FDA OKs Leukemia Treatment That Trains Cells To Fight Cancer

Today, the Food and Drug Administration approved a gene therapy known as vehicle T-cell therapy that genetically modifies a patient's own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer.

With as many as 3,100 patients under the age of 20 diagnosed with acute lymphoblastic leukaemia each year, the treatment known as Kymriah has been made available to all patients under the age of 25 in an attempt to try and treat the most common form of cancer in children.

The drug, called Kymriah, is a highly personalized cancer treatment called vehicle T-cell therapy (CAR is short for chimeric antigen receptor). "This is the first-ever true gene therapy treatment made available to the United States population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition". The customized treatment to be administered at certified medical centers involves drawing a patient's blood, genetically engineering their white blood cells, called T cells, turning them into a "living drug" that fights the disease, before re-dripping them back into the patient.

However, this type of therapy carries risk of severe side effects.

"The Novartis approval is also a momentous milestone in the path for cell therapies reaching patients - the final objective of those of us involved in developing therapies", said Catherine Bollard, President, ISCT and invited for the FDA Oncologic Drugs Advisory Committee Novartis CAR-T decision as an "invited subject matter expert".

"Of course, we have to talk about pricing from a national standpoint", he said.

The drug has shown promising remission and survival rates in clinical trials, the FDA said. "The real question is going to come when this therapy is poised to be expanded into other patients who are older, and providing the chance for 60 or 70 years of [additional] life is not what you're talking about, and then we're really going to have some tough value questions that we should be prepared to ask".

"The efforts involved in being able to provide this novel therapy to patients is unlike any other therapy now offered", says Colleen Dansereau, MSN, RN and CPN, who is director of clinical research nursing and gene therapy program manager at Dana-Farber/Boston Children's.

Epstein said he envisioned cell therapies having much shorter life cycles than traditional drugs. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

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Those updates could increase the number of patients that cell therapies can treat.

The treatment is the first of its kind approved in the U.S.

Kymriah is the first CAR-T cell therapy to get approved, and there are several more in the works.

They'd also have to be less toxic so that more people could consider it, instead of only those who have very few other options.

There could be side effects.

The high cost of the therapy reflects the fact that the CAR-T procedure is fairly intensive.

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment.

And then, to impact more people, the cell therapies will need to go beyond the blood cancers.

  • Myrtle Hill