With the addition of nine patients treated through expanded access programs, the integrated analysis of OTL-200 has demonstrated the following:Consistent with the results from the registrational study, patients treated with OTL-200 in the integrated data analysis demonstrated a reconstitution of arylsulfatase-A enzyme activity in the hematopoietic system
and stable engraftment of gene-corrected cells within one-month of receiving treatment.
In our study, most commonly affected organ system was the gastrointestinal system (51%) followed by immune system (20%), dermatological system (19%), hematopoietic system
(5%), cardiovascular system (4%), and central nervous system (1%).
Guidance for industry: biologics license applications for minimally manipulated, unrelated allogeneic placental/umbilical cord blood intended for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system
. Rockville, MD: Food and Drug Administration; 2014.
Driven by the interest in identifying underlying genetic determinants of drug response in a specific type of cancer of the hematopoietic system
, CeMM now reports on the mechanistic link between the LZTR1 gene, previously associated with a variety of rare disorders and rare cancers, and RAS.
The infected HSCs go on to reconstitute the hematopoietic system
with blood cells carrying the correct gene.
"We could not predict that prolonged fasting would have such a remarkable effect in promoting stem cell-based regeneration of the hematopoietic system
," added Prof Longo.
The MYC family genes, including MYC, MYCN, and MYCL1, are protooncogenes and known to be overexpressed or mutated in a plethora of different tumors, including that of the hematopoietic system
The numbers of all types of cells (neutrophils, monocytes and eosinophils) fluctuated within the normal physiological standard limits and therefore have no clinical significance as far as the effect of different drugs on the hematopoietic system
Recently, it was reported that rapid accumulation of excessive iron negatively affects the hematopoietic system
by damaging hematopoietic cells and hematopoietic microenvironment (18).
768 (%19.5) and vasculitis included) Nervous system and 663 (%16.9) cerebrovascular diseases Gastrointestinal system 512 (%13) Respiratory system 373 (%9.5) Cardiovascular system 356(%9.1) Urogenital system 340 (%8.6) Musculoskeletal disorders 268 (%6.8) (orthopedical post-ops, etc.) Hepatobiliary system 145(%3.7) Endocrine and metabolic system 145(%3.7) hematopoietic system
89 (%2.3) Infection diseases 82(%2.1) Others (traumas, traffic accidents, 192 (%4.9) intoxication, firearm, drowning in water, and genetic diseases) ICU: intensive care unit, n: number, H: Haseki Training and Research Hospital, DM: diabetes mellitus, HT: hypertension Table 2.
WHO 2007 categorises tumours of Central Nervous System into fifteen types which includes Astrocytic tumours, Oligodendroglial tumours, Ependymal tumours, Choroid plexus tumours, other neuroepithelial tumours, Neuronal and mixed neuronal glial tumours, tumours of the pineal region, embryonal tumours, tumours of cranial and paraspinal nerves, meningeal tumours, tumours of hematopoietic system
, germ cell tumours, familial tumour syndromes, tumours of sellar regions and metastatic tumours.
Therefore, not surprisingly G-CSF treatment is suggested as a potential therapeutic target for a wide range of diseases outside the hematopoietic system