Major depressive disorder (MDD) has a lifetime prevalence of over 16% and is associated
with reduced work productivity, disability, increased mortality, and increased rates of
suicide attempts and completed suicides. Unfortunately, ~34% fail to respond to standard
ADs (ADs). Environmental and patient-level factors that increase the risk of MDD could
pinpoint novel mechanisms underlying the disorder. One such factor may be relative
hypoxia. Persons with hypoxic medical conditions, such as asthma and chronic obstructive
pulmonary disease, are at higher risk of depression and suicide compared to those with
other chronic medical conditions. Smoking also promotes hypoxia and is linked to
increased risks of suicide and depression. Of special relevance to this study, living at
high altitude produces relative hypoxia even after months, and is linked to increased
risks of suicide and depression. Hypoxia could contribute to MDD in at least two ways.
First, brain bioenergetics are altered in both hypoxia and MDD. Hypoxia reduces several
neurochemical markers of brain activity, including phosphocreatine (PCr) and
n-acetylaspartate (NAA), and alters mitochondrial dynamics in the hippocampus. Proton
magnetic resonance spectroscopy (1H-MRS) shows that high-altitude residents have altered
whole brain pH and reduced inorganic phosphate to total phosphate (tP) ratios compared to
persons dwelling at sea-level. In depressed patients, phosphorus MRS (31P-MRS) shows
reduced nucleotide triphosphate (NTP) concentrations and decreased PCr concentrations; AD
response is associated with increases in PCr and NTP. Hypoxia could also promote MDD by
altering serotonin (5-HT) production. Chronic hypoxia reduces 5-HT in the forebrain and
brainstem in rodents. The conversion of tryptophan to 5-hydroxytryptophan (5-HTP) by
tryptophan hydroxylase is oxygen-dependent and slowed by hypoxia. Animal studies have
shown that selective serotonin reuptake inhibitors (SSRIs) are not as effective at
altitude, possibly because of inadequate 5-HT production. Reductions in 5-HT synthesis
and inefficiencies in bioenergetics could both contribute to altered brain functional
connectivity. MDD may disrupt cortical emotion regulation, and resting state functional
connectivity (fcMRI) studies suggest that depression involves reduced connectivity
between frontal cortical regions and the amygdala, while AD response correlates with
normalization of those connections. Alterations in connectivity associated with AD
response are correlated with changes in brain metabolites, suggesting a link to brain
bioenergetics.
This suggests two natural supplements as interventions for depression. Oral creatine
monohydrate (Cr) could improve bioenergetics in MDD, as Cr alters brain tCr, PCr, and NTP
levels. Moreover, Cr produces improvements in mood correlated with normalization of PCr
levels and structural connectivity. Alterations in 5-HT synthesis due to hypoxia could be
rectified by 5-HTP, as its conversion to 5-HT is not oxygen-dependent. 5-HTP elevates
brain 5-HT levels and has AD efficacy in clinical trials. The proposed study is a
two-phase, three-armed trial to evaluate whether SSRI/SNRI augmentation with the
supplements Cr, 5-HTP, or their combination (5-HTP+Cr) can enhance AD response in
treatment-resistant MDD. In the R61 phase, the study will assess the ability of the
interventions to alter biological signatures associated with depression, as measured by
31P-MRS, fcMRI, and changes in whole blood 5-HT. In the R33 phase, the study will attempt
to replicate the above findings with dose variation and evaluate their correlation with
clinical outcomes. The study will have the following aims:
Aim 1. Replicate the clinically meaningful changes in biological signatures (PCr:tP
ratios as measured by 31P-MRS, sgACC connectivity as measured by fcMRI, and whole blood
serotonin) demonstrated in the R61 phase, following the decision rule noted above.
Aim 2. Demonstrate that the changes in each of the above markers that is carried over
from the R61 phase are correlated with changes in depression as measured by the 17-item
Hamilton Depression Rating Scale, which would be studied in a subsequent efficacy trial.
Study results will help elucidate the potential efficacy of a novel combination of
nutritional supplements in persons with MDD, given strong epidemiologic and physiologic
evidence suggesting that relative hypoxia can contribute to depression through
alterations in brain bioenergetics and 5-HT synthesis. Target engagement will be
indicated by improvements in functional connectivity and frontal cortical energy
metabolism.