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Cancer research highlights from 2023

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By Mayo Clinic staff

Researchers at Mayo Clinic Comprehensive Cancer Center spent 2023 studying the biology of cancer and new ways to predict, prevent, diagnose and treat the disease. Their discoveries are creating hope and transforming the quality of life for people with cancer today and in the future. Here are some highlights from their research over the past year:

Mayo Clinic researchers link ovarian cancer to bacteria colonization in the microbiome.

A specific colonization of microbes in the reproductive tract is commonly found in people with ovarian cancer, according to a study from the Mayo Clinic  Center for Individualized Medicine . Published in  Scientific Reports  and led by  Marina Walther-Antonio, Ph.D. , a Mayo Clinic researcher, and Abigail Asangba, Ph.D., the discovery strengthens the evidence that the bacterial component of the microbiome — a community of microorganisms that also consists of viruses, yeasts and fungi — is an important indicator for early detection, diagnosis and prognosis of ovarian cancer . The study also suggests that a higher accumulation of pathogenic microbes plays a role in treatment outcomes and could be a potential indicator for predicting a patient's prognosis and response to therapy.  Read more .

Artificial intelligence is forging a new future for colorectal cancer and other digestive system diseases.

Colonoscopy remains the gold standard in detecting and preventing colorectal cancer , but the procedure has limitations. Some studies suggest that more than half of post-colonoscopy colon cancer cases arise from lesions missed at patients' previous colonoscopies. In 2022, Michael Wallace, M.D. , a Mayo Clinic gastroenterologist, published the results  of an international, multicenter study testing the impact of adding artificial intelligence (AI) to routine colonoscopies. His team, including James East, M.D. , a Mayo Clinic gastroenterologist, and other researchers from the U.S., the U.K., Italy, Germany and Ireland, found that incorporating AI into colonoscopies reduced the risk of missing polyps by 50%.  Read more .

A big step forward: Bringing DNA sequencing data to routine patient care.

The Tapestry study , an extensive genomic sequencing clinical research study, aims to complete exome sequencing (sequencing the protein-coding regions of a genome) for 100,000 Mayo Clinic patients. The results will be integrated into patients’ electronic health records for three hereditary conditions, and the amassed data will contribute to a research dataset stored within the Mayo Clinic Cloud on the Omics Data Platform. The overall hope of Tapestry is to accelerate discoveries in individualized medicine to tailor prevention, diagnosis and treatment to a patient's unique genetic makeup. It is poised to advance evidence that exome sequencing, when applied to a diverse and comprehensive general population, can proficiently identify carriers of genetic variants that put them at higher risk for a disease, allowing them to take preventive measures.  Read more .

Patients with multiple tumors in one breast may not need a mastectomy.

Patients who have multiple tumors in one breast may be able to avoid a mastectomy if surgeons can remove the tumors while leaving enough breast tissue, according to research led by the  Alliance in Clinical Trials in Oncology  and  Mayo Clinic Comprehensive Cancer Center . Patients would receive breast-conserving therapy — a  lumpectomy  followed by whole-breast  radiation therapy — rather than mastectomy . The study is published in the  Journal of Clinical Oncology . Historically, women with multiple tumors in one breast have been advised to have a mastectomy. Now, patients can be offered a less invasive option with faster recovery, resulting in better patient satisfaction and cosmetic outcomes, says  Judy Boughey, M.D. , lead author, Mayo Clinic breast surgical oncologist and the W.H. Odell Professor of Individualized Medicine. Read more .

Staging pancreatic cancer early with minimally invasive surgery shows positive results in patient prognosis.

A study published in the  Journal of the American College of Surgeons  reveals that performing a minor surgical procedure on patients newly diagnosed with  pancreatic cancer  helps to identify cancer spread early and determine the stage of cancer. The researchers add that the surgery ideally should be performed before the patient begins chemotherapy. "This is an important study because it supports that staging laparoscopy may help determine a patient's prognosis and better inform treatment so that patients avoid unhelpful or potentially harmful surgical therapy," says  Mark Truty, M.D. , a Mayo Clinic surgical oncologist who led the research.  Read more .

Mayo Clinic study reveals proton beam therapy may shorten breast cancer treatment.

In a trial published in  The Lancet Oncology , Mayo Clinic Comprehensive Cancer Center researchers uncovered evidence supporting a shorter treatment time for people with breast cancer . The study compared two separate dosing schedules of pencil-beam scanning proton therapy , known for its precision in targeting cancer cells while preserving healthy tissue to reduce the risk of side effects. The investigators found that both 25-day and 15-day proton therapy schedules resulted in excellent cancer control while sparing surrounding non-cancerous tissue. Further, complication rates were comparable between the two study groups. "We can now consider the option of 15 days of therapy for patients based on the similar treatment outcomes observed," says  Robert Mutter, M.D. , a Mayo Clinic radiation oncologist and physician-scientist. Read more .

Harnessing the immune system to fight ovarian cancer.

Mayo Clinic research is biomanufacturing an experimental, cell-based ovarian cancer vaccine and combining it with immunotherapy to study a "one-two punch" approach to halting ovarian cancer progression. This research begins with a blood draw from people with advanced  ovarian cancer  whose tumors have returned after standard surgery and chemotherapy. White blood cells are extracted from the blood, biomanufactured to become dendritic cells and returned to the patient. Dendritic cells act as crusaders that march through the body, triggering the immune system to recognize and fight cancer. "We're building on an earlier phase 1 clinical trial  that showed promising results  in terms of survival after the dendritic cell-based vaccine," says  Matthew Block, M.D., Ph.D. , co-principal investigator and Mayo Clinic medical oncologist. "Of the 18 evaluable patients in the phase 1 study, 11 had cancer return, but seven of them — 40% — have been cancer-free for almost 10 years. We typically expect 90% of patients in this condition to have the cancer return."  Read more .

New gene markers detect Lynch syndrome-associated colorectal cancer.

Researchers from Mayo Clinic Comprehensive Cancer Center and Mayo Clinic Center for Individualized Medicine have discovered new genetic markers to identify Lynch syndrome-associated colorectal cancer with high accuracy. Studies are underway to determine if these genetic markers are in stool samples and, if so, how this could lead to a non-invasive screening option for people with  Lynch syndrome . The research was published in Cancer Prevention Research , a journal of the American Association for Cancer Research. "This is an exciting finding that brings us closer to the reality that clinicians may soon be able to offer a non-invasive cancer screening option to patients with the highest risk of getting cancer," says  Jewel Samadder, M.D. , co-lead author of the paper and a Mayo Clinic gastroenterologist. Read more .

Mayo Clinic prepares to biomanufacture a new CAR-T cell therapy for B-cell blood cancers.

Mayo Clinic research has developed a new type of  chimeric antigen receptor-T cell therapy (CAR-T cell therapy)  aimed at killing B-cell blood cancers that have returned and are no longer responding to treatment. This pioneering technology, designed and developed in the lab of  Hong Qin, M.D., Ph.D. , a Mayo Clinic cancer researcher, killed B-cell tumors grown in the laboratory and tumors implanted in mouse models. The preclinical findings are published in  Cancer Immunology, Immunotherapy . "This study shows our experimental CAR-T cell therapy targets several blood cancers, specifically chronic lymphocytic leukemia," says Dr. Qin. "Currently, there are six different CAR-T cell therapies approved for treatment of relapsed blood cancers. While the results are impressive, not everyone responds to this treatment. Our goal is to provide novel cell therapies shaped to each patient's individual need."  Read more .

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Top Cancer Treatment Advances at MSK in 2023

By Julie Grisham Monday, December 18, 2023

Alicia Kalogeropoulos, shown here with her husband, Alex, was diagnosed with low-grade glioma at age 27. Her team at MSK gave her the opportunity to participate in a clinical trial of the experimental drug vorasidenib. The drug halted the growth of her tumor with no side effects.

Doctors at Memorial Sloan Kettering Cancer Center (MSK) pioneered advances in a variety of cancer treatment for patients in 2023. The results from many cutting-edge clinical trials this year are leading to promising new treatments for brain cancer, colorectal cancer, bladder cancer, endometrial cancer, and several different blood cancers.

In addition, the Food and Drug Administration (FDA) approved seven drugs in 2023 based on clinical trials in which MSK played a pivotal role.

“We are living in a transformative age for cancer care, where advances in the lab are moving into the clinic faster than ever before,” says MSK President and CEO Selwyn M. Vickers, MD, FACS . “Across MSK, our doctors, scientists, and other staff are focused on developing precision therapies that are effective against cancer while preserving patients’ quality of life during and after cancer treatment.”

Here are some of the most important developments of 2023, listed in chronological order:

Off-the-Shelf CAR T Cell Therapy for Multiple Myeloma Shows Promise 

One of the most advanced treatments for multiple myeloma is CAR T cell immunotherapy , but it can take a month or more to engineer customized immune cells for an individual patient. Now a new approach using donor cells brings the hope of giving off-the-shelf CAR T cell immunotherapy to patients in as little as five days. In a phase 1 clinical trial led by MSK multiple myeloma specialist Sham Mailankody, MBBS , patients were given CAR T cells already banked from other donors, as opposed to their own cells. The results, published in the January 23, 2023, issue of Nature Medicine , demonstrate that this type of CAR T cell donation can be safe and effective.

Testing CRISPR-Edited CAR T Cell Therapy in Lymphoma Clinical Trial

To make CAR T cell therapy even more precise and powerful, a clinical trial was launched at MSK testing CAR T cells that were made using a genome-editing tool called CRISPR/Cas9 . This latest advance was made possible by the work of Michel Sadelain, MD, PhD , Director of the Center for Cell Engineering and the Gene Transfer and Gene Expression Laboratory at MSK, in collaboration with MSK’s Cell Therapy and Cell Engineering Facility . Investigators believe the phase 1 clinical trial, led by hematologic oncologist Jae Park, MD , has the potential to change the future of immunotherapy .

Menin Inhibitors: Promising New Drug Treatment for Aggressive Leukemia

Revumenib, a drug in a new class called menin inhibitors, was developed based on research conducted at MSK. It targets certain genetic changes that are commonly found in acute myeloid leukemia  and acute lymphocytic leukemia . On March 15, 2023, MSK leukemia specialist and early drug development specialist Eytan Stein, MD , published results from the first-ever clinical trial of revumenib in Nature . More than half of patients with these molecular changes responded to the drug. Based on earlier data presented from this trial, the FDA had already granted revumenib Breakthrough Therapy Designation — indicating its potential over existing therapies to improve outcomes for patients.

For Advanced Endometrial Cancer, Chemotherapy Plus Immunotherapy Improves Outcomes

Endometrial cancer , which makes up about 90% of uterine cancers, is the fourth most common cancer in women and is one of the few cancers that is increasing in incidence and mortality. But until now, very few treatments have been developed specifically for endometrial cancer. Research published March 27, 2023, in The New England Journal of Medicine found that adding the immunotherapy drug pembrolizumab (Keytruda®) to standard chemotherapy greatly improves outcomes, whether or not patients had a genetic marker suggesting they were likely to respond to immunotherapy. The trial, which was also presented at the Society of Gynecologic Oncology’s annual meeting, was overseen by MSK physician-scientist Carol Aghajanian, MD .  

For Advanced, HER2-Amplified Bile Duct Cancers, Antibody Treatment Trial Shows Promising Results

Bile duct cancers are rare, aggressive types of gastrointestinal cancer that are difficult to treat. Research published June 2 in The Lancet Oncology found an antibody treatment called zanidatamab helped shrink tumors in some patients with bile duct cancers — specifically, a subset of people whose tumors make a high amount of the HER2 protein, which can cause cells to multiply too quickly. The phase 2b clinical trial, which also was presented at the American Society of Clinical Oncology’s annual meeting, was co-led by MSK gastrointestinal oncologist James Harding, MD .

Rectal Cancer Treatment Without Radiation: A New Option

A large multicenter clinical trial presented at the American Society of Clinical Oncology’s annual meeting studied treating locally advanced rectal cancer without radiation . Researchers found that after five years, people who received a type of chemotherapy called FOLFOX before surgery did just as well as those who underwent both chemotherapy and radiation before surgery. Skipping radiation could mean improvements in quality of life, including the preservation of fertility , sexual function, and more. The study, which was also published in The New England Journal of Medicine on June 4, 2023, was led by gastrointestinal oncologist Deb Schrag, MD, MPH , Chair of MSK’s Department of Medicine .

Experimental Drug for Low-Grade Glioma With IDH Gene Mutation Shows Promise

Brain tumors are among the deadliest and most difficult cancers to treat. But a new experimental drug called vorasidenib has been shown to slow the growth of low-grade diffuse gliomas with a certain gene mutation called IDH. A phase 3 clinical trial of people with low-grade gliomas containing the mutation found that the drug significantly slowed tumor growth — more than doubling the time before the cancer began to progress compared with a placebo. Results from a clinical trial demonstrating the drug’s potential were presented at the American Society of Clinical Oncology’s annual meeting and published in The New England Journal of Medicine on June 4, 2023. Ingo Mellinghoff, MD, FACP , Chair of MSK’s Department of Neurology , led the research.

Preventing Lynch Syndrome Cancers: New Study Suggests Immunotherapy Could Work

An analysis of patients with an inherited condition called Lynch syndrome found that immunotherapy drugs called checkpoint inhibitors may stop serious tumors from forming, suggesting a possible approach for preventing cancer. Lynch syndrome results from a DNA mismatch repair deficiency , which is caused by a gene mutation that prevents cells from repairing genetic damage. The study found that people who received an immunotherapy drug for tumors due to Lynch syndrome had fewer additional tumors in their internal organs than did patients treated with chemotherapy. The corresponding author of the paper, published October 16, 2023, in Nature Medicine , was MSK gastrointestinal oncologist and clinical geneticist Zsofia Stadler, MD .

New mRNA Pancreatic Cancer Vaccine Trial Starts Next Phase

An experimental approach to treating pancreatic cancer — an mRNA vaccine — has progressed to a phase 2 clinical trial . The new trial follows a phase 1 trial involving 16 MSK patients, reported May 10, 2023, in Nature , which found that patients who mounted an immune response to the vaccine had longer recurrence-free survival. The new study will investigate whether the therapeutic vaccine reduces the risk of pancreatic cancer returning after the tumor is removed by surgery . It will enroll approximately 260 patients at MSK and other sites around the world and is being led by MSK pancreatic cancer surgeon-scientist Vinod Balachandran, MD .

The FDA Approves 7 Drugs in 2023 Based on Significant Contributions From MSK Investigators

These new therapies treat cancers of the breast, urinary system, blood, lung, and soft tissues.

• For certain people with advanced or metastatic breast cancer (stages 3 and 4), the FDA approved the drug therapy elacestrant (Orserdu™) on January 27. Elacestrant was effective in patients whose tumors were ER-positive , HER2-negative ; had continued to grow after hormone therapy ; and had a mutation in a gene called ESR1 . The approval capped a decade of pivotal research led by MSK breast cancer specialist Sarat Chandarlapaty, MD, PhD . 
• For people with metastatic urothelial carcinoma — a cancer that primarily arises in the bladder and also occurs in other parts of the urinary system — the FDA approved the drug enfortumab vedotin (Padcev®) plus the immunotherapy drug pembrolizumab (Keytruda®) for two different patient groups on April 3 and December 15 . This greatly expands the number of people with bladder cancer who could benefit from these treatments. MSK genitourinary oncologist and bladder cancer specialist Jonathan Rosenberg, MD , led multiple clinical trials testing enfortumab vedotin alone or in combination with other drugs.
• For adults with previously untreated diffuse large B cell lymphoma , the FDA approved the drug polatuzumab vedotin (Polivy®) in combination with rituximab , cyclophosphamide , doxorubicin hydrochloride, and prednisone as a first-line therapy for adults with previously untreated diffuse large B cell lymphoma. This was the first new drug approval in this clinical setting in 20 years. It was based on the results of a study conducted in the United States and Europe. MSK’s Lymphoma Service , led by lymphoma specialist and cellular therapist Gilles Salles, MD, PhD , treated the most U.S. patients in the trial.
• For resistant forms of multiple myeloma , a rare blood cancer that can be difficult to treat, the FDA granted accelerated approval to elranatamab (Elrexfio™) on August 14. Elranatamab is a type of drug known as a bispecific antibody. It targets a protein on myeloma cells called BCMA, as well as a protein on the immune system’s T cells called CD3. MSK myeloma specialist and cellular therapist Alexander M. Lesokhin, MD, led the development and testing of this drug.
• For people with metastatic non-small cell lung cancer with a BRAF V600E mutation, the FDA approved a combination of the targeted drugs encorafenib (Braftovi®) and binimetinib (Mektovi®) on October 11. The combination of the drugs, which are both taken as pills, was already approved for some people with melanoma . The new approval was based on a multicenter phase 2 clinical trial led by MSK thoracic oncologists Gregory Riely, MD, PhD , and Michael Offin, MD .
• For the treatment of locally advanced or metastatic non–small cell lung cancer caused by a mutation called a ROS1 fusion, the FDA granted approval to repotrectinib (Augtyro™) on November 15. The drug appears to be effective even in patients who have developed resistance to other ROS1 inhibitors. The approval was based on results from a phase 1/2 trial led by MSK thoracic oncologist and early drug development specialist Alexander Drilon, MD .
• For adults with desmoid tumors that cannot be treated with surgery alone, the FDA granted approval to the drug nirogacestat (Ogsiveo™) on November 27. The approval was based on research led by investigators at MSK, including sarcoma expert Mrinal Gounder, MD . Nirogacestat is the first targeted therapy ever to be approved for desmoid tumors (also known as aggressive fibromatosis) , a type of soft-tissue tumor.

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Cancer Research Catalyst The Official Blog of the American Association for Cancer Research

Home > Cancer Research Catalyst > FDA Approvals in Oncology: January-March 2023 

FDA Approvals in Oncology: January-March 2023 

With the approval of new anticancer therapeutics, more treatment options become available for patients. Some therapies are new to the market, while some may have already been approved for other indications; some molecules are first in class, directed against a previously untargeted pathway or acting through a new mechanism, while others may be improved versions of drugs that already exist.  

To help our readers keep track of the cancer therapies approved by the U.S. Food and Drug Administration (FDA), make sense of their impact for patients, and put them in context of the current therapeutic landscape, Cancer Research Catalyst is launching a new blog series that will present a quarterly review of the latest from the FDA.  

Approvals issued from January to March 2023 included targeted therapies for B-cell malignancies, colorectal cancer, and pediatric brain cancer; additional options for hormone receptor (HR)-positive breast cancers; a new immune checkpoint inhibitor; and expanded indications for previously approved immune checkpoint inhibitors. 

Expanding the portfolio of targeted therapies for B-cell malignancies 

The development of molecularly targeted therapies has led to dramatic improvements in the care of patients with B-cell malignancies. Among these developments are Bruton’s tyrosine kinase (BTK) inhibitors, which block the function of the BTK enzyme that is involved in the survival of malignant B cells. The first BTK inhibitor, ibrutinib (Imbruvica), was approved in 2013, and additional inhibitors have followed.  

Approvals in January 2023 included an expanded approval for a next-generation BTK inhibitor and an accelerated approval for a reversible BTK inhibitor.  

• The first approval of the year, on January 19, was for the BTK inhibitor zanubrutinib (Brukinsa) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). This drug was first approved in 2019 for the treatment of mantle cell lymphoma (MCL) and then in 2021 for marginal zone lymphoma, based, in part, on the results of a clinical trial published in the AACR journal Clinical Cancer Research .   Zanubrutinib is a next-generation BTK inhibitor designed to have higher efficacy and an improved safety profile compared to previous versions. A recent commentary in the AACR journal Cancer Discovery reviewed the clinical evidence that established the superiority of zanubrutinib to ibrutinib for the treatment of relapsed or refractory CLL and SLL. 
• On January 27, another BTK inhibitor, pirtobrutinib (Jaypirca), was granted accelerated approval for the treatment of adult patients with MCL that has progressed following previous lines of therapy, including other BTK inhibitors. Pirtobrutinib is new to the market and is the only FDA-approved reversible BTK inhibitor, which means that it does not form a permanent bond with the BTK enzyme. Irreversible BTK inhibitors can lead to treatment resistance due to mutations in the portion of BTK that binds the inhibitor, therefore they cannot be used successively to treat patients. Pirtobrutinib was designed to remain active in the presence of acquired mutations and can be used after disease progression following treatment with existing BTK inhibitors, extending the benefit of targeting this pathway.  

Targeting HER2 in colorectal cancer 

On January 19, the FDA granted accelerated approval to the drug tucatinib (Tukysa) in a new combination regimen for certain patients with unresectable or metastatic colorectal cancer that has progressed following chemotherapy, a patient population that typically has limited treatment options.  

Tucatinib is a small molecule inhibitor of the HER2 receptor, whose overexpression leads to tumor cell proliferation, invasion, and metastasis. The first report of the preclinical activity of tucatinib in solid tumor models was published in the AACR journal Molecular Cancer Therapeutics , and tucatinib was later approved as a breast cancer treatment for patients with HER2-positive tumors .   

HER2 overexpression is found in approximately 3 to 5% of metastatic colorectal cancer. The latest indication for tucatinib extends its use to patients with HER2-positive unresectable or metastatic colorectal cancer in combination with the HER2-targeted monoclonal antibody trastuzumab (Herceptin).  

More therapeutic options for patients with hormone receptor-positive breast cancer 

Approvals issued in this first quarter expanded the armamentarium of therapies for estrogen receptor (ER)-positive breast cancer, including a new hormone therapy and two targeted therapies.  

• Hormone therapy is used as a treatment for ER-positive breast cancer to inhibit estrogen production or block its effects on cancer cells. On January 27, elacestrant (Orserdu) was approved for certain patients with locally advanced or metastatic ER-positive, HER2-negative breast cancer.   Elacestrant belongs to a class of hormone therapy drugs called selective ER degraders (SERDs), which block ER signaling by causing degradation of the receptor. Prior to the approval of elacestrant, fulvestrant (Faslodex) was the only approved SERD, which meant patients who stopped benefiting from fulvestrant were left with few treatment options. Unlike fulvestrant, which can only be given as an intramuscular injection, elacestrant can be administered orally. The approval of elacestrant was based on the results of a clinical trial presented in 2021 at the San Antonio Breast Cancer Symposium (SABCS), co-organized by the AACR. 
• On February 3, the FDA greenlighted sacituzumab govitecan-hziy (Trodelvy) for patients with locally advanced or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer that cannot be surgically removed and who have already received certain therapies. This approval expands on the 2020 approval of sacituzumab govitecan-hziy for metastatic triple-negative breast cancer .  Sacituzumab govitecan-hziy is an antibody-drug conjugate, a type of therapeutic consisting of a monoclonal antibody linked to a toxic payload. When the antibody portion binds its target protein on cancer cells, the toxic payload is internalized and kills the cancer cells with no or minimal harm to healthy cells. According to a study published in Molecular Cancer Therapeutics , the increased efficacy of sacituzumab govitecan-hziy compared to other similar drugs comes from its ability to efficiently penetrate tumors and rapidly release its payload after entering cells .   
• On March 3, abemaciclib (Verzenio) was approved in combination with endocrine therapy for patients with HR-positive, HER2-negative, early breast cancer who have a high risk of disease recurrence after surgery.  Abemaciclib is a targeted therapy that inhibits the function of two cell cycle-regulating proteins, CDK4 and CDK6. Besides abemaciclib, approved CDK4/6 inhibitors include palbociclib (Ibrance) and ribociclib (Kisqali).   CDK4/6 inhibitors have become standard of care in combination with hormone therapy for patients with metastatic HR-positive, HER2-negative breast cancer.   Abemaciclib was previously approved to treat progressive (2017) or untreated (2018) advanced HR-positive, HER2-negative breast cancers. In 2021, abemaciclib was approved as adjuvant treatment for early HR-positive, HER2-negative breast cancers with a high risk of recurrence as determined by expression of the Ki-67 biomarker.   The latest approval eliminated the Ki-67 testing requirement, expanding abemaciclib use to a broader patient population. Eligibility for this therapy is now based on the number of cancer-containing lymph nodes, tumor size, and tumor grade.   The expanded indication is based on a four-year follow-up of the phase III clinical trial that led to the 2021 approval of abemaciclib for certain early breast cancers. Results from this trial were presented at SABCS in 2020. 

Immunotherapy for lung, uterine, and skin cancer 

Immune checkpoint inhibitors (ICIs) release the immune system’s natural brakes to help T cells fight cancer and have become part of the standard-of-care for many cancer types.  

• ICIs were first approved for non-small cell lung cancer (NSCLC) in 2015 to treat patients with advanced disease that progressed after chemotherapy. Since then, the applications of immunotherapy for lung cancer treatment have been expanded from second line to first line and from advanced to earlier stages.  On January 26, the FDA greenlighted pembrolizumab (Keytruda) for the adjuvant treatment of NSCLC following surgery and platinum-based chemotherapy. Pembrolizumab is the second immune checkpoint inhibitor approved for this indication, following atezolizumab, which was approved in 2021.  
• On February 9, the FDA issued full approval of the ICI dostarlimab-gxly (Jemperli) for certain patients with recurrent or advanced endometrial cancer whose tumors have a DNA repair defect called mismatch repair deficiency (dMMR), making them more likely to respond to ICIs.    Dostarlimab-gxly had received accelerated approval for this patient population in 2021. That year, the AACR Cancer Progress Report featured the experience of an endometrial cancer survivor who benefited from dostarlimab-gxly treatment through participation in a clinical trial.   In addition to dostarlimab, another ICI used to treat advanced endometrial cancer is pembrolizumab, which was approved as a monotherapy to treat tumors with DNA repair defects and in combination with a targeted therapy for tumors without DNA repair defects. 
• On March 22, the FDA granted accelerated approval to a new ICI called retifanlimab-dlwr (Zynyz) for the treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma, a rare but aggressive non-melanoma type of skin cancer associated with a high risk of recurrence and metastasis.  This approval expands the portfolio of ICIs approved for the treatment of Merkel cell carcinoma, which includes avelumab (Bavencio) and pembrolizumab . 

A new therapy for childhood brain cancer 

On March 16, the FDA approved dabrafenib (Tafinlar) in combination with trametinib (Mekinist) for certain pediatric patients 1 year of age or older with low-grade glioma, the most common type of pediatric brain cancer.  

Surgery is the mainstay of treatment for pediatric low-grade glioma, and the survival rates of patients whose tumors are completely resected is high. When complete resection cannot be achieved, or in cases where surgery is not an option, chemotherapy is used to prevent recurrence or progression, but toxicity is a concern.  

The new approval marks the first targeted treatment option for patients who require systemic therapy and whose tumors harbor a specific mutation in the BRAF gene, known as V600E and associated with poor response to chemotherapy and radiotherapy. 

Dabrafenib and trametinib are molecularly targeted therapies that block the activity of the BRAF and MEK signaling proteins, respectively, to shut down the signaling pathway that is commonly activated in tumors with BRAF mutations. 

This combination was previously approved for patients 6 years of age and older with BRAF V600E-mutated unresectable or metastatic solid tumors, including low-grade glioma, that had progressed following prior therapy.  

For more details, including the full indications and the clinical trial results that led to each approval, visit the FDA approvals page on the AACR website. 

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• FDA Approvals in Oncology: April-June 2023
• FDA Approvals in Oncology: July-September 2023
• FDA Approvals in Oncology: October-December 2023