Phase I Trial of Immunotherapy Opens for Children with Relapsed or Treatment-resistant Leukemia
May 11, 2015
breakthrough method for turning patients' immune system against their cancer
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Dana-Farber/Boston Children's Cancer
and Blood Disorders Center has joined a clinical trial of immunotherapy for
children with relapsed or treatment-resistant acute lymphoblastic leukemia
(ALL). Led by Memorial Sloan Kettering Cancer Center (MSKCC), the trial is one
of several nationally that are evaluating cancer immunotherapy, a treatment
approach—hailed by Science magazine
as their Breakthrough of the Year in 2013—that triggers a patient's immune system
to attack his or her cancer cells. Dana-Farber/Boston Children’s is the only
site in New England to offer this experimental approach for children and
adolescents whose ALL has relapsed multiple times or never gone into remission.
The innovative trial starts with a patient's own infection-fighting
T-cells. These cells are collected and modified using gene therapy techniques
to produce a cancer-hunting protein called a chimeric antigen receptor (CAR).
Once infused back into the patient, the CAR then prompts the modified T-cells
to attack leukemia cells by seeking out and recognizing a protein they carry on
their surface. The CAR also spurs the modified T-cells to divide and grow once
they come in contact with ALL cells, fueling a more robust immune response.
"There has been evidence for many years that the immune system can
be used to help control leukemia, but we have yet to find the best way to
harness it to treat disease," said Lewis
Silverman, MD, clinical director of the Hematologic
Malignancies Center at Dana-Farber/Boston Children's and the local lead investigator on the
trial. "CAR-modified T-cell clinical trials have shown promising results
in adults and children with leukemias, and we are very excited to test this
treatment in children and adolescents with ALL.”
This pediatric CAR T-cell trial is available only at Dana-Farber/Boston
Children's and MSKCC. The trial is open to patients up to age 26 with relapsed
B-cell ALL who have not yet undergone a hematopoietic stem cell transplant. The
trial joins Dana-Farber/Boston Children’s already robust portfolio of clinical
trials for both leukemia and gene therapy.
Approximately 2,900 children in the United States are diagnosed each
year with ALL, the most common cancer of childhood. Although 80-90 percent of
patients go on to become long-term survivors, 15-20 percent of children who
achieve an initial complete remission later experience a relapse. The prognosis
for these patients depends on a number of factors, including the site of
relapse, length of time between initial diagnosis and relapse, and biological
features of the relapsed cells.
The Phase I trial is the first clinical step in the Food and Drug
Administration's process for approving a new drug or treatment. The goal is to
establish the treatment's safety in a small group of patients, with an eye
toward larger studies of safety and efficacy should the Phase I trial succeed.
Each trial participant will be followed very closely for side effects as well
as treatment response. Because the trial involves gene therapy, participants
will be followed for 15 years following treatment.
Find detailed information about the trial, including eligibility criteria.
Questions about eligibility should be directed to firstname.lastname@example.org.
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