Treatment of Wilson's Disease With Ammonium Tetrathiomolybdate

Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial

“…These challenges are not unique to this study and likely contribute to the lack of Cu balance studies in recent years, with the most recent study in patients with WD having been published over 30 years ago. 14 Because this study was designed as a short-term mechanistic evaluation of the effect of TMC on Cu elimination, clinical outcome data were not collected, and as a result, the relationship between increased fecal Cu excretion and WD outcomes such as neurologic improvement could not be assessed.…”

Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial

“…These challenges are not unique to this study and likely contribute to the lack of Cu balance studies in recent years, with the most recent study in patients with WD having been published over 30 years ago. 14 Because this study was designed as a short-term mechanistic evaluation of the effect of TMC on Cu elimination, clinical outcome data were not collected, and as a result, the relationship between increased fecal Cu excretion and WD outcomes such as neurologic improvement could not be assessed.…”

TDMQ20 as A Drug Candidate for Wilson’s Disease: Comparison with D-Penicillamine, Trientine, and Tetrathiomolybdate In Vitro and In Mice

“…Based on the daily dose of 12 mg/kg of bcTTM tested in mouse models, the predictable efficient dose in humans is expected to be roughly 1 mg/kg [ 72 ], corresponding to a daily dose of 0.22 mg/kg of Mo, i.e., 25 times higher than the calculated TDI. In fact, the daily dose of ammonium TTM evaluated in adult patients with WD was in the range of 120-240 mg [ 73 , 74 ], corresponding to 0.6–1.2 mg Mo/kg (body weight = 70 kg), i.e., 70–140 times higher than the TDI. Moreover, the accumulation of molybdenum in major organs (liver, spleen, kidneys, adrenals, brain) was evidenced after repeated oral administration of bcTTM to Sprague Dawley rats (15 or 60 mg/kg/d for 12 weeks) [ 75 ].…”

Ammonium tetrathiomolybdate relieves oxidative stress in cisplatin-induced acute kidney injury via NRF2 signaling pathway

“…As an effective copper chelator, TM has achieved good results in Wilson’s diease [ 45 ]. Due to the important role of copper in tumorigenesis and progression [ 46 ], TM has been proven to exhibit antineoplastic effects in various malignancies, including breast cancer [ 36 , 47 ], lung cancer [ 48 ], and ovarian cancer [ 49 ].…”

Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial

“…These challenges are not unique to this study and likely contribute to the lack of Cu balance studies in recent years, with the most recent study in patients with WD having been published over 30 years ago. 14 Because this study was designed as a short-term mechanistic evaluation of the effect of TMC on Cu elimination, clinical outcome data were not collected, and as a result, the relationship between increased fecal Cu excretion and WD outcomes such as neurologic improvement could not be assessed.…”

TDMQ20 as A Drug Candidate for Wilson’s Disease: Comparison with D-Penicillamine, Trientine, and Tetrathiomolybdate In Vitro and In Mice

“…Based on the daily dose of 12 mg/kg of bcTTM tested in mouse models, the predictable efficient dose in humans is expected to be roughly 1 mg/kg [ 72 ], corresponding to a daily dose of 0.22 mg/kg of Mo, i.e., 25 times higher than the calculated TDI. In fact, the daily dose of ammonium TTM evaluated in adult patients with WD was in the range of 120-240 mg [ 73 , 74 ], corresponding to 0.6–1.2 mg Mo/kg (body weight = 70 kg), i.e., 70–140 times higher than the TDI. Moreover, the accumulation of molybdenum in major organs (liver, spleen, kidneys, adrenals, brain) was evidenced after repeated oral administration of bcTTM to Sprague Dawley rats (15 or 60 mg/kg/d for 12 weeks) [ 75 ].…”

Ammonium tetrathiomolybdate relieves oxidative stress in cisplatin-induced acute kidney injury via NRF2 signaling pathway

“…As an effective copper chelator, TM has achieved good results in Wilson’s diease [ 45 ]. Due to the important role of copper in tumorigenesis and progression [ 46 ], TM has been proven to exhibit antineoplastic effects in various malignancies, including breast cancer [ 36 , 47 ], lung cancer [ 48 ], and ovarian cancer [ 49 ].…”

Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial

“…These challenges are not unique to this study and likely contribute to the lack of Cu balance studies in recent years, with the most recent study in patients with WD having been published over 30 years ago. 14 Because this study was designed as a short-term mechanistic evaluation of the effect of TMC on Cu elimination, clinical outcome data were not collected, and as a result, the relationship between increased fecal Cu excretion and WD outcomes such as neurologic improvement could not be assessed.…”

TDMQ20 as A Drug Candidate for Wilson’s Disease: Comparison with D-Penicillamine, Trientine, and Tetrathiomolybdate In Vitro and In Mice

“…Based on the daily dose of 12 mg/kg of bcTTM tested in mouse models, the predictable efficient dose in humans is expected to be roughly 1 mg/kg [ 72 ], corresponding to a daily dose of 0.22 mg/kg of Mo, i.e., 25 times higher than the calculated TDI. In fact, the daily dose of ammonium TTM evaluated in adult patients with WD was in the range of 120-240 mg [ 73 , 74 ], corresponding to 0.6–1.2 mg Mo/kg (body weight = 70 kg), i.e., 70–140 times higher than the TDI. Moreover, the accumulation of molybdenum in major organs (liver, spleen, kidneys, adrenals, brain) was evidenced after repeated oral administration of bcTTM to Sprague Dawley rats (15 or 60 mg/kg/d for 12 weeks) [ 75 ].…”

Ammonium tetrathiomolybdate relieves oxidative stress in cisplatin-induced acute kidney injury via NRF2 signaling pathway

“…As an effective copper chelator, TM has achieved good results in Wilson’s diease [ 45 ]. Due to the important role of copper in tumorigenesis and progression [ 46 ], TM has been proven to exhibit antineoplastic effects in various malignancies, including breast cancer [ 36 , 47 ], lung cancer [ 48 ], and ovarian cancer [ 49 ].…”