BONE-MARROW TRANSPLANTATION IN BATTEN-DISEASE (NEURONAL CEROID-LIPOFUSCINOSIS) - WILL IT WORK - PRELIMINARY STUDIES ON COCULTURE EXPERIMENTS AND ON BONE-MARROW TRANSPLANT IN LATE INFANTILE BATTEN-DISEASE
Bd. Lake et al., BONE-MARROW TRANSPLANTATION IN BATTEN-DISEASE (NEURONAL CEROID-LIPOFUSCINOSIS) - WILL IT WORK - PRELIMINARY STUDIES ON COCULTURE EXPERIMENTS AND ON BONE-MARROW TRANSPLANT IN LATE INFANTILE BATTEN-DISEASE, American journal of medical genetics, 57(2), 1995, pp. 369-373
Lymphocytes from a patient with preclinical late infantile Batten dise
ase were cultured alone and;with lymphocytes from donors, and the fate
of the curvilinear inclusions characteristic of the disease was monit
ored by electron microscopy. There was no evidence of transfer of defi
cient enzyme or factor that might have caused removal of the stored ma
terial, and the curvilinear profiles remained in the cultured cells wi
thout signs of degradation, Cells stimulated to divide with phytohaema
glutinin did not exhibit storage in culture suggesting that storage is
a function of the age of the cell, The patient received a bone marrow
transplant at 2 7/12 years while still clinically unaffected, and the
effect on lymphocytes and cells in skin and rectal biopsies was monit
ored by electron microscopy over a period of 9 months until the donor
marrow became displaced by the host cells, He has had one seizure and
now has neurophysiological evidence of late infantile Batten's disease
, Bone marrow transplant may have no effect on material already stored
but might prevent further build-up and halt the onset of the clinical
symptoms although very recent studies on early (fetal) transplants in
sheep with a form of Batten disease have shown no benefit. (C) 1995 W
iley-Liss,Inc.