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US clears first gene therapy for childhood leukemia

US regulators have approved the first treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia.

Experts hailed Wednesday’s move as ‘historic’, opening a new era in cancer care. 

The CAR-T cell treatment developed by Novartis Pharmaceuticals and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market.

It is one in a powerful but expensive wave of custom-made ‘living drugs’ being tested against blood cancers and some other tumors, too.

Hope for kids with leukemia: A patient, Emily Whitehead, five years after she became the first pediatric patient in the world to receive an experimental therapy at the Children’s Hospital of Philadelphia that has put her leukemia into long-term remission

HOW DOES CAR-T TREATMENT WORK? 

CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. 

In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancers. 

Millions of copies of the new T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and destroy cancer cells. 

Doctors call it a ‘living drug’ – permanently altered cells that continue to multiply in the body into an army to fight the disease. 

‘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.’

Novartis said it would charge $475,000 for the treatment, made from scratch for every patient. 

But, the company said there would be no charge if the patient didn’t show a response within a month.

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, reprogram them to harbor a ‘chimeric antigen receptor’ 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 tumors. 

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 reprogram a patient’s own cells to attack a deadly cancer,’ said FDA Commissioner Scott Gottlieb.

This first use of CAR-T therapy is aimed at patients desperately ill with a common pediatric cancer – acute lymphoblastic leukemia, or ALL – that strikes more than 3,000 children and young adults in the U.S. each year. 

While most survive, about 15 percent relapse despite today’s best treatments, and their prognosis is bleak.

In a key study of 63 advanced patients, 83 percent went into remission. 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 leukemia,’ said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, 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.

Most patients suffered side effects that can be grueling, even life-threatening. 

An immune overreaction called ‘cytokine release syndrome’ can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Also Wednesday, 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, ‘It isn’t a panacea.’

An IV bag of Novartis's drug Kymriah. On Wednesday, the Food and Drug Administration approved this first treatment that genetically engineers patients' own blood cells to seek and destroy childhood leukemia

An IV bag of Novartis’s drug Kymriah. On Wednesday, the Food and Drug Administration approved this first treatment that genetically engineers patients’ own blood cells to seek and destroy childhood leukemia

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

‘Unfortunately leukemia grows so rapidly that it can evade even the smartest of our technologies,’ he added.

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 U.S.,’ 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.’

Initially, Novartis’ CAR-T version – to be sold under the brand name Kymriah – will be available only through certain medical centers specially trained to handle the sophisticated therapy and its side effects.

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.

While this first use of CAR-T therapy only is aimed at a few hundred U.S. patients a year – relapsed ALL patients up to age 25 – it’s being tested as a treatment for thousands more. 

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.

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

‘CAR-T SAVED OUR LIVES’: THE FACES OF PATIENTS IN REMISSION AFTER GETTING GENE THERAPY

MIKE, 32, WHO TRAVELED TO PENNSYLVANIA FROM BRITAIN

Mike Brandon, 32, is now disease free after having CAR-T cell therapy last summer.

He was only able to have the breakthrough procedure after his wife Kate raised more than £450,000 through a social media campaign.

Mr Brandon, from Bristol, was diagnosed with acute lymphoblastic leukaemia in January 2014.

The illness was so advanced that his consultant told him that ‘all NHS routes were exhausted.’

He began CAR-T therapy in May 2016 at the University of Pennsylvania, and in October announced that he was cancer-free.

Mrs Brandon said at the time: ‘We have more tests ahead but, right now, we couldn’t be happier.

‘Prior to starting the trial, Mike’s bone marrow was made up of 90 per cent leukaemia cells and he was given weeks to live but thanks to the cutting edge CAR T-cell therapy he received in the US he is now cancer free.’

When launching the campaign, she had described her husband as a ‘kind, sincere and witty’ man who ‘deserved to have a chance at living life.’ 

Mike Brandon (pictured with his partner), from Bristol, was diagnosed with acute lymphoblastic leukaemia in 2014. He was announced cancer-free after CAR-T in 2016

Mike Brandon (pictured with his partner), from Bristol, was diagnosed with acute lymphoblastic leukaemia in 2014. He was announced cancer-free after CAR-T in 2016

ERIN, 6, WHO RAISED $180,000 TO GET TO SEATTLE FROM THE UK 

Six-year-old Erin Cross battled acute lymphoblastic leukemia for three years and had exhausted all other treatment options.

Her only chance of beating the disease was to have a bone marrow transplant – but needed to be in remission first.

And so her parents Sarah and Anthony discovered a clinic in the US which they believed to be their last hope, and traveled there in summer 2016.

After months of experimental tests, Erin was discharged from Seattle Children’s Hospital in September 2016.

A week later, back home in Chester, England, they received a phone call – to say she is officially in remission. 

AVA, 8, WHO WAS DECLARED ‘CANCER FREE’ AFTER RELAPSING 5 TIMES

When Ava Christianson was diagnosed with cancer at the age of four, doctors said she had the best odds.

She had the most common type of leukemia, acute lymphoblastic leukemia (ALL). In 90 per cent of cases, a conventional course of chemotherapy does the trick.

But her experience was different.

By the age of eight, Wisconsin-born Ava had tried all of the standard therapies – from chemo to a bone marrow transplant – and relapsed five times.

However, since she was first diagnosed researchers have made astonishing strides in the field of immunotherapy, training patients’ bodies to fight their tumors. 

Ava became the 18th person in history to try the new five-minute operation at the National Institutes of Health’s Clinical Center.

In August 2016, doctors gave her the all-clear. 

Ava Christianson, eight, was diagnosed with cancer at the age of four. She tried a new kind of therapy at the NIH clinic in Maryland last year, and was declared in remission

Ava Christianson, eight, was diagnosed with cancer at the age of four. She tried a new kind of therapy at the NIH clinic in Maryland last year, and was declared in remission

TWO LONDON BABIES ‘CURED’ OF TERMINAL LEUKEMIA BY GENE THERAPY

Two babies who were diagnosed with terminal leukemia were said to be cured thanks to CAR-T in London.

Layla Richards, 16 months, and an unidentified 11-month-old were injected with genetically engineered immune cells at London’s Great Ormond Street Hospital last year.

The immune cells were designed to attack cancer cells after previous attempts to treat the infants using traditional methods had failed.

In January this year, doctors described the children’s response to the treatment as ‘almost a miracle’ and ‘staggering’ – hailing it as the world’s first successful treatment of cancer.

The experiment raised the possibility of off-the-shelf cellular therapy using donor cells at low cost that could be dripped into patients’ veins at a moment’s notice. 

Read more at DailyMail.co.uk


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