2024: New Hope in Cancer Care
- Tragic stories about cancer often dominate the news cycle, but there is a lot of hope surrounding breakthrough medical advancements in the cancer space as well. SurvivorNet would like to highlight some of these stories — and explore what the future of cancer care may look like as well.
- Among some of the more exciting updates of 2024 are incredibly promising clinical trial results for advanced breast cancer patients and FDA approvals for glioma brain tumors, advanced prostate cancer, and more.
- Nearly 6 million cancer deaths have been prevented from 1975 to 2020, according to a study published in JAMA Oncology earlier this month — which looked at breast, cervical, colorectal, lung, and prostate cancers combined.
- With faster FDA approvals this year for cancer drugs — a total of 16 compared to nine last year — at this pace, millions more will surely be saved.
Here at SurvivorNet, we want to highlight some of those breakthroughs and the hope they are providing in the cancer community — from first of their kind drugs to promising clinical trials that could change the approach to cancer treatment for so many.
Read More1. 16 Cancer-Related FDA Accelerated Approvals
From 2009 to 2024, there were 67 accelerated approvals (for malignant hematology and oncology) by the U.S. Food and Drug Administration (FDA), most recently for colorectal cancer (cancer that begins in the colon or rectum), pancreatic cancer, metastatic non small-cell lung cancer (NSCLC) and sarcoma, which is a rare cancer in the connective tissues. There were a total of 16 approvals in 2024 alone, nearly doubling from nine approvals in 2023. “This listing includes accelerated approvals (AAs) for malignant hematology and oncology indications that have postmarketing requirement(s) for ongoing clinical trial(s) to verify clinical benefit,” the FDA notes.RELATED: New FDA Approval: Vyloy (Zolbetuximab) – A Promising Option for Advanced Gastric Cancer
Speedier drug approvals went into effect back in March 2020 during the Covid-19 pandemic, when a new FDA program called the Coronavirus Accelerated Treatment Program (CATP) launched due to unprecedented circumstances.
Though the accelerated approval process officially began in 1992, turnaround times have been getting faster, with the FDA reviewing data to new clinical trials within 24 hours, and turning around single-patient requests for expanded access to certain therapies “within three hours,” the site says.
In short, the FDA began reviewing new drug applications and clinical trial results faster than ever before.
The FDA’s site explained, “FDA approval of a drug means that data on the drug’s effects have been reviewed by CDER, and the drug is determined to provide benefits that outweigh its known and potential risks for the intended population.”
The CDER is the FDA’s Center for Drug Evaluation and Research.
Clinical Trials Bring Hope to Patients
Within the U.S., all new drugs must go through clinical trials before the FDA approves them. Although the rewards of clinical trials can be great, they also come with risks. Talking to your doctor about this before enrolling in a trial is important.
Some risks to consider include:
- The risk of harm and/or side effects due to experimental treatments
- Researchers may be unaware of some potential side effects of experimental treatments
- The treatment may not work for you, even if it has worked for others
Dr. Beth Karlan, a gynecologic oncologist at UCLA Health, previously explained to SurvivorNet that the goal with clinical trials is to advance cancer research to a point where the disease becomes akin to diabetes, where it becomes a manageable condition.
“Clinical trials hopefully can benefit you, but they also provide vital information to the whole scientific community about the effectiveness of these treatments,” Dr. Karlan said.
“They can be life-saving. In the last few years, we’ve seen many children and adults who have participated in trials and had miraculous results,” Dr. Karlan continued.
2. FDA-Approved Vorasidenib for Glioma
There is now more hope than ever for people with a type of tumor called glioma, which forms in the brain or spinal cord. Many gliomas, especially low- and early-grade gliomas, possess a genetic change called an IDH mutation. Recently, the FDA approved a drug called vorasidenib, which targets this mutation to improve treatment outcomes for patients with IDH mutant gliomas.
Types of Glioma
Gliomas can be divided into two types: IDH wild-type and IDH mutant gliomas. IDH mutant gliomas carry a mutation in the gene that codes for the IDH (isocitrate dehydrogenase) enzyme. Normally, this enzyme helps create energy for glial cells, the cells from which these tumors originate. However, when the IDH gene is mutated, it disrupts the normal function of the enzyme and can lead to the development of cancerous cells, eventually forming a glioma.
IDH mutant gliomas tend to grow more slowly and have a better prognosis compared to IDH wild-type gliomas. They are often lower-grade tumors, which means they progress more gradually. Unfortunately, despite treatment efforts—including surgery, chemotherapy, and radiation therapy—patients with IDH mutant gliomas are not typically cured.
Fortunately, new treatments have been developed that specifically target the IDH mutation found in these gliomas.
New FDA Approved Vorasidenib for IDH Mutant Gliomas
Vorasidenib: A New Hope
Dr. Alexandra Miller, Director of the Neuro-Oncology Division at NYU Langone Health, told SurvivorNet that vorasidenib is a “huge breakthrough for people with IDH mutant tumors.”
This drug was tested in a randomized clinical trial and showed impressive results. On average, it delayed the progression of IDH mutant gliomas by over 27 months, compared to just 11 months for patients who did not receive the drug. The trial results were so promising that the Food and Drug Administration (FDA) approved vorasidenib for use in grade 2 IDH mutant tumors.
Vorasidenib may also have long-lasting effects on patients. Researchers found that 87% of patients taking Vorasidenib did not need additional cancer treatment after two years, compared to only 27% of patients who did not take the drug.
What to Expect With Vorasidinib
Vorasidinib is an oral pill that is taken once daily.
Vorasidenib is what is known as an IDH inhibitor. The drug “is a type of precision medicine or targeted therapy that goes and affects just the tumor cells,” Dr. Miller explained. Therefore, this drug should not affect normal cells that are not expressing this mutation. This leads to fewer side effects for patients and makes the drug very tolerable.
Some common side effects include:
- Fatigue
- Headache
- Diarrhea
Some may also develop elevated liver enzymes on labs, which could indicate liver damage. This will be monitored by your doctor with routine blood work.
IDH Mutant Gliomas: The Future
The development and success of vorasidenib, now with FDA approval, offers hope for the future of targeted therapies for IDH mutant gliomas. Although it is not a cure, vorasidenib significantly improves the quality of life for patients with this challenging disease.
Ongoing research continues to explore ways to target the IDH mutation further. In the meantime, patients with IDH mutant gliomas should talk to their doctors about whether vorasidenib could be a beneficial part of their treatment plan.
3. Imlunestrant for Advanced Breast Cancer
Positive results from a clinical trial called the EMBER-3 Phase III trial indicate the promise of using a drug called imlunestrant to treat women with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer.
Estrogen receptors are sensitive to the hormone estrogen. When estrogen binds to these estrogen receptors, ER+ breast cancer can form. HER2- means that there are low to no HER2 proteins.
Trial results were announced by Lilly at the San Antonio Breast Cancer Symposium (SABC) in Texas on Dec. 11, with imlunestrant demonstrating improvement in a key metric, progression-free survival (PFS), when used as a standalone therapy in patients with an ESR1 genetic mutation (change) versus the standard of care endocrine therapy, reducing the risk of disease progression or death by 38%.
As the National Cancer Institute defines it, the ESR1 gene is “a gene that makes one of the two main types of estrogen receptor (ER) proteins. These proteins bind to and receive signals from the hormone estrogen.”
Imlunestrant used in combination with one of the drugs referred to as a CDK4/6 inhibitor (Verzenio) also reduced the risk of disease progression or death by 43% versus imlunestrant alone in all patients.
Seventy percent of patients are ER+. All [or the majority] will get first-line therapy with a CDK4/6 with endocrine therapy, our experts say. When they progress, doctors think about different ways of treating them based on their biomarkers (which, according to the American Cancer Society, are “genes, proteins, or other substances that can be tested to reveal important details about a person’s cancer.”) But here, the data is showing that regardless of biomarkers, there might be a benefit and this drug combination could be a second-line option for many patients.
“The data is telling us that regardless of the biomarker, we might be able to see the benefit and we might be able to offer this as a second option for many patients,” Dr. Komal Jhaveri, breast medical oncologist at Memorial Sloan Kettering Cancer Center and study author, told SurvivorNet about the research’s impact, which can help fill a void for patients with metastatic breast cancer.
Changing Treatment Paradigm in ER+ HER- patients
“Certainly, the impact these data might have is going to be for a very large group of patients,” Dr. Jhaveri continued. “Imlunestrant plus Verzenio does fill that void in many ways for patients because not only has it shown the longest PFS of any other contemporary phase III trial in that setting, but it’s also thought to be very, very safe with a low discontinuation rate of 6%.”
Dr. Jhaveri suggested this combination could be offered to a large group of patients, regardless of biomarker status, to delay chemotherapy.
The inclusion criteria of this study selected patients with postmenopausal status (either natural or surgical), who have progressed on or after previous endocrine therapies, such as aromatase inhibitors or selective estrogen receptor modulators, and individuals exhibiting resistance to first-line hormonal therapies.
Potential Advantages
The potential advantages of imlunestrant include enhanced efficacy — the drug may offer improved anti-tumor activity due to its potent estrogen receptor degradation — and the convenience of oral therapy. An oral SERD eliminates the need for intramuscular injections, potentially enhancing patient comfort and compliance.
With the data from the EMBER-3 trial, imlunestrant may be established as a new standard of care treatment or subsequent therapy in this setting of patients, and current treatment paradigms may be reshaped, providing new hope for patients who have limited options after standard endocrine therapies.
Doctors involved in the clinical research say the drug offers a comfortable and convenient alternative to injectable Selective estrogen receptor degraders (SERDs), potentially improving patients’ adherence and, consequently, quality of life.
“We’re trying to overcome the issues that we see with currently available agents … whether it’s tolerance issues or pharmacokinetic,” Dr. Jhaveri said. “We’re able to now see that there are these agents that are able to overcome these and will become a standard, a new standard for our patients which will actually have an impact on their survival, actually have an impact on their safety tolerability profiles and how they feel on it, and improve their quality of life.”
4. Orgovyx: An FDA-Approved Oral Drug for Advanced Prostate Cancer
Orgovyx is the first FDA-approved oral testosterone-lowering drug for advanced prostate cancer. Orgovyx works by blocking the action of a hormone called gonadotropin-releasing hormone (GnRH), which stimulates the production of testosterone in the testicles. By lowering testosterone levels, Orgovyx can slow down the growth of prostate cancer cells that depend on testosterone.
“Orgovyx is the first oral testosterone lowering agent that has been approved we have available for our patients,” Dr. David Wise, a medical oncologist with NYU Langone Health in New York City, told SurvivorNet. “There’s really been quite an evolution here in hormonal suppression.”
Who is a Good Candidate for Orgovyx?
Orgovyx is particularly helpful for patients with advanced prostate cancer because it does a good job of lowering testosterone quickly.
“Orgovyx is specifically aimed at patients where we need to rapidly decrease testosterone levels,” Dr. Wise said. “This makes it a strong option for those with aggressive, fast-growing or metastatic cancers that react quickly to changes in testosterone level.”
Dr. Wise added that the drug’s ability to swiftly reduce testosterone helps slow the progression of aggressive cancer types. Slower-acting treatments may not work as well.
What are the Benefits and Risks of Orgovyx?
One of the key advantages of Orgovyx is its rapid action in lowering testosterone levels, which is crucial for effective prostate cancer treatment, especially when the cancer is fast moving and aggressive.
Unlike some other drugs that lower testosterone levels, Orgovyx does not cause testosterone flares. This means that Orgovyx lowers testosterone levels without increasing them first, which can cause a temporary worsening of symptoms in some patients.
Orgovyx slows aggressive cancer growth without causing testosterone flares
Another advantage: unlike other GnRH antagonists that are given as injections, Orgovyx is a pill you take once a day. “The oral delivery mechanism of Orgovyx sets it apart from other options,” Dr. Wise said.
However, like all medications, Orgovyx can have side effects.
Common problems include:
- Hot flashes
- Fatigue
- Muscle and joint pain
- Diarrhea
- Constipation
- Decreased libido and erectile dysfunction
“While Orgovyx has typical side effects, its ease of administration is a significant benefit,” Dr. Wise added.
5. 6 Million Lives Saved Due to Prevention & Screening Efforts
New findings, published in JAMA Oncology, have estimated that 5.94 million deaths were prevented from breast, prostate, lung, colorectal, and cervical cancers combined.
The model-based study, which used population-level cancer mortality data and statistical models created by the Cancer Intervention and Surveillance Modeling Network between 1975 and 2020, found that cancer prevention and screening efforts “averted 8 of 10 of these deaths (4.75 million averted deaths).”
When looking at breast cancer cases, 1.03 million deaths, out of 2.71 million, were prevented during the time frame of the study. As for what averted the deaths, the study authors attributed 770,000 of those prevented deaths to treatment advances, with the rest due to mammography screenings. Overall, breast cancer screening led to 25% of breast cancer deaths averted.
In prostate cancer cases, prostate-specific antigen (PSA) testing led to 200,000 deaths being averted, while another 160,000 deaths were prevented thanks to treatment advances. Screening also led to a whopping to 56% percent of averted prostate cancer deaths.
Tobacco control was also found to have led a whopping 98% percent of averted deaths. Treatment advances helped the remaining averted deaths.
Take it From a Guy Who Looks at Diseased Lungs Every Day — Stop Smoking
Meanwhile, approximately 160,000 cervical cancer deaths were prevented thanks to Pap smears and human papillomavirus (HPV) testing. Removal of precancerous polyps also helped avert cancer deaths. Around 80 million Americans have HPV, or the human papillomavirus, according to the Centers for Disease Control and Prevention, and for most people it won’t cause any problems. HPV affects both men and women and in a small percentage it can lead to cancer most commonly of the cervix and head and neck.
The HPV vaccine is recommended to protect against these cancers. “The key with the vaccine is that you receive it before you have sexual encounters,” Dr. Jessica Geiger, a medical oncologist at Cleveland Clinic Cancer Center, previously told SurvivorNet. “So that’s why these vaccines are approved for young children … ages 9, 10, 11 years old, up to age 26.”
Why the HPV Vaccine is so Important in Preventing Cancer
As for averted colorectal cancer deaths, screening and the surgical removal of precancerous polyps also made had a major impact, as well as advances in treatment. Study authors found that 79% of deaths were averted during the study timeframe.
Contributing: SurvivorNet staff and our team of medical advisors
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