Covalent ERK inhibitors for suppressing cancer

BI-78D3, a small molecule, covalently binds to ERK1/2’s D-recruitment site, disrupting ERK signaling crucial for cancer cell growth. This shows promise for treating ERK-dependent cancers, including those resistant to BRAF inhibitors.

This disrupts ERK signaling crucial for cell proliferation and survival, particularly in certain cancers like melanoma with BRAF V600E mutations, including BRAF inhibitor-resistant ones. BI-78D3 selectively modifies ERK1/2 without affecting other kinases.

Background

The MAPK/ERK signaling pathway is essential for regulating cell proliferation and survival. This pathway plays a critical role in various cellular processes, and its dysregulation is implicated in many diseases, most notably cancer. More than half of melanomas harbor a constitutively active BRAF mutation (BRAFV600E) that leads to uncontrolled ERK signaling, driving tumor develop­ment and progression. This necessitates the development of effective therapies that can specifically target and modulate ERK activity to control these cancers.

Current approaches to target ERK signaling, such as BRAF and MEK inhibitors, have shown initial success in treating BRAF-mutant melanomas. However, the efficacy of these treatments is often short-lived due to the development of resistance mechanisms. These mechanisms frequently involve the reacti­vation of ERK signaling, either through mutations in ERK itself or through upregulation of other signaling pathways that bypass BRAF and MEK inhibition.

Furthermore, the use of ATP-competitive ERK inhibitors, while initially promis­ing, also faces the challenge of acquired resistance through mutations within the ATP-binding site of ERK. This highlights the need for alternative strategies to inhibit ERK that can overcome these resistance mechanisms and provide more durable therapeutic benefits.

Technology description

BI-78D3, a small molecule, offers a novel approach to cancer treatment by targeting the ERK signaling pathway, crucial for cancer cell growth and survival in certain cancers. It binds to the D-recruitment site (DRS) of ERK1/2, a location distinct from the typical ATP-binding site targeted by other inhibitors.

This binding is unique as BI-78D3 forms a covalent bond with a cysteine residue (C159) within the DRS. This covalent interaction disrupts ERK signaling in vivo, effectively inducing apoptosis in melanoma cells with BRAF V600E mutations, even those resistant to BRAF inhibitors. The compound demon­strates selectivity, modifying ERK1/2 without affecting other kinases like p38MAPK, JNK, or ERK5.

The technology is differentiated by its unique mechanism of action. Unlike ATP-competitive ERK inhibitors, BI-78D3 targets the DRS, a region crucial for ERK-protein interactions. This distinct binding site and covalent modification of C159 offer a potential advantage in overcoming resistance mechanisms that commonly arise with ATP-competitive inhibitors.

The selectivity of BI-78D3 for ERK1/2 over other kinases minimizes off-target effects, enhancing its potential as a therapeutic agent. Furthermore, BI-78D3 effectively induces apoptosis in BRAF inhibitor-resistant melanoma cells, suggesting its potential to address acquired resistance to existing therapies. This approach provides a new avenue for developing cancer therapies targeting ERK-dependent cancers.

Benefits

  • Targets ERK signaling pathway crucial for cancer cell proliferation and survival
  • Small molecule BI-78D3 covalently binds to ERK1/2’s D-recruitment site (DRS), disrupting ERK signaling.
  • Selective modification of ERK1/2 without affecting other kinases (p38MAPK, JNK, ERK5)
  • Induces apoptosis in melanoma cells with BRAF V600E mutations, including BRAF inhibitor-resistant cells
  • Offers a potential therapeutic strategy for ERK-dependent cancers, overcoming resistance to existing therapies

Commercial applications

  • Targeted cancer therapy
  • Melanoma treatment
  • BRAF-resistant cancer
  • ERK pathway modulation

Publication link

https://www.nature.com/articles/s41467-019-12996-8

 

Patent Information: