In today’s world, cancer stands as one of the most daunting challenges in modern medicine, affecting millions of lives across the globe. Beyond its physical toll, cancer inflicts profound emotional, financial, and societal burdens on individuals, families, and communities alike. Its diagnosis can shatter lives, disrupt families, and strain healthcare systems to their limits. 

Yet, amidst this landscape of adversity, breakthroughs in cancer treatment represent glimmers of hope, offering promise to those confronting this relentless disease. In recent years, we’ve witnessed remarkable strides in oncology research, expanding our understanding and paving the way for innovative therapies that hold the potential to fundamentally revolutionize cancer care. 

At the forefront of these advancements are targeted therapies – precise medicines engineered to zero in on specific molecular vulnerabilities within cancer cells, sparing healthy tissues and minimizing side effects. Bispecific antibodies, antibody-drug conjugates and CAR-T cell therapies exemplify the cutting-edge strategies reshaping the oncology landscape, offering new avenues for combating various forms of cancer. 

What’s more, the rise of immunotherapy marks a paradigm shift in cancer treatments, harnessing the body’s own immune system to recognize and destroy cancer cells. Checkpoint inhibitors, adoptive cell transfer, and cancer vaccines lead the charge in this transformative approach, unlocking unprecedented opportunities for durable responses and long-term remission. 

So, as we edge even closer to a future where cancer is no longer a source of fear but a conquerable challenge, let’s explore five recent breakthroughs and discoveries in the world of oncology…

1.    Compass Therapeutics’ BTC drug gets fast-tracked by the FDA

Compass Therapeutics, an oncology-focused biopharma, has made significant strides with its innovative new drug candidate, CTX-009. Recognizing the drug’s potential, the FDA has expedited its evaluation for use alongside paclitaxel to combat biliary tract cancers (BTC), a particularly aggressive form of gastrointestinal cancer. 

Each year, around 23,000 Americans are diagnosed with BTCs, yet treatment options remain sparse. Existing therapies often yield low response rates and limited improvements in patient survival, marking a pressing unmet healthcare need. 

The FDA’s decision to fast-track CTX-009 comes after Phase II results, which demonstrated a notable 37.5% response rate, an average progression-free survival rate of 9.4 months, and an average overall survival rate of 12.5 months—outperforming the majority of BTC therapies on the market. This remarkable efficacy stems from CTX-009’s unique mechanism of action, which targets two pivotal pathways, DLL4 and VEGF-A, intricately involved in the angiogenic process (the growth of new blood vessels in tumours) crucial for tumour vascularization.

However, CTX-009’s potential extends far beyond BTC. Preclinical and early-stage data indicate its efficacy against a spectrum of solid tumours, including colorectal, stomach, liver, pancreas and lung cancers. Intriguingly, even patients previously deemed resistant to conventional treatments have exhibited promising responses when administered CTX-009 monotherapy. 

With anticipation mounting, forthcoming Phase III trial results, slated for release by the end of the year, hold immense promise for the oncology landscape. Should the findings align with Compass Therapeutics’ earlier successes, CTX-009 could represent an enormous shift in cancer therapeutics, offering renewed hope and improved outcomes for patients grappling with various solid tumours. 

2.    Vor Bio set to expand AML treatment options with CD33-directed CAR-T cell therapy

Acute myeloid leukaemia (AML) may not be as widely recognized as some cancers, but its impact is devastating. Typically striking individuals around 68 years old, AML is a relentless disease that ravages blood and bone marrow and spreads rapidly. Sadly, current treatment options are limited, with nearly 70% of AML patients succumbing within five years of diagnosis. That means that for those battling the disease, life revolves around gruelling treatments, hospital stays and reliance on dedicated caregivers. 

At the moment, the only curative therapy for AML patients is a procedure known as HSCT, a stem cell transplant that aims to eradicate cancerous bone marrow, paving the way for healthy cells to regenerate. However, this approach, which involves conditioning patients with chemotherapy before introducing stem cells from a donor, isn’t failproof: a staggering 30-40% of recipients experience relapse. 

Enter Vor Bio, a cell and gene-focused biotech armed with a groundbreaking new therapy known as VCAR33. They aim to offer a fighting chance to typically elderly AML patients through targeted CAR-T technology, with the sophisticated precision VCAR33 utilizing healthy donor cells, meticulously engineered to hone in on a specific biological target, an antigen known as CD33, commonly found on cancerous myeloid cells. 

Preliminary clinical findings of Vor’s VCAR33 therapy offer promising insights. Unlike traditional approaches that indiscriminately wipe out all CD33 in the blood marrow system, Vor’s method appears more nuanced, potentially rendering it safer and more effective than other treatments on the market. 

In fact, haematology and blood cancer experts have pointed to CD33 as a promising target for experimental AML treatments, given how commonly the antigen is expressed in cancer patients’ cells. Even pharmaceutical giant Pfizer has ventured into this arena with their FDA-approved therapy, Mylotarg, which is offered to AML patients with reoccurring cancer who don’t respond to other treatments, albeit with the risk of potentially life-threatening side effects for some patients. 

Still, Vor’s approach offers a fresh perspective. In January, the team announced the first AML patient received VCAR33 in a Phase I/II clinical trial, marking a pivotal moment in their quest for an effective solution. The trial, targeting patients with refractory or relapsed AML post-transplant, holds the promise of unleashing the potent anti-leukaemia activity of CD33-targeted CAR-T cells derived from healthy donors, offering patients the prospect of a brighter, healthier future in the fight against AML. 

3.    Abbvie receive full FDA approval for their ovarian cancer treatment, Elahere

Earlier this quarter, the FDA granted full approval to Abbvie and ImmunoGen’s pioneering cancer drug, Elahere, marking a significant milestone in the fight against cancer. This medication, which belongs to a cutting-edge class of cancer therapies dubbed ‘biological missiles’ for their remarkable precision in targeting cancer cells, is now available for patients battling specific types of cancer in the ovaries, fallopian tubes, or peritoneum who have already undergone one to three prior treatment regimens. 

For context, ovarian cancer stands as the leading cause of death from gynaecological cancers in the US, with roughly 20,000 patients diagnosed each year and only 7,000 of those patients surviving. Typically, patients with these cancers undergo surgery followed by platinum-based chemotherapy. However, resistance often develops, which is challenging to treat, necessitating alternative treatments like Elahere, as emphasized by Abbvie in a recent press release. 

So, back to Elahere’s approval process… In 2022, the drug received accelerated approval from the FDA, which the American Association for Cancer Research dubbed a milestone. That’s because Elahere became the first antibody-drug conjugate ever approved for platinum-resistant ovarian cancer, a condition with a typically poor prognosis. Then, even better news followed, as compelling data from a 2023 study prompted the FDA to grant full approval to Elahere. 

The pivotal Phase III trial compared Elahere to standalone chemotherapy, enrolling 453 patients with varying treatment histories – 14% having had one prior line of therapy, 39% having had two prior lines of therapy, and 47% having had three prior lines of therapy. 

The results were striking, showcasing a statistically significant and clinically meaningful improvement in overall survival with Elahere, including a 33% reduction in the risk of death and a 35% reduction in the risk of tumour progression, accompanied by fewer reported adverse effects. 

In fact, Elahere demonstrated an impressive objective response rate of 42.3%, with 12 complete responses observed, contrasting with the chemotherapy group’s response rate of 15.9% with no complete responses. However, it’s important to note that despite its efficacy, Elahere comes with a boxed warning, signalling potential severe side effects, particularly ocular issues, which were observed in 59% of patients. 

Overall, Elahere’s approval offers hope to thousands of patients diagnosed annually, representing the first drug to show an overall survival benefit in this patient population. With standard treatment options falling short in efficacy and burdened with significant toxicities, Elahere stands as a symbol of progress in the pursuit of better outcomes for patients with platinum-resistant ovarian cancer.

4.    AstraZeneca and Daiichi Sankyo’s Enhertu shows greater promise in new breast cancer trial

Last year, the European Commission gave the nod to Enhertu, a joint effort between AstraZeneca and Daiichi Sankyo, as a treatment for unresectable or metastatic HER2-low breast cancer after clinical trials showed a promising 50% reduction in the risk of disease progression or death. This approval targeted adult patients who previously underwent chemotherapy in the metastatic setting or experienced disease recurrence during or within six months of completing adjuvant chemotherapy. 

Now, following on from this success, AstraZeneca and Daiichi Sankyo share upbeat findings from a late-stage study of Enhertu in a specific group of breast cancer patients. The Phase III trial pitted Enhertu against chemotherapy in patients with hormone receptor-positive, HER2-low or HER2-ultralow advanced or metastatic breast cancer. 

For reference, breast cancer ranks as the second most common cancer globally and is a leading cause of cancer-related deaths, with over two million diagnoses in 2022 alone. Among breast cancer subtypes, hormone receptor-positive HER2 negative is the most prevalent, with an estimated 65% of cases having HER2-low expression and an additional 25% potentially having HER2-ultralow expression. 

Before Enhertu’s approval for HER2-low metastatic breast cancer last year, no targeted therapies were specifically greenlit for these patients. Furthermore, still no approved targeted therapies for those with HER2-ultralow expression exist. 

However, the latest Phase III trial showed statistically significant and clinically meaningful improvement in progression-free survival compared to chemotherapy in patients with hormone receptor-positive, HER2-low metastatic breast cancer after endocrine therapy. This improvement held true for both the HER2-low and HER2-ultralow expression groups. 

Notably, Enhertu also hinted at a possible overall survival improvement over chemotherapy in HER2-low metastatic breast cancer and across the entire trial population. However, these findings are preliminary, and the trial will further evaluate overall survival rates. 

In essence, these promising findings hint at a new era in breast cancer care, with Enhertu potentially setting a new standard for patients with HER2-low and HER2-ultralow metastatic breast cancer, offering a glimmer of hope for patients worldwide. 

5.    iOncologi and the University of Florida reveal seven years’ worth of findings for mRNA vaccine targeting glioblastomas

Glioblastomas, the most aggressive and fastest-growing type of brain tumour, are the most common malignant brain tumours in adults, known for their low survival rates. Typically, patients face an average survival time of just 12-18 months, with only 25% surviving beyond one year and a mere 5% surviving beyond five years. 

But now, a collaboration between researchers from the University of Florida and a spin-out biotech, iOncologi, offers a beacon of hope. They’ve recently unveiled groundbreaking results from over seven years of experiments, leading to a first-of-its-kind Phase I trial involving four human patients and ten dogs spontaneously developing brain cancer. 

What sets the trial apart is the use of an innovative mRNA vaccine, a novel approach to cancer treatment. Traditional mRNA vaccines target immune cells via lipid nanoparticles, triggering an immune response. However, this new vaccine, developed by iOncologi and the University of Florida, takes a different route. Wrapped in RNA lipid particle aggregates (RNA-LPAs), it mimics a virus, activating a protein called RIG-I in stromal cells found in tissues and tumour microenvironments. Injected into the bloodstream, it prompts the release of cytokines, chemokines, dendritic cells, and lymphocytes, training the immune system to combat the patient’s cancer. 

This systemic injection method overcomes a significant challenge in cancer vaccines: eliciting a robust immune response. In fact, within six hours, researchers saw blood markers associated with immune activation surge, transforming previously ‘cold’ tumours, inaccessible to the immune system, into ‘hot’ ones in just 48 hours. 

From there, results demonstrated that the human participants survived or lived without disease for ‘longer than expected’, and the canine participants lived for an average of 139 days– far longer than the typical 30–60-day survival time. 

As the team progresses into an expanded Phase I study, including up to 24 pediatric and adult patients, they’ll finetune dosing and study the vaccine’s side effects. Subsequently, a Phase II trial will enrol 25 pediatric patients, with plans to explore the vaccine in combination with other immunotherapies.

All in all, the research provides hope for the future of glioblastoma therapies, not only for humans but also for our beloved furry friends too.