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Contracts offered to recruitment agencies to help NHS boost workforce by 5,000 GPs.

Last year’s winter flu jab worked well for children and adults – but not for the over-65s, data shows.

Opening a new era in cancer care, US health officials have approved a breakthrough treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukaemia.

The Food and Drug Administration (FDA) called the approval historic, the first gene therapy to hit the US market. Made from scratch for every patient, it’s one of a wave of “living drugs” under development to fight additional blood cancers and other tumours, too. 

This treatment method came through after a new drug was approved for the treatment of B-cell acute lymphoblastic leukaemia (ALL), recently reported on Health24.

Brand new treatment method

Novartis Pharmaceuticals set the price for its one-time infusion of so-called “CAR-T cells” at $475 000 (±R6 189 000), but said there would be no charge for patients who didn’t show a response within a month.

“This is a brand new way of treating cancer,” said Dr Stephan Grupp of Children’s Hospital of Philadelphia, who treated the first child with CAR-T cell therapy – a girl who’d been near death but now is cancer-free for five years and counting. “That’s enormously exciting.”

Gene therapy techniques used

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient’s blood, reprogramme them to harbour a “chimeric antigen receptor” or CAR that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

It’s a completely different way to harness the immune system than popular immunotherapy drugs called “checkpoint inhibitors” that treat a variety of cancers by helping the body’s natural T cells better spot tumours. CAR-T cell therapy gives patients stronger T cells to do that job.

“We’re entering a new frontier in medical innovation with the ability to reprogramme a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb.

Majority of study subjects in remission

The first CAR-T version, developed by Novartis and the University of Pennsylvania, is approved for use by several hundred patients a year who are desperately ill with acute lymphoblastic leukaemia, or ALL. It strikes more than 3 000 children and young adults in the US each year and while most survive, about 15% relapse despite today’s best treatments.

In a key study of 63 advanced patients, 83% went into remission soon after receiving the CAR-T cells. Importantly, it’s not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare long-term.

Still, “a far higher percentage of patients go into remission with this therapy than anything else we’ve seen to date with relapsed leukaemia,” said Dr Ted Laetsch of the University of Texas Southwestern Medical Centre, one of the study sites. “I wouldn’t say we know for sure how many will be cured yet by this therapy. There certainly is a hope” that some will be.

Treatment for side-effects

Most patients suffered side effects that can be gruelling, even life-threatening. An immune overreaction called “cytokine release syndrome” can trigger high fevers, plummeting blood pressure and in severe cases organ damage, side effects that require sophisticated care to help patients without blocking the cancer attack. The FDA designated a treatment for those side effects.

“This is remarkable technology,” said Dr Mikkael Sekeres of the American Society of Hematology. But, he cautioned that CAR-T “isn’t a panacea”.

Among concerns, sometimes leukaemia can develop resistance, and sometimes patients worsen while waiting for their new cells, said Sekeres, who directs the Cleveland Clinic’s leukaemia programme and wasn’t involved with CAR-T testing.

“Unfortunately leukaemia grows so rapidly that it can evade even the smartest of our technologies,” he added.

Patient safety important

To better ensure patient safety, the FDA is requiring Novartis to offer CAR-T therapy only through medical centres specially trained and certified to handle the complicated treatment. Novartis expects to have 32 centres around the country, mostly in large cities, running by year’s end, with the first 20 offering care within the next month.

Patients’ collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks. The $475,000 price tag doesn’t include the cost of needed hospitalisations, travel to a certified hospital and other expenses.

On a conference call, Novartis executives said the company is working with the Medicaid programme and private insurers and expects broad coverage, and will offer some financial assistance with such things as copay and travel costs. But they didn’t promise all patients would be able to get the therapy.

Replacement for bone marrow

For some patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study.

“I don’t want to be an apologist for high drug prices in the US,” Grupp stressed. But if it’s the last treatment they need, “that’s a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them.”

“This is a turning point in the fight” against ALL, said Penn’s Dr Carl June, who pioneered the therapy.

But he and other researchers say thousands more patients eventually may benefit. Kite Pharma’s similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma.

Treatment for other tumours

Scientists around the country also are trying to make CAR-T therapies that could fight more common solid tumours such as brain, breast or pancreatic cancers – a harder next step.

“Although narrow in scope, today’s FDA ruling is a milestone,” said Dr David Maloney of the Fred Hutchinson Cancer Research Center in Seattle, whose team has worked with Juno and is researching CAR-T in a variety of cancers. “Approvals are an important step, but they’re just the beginning.” 

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Viagra is known to give men’s bedroom performance a boost. 

Previous research claimed that the advantages of this drug were not only experienced between the sheets, but that it could also help prevent a heart attack.

However, new research has shown that this is not true.

Not good for pulmonary hypertension

Recent studies suggest that the impotence drug Viagra (sildenafil) might help ease the problem, known as “pulmonary hypertension linked to valvular heart disease”. This happens when one of the heart’s valves goes awry, which can lead to dangerously high blood pressure in the nearby lungs.

But the latest research suggests the medicine might actually do the opposite – and raise patients’ heart risks instead.

In what he called a “surprise” finding, “six-month treatment with sildenafil leads to worse clinical outcomes than placebo,” said lead researcher Dr Javier Bermejo, a cardiologist at University Gregorio Maranon General Hospital in Madrid, Spain.

Finding a surprise

The bottom line: “Long-term usage of Viagrta (sildenafil) for treating residual pulmonary hypertension in patients with valvular heart disease should be avoided.”

Bermejo spoke in a news release from the annual meeting of the European Society of Cardiology, in Barcelona, Spain. He presented his team’s findings at the meeting.

One US heart expert agreed that the finding is a surprise, but noted that many people suffer from pulmonary hypertension, so more study is needed.

“Pulmonary hypertension is defined as increased blood pressure in the lungs,” said Dr Satjit Bhusri, a cardiologist at Lenox Hill Hospital in New York City. “This high pressure can be due to an abnormality within the lungs or as a consequence of long-term high pressures from the heart – such as a structural problem with a heart valve – that are transmitted to the lungs.”

Surgery can correct the valvular issue, he said, and while “the high pressures in the lungs can get better, they may also remain elevated.”

Bhusri noted that in prior studies, Viagra had shown promise in treating this lingering pulmonary high blood pressure. Viagra is a powerful drug that works by widening the blood vessels, and it can have a strong effect on blood flow.

Bermejo said Viagra is often used “off-label” to help treat pulmonary hypertension, even though it’s not approved for such use.

But does the drug actually help? To help answer that question, Bermejo’s team tracked the effectiveness of Viagra in a clinical trial involving 200 patients treated in 17 different hospitals.

Viagra patients worse off

Each of the patients randomly received 40 milligrams of Viagra three times daily or a placebo for a period of six months. Neither the patients nor the researchers knew which patients received the drug.

Over the course of the study, the researchers tracked deaths, hospital admissions for heart failure, tolerance for physical activity and reports of well-being among the participants.

The result: Patients treated with Viagra actually fared worse than those taking the placebo, the Spanish team reported. By six months, 33% of the Viagra patients were worse off than when the study began, compared to 15% of those in the placebo group.

“Compared to patients taking placebo, the chance for worse clinical outcomes … was more than twice as high in those taking sildenafil,” Bermejo noted.

profound negative effects

And the negative effects appeared to affect nearly everyone. “We were unable to identify any particular subset of patients who could potentially benefit from sildenafil,” he said.

For example, patients treated with Viagra had more severe heart symptoms and earlier and more frequent hospital admissions due to heart failure, the researchers said. In fact, these patients had double the risk of needing admission to a hospital than those in the placebo group.

For his part, Bhusri said he was surprised by the finding since, to his knowledge, “there is no physiologic reason to expect such” outcomes from using Viagra in this way.

For that reason, he said, “there needs to be further studies to answer the why and how before we change our approach to patients with elevated lung pressures.”

Dr Puneet Gandotra directs the cardiac catheterization lab at Northwell Health’s Southside Hospital in Bay Shore, New York. He said that while the exact cause of worsening patient outcomes isn’t clear, it’s possible that Viagra interacted in a negative way with other medications the patients were taking.

Experts note that findings presented at medical meetings should be considered preliminary until published in a peer-reviewed medical journal.

Not all bad news

While the above research does show the negative effects of Viagra, it’s important to realise that there are benefits. For example, a recent study showed that Viagra may reduce type 2 diabetes. If you are unsure whether you should be using Viagra or not, discuss this with your doctor.

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