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Breakthrough Therapy Designation for Novel Immunotherapy

Posted by andreahgreif in The LLS Blog, 08 July 2014 · 4,775 views

Today we learned the exciting news that the FDA has granted Breakthrough Therapy designation for an investigational immunotherapy that LLS has played a significant role in helping to advance. The therapy, CTL019, also known as chimeric antigen receptor (CAR) therapy, is an approach that genetically engineers patients’ immune T cells and reintroduces them into the body to kill cancer cells. The work is being led by Carl June, M.D., at University of Pennsylvania, who, along with his team, LLS has been funding for more than 16 years with a commitment of more than $21 million through 2017.

The breakthrough designation puts the therapy on track for expedited review to bring it more quickly to patients.

The breakthrough designation is specifically for clinical trials testing the approach for pediatric and adult patients with relapsed or refractory acute lymphoblastic leukemia (ALL). Trials employing this approach are also under way for relapsed and refractory chronic lymphocytic leukemia (CLL), as well as for patients with non-Hodgkin lymphoma and myeloma.

CTL019 is the first genetically-engineered cancer immunotherapy to receive the breakthrough designation and only the fifth biologic agent so far.

Early-stage clinical trials at the Hospital of the University of Pennsylvania and the Children’s Hospital of Philadelphia have already shown encouraging results for CAR therapy. To date, 89 percent of ALL patients who were not responding to conventional therapies went into complete remission after receiving the engineered T cells.

During the 2013 annual meeting of the American Society of Hematology the Penn research team announced study results of the first 27 ALL patients (22 children and five adults) treated with CTL019. The first pediatric ALL patient to receive the Penn therapy celebrated the second anniversary of her cancer remission in May, and the first adult patient remains in remission one year after receiving the therapy. That first pediatric patient is Emily Whitehead, now 9, who LLS had the pleasure of hosting, along with her parents, at our conference, “Leading the Way to a World Without Blood Cancers,” in Washington D.C. in May.

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