NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthful patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little damsel was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the fine privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a view to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

This achievement in immunotherapy, tho’ quintessentially modern, is the culmination of more than a century of scientific research. It all commenced back in 1890, when Fresh York-based surgeon William Coley found a handful of patients who survived evidently fatal cancers after they suffered an acute infection. He developed a toxin—“Coley’s toxin”—that he used stimulate patients’ immune systems to treat their cancer. He believed his treatment worked. The broader medical profession was skeptical, and failure was all too common.

Over the decades, medical interest in immunotherapy waxed and waned; and researchers developed an array of immunotherapeutic interventions, to varying effect. Ultimately, a number of basic science advances in immunology, cell biology, and genetics were needed to make real progress in immunotherapy possible.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the uncommon case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Three]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest investigate demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials showcased the treatment to be enormously promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Many questions must be addressed before we can herald immunotherapeutic approaches to cancer an unqualified success. There are still too many severe reactions, too many non-responses or relapses, and, potentially, a very high price tag for their widespread use, which will be truly challenging to scale up. But we’re off to a promising begin.

Emily keeps smiling. Her smile gives me hope. Watching her grow from a youthfull lady fighting with a panicking disease into a poised youthfull woman who looks forward to beginning the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m anxious to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress proceeds to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of utterly hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little female was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the excellent privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

This achievement in immunotherapy, however quintessentially modern, is the culmination of more than a century of scientific research. It all commenced back in 1890, when Fresh York-based surgeon William Coley found a handful of patients who survived evidently fatal cancers after they suffered an acute infection. He developed a toxin—“Coley’s toxin”—that he used stimulate patients’ immune systems to treat their cancer. He believed his treatment worked. The broader medical profession was skeptical, and failure was all too common.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Three]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest explore demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials displayed the treatment to be enormously promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Observing her grow from a youthfull dame fighting with a panicking disease into a poised youthful woman who looks forward to embarking the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m impatient to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most titillating areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthful adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little chick was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the superb privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a glance to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

This achievement in immunotherapy, tho’ quintessentially modern, is the culmination of more than a century of scientific research. It all embarked back in 1890, when Fresh York-based surgeon William Coley found a handful of patients who survived evidently fatal cancers after they suffered an acute infection. He developed a toxin—“Coley’s toxin”—that he used stimulate patients’ immune systems to treat their cancer. He believed his treatment worked. The broader medical profession was skeptical, and failure was all too common.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the uncommon case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Trio]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest explore demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials showcased the treatment to be utterly promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthfull damsel fighting with a scaring disease into a poised youthfull woman who looks forward to embarking the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m antsy to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most titillating areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthfull patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little dame was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the fine privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a look to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

This achievement in immunotherapy, however quintessentially modern, is the culmination of more than a century of scientific research. It all embarked back in 1890, when Fresh York-based surgeon William Coley found a handful of patients who survived evidently fatal cancers after they suffered an acute infection. He developed a toxin—“Coley’s toxin”—that he used stimulate patients’ immune systems to treat their cancer. He believed his treatment worked. The broader medical profession was skeptical, and failure was all too common.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Trio]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest examine demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials demonstrated the treatment to be utterly promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthfull chick fighting with a panicking disease into a poised youthfull woman who looks forward to embarking the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m anxious to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress proceeds to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little chick was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the fine privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a look to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest probe showcased that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthful doll fighting with a scaring disease into a poised youthful woman who looks forward to commencing the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m impatient to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most titillating areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of utterly hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthfull patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little damsel was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the good privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest examine displayed that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials showcased the treatment to be utterly promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthful female fighting with a scaring disease into a poised youthful woman who looks forward to commencing the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m antsy to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress proceeds to be made against the Emperor of All Maladies, cancer. One of the most titillating areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of utterly hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthful adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little damsel was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the superb privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a glance to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Three]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest explore showcased that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthful lady fighting with a panicking disease into a poised youthful woman who looks forward to commencing the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m antsy to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of utterly hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthful adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthfull patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little female was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the good privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a look to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Three]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest examine showcased that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials displayed the treatment to be utterly promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Observing her grow from a youthful woman fighting with a panicking disease into a poised youthfull woman who looks forward to beginning the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m impatient to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress proceeds to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthfull patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little woman was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the superb privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a glance to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Trio]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest probe showcased that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

Emily keeps smiling. Her smile gives me hope. Watching her grow from a youthfull dame fighting with a panicking disease into a poised youthful woman who looks forward to embarking the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m impatient to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress resumes to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of utterly hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthful adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little lady was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the superb privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

CAR-T cells have been called “a living drug” because doctors collect and manipulate a patient’s own immune cells to treat his or her cancer. Specifically, the harvested T cells are genetically engineered to produce fresh surface proteins (the CARs, or chimeric antigen receptors) that permit them to recognize and attack cancer cells more effectively. After expanding the number of these enhanced T cells, doctors infuse them back into patients to soup up their immune systems. The capability of T cells to attack a cancer cell is a glance to behold. Our capability to corset and direct that power is a true miracle of modern medicine.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Three]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest explore demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials showcased the treatment to be utterly promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Eyeing her grow from a youthful chick fighting with a scaring disease into a poised youthfull woman who looks forward to beginning the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.

I’m impatient to see where our immunotherapy researchers take us next!

FDA Approves Very first CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia, NIH Director – s Blog

NIH Director's Blog

Caption: Cancer survivor Emily Whitehead with her dog Lucy.

Credit: Emily Whitehead Foundation

Tremendous progress proceeds to be made against the Emperor of All Maladies, cancer. One of the most arousing areas of progress involves immunotherapy, a treatment strategy that harnesses the natural capability of the body’s own immune cells to attack and kill tumor cells. A lot of enormously hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its very first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and youthfull adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about ninety percent of youthfull patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little damsel was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents fresh hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,Two]. Moving forward with extra clinical tests, they treated Emily—their very first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the good privilege of getting to know Emily and her parents over the last few years.

Caption: CAR-T cell therapy involves genetically engineering immune T cells to recognize specific proteins, or antigens, on tumor cells and attack them.

Credit: Carl June, University of Pennsylvania, Philadelphia

This achievement in immunotherapy, tho’ quintessentially modern, is the culmination of more than a century of scientific research. It all began back in 1890, when Fresh York-based surgeon William Coley found a handful of patients who survived evidently fatal cancers after they suffered an acute infection. He developed a toxin—“Coley’s toxin”—that he used stimulate patients’ immune systems to treat their cancer. He believed his treatment worked. The broader medical profession was skeptical, and failure was all too common.

These advances came largely in the 1970s and 1980s—many with NIH support. They were recognized by visionaries, such as NIH’s own Steven Rosenberg, as providing fresh insights into longstanding questions about how to train our immune systems to recognize and fight cancer. Rosenberg, still at NIH’s National Cancer Institute (NCI), had been thinking about immunotherapy since the 1960s, when he witnessed the infrequent case of a patient with terminal cancer who appeared to be cured by a separate infection. Drawing upon fresh scientific skill, Rosenberg had early success in the 1980s with a precursor to CAR-T therapy [Trio]. More recently, he developed and successfully administered his own version of CAR-T [Four].

With immunotherapy, as with so many of our medical interventions, the path from original discovery or observation to effective treatment is long, circuitous, and densely populated. In fact, a fascinating latest probe demonstrated that the development of another form of cancer immunotherapy, so-called “checkpoint inhibitors,” arose from the work of some 7,067 scientists, toiling over the course of a century [Five]. Immunotherapy is indeed the achievement of many minds. At the same time, the commitment and passion of researchers such as June and Rosenberg—and James Allison of the University of Texas MD Anderson Cancer Center, Houston, for checkpoint inhibitors—have been essential to pushing past obstacles and skeptics, and bringing us to this remarkable moment in medicine’s history.

As it turns out, the very same year that June and his colleagues successfully treated Emily, they partnered with the Swiss-based pharmaceutical company Novartis to develop commercially a version of CAR-T therapy, now called Kymriah™ (tisagenlecleucel). Further trials demonstrated the treatment to be enormously promising. In July, an FDA advisory committee met to consider whether to recommend approval of the therapy. With his daughter by his side, Emily`s father, Thomas, gave impassioned and eloquent testimony on CAR-T’s behalf. In the end, the committee voted unanimously to recommend that the FDA approve the treatment. And, today, the FDA followed that recommendation and gave formal approval.

Emily keeps smiling. Her smile gives me hope. Watching her grow from a youthfull dame fighting with a panicking disease into a poised youthfull woman who looks forward to embarking the seven th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.