Search bioRxiv⌕ Search

bioRxiv · 10.1101/2025.10.24.684408

Targeting the Alk4 pathway protects against age-related bone loss.

Abstract

Osteoporosis is a chronic age-related condition in which imbalanced activities between bone-forming osteoblasts and bone-resorbing osteoclasts lead to the progressive loss of bone volume and quality. While drugs that target osteoclastic activity have been developed, there remains a lack of efficient therapies that increase osteoblast number and function in aging bones. Here, we investigated if Activin, known to increase in the circulation with age, plays a primary role in bone loss associated with aging. We showed that, in mouse femurs, levels of Activin signaling progressively increased with age and strongly correlated with the loss of trabecular bone. Furthermore, mice lacking the type I receptor for Activin, namely Alk4, in osteoblast progenitors (Alk4 cKO mice) had increased trabecular bone acquisition, osteoblast number, and bone formation rate. In addition, Alk4 cKO male mice were protected against early age-related trabecular bone loss observed at 1 year of age. These results indicate that Activin signaling inhibits bone formation and osteoblast activity and is likely associated with osteoporosis. To further test this, we injected 2-year-old male mice with a ligand trap (Alk4-Fc) to capture circulating Activin. Alk4-Fc protected against loss of trabecular bone in femurs and L5 vertebrae. Interestingly, Alk4-Fc also prevented a decrease in muscle mass in gastrocnemius, quadriceps, and triceps suggesting that circulating Activins also play a role in sarcopenia. In summary, our preclinical mouse models reveal that circulating Activins play a primary role in age-related bone loss and can be efficiently targeted to alleviate osteoporosis and sarcopenia in aging mice. One Sentence SummaryIn this study, we discovered a new way of preserving trabecular bone mass in aging mice by inhibiting activity of Alk4 pathway.

Explore related subjects

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

Maridas, D., Gamer, L., Chim, S. M., Burton, K. M., Van Doren, E., Kirshen, K., Keles, E., Avalos, K., Khetani, R. S., Rosen, V.. 2025-10-24. Targeting the Alk4 pathway protects against age-related bone loss.. https://doi.org/10.1101/2025.10.24.684408

Cite the original work for its findings. Save a collection to share your selection of sources.

KEEP EXPLORING

Related preprints

Sequence context and partner availability specify divergent cell fates from a single transcription factor

Transcription factors drive the gene regulatory networks that control developmental transitions, yet which of their many targets a cell uses remains unresolved. Expressing Cebp, Cebp{beta} or Cebp{delta} identically, alone or with Oct4, Sox2, Klf4 and cMyc, gave four outcomes: adipocyte from fibroblasts and neural stem cells, trophectoderm from embryonic stem cells, and myeloid from B cells. Raising Cebp levels changed how many sites it reached, not which sequence it read: clustered motifs maintained somatic enhancers, while spillover onto low-copy sites created de novo enhancers carrying composites of Cebp and cell-type-restricted partners. In fibroblasts, Cebp also redistributed Oct4, Sox2 and Klf4 from early targets, blocking iPSC formation. Supplying the predicted partners rewrote the output: Spi1 redirected Cebp into parallel adipogenic and myeloid programs, via a transient PpargCD45 state using separate enhancers for each; TFAP2C abolished adipogenic conversion. Motif density and partner composites therefore assemble a factor's instructive output in the receiving cell.

cell biology↗

High-content screening identifies FDA-approved activators of mitophagy

Mitochondrial diseases are highly heterogeneous disorders caused by defects in oxidative phosphorylation, for which only limited therapeutic options are available. The elimination of dysfunctional mitochondria via mitophagy activation is an attractive strategy to treat several mitochondrial diseases, irrespective of the underlying genetic defect. To identify pharmacological activators of this pathway, we performed a high-content screening of 1,971 FDA-approved compounds using cellular reporters of TFEB nuclear translocation and mitophagy. This screening identified 294 compounds that promoted TFEB translocation, nine of which increased the number of mitolysosomes without compromising cell viability or damaging mitochondria. Among the selected compounds, Meptazinol and Ponatinib emerged as the top-ranked mitophagy inducers in fibroblasts from patients with complex I and IV deficiency, respectively, and were prioritised for further characterisation. However, they did not rescue the bioenergetic defect, suggesting that the stimulation of mitophagy per se is not sufficient to ameliorate the OXPHOS defect in mitochondrial diseases. Overall, our findings establish a scalable platform for the identification of pharmacological activators of TFEB and identify candidate compounds for therapeutic modulation of mitophagy.

cell biology↗

Matrix Gla Protein deficiency reveals a previously unrecognized mucosal calcification in the mouse airways

Tracheal calcification is a common finding that is considered to have limited clinical significance when detected most often in elderly individuals. Despite its frequent occurrence, the cellular and molecular mechanisms at the origin of tracheal calcification remain largely unexplored. In this context, we aimed to characterize the development of tracheal calcification, and to determine the role of matrix Gla protein (MGP), a major inhibitor of ectopic calcification whose genetic or pharmacologic deficiency is associated with premature tracheal calcification in humans. We found that wild-type mice undergo an unexpected early physiologic calcification of the tracheal cartilage rings beginning around one month after birth. This calcification progresses in a dynamic rostro-caudal sequence through a process involving terminal differentiation of tracheal chondrocytes. These findings challenge the prevailing view that tracheal cartilage is a permanent hyaline cartilage and instead reveals an early and highly organized physiologic calcification process. Furthermore, our findings further identify MGP as a key regulator of calcification within the respiratory tract. Indeed, MGP deficiency not only markedly accelerates tracheal cartilage calcification through a similar pattern and mechanism but also induces a previously unrecognized calcification of the tracheal mucosa, a lesion absent in wild-type mice. These alterations could partly explain the respiratory manifestations observed in MGP-deficient patients having Keutel syndrome, which include dyspnea, cough, stridor, and recurrent infections, symptoms previously being mainly attributed to calcification of the tracheal cartilage rings.

cell biology↗