FDA approves a breakthrough hailed as a ‘new frontier’ in medicine

FDA approves a breakthrough hailed as a ‘new frontier’ in medicine.

Kymriah

The Federal Drug Administration (FDA) in the United States has approved a treatment that has been hailed as opening a ‘new frontier’ in medicine.

The treatment is called CAR T-cell therapy and uses a patient’s own genetically modified cells to attack a type of leukaemia.

“This is a dream come true,” said Henry Fung, Director of Philadelphia’s Fox Chase Cancer Center at Temple University Hospital’s Bone Marrow Transplant Program. “This is the breakthrough of the century.”

Dr Fung believes the treatment could help to defeat an illness that resists conventional treatments like chemotherapy and radiation, leaving patient’s facing death.

This new approach to fighting cancer harnesses the body’s immune system – a long-sought goal of medical researchers.

Dr Hetty Carraway, an acute leukaemia doctor at the Cleveland Clinic, says the newly approved therapy could be the first step to a new way of treating cancer.

“If it can bring this kind of paradigm to other types of cancers, that’s where I think the larger implications are,” said Dr Carraway.

Currently, the FDA is looking at 76 new drug applications related to CAR T-cell therapy for ailments such as genetic disorders, autoimmune diseases, diabetes, cancer and HIV.

"New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses," said FDA Commissioner Scott Gottlieb.

The new treatment goes by the brand name ‘Kymriah’. It is also known as chimeric antigen receptor T-cell therapy.

How does it work?
Doctors extract a patient’s white blood cells (T cells), freeze them and and ship them off to a laboratory where they’re genetically engineered to attack a specific protein in cancerous B cells.

“This is really combining everything together,” said Dr Fung. “This is truly using patients’ own immune cells to fight cancer.”

There are some drawbacks, such as the cost, which is just under $600,000, and the side effects, which include cytokine disease, anaemia, nausea, diarrhoea, and headaches. 

“We know and expect that type of side effect will happen, and we know that we can successfully manage it,” said Dr Carraway.

Other questions also remain, such as how long the CAR T-cells remain in the body. In some patients, they’ve lasted up to five years, while in others, they’ve died out in a few weeks.

That leaves the final question: whether cancer will return if the CAR T-cells are gone.

Read more at WebMD

Have you heard about this treatment before? How much would you pay for a fighting chance at beating leukaemia?

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    COMMENTS

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    Jennie
    4th Sep 2017
    10:54am
    Sounds very good but the key words here could be, "How much would you pay for a fighting chance at beating leukaemia?" Depends what you can afford...
    Puglet
    4th Sep 2017
    12:06pm
    Car-T cell therapy is a POTENTIAL medical break through and is very exciting for everyone but especially for the companies who have patented this therapy. Calling it the medical break through of the century is ridiculous and is just an advertising gimmick. In 10 years when a few thousand people have survived a few years we'll know just how good it is. The therapy will only be used for patients for whom all other treatments do not work. It's very unethical to suggest to patients desperate to stay with their loved ones a bit longer that a 600,000 dollar therapy may mean they may survive for a few years although an unspecified number will die in a few weeks especially since most families will need to go into severe debt for a treatment which may or may not work. Of course the definition of 'work' is different for everyone so I can't pass judgement.
    Jennie
    4th Sep 2017
    12:17pm
    Good comment Puglet. And just thinking of those American families who sell their homes in order to pay for leukaemia treatment for their children. Also what about the side effects? They could kill you. Can those side effects really be controlled? I don't believe so.
    KSS
    4th Sep 2017
    12:45pm
    "There are some drawbacks, such as the cost, which is just under $600,000, and the side effects, which include cytokine disease, anaemia, nausea, diarrhoea, and headaches."

    Pretty significant drawbacks don't you think? Especially when you don't know what happens after the CAR T cells have been replaced.
    Patriot
    4th Sep 2017
    1:03pm
    Quality of life overshadows quantity ANY Time!
    Once quality is gone: "Stuff the Quantity".
    arbee
    5th Sep 2017
    2:36pm
    Who do you think should be paying the $600,000.00? The government, which means taxpayers, because there are limits to how much can be spent on health and every dollar spent on this would mean a dollar less spent elsewhere on health.
    Jenny
    4th Sep 2017
    2:37pm
    I could never compromise my family's financial stability in order to gain a few more years, especially when the treatment is unproven.
    ROB
    4th Sep 2017
    4:28pm
    Wow! When we already have a product to successfully raise Glutathione and Carnosine levels to heal and boost the immune system, with extensively studies proving the effectiveness and safety of this technology that has NO Harmful side effects and costs under 1200 TIMES LESS than the "Breakthrough of the century", then one wonders at the madness of all this?
    If I ever had any of the issues that this ‘Kymriah’ is attempting to “fix” I know what I would be using and that is something we have already found proven, simple to apply and already being used worldwide by millions of people for all ages.
    Jolly
    5th Sep 2017
    7:14am
    So Rob, what is this magic cure that only you know about but have neglected to tell us about.
    ROB
    5th Sep 2017
    4:52pm
    The short answer Jolly is that we use a new technology the same as now used in hospitals in Europe and elsewhere about the world. It is not openly advertised in Australia despite being readily available here and even accepted by the TGA. This is because it can easily be misunderstood until one has the opportunity to learn more. It is not a "Magic cure" but something already found to improve organ function with controlled clinical measurements - something other "break throughs" do not appear to have? We make the suggestion to keep an open mind and look for something already proven as safe, low in cost, effective and without harmful side effects. You are welcome to enquire if you would like further information on availability, research etc.

    Rob wellnessplus@adam.com.au
    Jennie
    5th Sep 2017
    6:15pm
    Rob it seems that you are advertising a product, hence the secrecy. Do you sell this? Is this your business?
    ROB
    5th Sep 2017
    6:43pm
    Hi Jennie, our task around the world is for assisting people with the use and application of the product toward a satisfactory result and my partner and I do this, ongoing, world wide free and of charge. Yes, we can assist with purchase if needed anywhere but there are also hundreds of thousands of others we can direct enquirers to. Our primary aim is for assisting people gain a far better quality of life. We do not promote for selling through YLC as that would conflict with the aims of YLC. However in already personally assisting thousands of people around the world I feel it is something others may like to know about?