Note for: Advances and perspectives of PARP inhibitors

Note for: Advances and perspectives of PARP inhibitors

Doi: 10.1186/s40164-019-0154-9

Overview:

-          Continuous DNA replication in cancer cells leads to higher demand of DNA repair components

-          Growing evidence -- > broader population of patients -- > benefit from PARPi beside one who has BRCA1/2 mutated tumors

-          Potential patients for PARPi

o   Defective in replication stress

o   HR deficiency

-          Summarizing

o   Advanced of PARPi in clinical application

o   PARPi based combination strategies in preclinical and clinical studies

Cancer characteristic

-          Generation of mutations

-          Neoantigens

-          Genome integrity

To survive

-          Maintain specific DNA damage repair pathways to control DNA damage events

-          Thus, targeting DNA repair pathways -- > ideal targets for cancer treatment

Rational

-          Different of HR status between cancer (HR-defective) and normal cells

o   Initiate PARP -- > cell replicate -- > DSB -- > cancer cells repair DNA through NHEJ -- > toxic

o   Initiate PARP -- > cell replicate -- > DSB -- > normal cells repair DNA majorly through HR -- > survive

Background of PARP

-          PARP1 -- > more related to DNA damage repair -- > generate PAR after DNA damage event

-          6  main domains

o   Three zinc finger domains

o   One BRCA1 c-terminus domain (auto-modification domain)

o   WGR- rich domain

o   One catalytic domain

§  One helical domain (HD)

§  One ADP-ribosyltransferase catalytic domain (ART)

During undamaged DNA

o   HD inhibits binding between PARP1 + beta-NAD

During SSB (single strand break)

o   PARP1 recognize the SSB through Zn-finger domains

o   Auto-inhibitory function of HD -- > stop (through conformational change)

o   Catalytic function of ART activates

o   Promoting the recruitment of DNA repair effectors and chromatin remodeling

o   Auto-PARylation on PAPR1 -- > cause dissociation of PARP1 from DNA chains -- > restore auto-inhibitory status of PARP1


Clinical development of PARP inhibitors

-          The mode of mechanism in term of killing cancer cells is not clear

-          It proposes that trapping PARP on damaged DNA -- > induce apoptosis in cancer cells rather than inhibiting the catalytic site -- > this comes from the observation of talazoparib which show the most cytotoxicity potency with the highest ability to trap PAPR1 with DNA

FDA-approved PARP inhibitors

-          Olaparib

o   First PAPRi entering clinical practice

o   Used with gBRCA1/2m ovarian cancer

o   Used for maintenance treatment for patients who are sensitive to platinum-based therapy

-          Rucaparib

o   Same as olaparib -- > used as maintenance treatment for patients who are sensitive to platinum-based therapy

-          Niraparib

o   Maintenance treatment of Pt-sensitive epithelial ovarian, fallopian tube, or primary peritoneal cancer patients

-          Talazoparib

o   Among PARPis -- > this one has the most PAPR1 trapping and cytotoxic potency

PAPR inhibitors in clinical trials

-          Veliparib has not been approved by FDA for cancer treatment

-          Combination therapy with Pt-based drug

Beyond BRCA1/2 mutated cancer

HR deficient cancer

-          Looking the mutation in genes which give rise the same phenotype as of gBRCA1/2m

-          Thus, PARPi could be used

-          Those genes are related to HR

o   RAD51

o   ATM

o   ATR

o   PALB2

o   Fanconi anemia gene family

-          Identification of HR deficiency in cancer cells -- > complex work

-          Better to find “measurable way to indicate the HR activity” -- > thus, PAPRi could be initiated, for example,

o   Large scale chromosomal rearrangements

o   Loss of heterozygosity

Replication stress

-          SCLC -- > HR proficiency but sensitive to PARPi -- > later, it founds out that this cancer type is attributed to high replication stress -- > drive by 1.mutation of tumor suppressor gene 2. Amplification of oncogenes

PARPi-involved combination therapy

-          Acting as sensitizers for chemotherapies, immunotherapies, and targeted therapies -- > limiting DNA damage repair

o   PARPi + genotoxic chemotherapy

o   PARPi + immune checkpoint inhibitor

§  PARPi generate more neoantigen (more damage, more mutations), thus, increase the immunogenicity of cancers

 





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