors say they are unlikely or very unlikely to get vaccinated against COVID-19. The most common reasons cited for this hesitancy were concerns about side effects and a belief that the vaccines have not been tested properly.

Dr. Collins reinforced that decades of research enabled the rapid development of these lifesaving vaccines. While there is no reason to believe COVID-19 vaccines are any less safe in patients with blood cancers, LLS created a patient registry to increase the understanding of how COVID-19 vaccines affect blood cancer patients. We gathered results from over 1,000 patients who participated in the registry and found that vaccine side effects for blood cancer patients were mild and comparable to that of the general population. Read more about the results here.

Liz Hamel from the Kaiser Family Foundation (KFF) highlighted the KFF’s ongoing public opinion surveys showing that the “moveable middle,” people who are neither very likely nor very unlikely to get the COVID-19 vaccines, has decreased over time from 49% to 17%. This is a trend LLS hopes to see in blood cancer patients when we repeat our survey findings.

If you are a blood cancer patient or survivor who is hesitant about the vaccine, please speak with your healthcare professional about getting vaccinated.

A recording of the forum is available here.

The future of CAR T

LLS is proud of its long and significant role in funding the discovery and advancement of CAR T cell-therapy, which has been highly successful in treating very ill patients with blood cancers, who otherwise facing very poor prognoses. Five CAR T therapies are currently approved in the United States and the first CAR-T therapy for multiple myeloma was recently approved. 

Despite the efficacy of CAR T-cell therapy in some patients, the treatment has some limitations, including disease relapse.

Dr. Sanaz Ghafouri (University of California Los Angeles Medical Center) presented results from a phase 1 clinical trial of a bispecific or “two armed” CD19/CD20 CAR T treatment, which target two tumor antigens at once. This experimental treatment uses “naïve memory T cells,” that enable the body to fight off new, unrecognized diseases.

The analysis included five patients with B-cell malignancies that were positive for both CD19 and CD20 tumor antigen expression. Naïve memory T cells were extracted from each patient, engineered to express an anti-CD19/CD20 CAR, expanded, and infused back into the patient.

After a median follow-up of 13 months, four of the five patients had complete remission. Median progression-free survival and overall survival were not reached at the time of follow-up, and all responding patients continued to have CAR T-cell persistence at the time of data cutoff. These preliminary results show promise for blood cancer patients who have limited treatment options.

Early promise for NK killer cells

Immunotherapies using the immune system’s natural killer, or “NK” cells, represent an emerging field that is targeting more hard-to-treat cancers. Dr. Katy Rezvani (MD Anderson Cancer Center) presented data from an early-stage trial of four lymphoma patients who received NK cells “loaded” with the innate cell engager AFM13, designed to direct NK cells to the tumor site where they attack and kill cancer cells.

The patients enrolled in the study had disease progression, despite treatment with at least four, and as many as 14 different drugs before entering the trial. After receiving NK cell treatment primed with the experimental antibody drug, AFM13, all four patients achieved at least a partial remission and two went into complete remission.

While it is unclear how long these responses will last, these initial results are encouraging and indicate AFM13 may have the potential to help NK cells target and destroy cancer cells. While this trial is too small to draw safety conclusions, there were no observed events of cytokine release syndrome, neurotoxicity syndrome or graft-versus-host disease among the four treated patients.

LLS recognized the potential of AFM13 early and partnered with Affimed back in 2013 through our Therapy Acceleration Program (TAP). TAP provided $4.4 million co-funding supporting two AFM13 clinical trials to treat Hodgkin lymphoma patients. LLS is continuing to fund the development of NK cells. Dr. Rezvani is currently funded by LLS through a Translational Research Program grant to develop a similar, NK-cell based immunotherapy for multiple myeloma.

Positive Data for PI3K Inhibitor

PI3K are a group of kinase proteins that act like switches inside cells. In some cancers PI3K is permanently switched “on,” which means that the cancer cells grow uncontrollably. Researchers are developing treatments that block, or inhibit PI3K, to stop the cancer cells from growing.

Since 1999, LLS research grants have contributed to the understanding of the PI3K pathway, which is implicated in almost all human cancers. There are three PK13 inhibitors currently FDA approved to treat certain types of blood cancer.

Dr. Matthew J. Matasar (Memorial Sloan Kettering Cancer Center) presented results from the phase 3 CHRONOS-3 trial, which added the PI3K inhibitor copanlisib to the antibody rituximab in patients with relapsed indolent non-Hodgkin lymphoma (NHL), including those unfit for chemotherapy.

Among more than 450 patients who received rituximab plus copanlisib, the median progression-free survival was 21.5 months, compared to 13.8 months for those that who received rituximab plus a placebo. The overall response rate was 81% in patients who received the copanlisib combination, with 34% of them having a complete response. In comparison, overall and complete response rates for rituximab plus placebo were 48% and 15%, respectively, Side effects were lower in this trial compared with the previously tested oral PI3K inhibitors, which investigators believe is due to the intermittent intravenous dosing schedule for copanlisib.

Cancer Health Disparities

Several sessions at AACR focused on cancer health disparities and tools being developed to help underserved populations access the benefits of precision health prevention and treatment. This is a major focus area for LLS, which is working on multiple fronts towards health equity for all.  

To get the full benefit of cancer research, we need to increase participation among individuals who represent the diversity of the U.S. population. To overcome these urgent challenges, LLS created the IMPACT (Influential Medicine Providing Access to Clinical Trials) research grants, which will award funding to major cancer research and treatment centers to establish a “hub and spoke” infrastructure to expand access to clinical trials in their region. LLS has selected three projects for the first round of funding and will announce further details in the coming weeks.

Among the many interesting presentations at AACR, Dr. Tiffany A. Wallace (National Cancer Institute) provided an overview of the complex and interrelated factors that contribute to cancer health disparities in the U.S. These factors may include inequalities in transportation, housing, education, income, employment and access to quality healthcare. She also discussed the poor representation of racial and ethnic minority populations in clinical trials, a topic of critical importance to LLS.

Clinical trials are vital to improving cancer treatment, but only about 5-8% of cancer patients participate in them with even lower participation rates among underrepresented groups, including those without insurance or with low income; Black, Indigenous, and people of color; patients from rural areas; young adults and the elderly.

I look forward to attending and reporting on further advancements for blood cancer patients from the next major medical meeting, ASCO 2021 taking place from June 4-8. 

Because you volunteer, your impact has a ripple effect

By Teri Johnson, Senior Vice President, Volunteer Engagement at LLS | April, 2021

When you commit time and talent to a nonprofit’s mission – no matter how you choose to get involved – you become a volunteer, and the impact of your efforts runs far deeper than the specific project or task you work on. For example, volunteers don’t always realize that the hours they spent stocking shelves meant hundreds of families would have food for two weeks. Or that the time spent fundraising not only raised critical mission dollars but also added value upward of $1,250 to the organization’s bottom line. When you consider that many corporate partners, philanthropists, and foundations want to know the impact of volunteerism before sponsoring, granting, or donating, it’s clear just how much your volunteer time matters.

Your volunteer time creates a powerful ripple effect that touches countless lives and sustains vital work in our communities. At LLS, we see this first-hand every day.

This past year has challenged us all as we’ve had to adapt to a new normal. For patients and caregivers, the pandemic has brought added distress, new financial worries, and loneliness on top of navigating their cancer care and treatments. The pandemic could have stopped LLS in our tracks, but it didn’t. Together, we forged ahead with our mission because patients and families couldn’t wait for our help. And while the uncertainty continues today, here’s what I do know.

Since the earliest days of the pandemic, volunteers stood in partnership with our staff and expressed unwavering support for other volunteers, our patients, families, and our communities. Even though we couldn’t be in the same room, attend events together, celebrate successes, and give high-fives, LLS volunteers showed up. You rolled up your sleeves to provide critical support for patients, raise your voices for equal access to care, fundraise in support of research, and so much more. Our volunteers dedicated more than 200,000 hours from March 2020 to April 2021 – an incredible feat during challenging times.

To put this into perspective, the value of that time is roughly equivalent to the following activities:

• 108,800 free educational booklets for blood cancer patients and healthcare professionals
• 54,000 translational services for a Specialist to communicate with non-English speaking patients
• 18,133 critical lab supplies for LLS-funded researchers
• 10,880 financial assistance for eligible blood cancer patients through our Urgent Need Program

When blood cancer patients and families needed us more than ever, LLS volunteers, alongside our staff, were unstoppable. Across the country, volunteers support every facet of our organization – from bolstering our regions’ efforts to raising critical funds to providing direct support to patients and families and advocating for policy changes at the state and federal level. That’s why LLS volunteers are truly superheroes. And your passion, time, and talent are superpowers in helping LLS beat blood cancers. In fact, while our bedrooms became offices and our kitchens became schools, and we all learned to unlock new skills, LLS volunteers helped us achieve remarkable progress across our mission.

FDA Approval of New Treatment Option for Children with Secondary Acute Myeloid Leukemia is a Welcome Step Forward

By Gwen Nichols, MD, LLS Chief Medical Officer | March, 2021

Here at The Leukemia & Lymphoma Society (LLS), we applaud every advance for children with blood cancers. Today's reason to celebrate is the news that the U.S. Food and Drug Administration (FDA) expanded the approval of daunorubicin and cytarabine (Vyxeos®) to include treatment of pediatric patients aged one year and older with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). This is a welcome and necessary advancement in support of some of our most vulnerable patients.

The therapy was previously approved in 2017 for adult patients who acquired AML after being treated for a previous cancer or whose AML developed after progressing from a diagnosis of myelodysplastic syndrome. LLS recognized this therapy's potential back in 2009 when it invested in it through its Therapy Acceleration Program® (TAP), which invests in cutting-edge companies working in areas of high unmet need for blood cancer patients. LLS partnered with Celator Pharmaceuticals, which Jazz Pharmaceuticals later acquired, on the clinical development of daunorubicin and cytarabine (Vyxeos®) for adults with AML. 

The approval is supported by safety data from two trials conducted by the Children's Oncology Group (COG) and Cincinnati Children's Hospital (CCH), which established safety for relapsed/refractory patients. Both studies did report adverse reactions, including bleeding events, fever, and rash. The efficacy data was based on the CPX351-301 study in adults with secondary AML, which LLS TAP supported. 

While we celebrate today’s news and what it means for children with AML and their families, and all the work being done to provide safe and effective treatments, LLS remains focused on how much more there is to do. Today, many children do not survive pediatric blood cancer, and survivors are often left with lifelong complications from treatment. When it comes to AML, the five-year survival rate for children younger than 15 years old diagnosed is only 68.7 percent.

Children have been treated with a one-size-fits-all approach for too long, despite cancer behaving differently in children than in adults. For many childhood cancers, chemotherapies developed decades ago remain the standard of care and simply don’t work well enough. In this era of precision medicine – giving the right treatment to the right patient at the right time – it’s critical for children to receive new therapies designed just for them.

LLS is determined to usher in a new era of drug development for pediatric blood cancers that puts children at the center of their care. Through the LLS Children’s Initiative, we seek to shift the standard of care for children, moving them away from toxic chemotherapies and toward effective and safe precision medicine treatments that target their cancer without harming the rest of their bodies. LLS is currently laying the groundwork for LLS PedAL, the first global precision medicine clinical trial for children with relapsed acute leukemia, which is being informed by its success with the Beat AML® Master Clinical Trial that was launched in 2016 for newly diagnosed AML adult patients 60 or older. Further, LLS is funding more than 30 research grants focused on deepening our understanding of the unique genetic causes and molecular biomarkers of pediatric blood cancer and how best to target them.

LLS is striving to raise $100 million for The LLS Children’s Initiative, which is a multi-year collaboration to expand our investment in leading-edge research, provide a comprehensive array of free information, resources, and financial support for affected children and families, and support policies that break down barriers to accessing care and developing new treatments.

Our goal is not only for children to survive blood cancer but for them to thrive in their lives after treatment.