Heart attacks and strokes are among the leading causes of death globally, with millions suffering from cardiovascular diseases (CVD) every year. There are more than seven million people in the UK alone, with about 100,000 patients experiencing heart attacks annually. However, a group of researchers at University College London (UCL) estimate that one 'polypill' taken daily day could eliminate a majority of these cases dramatically lowering death tolls.

The proposed polypill, a combination of a statin and three blood pressure-lowering drugs, has been under study for over two decades. Experts argue that introducing this pill universally for individuals aged 50 and above could be more effective than the current NHS Health Check, which assesses risk factors every five years for those aged between 40 and 74.

Studies have repeatedly proven the effectiveness of the polypill in preventing CVD. A groundbreaking 2019 study in The Lancet found that five years' use of the polypill cut the risk of heart attack and stroke by a third. In addition, previous modelling analyses have estimated that if given universally to people over 55, the polypill might be able to prevent 80% of heart attacks and strokes.

Today, the NHS Health Check follows a risk-based model in which patients are tested for CVD risk factors and treated with drugs accordingly. Yet, as per UCL's study, this system has serious flaws:

Low Uptake: Just 40% of those eligible for the NHS Health Check choose to have it, leaving a considerable number of at-risk patients undiagnosed and untreated.

Ineffective Prediction of Risk: The majority of heart attacks and strokes happen to people at average risk levels, thus making it challenging to identify the need for intervention effectively.

Limited Effectiveness: Even at maximum take-up, the NHS Health Check programme is predicted to have fewer health impacts compared to a polypill initiative applied to the whole population.

Simplicity and Affordability of the Polypill Strategy

One of the big benefits of the polypill is that it is so easy. In contrast to the existing screening-based model, the polypill scheme would not involve complicated medical tests or lengthy risk assessments. Instead, people reaching 50 would just have to fill out a few questions to determine possible side effects before they were prescribed.

Professor Aroon Hingorani of the UCL Institute of Cardiovascular Science, one of the strongest proponents of this scheme, says:

"Finally, the time is now to do much better on prevention. A population approach would prevent a lot more heart attacks and strokes than is done today with a strategy of trying to target a smaller group only."

Aside from the possible health implications, the polypill is also an economic solution. The drugs used are off-patent, thus cheap to produce and distribute. With the vast economic cost of managing CVD-related illnesses, a preventive model could result in substantial cost-saving for the NHS in the future.

The polypill has been proven to be effective by numerous international trials. In 2019, a randomised trial in rural Iran discovered that participants who took the polypill for five years had a 34% reduced risk of having a heart attack or stroke compared to non-participants.

Likewise, modelling research has indicated that even if only 8% of people aged over 50 took up the polypill regimen, it would still be more beneficial to their health than the NHS Health Check programme.

Is This a Case of Over-Medicalisation?

One of the main objections to the polypill strategy is the suggestion that it might result in the unnecessary medicalisation of a significant proportion of the population. But, it is argued, it should be considered as a preventative measure, not as mass medication.

Professor Sir Nicholas Wald of UCL's Institute of Health Informatics explains:

"Instead of being a 'medicalisation' of a significant proportion of the population, a polypill programme is a prevention measure to prevent an individual from becoming a patient."

He compares it with public health measures like water fluoridation or compulsory seatbelts—interventions that have been shown to have a significant impact in reducing public health danger at low individual cost.

With the evidence in favour of the polypill's effectiveness and viability overwhelming, experts are calling on the NHS to act now. It is their belief that substituting the NHS Health Check with a polypill-based prevention program could be the UK government's flagship policy under its pledge to put disease prevention ahead of cure.

As Professor Hingorani points out, "The status quo is not a justifiable option." With CVD still a major cause of death globally, taking a population-wide polypill approach could be a turning point for preventative medicine, potentially saving thousands of lives annually. The question now is whether the NHS will take up this call and establish a policy with the potential to transform the prevention of cardiovascular disease on a national level.

A tiny infection under a newborn’s fingernail turned out to be an important clue that led doctors to an unexpected and life-threatening diagnosis of an aggressive brain tumour.

Joey Sharp, from Penicuik in Midlothian, Scotland, was just 11 days old when his parents took him back to hospital after he began struggling to feed, losing weight and developing persistent jaundice. He was also showing signs of twitching intermittently.

A Tiny Nail Problem Led Doctors To Look Further

While examining the newborn, doctors noticed what appeared to be a very small infection in one of his fingernails. According to Joey’s mother, Sam Sharp, the abnormality was so tiny that it looked almost like a grain of sand beneath the nail.

But while doctors were investigating the problem, an ultrasound revealed a tumour in Joey’s brain.

The discovery came as Joey’s other symptoms were becoming increasingly concerning. What initially looked like a minor problem with his finger became part of a much larger medical investigation.

Joey was subsequently diagnosed with glioblastoma, an aggressive form of brain cancer.

Also read: BBC News Presenter Maryam Moshiri Reveals Incurable Blood Cancer: What Is Polycythemia Vera?

Why Were Doctors Looking At His Finger?

The nail infection itself was not reported as the cause of the brain tumour. Rather, investigating the unusual finding was part of the baby's broader medical assessment after he returned to hospital with several unexplained symptoms.

In newborns, symptoms can sometimes be difficult to interpret because babies cannot describe what they are experiencing. Problems such as poor feeding, weight loss, jaundice, and abnormal movements can have many possible causes.

In Joey's case, doctors' investigations ultimately led them to perform imaging that uncovered the brain tumour.

The story is therefore less about a nail infection “causing” a brain tumour and more about how an apparently minor physical finding helped doctors continue investigating a very sick newborn until they found the underlying problem.

Also read: FDA Approves New Breast Cancer Treatment That Targets Resistance Before Cancer Progresses

The Newborn Was Diagnosed With Glioblastoma

For Joey, treatment was intensive. He underwent three brain surgeries and nine rounds of chemotherapy. Two operations were performed to remove the tumour, while a third was needed to deal with scar tissue that was preventing medication from controlling his seizures.

At one point, Joey was experiencing more than 30 epileptic seizures a day and required feeding tubes.

What Symptoms Glioblastoma Can Cause In Newborns?

In babies, brain tumours may announce themselves through changes in head growth, feeding, alertness, vomiting or development rather than the classic headaches adults experience. According to reports on congenital glioblastoma, possible signs include:

• Rapidly increasing head size (macrocephaly) is one of the more important clues in infants
• Bulging or tense soft spot (fontanelle) due to increased pressure inside the skull
• Persistent vomiting, particularly when accompanied by other neurological changes
• Unusual sleepiness or lethargy
• Poor feeding or difficulty feeding
• Irritability or unusual changes in behaviour
• Seizures
• Weakness or reduced movement of one side of the body
• Abnormal eye movements or visual problems
• Delayed development
• Poor weight gain

Despite the severity of his illness and the treatment he required as a baby, Joey survived. He also participated in clinical trials designed to help researchers better understand how chemotherapy can be used to treat babies with brain tumours. He is now reported to be cancer-free.

In a world first, an experimental treatment has been administered to a 26-year-old woman, the daughter of American hedge fund manager Bill Ackman, in which mitochondria from her leg were injected into her eyes.

While the treatment did not restore vision, it produced a temporary return of the pupils’ response to light, offering a potentially important finding, according to scientists from the Icahn School of Medicine at Mount Sinai in a paper published as a preprint on Research Square.

The eye study, posted as a preprint on August 10, has not yet undergone peer review. The treatment remains highly experimental and involved only one patient.

“It was my daughter. Mitochondria has amazing potential,” Ackman said in a post on X, adding that the family had seen a sustained benefit from the initial injection, although it was weaker than the immediate response.

Brain Hemorrhage Left Her Unable To See

In February this year, Ackman’s daughter Lucy collapsed in her Brooklyn apartment after suffering a brain hemorrhage, leaving her unable to move, speak or see, the billionaire said in a post on X.

While doctors performed emergency surgery, the injury damaged her optic nerve and left her unable to see. Her pupils also stopped responding to light.

Despite the progress in other areas, Lucy’s vision has remained severely affected.

After receiving emergency approval from the US Food and Drug Administration, doctors at Mount Sinai extracted mitochondria — the energy-producing structures inside cells — from Lucy’s leg muscle and injected them into the fluid of both eyes, according to Nature.

“Over the last six months, she has recovered her cognition – she understands everything including her circumstance – is able to walk a hundred or more steps at a time with assistance, is making progress with sounds, vowels and consonants and the beginnings of speech, but she remains unable to see,” Ackman said.

Mitochondrial Transplant Showed Temporary Visual Response

The researchers reported that Lucy, who had optic nerve damage in both eyes for three months, received one mitochondrial injection in each eye, 24 hours apart.

Importantly, neither eye developed inflammation. Within days of the injections, her pupils began responding to light again — something that had not happened in 45 tests over the previous 71 days.

She also reported seeing shapes and shadows through her left eye.

However, the response faded after about four weeks, and the treatment did not restore normal vision.

“We are working on a method to inject her eyes with more frequency,” Ackman wrote on X.

When Can Mitochondrial Transplants Help?

According to the researchers, mitochondrial transplantation involves delivering healthy, functioning mitochondria into tissue where the mitochondria have been damaged.

The approach has been studied in the human heart and brain, but had not previously been used in the eye.

"To our knowledge, this is the first administration of isolated mitochondria to the human eye. The procedure was performed to the filed specification in both eyes," the researchers said in the paper.

Retinal ganglion cells, which are involved in transmitting visual information from the eye to the brain, depend heavily on mitochondria to produce energy. In studies involving rodents, mitochondria injected into the vitreous — the gel-like substance inside the eye — have been taken up by these cells and improved their survival after optic nerve injury.

In Lucy’s case, the injection of her own mitochondria into the eyes was reported to be safe and was associated with the temporary return of pupillary responses to light. However, it did not restore normal vision, and the visual response faded after about four weeks.

Because the report involves only one patient and has not yet undergone peer review, much more research is needed to determine whether mitochondrial transplantation could eventually become a treatment for optic nerve damage or other forms of vision loss.

The US Food and Drug Administration has approved a new breast cancer treatment that aims to act on signs of treatment resistance before the cancer starts progressing.

The US FDA has granted accelerated approval to Etcamah (camizestrant), in combination with a CDK4/6 inhibitor, for adults with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer whose tumours develop an ESR1 mutation while being treated with an aromatase inhibitor and a CDK4/6 inhibitor.

All About The New Breast Cancer Treatment

The new approach uses a blood test to detect circulating tumour DNA (ctDNA) carrying an ESR1 mutation. If the mutation is detected, doctors can switch treatment to camizestrant rather than waiting for visible disease progression on scans.

The FDA described this as its first cancer therapy approval guided by detection of a resistance mutation in circulating tumour DNA before imaging shows progression of the disease.

Also read: Have Dense Breasts? What Women Should Know About Their Breast Cancer Risk

What Did The Clinical Trial Find?

The approval was based on results from the Phase III SERENA-6 trial, which included 315 patients with HR-positive, HER2-negative locally advanced or metastatic breast cancer.

All participants were receiving an aromatase inhibitor plus a CDK4/6 inhibitor as their initial endocrine based treatment and had no evidence of disease progression when an ESR1 mutation was detected through blood testing.

Patients were randomly assigned to either switch to camizestrant while continuing their CDK4/6 inhibitor or continue their existing aromatase inhibitor with the CDK4/6 inhibitor.

Survival period without progression was 16 months with camizestrant compared with 9.2 months with standard treatment. The risk of disease progression or death was reduced by 56% with the camizestrant combination.

Also read: Alcohol-Linked Cancer Deaths Doubled In US: Colorectal Leads In Men, Breast Leads In Women

Importance Of ESR1 Mutations

ESR1 mutations are one way hormone receptor-positive breast cancers can adapt to treatment. According to the FDA, fewer than 5% of patients have an ESR1 mutation when HR-positive metastatic breast cancer is first diagnosed. After disease progression on an aromatase inhibitor, however, the mutation is found in nearly 40% of patients.

This means that detecting the mutation earlier could potentially give doctors a chance to change treatment while the cancer is still controlled.

Dr Kevin Kalinsky, an investigator on SERENA-6, said, "The approach allows doctors to change treatment at an earlier opportunity ahead of disease progression rather than waiting until the cancer becomes harder to treat."

Risks And Limitations

The FDA approval is accelerated, meaning continued approval may depend on confirmatory studies that will verify clinical benefit.

The regulator specifically noted that it has not yet been established whether intervening when an ESR1 mutation is detected, before radiographic progression, ultimately translates into a meaningful overall survival benefit.

The FDA has simultaneously approved the Guardant360 CDx blood test as a companion diagnostic to identify patients whose tumours carry the relevant ESR1 mutations.

The significance of the approval therefore goes beyond a new drug. It represents a shift toward using molecular clues in the bloodstream to detect treatment resistance and change therapy before cancer progression becomes visible on a scan.