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14 - CAR T-cell Therapy: Cures for ALL and More?

Published online by Cambridge University Press:  19 January 2018

Philip A. Rea
Affiliation:
University of Pennsylvania
Mark V. Pauly
Affiliation:
University of Pennsylvania
Lawton R. Burns
Affiliation:
University of Pennsylvania
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Summary

Doug Olson was first diagnosed with chronic lymphocytic leukemia (CLL) in 1996. Come 2009, after six years of chemotherapy and five years of remission, the 64-year-old was informed that his cancer was back and no longer responding to therapy. His only hope of surviving with a refractory CLL diagnosis for more than another two years was a bone marrow transplant, which carried a 22 percent risk of treatment-related mortality and at best a 48 percent chance of complete remission.

Not long after Doug's grim diagnosis, at the other end of the age spectrum, Emma (also known as Emily) Whitehead, a five-year-old, was diagnosed with acute lymphocytic leukemia (ALL). For many children afflicted with ALL the prognosis is not as bleak as it could be. Two years of intensive chemotherapy followed by consolidation therapy is often sufficient to rid them of the disease. Alas, that was not to be the case for Emma. After her second relapse in 2012, Emma's ALL was deemed to be refractory.

In one last ditch effort, Doug and Emma were among the first to undergo chimeric antigen receptor (CAR) T-cell therapy at the University of Pennsylvania and the Children's Hospital of Philadelphia (CHOP), respectively. As of now, some six years later, both Doug and Emma remain cancer-free.

EMPEROR OF ALL MALADIES

Cancer, the emperor of all maladies, still kills more than eight million people per year and accounts for 13 percent of all deaths worldwide. Great strides have undoubtedly been made in treating this disease, or more correctly this group of diseases, but the frustrating truth is that progress has been patchy and much remains to be done. Some cancers respond well to treatment if caught early, practically all have a poor prognosis if caught late, and there are many that pose a huge therapeutic challenge regardless of when they are caught.

We all know it: more often than not, cancer treatment comes at a great price, whether measured in patient suffering and years of life lost or the sheer expense of surgery, chemotherapy, and/or radiation therapy. Add to that the psychological torment of having to live for months or years in fear of still more of the same again – relapse. Never before, as populations increase and age, has the need been so great for novel truly curative cancer therapies that do patients more good than harm.

Type
Chapter
Information
Managing Discovery in the Life Sciences
Harnessing Creativity to Drive Biomedical Innovation
, pp. 417 - 448
Publisher: Cambridge University Press
Print publication year: 2018

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