Correspondence
A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
N Engl J Med 2003; 348:255-256January 16, 2003
- Article
To the Editor:
We recently reported (April 18 issue)1 the sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease. These results have since been confirmed in four additional patients with typical X-linked severe combined immunodeficiency. Of the first four successfully treated patients, three continue to do well up to 3.6 years after gene therapy, whereas a serious adverse event occurred in the fourth patient. At a routine checkup 30 months after gene therapy, lymphocytosis consisting of a monoclonal population of Vγ9/Vδ1, γ/δ T cells of mature phenotype was detected. One proviral integration site was found, located on the short arm of chromosome 11 within the LMO-2 locus, as determined with the use of linear-amplification mediated polymerase-chain-reaction analysis.2 This proviral integration within the LMO-2 locus was associated with aberrant expression of the LMO-2 transcript in the monoclonal T-cell population. Aberrant expression of LMO-2 has been reported in acute lymphoblastic leukemia arising from T cells with α/β receptors, usually with the chromosomal translocation t(11;14).3 Tests for replication-competent retrovirus were repeatedly negative in our patient's lymphocytes.
Between 30 and 34 months after gene therapy, the patient's lymphocyte count rose to 300,000 per cubic millimeter, and hepatosplenomegaly developed. Further investigations showed the presence of a t(6;13) translocation, which had not been detected 30 months after the therapy. Treatment with a chemotherapy regimen based on a high-risk protocol for acute lymphocytic leukemia (a protocol of the Dutch Childhood Leukemia Study Group) was initiated and has resulted, to date, in a dramatic reduction in the abnormal cells.
We interpret these findings as the consequence of the insertional mutagenesis event, a risk that is potentially associated with retrovirally mediated gene transfer and that has previously been considered to be very low in humans.4 For this reason, a thorough reassessment of the potential risk of retrovirally mediated gene therapy is warranted. It is likely that additional factors may have contributed to the adverse event in our patient, including a varicella–zoster virus infection five months before clinically detectable lymphoproliferation, which may have stimulated immune reactivity of the γ/δ T-cell clone, or a selective growth advantage conferred by γc expression in the transduced cells. Genetic predisposing factors for childhood cancer are also possible, since medulloblastomas have developed in the proband's sister and a first-degree relative.
We have proposed to the French regulatory authorities a halt to our trial until further evaluation of the causes of this adverse event and a careful reassessment of the risks and benefits of continuing our study of gene therapy in patients with X-linked severe combined immunodeficiency can be completed. The latter will include a comparison with the outcome of the only available alternative therapy, haploidentical stem-cell transplantation.5
5 ReferencesSalima Hacein-Bey-Abina, Pharm.D., Ph.D.
Necker University Hospital, 75015 Paris, FranceChristof von Kalle, M.D., Ph.D.
Children's Hospital Research Foundation, Cincinnati, OH 45229Manfred Schmidt, Ph.D.
Freiburg University Medical School, 79106 Freiburg, GermanyFrançoise Le Deist, M.D., Ph.D.
Necker University Hospital, 75015 Paris, FranceNicolas Wulffraat, M.D.
Wilhelmina Children's Hospital University Medical Center, 3508 AB Utrecht, the NetherlandsElisabeth McIntyre, M.D., Ph.D.
Isabelle Radford, M.D., Ph.D.
Necker University Hospital, 75015 Paris, FranceJean-Luc Villeval, Ph.D.
Christopher C. Fraser, Ph.D.
Millennium Pharmaceuticals, Cambridge, MA 02139Marina Cavazzana-Calvo, M.D., Ph.D.
Alain Fischer, M.D., Ph.D.
Necker University Hospital, 75015 Paris, France
fischer@necker.fr 1
Hacein-Bey-Abina S ,Le Deist F ,Carlier F , et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-1193
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Schmidt M ,Zickler P ,Hoffmann G , et al. Polyclonal long-term repopulating stem cell clones in a primate model. Blood 2002;100:2737-2743
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Rabbitts TH ,Bucher K ,Chung G ,Grutz G ,Warren A ,Yamada Y . The effect of chromosomal translocations in acute leukemias: the LMO2 paradigm in transcription and development. Cancer Res 1999;59:Suppl:1794s-1798s
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Stocking C ,Bergholz U ,Friel J , et al. Distinct classes of factor-independent mutants can be isolated after retroviral mutagenesis of a human myeloid stem cell line. Growth Factors 1993;8:197-209
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Buckley RH ,Schiff SE ,Schiff RI , et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-516
Full Text | Web of Science | Medline
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