Clinical trials represent the forefront of our knowledge in cancer care. Patients participating in these trials are receiving the best of the best in cancer treatment – and advancing our ability to fight cancer in the future. The popular perception is that clinical trials are limited to university or hospital systems, but this couldn’t be further from the truth. At community oncology clinics around the nation, clinicians and patients are breaking ground in the fight against cancer with cutting edge clinical trials.

On May 12 at 12 p.m. ET, Katie Goodman, Vice President, Clinical Research, American Oncology Network (AON) and Rose Gerber, Director of Patient Advocacy & Education, COA, shared information from the provider and patient perspective on clinical trials, including understanding the different types of clinical trials, the rigorous safety review process, and who is eligible to take part in a trial (hint: just about anybody!). From contributing to advancements in care for patients to expanding one’s health outcomes, clinical trials provide opportunities for patients to make an impact on their own health care and on the lives of others going forward.

This event was part of our CPAN Advocacy Chats series – 30-minute, monthly educational webinars on key cancer issues and the policies that impact them. Each month features a new topic and special guests joining us. Patients, survivors, caregivers, and other cancer care advocates are all invited to join us for these free, interactive, virtual events!

CPAN Advocacy Chat: Clinical Trials in the Community Setting

Patients with previously untreated, unresectable, advanced esophageal squamous cell carcinoma (ESCC) had significantly better overall survival (OS) when treated with Opdivo (nivolumab) in addition to either chemotherapy or Yervoy (ipilimumab), as opposed to patients treated with chemotherapy alone.

The first results of the CheckMate-648 study, which notably marked Opdivo as the first PD-1 inhibitor to durable responses and better OS in patients with ESCC, were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.

“So we actually have not just one, but two new treatment options for our patients,” said Dr. Ian Chau, consultant medical oncologist Royal Mardsen Hospital in the United Kingdom and lead study author, in an interview with CURE®. “And apart from improving their survival, we also noticed it’s not just related to the average survival, we've noticed that there were longer term survivors in the immunotherapy-containing arms.”

Chau also noted that tumor shrinkage achieved by the immunotherapy in the study lasted longer than tumor shrinkage from chemotherapy alone.

Cancer of the esophagus carries a poor prognosis. Each year globally, over half a million of patients will die from this disease. And in this particular study, we look at a subtype … called squamous cell cancer that is actually the most predominant type in this disease. And for decades, there has been no improvements in our treatment outcome for our patients. And for patients where their cancer has already spread or has already gone to other places, the average lifespan is only about 10 months or less. So there really is a lot that we need to improve on. So we performed this study, which is called CheckMate-648 study, where we have patients with this disease who are newly diagnosed with cancer, which unfortunately has already presented a stage where it has spread. All patients who may previously have had more localized disease, had some treatment, but unfortunately, the cancer had come back. 

And what we've found is that patients who have received immunotherapy, so the group who have either added immunotherapy to chemotherapy, or the group where they have two immunotherapy drugs, both of these groups have shown a significantly better survival compared to patients who have chemotherapy alone, which is what we have been treating patients (with) for decades. So we actually have not just one, but two new treatment options for our patients. And apart from improving their survival, we also noticed it’s not just related to the average survival, we've noticed that there were longer term survivors in the immunotherapy-containing arms. And we also note in this study that for patients who have tumors shrinkage, which was achieved by immunotherapy, the tumors shrinkage lasts for much longer than the tumor shrinkage that was achieved by chemotherapy alone.

For more news on cancer updates, research and education, don’t forget to 

Opdivo was the first PD-1 inhibitor to show durable responses in patients with esophageal squamous cell carcinoma.

New Potential Immunotherapy Treatment Options in Advanced Esophageal Squamous Cell Carcinoma

Health insurance status may affect kidney cancer outcomes, according to recent research.

Patients with metastatic renal cell carcinoma (RCC) with primary Medicaid or no medical insurance had worse survival outcomes compared with those with primary Medicare, according to findings from a recent study.

Results from this study, which were presented at the 

, also demonstrated that patients with secondary insurance had superior survival without disease progression versus those with only primary insurance.

“Overall, our findings suggest that insurance status may serve as a determinant of clinical outcomes,” the study authors wrote in the abstract. “These hypothesis-generating data warranted external validation in prospective studies.”

Researchers analyzed data from 645 patients (73% men; median age at diagnosis, 60 years) with metastatic RCC from two National Cancer Institute-designated comprehensive cancer centers who received a diagnosis between 1990 and 2022.

The available insurance information was categorized as Medicare (53.3%), private insurance (38.8%) and Medicaid/no insurance (7.9%). In addition, secondary insurance was defined as either the absence or presence of secondary medical coverage.

Several outcomes were assessed including progression-free survival (the time during and after treatment when a patient with cancer is alive without disease worsening) and overall survival (the time when a patient with cancer is still alive). These outcomes were examined to determine the potential impact of insurance status.

The most common first-line treatments given to patients with metastatic RCC in this study were monotherapy with targeted agents (66.4%), targeted/immunotherapy combinations (10.2%) and dual immunotherapy (13.6%).

The median progression-free survival for all patients in the study was 6.6 months. When assessed by insurance status, the median progression-free survival was 7.7 months for patients with Medicare, 5.5 months for those with private insurance and 4.9 months for patients with Medicaid/uninsured. Researchers noted a significant difference in progression-free survival among the insurance groups.

Secondary insurance status also showed a statistically significant difference in median progression-free survival, with 8.1 months in patients with secondary insurance compared with 6.1 months for those without it.

For overall survival, the median for all patients in the study was 36.8 months. There was a significant difference in overall survival in patients with primary Medicare (49 months), private insurance (28.5 months) or Medicaid/no insurance (21.6 months).

Overall survival did not significantly differ in patients with or without secondary insurance.

Superior median progression-free survival and overall survival were observed in patients with primary Medicare versus Medicaid/no insurance. Researchers noted that patients with private insurance had a lower risk for progression and death versus those with Medicaid/no insurance, although the difference was not statistically significant.

Patients with Medicaid or no insurance may have worse survival outcomes compared with Medicare or private insurance in those with metastatic renal cell carcinoma.

Insurance Type May Indicate Survival Outcomes in Metastatic RCC

The phone rang and it was the call that everyone who has gone through cancer will never ever forget.

“Hello, This is Dr. So-and-so, I’m sorry to tell you that you have breast cancer.” This realization did not seem real. My world collapsed; my body collapsed!

“Not me! It can’t be me! I’m young and healthy. I’m a mom. I’m a wife. I’m a daughter. I have a daughter. I am going to die!” I thought, though that was not the realization I had.

This month marks six years since my cancer diagnosis. Today my words to anyone (including myself) would be: No, you are not going to die. Well, we are all going to die one day but right now, you are going to fight. You are going to survive. That was the realization!

I like to call myself a survivor. The truth is that cancer does not mean life is over. It comes in all shapes and forms, but with a positive mind and spirit, you can find strength from within. In the beginning of my diagnosis, I wasn’t open to accepting help or advice. When I look back now, I realize that it was the support and guidance that I received from other survivors that helped me the most. There is no one better to help guide you than someone who has been in your shoes.

I was the 1 in 8 chosen to get breast cancer. It was a stressful time, that I won’t deny. For me, once I allowed myself to grieve, I was able to get mad and prepare myself for the fight. I realized the importance of listening very carefully to my doctors, educating myself and asking lots of questions. That would ultimately help cure my cancer. I also determined that by accepting what I have been dealt, it allowed me to put my energy into being well again. I may have lost my breasts, but I was put back together by an amazing team of doctors. If I wasn’t clear headed, I never would have been able to move forward and make the important life decisions that I was faced with making.

I took the news of my diagnosis very hard. For me, the worst part was the mental and emotional breakdown, and not the physical part or the cancer itself. After I went through the grieving, anxiety and depression, I somehow had a fire within me telling me that I was not going to die. I will have a strong body again. I will shower myself with good vibes and I will heal — and that’s just what I did.

The traumatic and emotional part of cancer will always stay with me, but cancer itself has made me who I am today. I am stronger. I am wiser. I no longer take things for granted and most importantly, I choose to be kind because you never know what a person is going through. It could be cancer.

I will forever be grateful for the life I have ahead of me. I am a stronger person. I am a changed person, but I fought, and I won and I’m a survivor.

One realization to me that I did not know before, but I know now is: Life isn’t about waiting for the storm to pass. It’s about learning to dance in the rain.

You need to have hope and believe you will make it to tomorrow and the day after ... and the day after that… and live life to your fullest. You are a warrior and a survivor too.

This post was written and submitted by an anonymous writer. The article reflects the views of the writer and not of CURE®. This is also not supposed to be intended as medical advice.

My cancer diagnosis — and the emotional effects that came with it — made me who I am today. 

Cancer Helped Me Choose Kindness

Treatment with eflornithine plus lomustine improved survival in patients with grade 3 IDH-mutant astrocytoma, a type of brain cancer. 

Eflornithine and lomustine improved survival compared with lomustine alone in patients with grade 3 IDH-mutant astrocytoma whose disease progressed after radiation and adjuvant temozolomide treatment, according to a press release from Orbus Therapeutics, the manufacturer of eflornithine.

Findings from this subpopulation of the STELLAR study were also recently presented at the Society for Neuro-Oncology Annual Meeting.

According to the National Cancer Institute, astrocytoma is a type of glioma (or 

) that forms in star-shaped cells — astrocytes — in the brain and spinal cord. Although they can be either noncancerous or cancerous, those considered grade 3 are malignant, meaning they can grow and spread quickly. An IDH mutation can make tumor cells grow more quickly and aggressively.

Adjuvant: additional treatment given after the primary treatment to reduce the risk of cancer recurrence.

Progression-free survival (PFS): the time during and after treatment when a patient with cancer lives with the disease without worsening.

Overall survival (OS): the time when a patient with cancer is still alive.

Myelosuppression: a condition when bone marrow activity is decreased, resulting in fewer white blood cells, red blood cells and platelets.

“For patients with recurrent grade 3 astrocytoma, a rare and fatal disease, there are very few available treatment options,” Bob Myers, Chief Executive Officer of Orbus, said in the release. “The last FDA approval of a chemotherapeutic agent for these patients was 25 years ago.”

In the STELLAR study, researchers compared the safety and efficacy of eflornithine plus lomustine with lomustine alone in patients with anaplastic astrocytoma whose disease recurred after radiation, surgery and adjuvant temozolomide chemotherapy, according to the release. In particular, researchers enrolled 343 patients, of whom 194 had grade 3 IDH-mutant astrocytoma.

The survival improvements with the combination treatment were clinically more meaningful in patients with IDH-mutant astrocytoma than with the overall study population, according to the release. For all patients in the STELLAR study, 343 patients 

 and lomustine were 94% as likely to die as those who received the standard treatment with lomustine.

In contrast, in the 194 patients with recurrent grade 3 IDH-mutant astrocytoma, eflornithine plus lomustine contributed to clinically meaningful improvements in progression-free survival (PFS) and overall survival (OS). The median OS was 34.9 months in patients treated with eflornithine plus lomustine compared with 23.5 months in those treated with lomustine alone. Regarding PFS, the median was 15.8 months in the combination group compared with 7.2 months in the lomustine-alone group.

“The subset results for the patients with recurrent grade 3 astrocytoma in the STELLAR study represent the first positive outcome to show significant increases in overall survival and progression-free survival in a very difficult-to-treat form of malignant glioma,” Dr. Howard Colman, the Jon M. Huntsman Presidential Chair in Neuro-Oncology at the Huntsman Cancer Institute at the University of Utah in Salt Lake City, said in the release. “These findings from the STELLAR study are very impressive and provide caregivers and patients with hope for a new treatment option for this fatal disease.”

The release also noted that treatment with eflornithine plus lomustine was well-tolerated, with no unexpected safety signals observed throughout the trial. The most common grade 3 (severe) or worse side effects from treatment were related to hearing impairment and myelosuppression. The patients with grade 3 or worse blood-related side effects or organ function-related side effects were to the extent of what researchers expected, the release explained.

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Eflornithine combined with lomustine significantly improved survival outcomes in patients with recurrent grade 3 IDH-mutant astrocytoma compared to lomustine alone. This combination therapy demonstrated clinically meaningful improvements in both progression-free survival and overall survival, with median overall survival extending to 34.9 months versus 23.5 months for lomustine alone. The STELLAR study's findings mark a significant advancement in treating this aggressive and rare form of malignant glioma, offering new hope for patients. The treatment was well-tolerated, with expected side effects primarily involving hearing impairment and myelosuppression.

Survival improved in patients with recurrent grade 3 IDH-mutant astrocytoma who were treated with eflornithine and lomustine.

Combination Treatment May Improve Survival in Recurrent Brain Cancer Subtype

The Food and Drug Administration (FDA) granted a breakthrough therapy designation to Enhertu (trastuzumab deruxtecan) for the treatment of certain patients with unresectable or metastatic breast cancer.

The breakthrough therapy designation, which will speed up the development and FDA’s review of the drug, is for adults who have unresectable or metastatic HER2-low breast cancer who underwent prior systemic therapy for metastatic disease or who experience recurrence during or within six months of finishing adjuvant (post-surgical) chemotherapy. If patients have hormone receptor- (HR) positive disease, they need to have received or be ineligible for endocrine therapy to be treated with Enhertu.

The FDA based its decision on findings from the global, randomized 

 which compared Enhertu with physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive, HR-negative and HER2-low unresectable and/or metastatic breast cancer who had previously undergone one or two prior lines of chemotherapy.

The main goal of DESTINY-Breast04 was to see if Enhertu bested chemotherapy when it came to progression-free survival (time from treatment until disease worsens) in patients with HR-positive, HER2-low metastatic breast cancer. The secondary goal was to see if progression-free survival could be improved in HER2-low metastatic breast cancer, regardless of HR status, as well as overall survival.

“Historically, only patients with HER2-positive metastatic breast cancer were shown to benefit from HER2-directed therapy. DESTINY-Breast04, in which Enhertu showed a clinically meaningful survival benefit in patients with HER2-low metastatic breast cancer, is the first trial to demonstrate that selecting patients for treatment based on low expression of HER2 has the potential to change the diagnostic and treatment paradigms for these patients,” said Ken Takeshita, global head, R&D, Daiichi Sankyo, one of the pharmaceutical companies that is developing Enhertu, along with AstraZeneca, in a press release.

The release cited the positive high-level results seen from DESTINY-Breast04, which showed that Enhertu led to a meaningful improvement in progression-free survival and overall survival in patients with HER2-low unresectable and/or metastatic breast cancer in all randomized patients with HR-positive and HR-negative disease. Additionally, there were no new side effects that were observed from the drug.

More detailed data from DESTINY-Breast04 will be presented at an upcoming medical meeting, according to the release.

Chemotherapy is the only treatment that is currently available for patients with HR-positive disease who progressed on hormone therapy, and there are few targeted treatments available for those with HR-negative breast cancer, highlighting the unmet need that Enhertu could potentially fill.

“Enhertu continues to show transformative potential, and this milestone represents an important advance for patients with HER2-low metastatic breast cancer who are in urgent need of new treatment options and better outcomes,” Susan Galbraith, executive vice president, Oncology R&D, AstraZeneca said in the release.

After Enhertu showed promising clinical trial results, the Food and Drug Administration agreed to speed up the development and review of the drug for certain patients with unresectable or metastatic breast cancer. 

Enhertu Granted Breakthrough Therapy Designation for Unresectable/Metastatic Breast Cancer Treatment

The Food and Drug Administration (FDA) has granted priority review to an application seeking approval for futibatinib for the treatment of previously treated locally advanced or metastatic cholangiocarcinoma, a cancer of the bile ducts of the liver, that harbors FGFR2 genetic mutations, according to a press release.

Of those diagnosed with cholangiocarcinoma, according to the release, 10% to 16% of have tumors that express this particular genetic mutation.

“Given the lack of an accepted standard chemotherapy following the failure of first-line treatment, futibatinib could represent a significant opportunity for a targeted therapy in this subset of patients with (cholangiocarcinoma), which has driven our pursuit with this investigational compound,” Volker Wacheck, vice president of Clinical Development at Taiho Oncology, said in the release. “We look forward to working with the FDA as they consider the application for futibatinib under priority review.”

The FDA grants drugs priority review that, if approved, would lead to significantly improved outcomes compared to the standard-of-care treatment options. By granting a treatment a priority review, the FDA agrees to speed up the review of the agent. The agency plans on deciding on whether to approve the drug by the end of September.

The filing for the expedited approval is based on findings from the phase 2b FOENIX-CCA2, which demonstrated that among 103 patients, treatment with futibatinib induced an objective response rate (the percentage of patients whose disease decreased or disappeared) of 41.7%. The results also showed that the median duration of response in patients wo received the orally delivered drug was 9.7 months. Of note, 72% of the treatment responses lasted for at least six months.

Some of the most common side effects associated with treatment included abnormal levels of phosphorous in the blood (85%), which could be a sign of kidney damage, as well as alopecia (33%) and dry mouth (30%).

Migraine was the only side effect deemed to be serious in nature that was reported in more than one patient.

The FDA plans to decide on whether to approve futibatinib for the treatment of patients with previously treated, locally advanced or metastatic cholangiocarcinoma that harbors a certain genetic mutation by September.

FDA to Speed Up Review of Futibatinib to Treat Certain Patients With Cholangiocarcinoma

Adcetris (brentuximab vedotin) with four cycles of combination chemotherapy may improve complete response rates and two-year survival in patients with early-stage, unfavorable-risk Hodgkin lymphoma, according to a study published in the 

This treatment approach may lead to a reduction or elimination of radiotherapy specifically for patients who are positron emission tomography (PET)-negative, meaning that there is no evidence of malignant disease.

“Given the limited number of patients per cohort and the nonrandomized design, the current pilot study was not designed to definitively compare the four treatment arms,” the study authors wrote. “However, the outcomes establish (four cycles of Adcetris and doxorubicin, vinblastine and dacarbazine) as a highly active and well-tolerated regimen.”

Researchers aimed to assess whether a short course of Adcetris with doxorubicin, vinblastine and dacarbazine chemotherapy can allow the safe reduction or elimination of consolidative radiation in patients with early-stage Hodgkin lymphoma. To do so, 117 patients (median age, 32 years; 50% men) ages 18 to 60 years with early-stage Hodgkin lymphoma were treated with four cycles of Adcetris and chemotherapy including doxorubicin, vinblastine and dacarbazine. Researchers assembled four different groups with different unfavorable risk factors measurements to determine bulky disease. These groups received varying doses of radiotherapy:

Cohort 1 (30 patients): 30-Gy involved-site radiotherapy,

Cohort 2 (29 patients): 20-Gy involved-site radiotherapy,

Cohort 3 (29 patients): 30-Gy consolidation-volume radiotherapy and

Several factors were assessed throughout the study including safety in Cohort 1 and complete response rates according to PET scans for the remaining cohorts. Follow-up was conducted for a median of 3.8 years.

The complete response rate was 93% in the group that received 30-Gy involved-site radiotherapy, 100% in those that received 20-Gy involved-site radiotherapy, 93% in patients who received 30-Gy consolidation-volume radiotherapy and 97% in those who did not received radiotherapy.

Overall progression-free survival (the time a patient lives with cancer without disease worsening) at two years for the entire patient population was 94% with overall survival (the time a patient with cancer is still alive) of 99%. At two years, progression-free survival was 93% in patients treated with 30-Gy involved-site radiotherapy, 97% in those treated with 20-Gy involved-site radiotherapy, 90% in patients treated with 30-Gy consolidation-volume radiotherapy and 97% in those who did not receive radiotherapy.

Patients experienced side effects including neutropenia (low number of white blood cells called neutrophils in the blood; 44%), febrile neutropenia (fever with neutropenia; 8%) and peripheral neuropathy (pain, numbness and weakness in the hands and feet; 54%). Side effects were largely reversible.

“The encouraging outcomes associated with short-course (Adcetris and doxorubicin, vinblastine and dacarbazine) chemotherapy alone in this study provided support for the UK RADAR study, an ongoing randomized clinical trial comparing (doxorubicin, bleomycin, vinblastine, dacarbazine) and (Adcetris and doxorubicin, vinblastine and dacarbazine) for (early-stage Hodgkin lymphoma),” the study authors wrote. “In (the) future, other novel agents including checkpoint inhibitors will be studied to enable omission of (radiotherapy) and reduce long-term toxicity in bulky (early-stage Hodgkin lymphoma).”

For patients with early-stage, unfavorable-risk Hodgkin lymphoma, this treatment option may allow for the reduction or elimination of radiotherapy in patients without malignant disease.

Adcetris Plus Combo Chemotherapy May Impact Survival and Response to Treatment in Subset of Patients With Hodgkin Lymphoma

A dose reduction of Xpovio (selinexor), when given in combination with Velcade (bortezomib) and dexamethasone, led to promising outcomes in patients with multiple myeloma, highlighting the importance of decreasing doses (when appropriate) to optimize patient outcomes.

The findings came from the BOSTON trial, and will be presented at the 

2021 American Society of Hematology Annual Meeting

. In an interview with CURE®, Dr. Sundar Jagannath, Director of the Center of Excellence for Multiple Myeloma and a professor of medicine at The Tisch Cancer Institute in New York, explained why reducing the dose of a drug doesn’t mean patients are getting less benefit.

“Because you adjust the dose of the drug, the efficacy doesn't go down,” Jagannath said. “And because now it is tolerable, actually, the patients stay on the drug longer. And because they stay on the drug longer, they are able to control the disease much longer and more deeper responses are achieved, because they are able to stay on the drug longer. And therefore, the duration of response is better.”

What we were surprised to find (was) that the people who got dose reduced because of side effects, as well as the patients who didn't get dose reduced because they didn't experience side effects, there was not a big difference in terms of age, sex of the patient, or type of disease or something like that. So, they were comparable.

The next question that comes up, “OK, so at least they were comparable. So how did the patients who got dose reduced do as compared to patients who didn't have dose reduction?”

(The) most important thing is, was there a reduction in the efficacy as you reduce the dose? And to our surprise, we found out that the patients who had dose reduction actually had a higher response rate; they had a response rate of 81.7%, as compared to 62%.

And not only that, (but) the duration of the response was longer. So that outcome was better — the outcome measured in terms of progression-free survival, or overall survival. So it was actually the progression free survival improved to 17 months compared to nine months.

So this is very important, that means you are able to manage the patient with this new drug in the combination to benefit the patient. You can adjust the dose of the drug, and because you adjust the dose of the drug, the efficacy doesn't go down. And because now it is tolerable, actually, the patients stay on the drug longer. And because they stay on the drug longer, they are able to control the disease much longer and more deeper responses are achieved, because they are able to stay on the drug longer. And therefore, the duration of response is better.

So what we found (were) surprises — dose adjustment actually benefited the patient. So the message (that) is going to be put out in the American Society of Hematology Meeting, is for hematologist oncologists, and to let them know because they are all physicians that yes, you can use this drug safely, you can dose reduce without worrying about efficacy going down. So that was the importance of this particular abstract.

In recent trial results, a dose reduction of Xpovio (selinexor) was associated with longer progression-free survival and duration of response while also reducing side effects in patients with multiple myeloma.

Dose Reduction of Xpovio Improves Safety and Optimizes Patient Outcomes in Myeloma

It’s been nearly a decade since Robyn Stacy-Humphries, a 58-year-old radiologist from Charlotte, North Carolina, first learned she had diffuse large B-cell lymphoma (DLBCL). Today, she’s in remission — a fact she attributes to her treatment with chimeric antigen receptor (CAR)-T cell therapy.

Through the years, Stacy-Humphries’ treatment has included everything from six cycles of R-CHOP chemo/biotherapy (cyclophosphamide, doxorubicin, vincristine, prednisone and Rituxan [rituximab]) — the standard of care for DLBCL — to R-ICE (Rituxan, ifosfamide, carboplatin and etoposide), intrathecal chemotherapy (chemotherapy injected into the spinal fluid), myeloablative chemotherapy, high-dose BEAM chemotherapy (carmustine, etoposide, cytarabine and melphalan), rescued with an autologous stem cell transplant (a transplant using her own stem cells), and head and neck radiation. Although she’d gone into remission prior to her stem cell transplant, the cancer returned.

In April 2016, nine months after her stem cell transplant, Stacy-Humphries’ DLBCL returned again. With no matched donor on the horizon for an allogeneic transplant (a transplant using donor stem cells) — the next standard treatment for her cancer — she knew she was running out of time.

That’s when she and her husband got to work, researching clinical trials for an emerging treatment known as CAR-T cell therapy. She landed a spot in the phase 2 JULIET trial of Kymriah (tisagenlecleucel), a CAR therapy made by Novartis, at The Ohio State University’s Comprehensive Cancer Center.

“My early-phase clinical trial was like jumping out of a plane and hoping the parachute opens,” Stacy-Humphries says.

“I was one of the first 200 people in the world to receive this treatment. And the trial I was in was the basis for the FDA approval of the CAR-T cell product.”

Four years after receiving CAR-T cell therapy, she remains in remission and optimistic. “I’m likely to stay in complete remission,” she says. “But no one will actually give you a guarantee.”

CAR-T cell therapy is an immunotherapy, a type of therapy that helps a patient’s own immune system fight their cancer. Doctors draw blood from the person with cancer. The person’s T cells, white blood cells that develop from stem cells and help fight infection, are then separated from the blood in a laboratory that makes the CAR-T cell product. There, scientists change the genetic make-up of the T cells, making them produce customized receptors called chimeric antigen receptors that recognize specific proteins on tumor cells. These engineered T cells are then grown, or expanded, in the lab.

Once the CAR-T cell product has expanded, the person with cancer receives chemotherapy known as lymphodepleting treatment. This treatment clears the way in the body for an infusion of the re-engineered T cells, which multiply further in the patient’s body. The CARs then help the person’s T cells find a certain protein, known as an antigen, on the surface of cancer cells and destroy the cancer.

In 2017, the Food and Drug Administration (FDA) approved two types of CAR-T cell therapies. Kymriah was approved for patients up to age 25 with B-cell precursor acute lymphoblastic leukemia (ALL) who relapsed after two or more treatments, while Yescarta (axicabtagene ciloleucel) was approved for adult patients with large B-cell lymphoma who have relapsed after, or not responded to, two types of treatment.

In 2018, the FDA also approved Kymriah for adults with large B-cell lymphoma, including DLBCL, who have relapsed after or not responded to two or more standard therapies. In July 2020, the FDA approved Tecartus (brexucabtagene autoleucel) for adults with mantle cell lymphoma who have not responded to other treatments or relapsed after those treatments.

According to Dr. Frederick Locke, a medical oncologist and translational researcher in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center in Tampa, Florida, which was part of the clinical trial that supported the approval of Tecartus, having CAR-T cell therapy as an option for patients with mantle cell lymphoma is critically important.

“We have therapies for relapsed refractory mantle cell lymphoma that can work, but when patients relapse after getting those treatments, the disease can progress very rapidly,” he says.

CAR-T cell therapy offers hope for some patients with certain blood cancers who have not responded to conventional therapies, Locke adds.

“When we look at outcomes in patients with refractory diffuse large B-cell lymphoma, we know from robust historical data that patients who didn’t respond to their last line of chemotherapy or relapsed after an autologous stem cell transplant have a very low chance of responding to more therapy — conventional therapy, that is,” he says, adding that about 1 in 4 patients can expect to respond to another line of chemotherapy and less than 1 in 10 can expect their lymphoma to disappear after additional conventional therapy.

“These are the same patients who are now eligible for CAR-T cell therapy and the results are remarkable,” he says.

Locke adds that 30% to 40% of patients with DLBCL who have CAR-T cell therapy achieve durable remission that stretches beyond three years and counting. “These are results we would not expect and would not have seen with existing therapies for refractory diffuse large B-cell lymphoma,” he says, noting that data from randomized controlled clinical trials on the long-term impact of CAR-T cell therapy are still needed.

CAR-T cell therapy is complicated; it’s also potentially expensive for those who qualify for treatment. In a July 2020 comment on the Centers for Medicare & Medicaid Services (CMS) Hospital Inpatient Prospective Payment System proposed rule, the American Society of Clinical Oncology noted the average cost of a CAR-T cell product at $373,000, with the estimated cost of CAR-T cell therapy and related services at $419,238.

Medicare covers a portion of CAR-T cell therapy provided in health care facilities that are enrolled in FDA risk evaluation and mitigation strategies (REMS), a federal program that oversees the safe use of drugs, for FDA-approved uses. Medicare may also cover a portion

of some off-label uses of CAR-T cell therapy that meet certain recommendations. While private health insurance plans may cover some or all of the cost of the product for patients who qualify for FDA-approved treatment, there are additional costs related to CAR-T cell therapy for which patients might bear a heavy financial burden.

These additional costs include a pre-therapy consult, chemotherapy to deplete the lymphocytes, and collection procedures, as well as expenses during recovery and ongoing monitoring according to Dr. Stuart Goldberg, chief of the Division of Outcomes and Value Research at the John Theurer Cancer Center at Hackensack University Medical Center in Hackensack, New Jersey.

“It also involves doctor visits, two weeks in the hospital, post-procedure care, all of the X-rays, CT scans, etc.,” he says. “None of that is the $400,000 cost of the CAR T. And CAR-T cells aren’t available in your backyard. So, you’re talking about hotel rooms, transportation, time off work for loved ones and caregivers. There are other costs that sneak in that no one talks about.”

“Everybody thinks that cancer patients are most worried about pain,” he says. “It turns out that more cancer patients are worried about the cost of care. When patients come in to see us, we ask about side effects like fever, pain and nausea. But most times when we see a patient, we rarely talk to them about whether they can pay the bills.”

According to Goldberg, patients who go through expensive treatments such as bone marrow transplant or CAR-T cell therapy talk to a financial counselor as part of the consult. “They map out what the out-of-pocket costs might be, and what costs their insurance might cover,” he says. “If we identify someone who we think may have some difficulty, we start to plug them in to various support programs that might be available. But even then, we cannot anticipate all the costs of travel, hotels, lost wages and other non-medical expenses that could burden the patient and their family.”

Stacy-Humphries says it’s important to keep costs — and outcomes — in perspective when evaluating treatment options. While much of her CAR-T cell cost was covered as part of her clinical trial, she had to pay out-of-pocket costs for all of the tests required to get into the trial before she met her insurance’s high deductible, she says. “But those were the same tests that would be required to have an allogeneic transplant,” she says, noting that her own research puts the cost of an allogeneic transplant, over time, at about $800,000. “And it’s not like chemotherapy is free. It’s very expensive.”

Dr. David Siegel, chief of the Myeloma Division at the John Theurer Cancer Center at Hackensack University Medical Center, says the standard of care for multiple myeloma, a four-drug combination given to newly diagnosed patients with the intention of administering it indefinitely in the absence of progression, is incredibly expensive.

“The cost of these drugs is tens of thousands of dollars a month,” he says. “If you’re a lucky patient and you stay in remission for three or four years, which is certainly not out of the realm of what’s expected, it’s $300,000 a year to give these drugs.” The cost of CAR-T cell therapy, which is not yet FDA approved for myeloma treatment, seems relatively inexpensive by comparison if a patient then doesn’t need to be treated for two years, he adds.

Treating potential side effects of CAR-T cell therapy may add additional costs. Some patients develop cytokine release syndrome, an immune system response that causes high fever and low blood pressure, or neurotoxicity, brain impacts such as seizures or confusion. Although these medical toxicities can usually be controlled, the additional treatments may add to the “financial toxicity.”

Some nonprofit organizations offer assistance for eligible patients — or can help connect patients to programs that help with paying for care, travel and lodging. The Leukemia & Lymphoma Society (lls.org) offers a co-pay assistance program for eligible patients, while CancerCare (cancercare.org) offers travel and other assistance for eligible patients.

Pharmaceutical companies may also offer patient assistance programs; for example, Novartis offers Kymriah Cares. According to Julie Masow, global head of Novartis Oncology External Communications in Cambridge, Massachusetts, assistance and support for eligible patients may include benefit and network investigation, co-pay assistance and travel support. Patients seeking help with private insurance to help cover their portion of financial responsibility for Kymriah should speak to their doctor or treatment center about eligibility and submitting a request for co-pay assistance.

Patients participating in clinical trials find that many CAR-T cell therapy expenses are covered. Chrissy Degennaro, 50, of Ocean City, New Jersey, was 36 when she received a multiple myeloma diagnosis. She has participated in two CAR-T cell clinical trials — one in 2017 and one in 2018 — with Celgene, now a Bristol Myers Squibb company.

During the first trial, when her engineered T cells were infused, Degennaro spent two weeks in the hospital, followed by two weeks in a hotel nearby. A bone marrow biopsy two weeks after completing treatment showed no signs of myeloma, she says.

Her treatment and hotel costs were covered during the trial, she says.

“Participating in the study made it affordable,” Degennaro says. “It wasn’t the financial burden on me that it could have been.”

A year later, her myeloma returned, so she had a second CAR-T cell treatment and was in remission for a few months. Her myeloma has since returned.

“To have even six months off treatment is a treat,” Degennaro says, adding that today, she takes a combination of selinexor, Velcade (bortezomib) and dexamethasone. “Some people say CAR T is not worth it. It was totally worth it for me. It gave me all that time off treatment.”

Weighing the cost of CAR-T cell therapy in treating blood cancers is a finical burden for many patients with blood cancer. 

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