FDA Approval of a “Living Drug” Heralds a New Era in Cancer Care

The FDA approves the first “living drug” cell therapy for childhood leukemia.


Update: Just a week after the FDA approved  Novartis’ Kymriah CAR-T cell therapy, the FDA has put on hold further testing of biopharmaceutical company Cellectis’ own CAR-T drug after the recent death of a subject in their clinical trials.

--

On August 30, the FDA made history by approving the first “living drug” in the U.S. It genetically modifies the patient’s own blood cells to enlist them as tiny warriors that seek out and destroy cancer cells, in this case, those associated with childhood leukemia. The newly approved drug, Kymriah, is a CAR-T cell treatment developed by Novartis and the University of Pennsylvania. Its arrival heralds a new chapter in the treatment of blood cancers and some tumors. In the FDA press release, Commissioner Scott Gottlieb says, “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.”

“This is a brand-new way of treating cancer,” Dr. Stephan Grupp of Children’s Hospital of Philadelphia tells AP, “That’s enormously exciting.” Grupp is Emily Whitehead’s doctor — she was the first patient to receive the cell therapy, and though she was as close to 48 hours from organ failure when first enrolled in the experimental treatment, Whitehead has now been cancer-free for five years.

Whitehead and her parents today (CHILDRENS HOSPITAL OF PHILADELPHIA)

Whitehead’s family discusses their difficult decision. (CHILDRENS HOSPITAL OF PHILADELPHIA)

CAR-T was first developed by a group of doctors some five years ago and gained notice when Whitehead began treatment. With CAR-T, some T-cells are removed from a patient’s blood. A chimeric antigen receptor (CAR) receptor is added to them, and the enhanced T-cells are then returned to the patient’s blood stream, where they’re newly capable of hunting down and destroying cancer cells. Kymriah handles this turbocharging within the patients body.

(STILIYAN PETROV FOUNDATION)

One of the doctors who developed the method, Carl June, notes that the therapeutic effects of CAR-T can be startling. In 2016, he told Popular Science about one patient who, after seeming at first not to respond, suddenly got better. When his blood was analyzed, it turned out that every single CAR-T cell he had had descended from a single T-cell. June recalled, "We infused 100 million into him, and just one of them did all the heavy lifting."

What’s perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer.

As with all treatments, there is a risk. When the body’s immune system kicks into high gear to fight the cancer, extremely high fevers can occur as well as the production of proteins that produce inflammation. Though these signify that the treatment’s actually working, they’re also dangerous conditions in and of themselves, and require carefully monitoring and treatment. This is one of the reasons Kymriah's cleared for use only under very specific circumstances.

It’s too soon to tell how widely applicable CAR-T will be — it’s especially suited to leukemia since infusing patients with T-cells is relatively simple. Research is therefore focusing for now on refining the therapy for patients of this disease. CAR-T treatment of other cancers will have to wait for the time being.

​There are two kinds of failure – but only one is honorable

Malcolm Gladwell teaches "Get over yourself and get to work" for Big Think Edge.

Big Think Edge
  • Learn to recognize failure and know the big difference between panicking and choking.
  • At Big Think Edge, Malcolm Gladwell teaches how to check your inner critic and get clear on what failure is.
  • Subscribe to Big Think Edge before we launch on March 30 to get 20% off monthly and annual memberships.
Keep reading Show less

Why are so many objects in space shaped like discs?

It's one of the most consistent patterns in the unviverse. What causes it?

Videos
  • Spinning discs are everywhere – just look at our solar system, the rings of Saturn, and all the spiral galaxies in the universe.
  • Spinning discs are the result of two things: The force of gravity and a phenomenon in physics called the conservation of angular momentum.
  • Gravity brings matter together; the closer the matter gets, the more it accelerates – much like an ice skater who spins faster and faster the closer their arms get to their body. Then, this spinning cloud collapses due to up and down and diagonal collisions that cancel each other out until the only motion they have in common is the spin – and voila: A flat disc.

Scientists study tattooed corpses, find pigment in lymph nodes

It turns out, that tattoo ink can travel throughout your body and settle in lymph nodes.

17th August 1973: An American tattoo artist working on a client's shoulder. (Photo by F. Roy Kemp/BIPs/Getty Images)
popular

In the slightly macabre experiment to find out where tattoo ink travels to in the body, French and German researchers recently used synchrotron X-ray fluorescence in four "inked" human cadavers — as well as one without. The results of their 2017 study? Some of the tattoo ink apparently settled in lymph nodes.


Image from the study.

As the authors explain in the study — they hail from Ludwig Maximilian University of Munich, the European Synchrotron Radiation Facility, and the German Federal Institute for Risk Assessment — it would have been unethical to test this on live animals since those creatures would not be able to give permission to be tattooed.

Because of the prevalence of tattoos these days, the researchers wanted to find out if the ink could be harmful in some way.

"The increasing prevalence of tattoos provoked safety concerns with respect to particle distribution and effects inside the human body," they write.

It works like this: Since lymph nodes filter lymph, which is the fluid that carries white blood cells throughout the body in an effort to fight infections that are encountered, that is where some of the ink particles collect.

Image by authors of the study.

Titanium dioxide appears to be the thing that travels. It's a white tattoo ink pigment that's mixed with other colors all the time to control shades.

The study's authors will keep working on this in the meantime.

“In future experiments we will also look into the pigment and heavy metal burden of other, more distant internal organs and tissues in order to track any possible bio-distribution of tattoo ink ingredients throughout the body. The outcome of these investigations not only will be helpful in the assessment of the health risks associated with tattooing but also in the judgment of other exposures such as, e.g., the entrance of TiO2 nanoparticles present in cosmetics at the site of damaged skin."

Photo by Alina Grubnyak on Unsplash
Mind & Brain

Do human beings have a magnetic sense? Biologists know other animals do. They think it helps creatures including bees, turtles and birds navigate through the world.

Keep reading Show less