A Breakthrough Therapy’s Food for Thought

Last Wednesday, the Food and Drug Administration approved a new kind of treatment for a type of cancer known as B-cell acute lymphoblastic leukemia (ALL) in patients under the age of 25 who have not been helped by other treatments or who have relapsed.

ALL is a disease of the bone marrow and blood, in which the body makes abnormal lymphocytes, a type of white blood cell. The illness is the most common childhood cancer in the U.S. The National Cancer Institute estimates that 3,100 people 20 and younger are diagnosed with ALL each year.

The federal agency called its decision “historic,” since the treatment, called Kymriah, is the first one to be approved in the U.S that is based on genetic modification.

That is to say, it relies on taking a patient’s own cells — immune agents called T-cells —and then genetically altering them to attack the deadly cancer, after which the “reprogrammed” cells are reintroduced into the patient.

What happens then, at least in a surprisingly large number of cases, is that the modified T-cells multiply in the hundreds of millions and proceed to kill the cancerous ones. In one trial, 83 percent of children who got the treatment went into remission within three months.

Even though a mere single “dose” is enough to send patients into remission, it is an expensive treatment — $475,000 per shot — because of the years of research that yielded the therapy and the complex technology necessary to achieve the genetic engineering.

Hopes are high, although it can’t be known at this point whether successfully treated patients will remain in remission.

Nor is the treatment itself without dangers. An immune overreaction 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. And so, the FDA is requiring Novartis, the company that along with the University of Pennsylvania developed the treatment, to offer it only through medical centers specially trained and certified to handle the complicated treatment.

But the therapy clearly represents a breakthrough, in its use of the body’s own cells to fight a disease. “We’re entering a new frontier in medical innovation,” said FDA Commissioner Scott Gottlieb, “with the ability to reprogram a patient’s own cells to attack a deadly cancer.”

The implication of that fact should not be lost on anyone, especially not on those of us who recognize the Creator of those cells, “asher yatzar es haadam b’chochmah.”

The amazement that people feel when therapies like the newly approved one for ALL arrive on the scene, is really an amazement at the “natural” workings of our bodies. Immune cells are nothing new; they are part of what we are all born with. Only when things go wrong are we forced to recognize the life-saving role such cells play every instant of our lives.

Human beings tend to perk up only at something novel. But we should all be in awe of nisecha sheb’chol yom imanu no less. All that Novartis has done — though it is a technical feat well worthy of acclaim — is harness the body’s natural ability and put it to use. Manipulating the genetic material of a cell is a great accomplishment. But it remains, in the end, a manipulation, a utilization of the existent miracles of cells and of DNA to effect a medical benefit.

Scientists around the country also are currently trying to create therapies like the one for ALL to fight more common solid tumors. There is great medical promise, too, in therapies that utilize stem cells — undifferentiated embryonic cells that can form practically any tissue of the body — to treat any number of diseases, as the cells can theoretically be coaxed to develop into pancreatic cells, which could cure diabetes; into dopamine-secreting brain cells, which could reverse Parkinson’s disease; into muscle, heart, liver or blood cells, which could yield treatments for muscular dystrophy, cardiac disease, liver failure and cancers.

While such developments are currently only on the distant horizon, every healthy person has sufficient numbers of all those very cells working constant miracles, cells that once developed from embryonic stem cells of their own, the ones we are all born with.

Once upon a time, heart transplants were flabbergasting. But, at least to thoughtful people, they were never remotely as amazing as hearts.

Developments like the new treatment for ALL understandably seize our attention, and we rightly feel gratitude to the researchers and doctors who helped make it possible. But such developments should also leave us with a deeper awareness of the manifold miracles we routinely experience, a deeper realization of the fathomless debt we owe our Creator.