More than three years after the first chimeric antigen receptor (CAR) T-cell therapy achieved U.S. Food and Drug Administration (FDA) approval, the revolutionary approach that has upended blood cancer treatment continues to generate excitement.
LLS support over more than two decades helped lay the groundwork for this approach to therapy that has been a game-changer for patients who have relapsed multiple times and seemingly exhausted their treatment options. The method works by infusing immune (T) cells with cancer-attacking synthetic receptors that seek out and bind to a specific protein on the surface of the cancer cells, causing their destruction. The CAR-T cells continue to expand and fight the cancer cells, becoming a living drug within the patient’s body. Pediatric acute lymphoblastic leukemia patients and adults with large B-cell lymphomas have benefited from long-lasting remissions from the first CAR-Ts approved. The latest CAR-T approval was in August for patients with mantle cell lymphoma.
Consensus is building that the next CAR-Ts on the horizon will be for multiple myeloma patients and follicular lymphoma and marginal zone lymphoma patients who have relapsed multiple times. During this week’s virtual 62nd American Society of Hematology (ASH) annual meeting, a slew of presentations makes clear several of these are likely to get approved in early 2021.
The myeloma CAR-Ts all target a protein called BCMA found on cancerous plasma cells in myeloma patients. BCMA has emerged as a prime target for myeloma treatment, and the first BCMA-targeting therapy, an antibody-drug conjugate (an antibody bound to a cytotoxic agent), was approved by the FDA in August – read more here.
Of the multiple companies vying in the BCMA-targeting CAR-T space, Johnson & Johnson’s product, ciltacabtagene autoleucel (cilta-cel), appears to be the frontrunner, with the highest response rates and longest-lasting CAR-T cells. In a study of 97 patients, 97 percent responded to the treatment, and 67 percent had a complete response, meaning no cancer cells were detectable. At just over one year (the median follow-up, meaning at least 50 percent of the patients had been on the trial), 77 percent of the patients were still alive and their cancer had not gotten worse.
Johnson & Johnson plans to submit its data to the FDA within the next few weeks. What's more, Bristol Myers Squibb/Bluebird’s idecabtagene vicleucel (ide-cel) – also promising while not as long-lasting in trials to date – is already before the FDA with a decision anticipated in March. While not involved in the study present at the ASH meeting, an LLS-funded researcher at Emory University, Madhav Dhokapkar, MBBS, is studying this therapy to find ways to make it more durable, safer, and more effective. Many CAR-T products from Chinese companies were also presented at the meeting.
The length of time that BCMA-targeting CAR-T cells can last in patients has been questionable, so the results presented at this ASH meeting are encouraging. At the same time, the deaths of several patients on trials have raised concerns about potential toxicities, including dangerous neurological side effects and another extreme immune response called cytokine release syndrome. Despite this, the FDA might determine that the benefits for patients without other treatment options outweigh the risks.
Patients with follicular (FL) and marginal zone lymphoma (MZL), two slow-moving “indolent” forms of non-Hodgkin lymphoma, also have reason for hope. Results from a clinical trial entitled ZUMA-5, testing a CAR-T developed by Kite Pharma, a Gilead company, showed high response rates for patients with both forms of the disease. LLS helped Kite Pharma launch their CAR-T program through a partnership entered in 2015.
Of 104 patients evaluated, 84 with FL and 20 with MZL, 92 percent had an objective response, meaning their cancer cells were reduced; 76 percent of patients had a complete response, meaning the cancer became undetectable after a median follow-up of 17.5 months (the time when 50 percent of the patients have been on trial).
Researchers across the globe – with support from LLS – continue to work tirelessly to usher in the next generation of CAR T-cell therapies so that they're safer and more efficacious for more patients with blood cancers.
With the COVID-19 pandemic driving yet another science conference to the virtual world, it is fitting that the 62nd ASH Annual Meeting and Exposition officially kicked off today with a virtual fireside chat via live stream featuring none other than Anthony Fauci, MD, director of the National Institute of Allergy and Infectious Diseases, and a member of the administration’s coronavirus task force.
The ASH meeting typically fills massive convention centers each December. Each year, 40,000 scientists, clinicians and other healthcare professionals, pharmaceutical and biotechnology executives, media, and communications specialists, gather to share and learn about the latest advances in the blood cancers and other blood diseases, including sick cell anemia and hemophilia
Dr. Fauci and ASH president Stephanie Lee, MD, discussed the impact of COVID-19 on patients with blood cancers. Despite cancer patients being excluded from clinical trials for impending COVID-19 vaccines, Dr. Fauci recommended that patients who are immunocompromised, including blood cancer patients, receive the vaccine when it becomes available. He also said that all healthy people should get the vaccine to create the umbrella effect of herd immunity. Dr. Fauci also said that “we can crush the virus” if billions of people around the globe have access to the vaccine. In the meantime, he stressed it is urgent to continue to practice responsible public health measures like wearing masks and social distancing.
Here at The Leukemia & Lymphoma Society (LLS), we hear from blood cancer patients and caregivers each day about the profound effects of the COVID-19 pandemic on their cancer care, treatment, and daily lives. So many of us are eager for a vaccine so that we can protect ourselves and our loved ones and see a gradual return to normal. While it’s been an incredibly trying year, there are bright spots ahead as we look toward safe, effective vaccines on the horizon. While we may not have all the answers at this time, I’m addressing a few FAQs from blood cancer patients and caregivers based on what we know right now. I encourage you to discuss COVID-19 vaccines further with your oncologist.
I also want to stress that it’s normal to experience feelings of anxiety, fear, and uncertainty as you continue to navigate your care during these challenging times. LLS offers a wide array of free education and support that can help. LLS Information Specialists – highly trained oncology professionals – are here to provide one-on-one support at (800) 955-4572 or by live chat/email. Taking care of yourself is so important, so make sure you’re taking advantage of telehealth, following preventive measures from the CDC, and talking to your healthcare provider about getting a flu shot.
Last updated 12/2/2020. For the latest updates, please check our FAQ page here.
1. When will a COVID-19 vaccine be available?
An approved or authorized vaccine to prevent COVID-19 is not available yet in the U.S., but there is encouraging news. Many COVID-19 vaccines are under development, and some have shown promising results so far in clinical trials. For example, Pfizer and Moderna recently applied for emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for their COVID-19 vaccines. Both companies reported their vaccines are around 95 percent effective according to preliminary results; however, these vaccines were not tested among cancer patients. A limited quantity of vaccines may be available for individuals at the highest risk, including frontline healthcare workers as well as residents and staff of nursing homes and long-term care facilities, before the end of the year. A federal advisory group has recommended these groups receive COVID-19 vaccines first, and these recommendations are currently under review by the director of the U.S. Centers for Disease Control and Prevention (CDC). If approved, these recommendations for the highest priority groups (Phase 1a) will become official CDC guidance. It will take some time – likely late spring or early summer 2021 – before COVID-19 vaccines become more widely available.
2. Will COVID-19 vaccines be safe for blood cancer patients? Should I get one right away or wait?
Vaccines are being developed and tested in record time, and the pace of progress gives us all reason to be optimistic. But since most COVID-19 vaccine clinical trials have not yet included cancer patients, it’s too soon to know if or how well these vaccines might work for blood cancer patients. In general, cancer treatment can affect how a patient’s immune system responds to vaccines. And certain types of vaccines – such as those containing live viruses (e.g., measles-mumps-rubella vaccine) – are not recommended for people with cancer. That’s why it’s important to talk to your oncologist about which vaccines are safe for you.
The good news is, none of the COVID-19 vaccines currently in development in the U.S. use the live virus, according to the CDC; a variety of new approaches are being studied. Ultimately, more research is needed to determine the safety and efficacy of any new COVID-19 vaccine for cancer patients. Over the coming months, clinical trials will continue to expand, giving the medical and scientific communities more data on how well these vaccines work and who should receive them. For example, because children’s immune systems are different from those of adults, pediatric clinical trials are essential in order to establish safety and efficacy of COVID-19 vaccines among children. As some pediatric trials are just getting started, it will be some time before we see a vaccine available for kids – likely later into 2021. While we don’t know when these trials will begin including children with cancer, it’s a good idea to begin discussing vaccination plans with your child’s oncologist.
LLS continues to monitor for developments closely. We are keeping abreast of the ongoing studies, and advocating for the inclusion of cancer survivors in clinical trials when it is safe to do so. In the meantime, I encourage you to discuss with your oncologist what makes sense for you based on where you are in your treatment plan.
3. What’s an emergency use authorization (EUA)?
A vaccine can be distributed in the U.S. only after an approval or an emergency use authorization (EUA) from the FDA. During a public health emergency, the FDA can issue an emergency use authorization (EUA) to OK certain medical products more quickly based on the best available evidence. Through EUAs, the FDA can make medical products available to the public without waiting for all of the evidence needed for full approval or clearance. Since the start of the pandemic, the FDA has authorized hundreds of coronavirus tests and a few treatments, including the second antibody treatment, most recently. The agency has also provided guidance to vaccine developers, outlining the efficacy and safety conditions that need to be met for a COVID-19 vaccine to receive EUA.
4. Should my caregiver or loved ones get a COVID-19 vaccine? If so, how soon and will it be safe?
While we don’t know when a COVID-19 vaccine will be available, I encourage caregivers and other family members to start a conversation about COVID-19 vaccination with their healthcare providers as early as possible. Clinical trials are evaluating COVID-19 vaccines in tens of thousands of participants to generate the scientific data needed by the FDA to determine safety and effectiveness. Data so far suggest the vaccines are safe, with side effects similar to other vaccines that trigger an immune response, including injection site pain, fatigue, and the occasional fever. Caregivers should be certain to tell their healthcare provider that they care for a blood cancer patient.
5. I’m a blood cancer survivor. Should I consider getting a COVID-19 vaccine right away?
I encourage blood cancer survivors to talk to their oncologists about COVID-19 vaccines. Preliminary data from clinical trials suggest these vaccines are safe and effective among older adults, who do not make as many protective antibodies as younger people. This is promising news. While we have every reason to believe these vaccines will be safe for individuals who have completed cancer treatment, more research is needed. LLS will be closely monitoring for developments over the coming months. In the meantime, I encourage you to discuss a vaccination plan with your care team. You can find general information about immunizations for survivors here.
6. Why is a vaccine taking so long?
Because COVID-19 is caused by a novel virus, entirely new vaccines must be developed and tested. Typically, vaccine development takes years – sometimes decades – from discovery research and pre-clinical testing to clinical trials, regulatory approval, and production and delivery. Thankfully, unprecedented collaborations among government agencies, academic institutions, researchers, nonprofits, and biotech companies are speeding this process. Multiple groups are working together to make sure that safe, effective COVID-19 vaccines become available as quickly as possible.
7. Can you explain herd immunity?
Herd immunity is a concept used for vaccination, in which a population can be protected from a virus if a certain threshold of vaccination is reached. With herd immunity, if a vast majority of people are vaccinated, it reduces how much a virus can spread throughout the whole population, helping to protect vulnerable groups who cannot get vaccinated. While the availability of COVID-19 vaccines would mark a significant step forward, it will be a while before herd immunity is achieved given the large numbers of people who would need to be vaccinated and how quickly the virus spreads through asymptomatic carriers. Further, more data will be needed to evaluate how well the vaccines prevent infection and induce immunity over time. It’s important to remember that herd immunity refers to community protection – as opposed to individual protection – so it’s not a substitute for appropriate vaccination of people at risk in the population.
8. How will COVID-19 vaccines be distributed, and how do I get one once available?
Likely, vaccine distribution will follow a phased approach, where the first people to receive them will be frontline healthcare workers and other individuals at the highest risk, such as residents and staff of nursing homes and long-term care facilities. The CDC is collaborating with health departments and partners on vaccination programs that can accommodate different vaccines and scenarios. As part of this, the CDC is also working with state, tribal, territorial, and local jurisdictions on vaccination plans for their respective areas. What’s more, the CDC is developing COVID-19 vaccination recommendations based on input from the Advisory Committee on Immunization Practices (ACIP), composed of medical and public health experts. Key to these recommendations is guidance on who should receive a COVID-19 vaccine first if supply is limited. These recommendations are expected to be approved by the director of the CDC soon. Distribution for COVID-19 vaccines is a complex endeavor, particularly given ultra-cold storage requirements and two-dose regimens, but collaboration is leading the way. It’s anticipated that these vaccines will become more widely available to the general public in late spring or early summer 2021. Be sure to speak with your oncologist about whether a COVID-19 vaccine might be safe for you.
9. How do these new vaccines work?
Researchers are studying various approaches. While we don’t yet know how these approaches compare in terms of safety and efficacy, the medical and scientific communities are learning more each day. One promising approach involves a new technology called messenger RNA (mRNA). Unlike traditional vaccines that rely on weakened or inactive viruses or proteins of those viruses, mRNA vaccines use a snippet of the virus’s genetic code to instruct cells to build viral proteins. In this case, the cells learn how to make the so-called “spike protein” found on the surface of the virus that causes COVID-19. As a result, an immune response gets triggered, which produces antibodies, helping the body recognize and fight infection if exposed to the virus naturally. This is the first time this technology has been used for a vaccine. And the lightning speed at which the science is progressing is truly remarkable.
10. How do I cope with pandemic fatigue while we wait for vaccines?
If you’re experiencing feelings of anxiety, stress, fear, sadness, and grief, know that you are not alone. Months of isolation and uncertainty can take a toll on anyone, and we’re hearing from many patients and caregivers who are going through it right now. Taking care of yourself is one of the most important things you can do, and support is available to you. LLS has so many free resources to help you find the support that is right for you, including online chats, our social network LLS Community, a peer-to-peer support program, and so much more. LLS Information Specialists are here to help you and any blood cancer patients, survivors, caregivers, family members, and healthcare professionals at (800) 955-4572 or by live chat/email here.
Find more free blood cancer information, education, and support from LLS.