Promising Highly Targeted Therapies for Cholangiocarcinom

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1		Masaki Kuwatani	--	2184	2023-07-24 11:24:46	\|
2	format correction	Dean Liu	+ 2 word(s)	2186	2023-07-25 02:26:44	\|

This entry is adapted from the peer-reviewed paper 10.3390/cancers15143686

To overcome the poor prognosis of cholangiocarcinoma (CCA), highly targeted therapies, such as antibody-drug conjugates (ADCs), photodynamic therapy (PDT) with/without systemic chemotherapy, and experimental photoimmunotherapy (PIT), have been developed. Three preclinical trials have investigated the use of ADCs targeting specific antigens, namely HER2, MUC1, and glypican-1 (GPC1), for CCA. Trastuzumab emtansine demonstrated higher antiproliferative activity in CCA cells expressing higher levels of HER2. PDT is effective in areas where appropriate photosensitizers and light coexist. Its mechanism involves photosensitizer excitation and subsequent reactive oxygen species production in cancer cells upon irradiation. Hematoporphyrin derivatives, temoporfin, phthalocyanine-4, talaporfin, and chlorine e6 derivatives have mainly been used clinically and preclinically in bile duct cancer. PIT is the most novel anti-cancer therapy developed in 2011 that selectively kills targeted cancer cells using a unique photosensitizer called “IR700” conjugated with an antibody specific for cancer cells. PIT is currently in the early stages of development for identifying appropriate CCA cell targets and irradiation devices. Future human and artificial intelligence collaboration has potential for overcoming challenges related to identifying universal CCA cell targets. This could pave the way for highly targeted therapies for CCA, such as ADC, PDT, and PIT.

cholangiocarcinoma biliary tract cancer antibody-drug conjugate photodynamic therapy

1. Introduction

The biliary tree is complicated and finely branched into the liver, which is anatomically classified into small intrahepatic (ϕ20–300 μm), large intrahepatic (300–800 μm), and extrahepatic (>800 μm) bile ducts, including the gallbladder ^[1]. Its numerical bifurcations can cause difficulty in the detection of and approach to early biliary lesions, especially in the periphery, leading to a poor prognosis of cholangiocarcinoma (CCA), with a 5-year relative survival rate of 7–20% ^[2]^[3]^[4]. To improve prognosis, novel therapeutic approaches to CCA are necessary.

There have been a number of clinical and basic studies on CCA regarding etiology, clinicopathology, molecular biology, and immunology that have clarified the characteristics and targeted points of CCA to some extent. Currently, there are six primary modalities of anticancer treatment for CCA: (1) surgical therapy; (2) chemotherapy; (3) radiotherapy, including brachytherapy and proton radiotherapy; (4) immunotherapy, including immune checkpoint inhibitor and chimeric antigen receptor (CAR) T-cell therapy, (5) phototherapy, and (6) combination therapy, such as antibody-drug conjugate (ADC) therapy (2 + 4), photodynamic therapy (PDT; 2 + 5), and photoimmunotherapy (PIT; 4 + 5). Surgical therapy has developed with the increase in skilled surgeons, high-resolution imaging modalities, and the introduction of portal vein embolization ^[5]. Chemotherapy has been developed with an understanding of the biology and molecular mechanisms of CCA ^[6]. Radiotherapy has also improved with internal small, sealed sources ^[7]^[8] and proton beams ^[9], mainly with palliative and supportive intent. Immunotherapy has evolved and improved with the advent of immune checkpoint inhibitors, such as durvalumab (a PD-L1 antibody) ^[10] and CAR-T therapy ^[11]. Among them, highly targeted therapies such as ADC, PDT, and PIT are attracting much attention for precision medicine because of their expected high effectiveness and low invasiveness.

To provide highly targeted therapies, previous researchers revealed that CCA abnormally expresses some possible antigens/targets or molecules on the cell surface, such as epidermal growth factor receptor (EGFR), HER2 (c-erbB-2), and vascular endothelial growth factor receptor (VEGFR) ^[12].

In addition, recent genetic investigations of biliary tract cancer have revealed a range of genetic mutations and alterations specific to the primary cancer site. These include fibroblast growth factor receptor-2 (FGFR2) fusion and IDH1/2 in intrahepatic CCA (ICC); HER2 (erbB-2), PRKACA, and PRKACB in extrahepatic CCA (ECC: perihilar and distal CCA); EGFR, ERBB3, and PTEN in gallbladder cancer; and KRAS, SMAD4, and TP53 shared in ICC and ECC ^[13]^[14]. Some of these genetic mutations are also common in pancreatic cancer, which is attributed to the adjacent embryologic relationship between the biliary tree and pancreas ^[15]. These genetic variations often present challenges and emphasize the importance of implementing precision medicine approaches tailored to the specific cancer site.

2. Antibody-Drug Conjugate

2.1. Preclinical Studies of ADCs for CCA

There have been three preclinical trials of ADCs for CCA using murine models whose targeted antigens were human epidermal growth factor receptor 2 (HER2) ^[16], mucin 1 (MUC1) ^[17], and glypican-1 (GPC1) ^[18] (Table 1). HER2 is a member of the EGFR family. Protein dimers of HER2 with HER family receptors, such as EGFR, HER2, HER3, or HER4, accelerate cell proliferation and prolong cell survival. Amplification or overexpression of HER2 is predominantly associated with tumorigenesis in the breast (15–30%), stomach, and esophagus (10–30%) ^[19]^[20]^[21]. HER2 overexpression is also observed in 4.8% (95% confidence interval [CI] 0–14.5%) of ICC, 17.4% (95% CI 3.4–31.4%) of ECC, 19.1% (95% CI 11.2–26.8%) of gallbladder cancer, and 27.9% (95% CI 0–60.7%) of ampullary carcinoma ^[22]. Yamashita-Kashima et al. indicated that the anti-proliferative activity of the ADC trastuzumab emtansine (T-DM1) was higher in CCA cell lines with higher levels of HER2 expression and in proportion to HER2 status ^[16]. Shinoda et al. showed that the cytotoxicity of lymphokine-activated killer cells (a heterogeneous population consisting of NK, NKT, and T cells) against CCA can be reinforced by staphylococcal enterotoxin A (SEA) conjugated with an antibody directed to MUC1 (MUSE11) in CCA cells ^[17]^[23]. SEA is one of the superantigens that bind outside of the peptide-binding groove of MHC class II molecules and activate T cells expressing a certain Vβ type of T cell receptor ^[24]^[25]. The transmembrane glycoprotein MUC1 is a mucin family member that can act as a lubricant, moisturizer, and physical barrier in normal cells. MUC1 is also an epithelial mucin antigen that is widely expressed in adenocarcinomas, such as those arising in the pancreas, stomach, ovaries, and bile ducts ^[26]^[27]^[28]. Thus, the SEA-MUSE11 conjugate can work via the above-mentioned phase 4 mechanism alone.

Table 1. Targets for cholangiocarcinoma cells.

		Preclinical Study			Clinical Study
Name	Type	ADC	ADC-Payload	PIT	ADC	ADC-Payload
HER2	Receptor	✓	Emtansine (DM1)	✓	✓	Emtansine (DM1) Deruxtecan (DXd)
EGFR	Receptor			✓
FGFR2	Receptor				✓	Ixadotin
MUC1	Secretion (mucin)	✓
Glypican-1 (GPC1)	Secretion (proteoglycan)	✓	Monomethyl auristatin F (MMAF)
CD133/ prominin-1	Secretion (glycoprotein)	✓	Maleimidocaproyl-valine-citrulline-p-aminobenzoyl-MMAF (vcMMAF)
TROP2	Secretion (glycoprotein)			✓

HER2—human epidermal growth factor receptor 2; EGFR—epidermal growth factor receptor; FGFR2—fibroblast growth factor receptor-2; MUC1—transmembrane glycoprotein mucin 1; TROP2—trophoblast cell surface antigen 2. ✓ indicates the existence of previous reports.

GPC1, a cancer antigen, is a heparan sulfate proteoglycan that is linked to the cell surface by a glycosylphosphatidylinositol anchor and promotes tumor growth, metastasis, and invasion by acting as a co-receptor ^[29]. Yokota et al. reported high expression of GPC1 in 47% of patients with ECC by immunohistochemical staining and that MMAF-conjugated anti-GPC1 antibodies showed potent tumor growth inhibition against GPC1-positive CCA cells in vitro and in vivo ^[18]. MMAF is a tubulin-polymerizing inhibitor and is also clinically used as a payload in ADCs for relapsed or refractory multiple myeloma. Therefore, anti-GPC1 ADCs should be clinically investigated and developed, especially for ECC, considering other payloads such as DM1 and DXd.

CD133/prominin-1 is a pentaspan transmembrane glycoprotein overexpressed in various solid tumors, including colorectal and glioblastomas. In a study by Smith et al. ^[30], CD133 was found to be highly expressed in ≥50% of pancreatic, gastric, and intrahepatic CCA. In addition, anti-CD133 ADC (maleimidocaproyl-valine-citrulline-p-aminobenzoyl-MMAF [vcMMAF]) treatment resulted in a significant delay in Hep3B (hepatocellular carcinoma) tumor growth in SCID mice. Thus, the anti-CD133 antibody-vcMMAF conjugate could also be effective for CCA treatment.

2.2. Human Studies of ADCs for CCA

Seven ADCs have been approved for solid tumors worldwide. Among these, three are approved for breast cancer targeting HER2, two for gastric cancer targeting HER2, and one each for head and neck cancers targeting EGFR/tissue factor and urothelial cancer targeting nectin-4 (including duplicate indications). In addition, there have been three clinical trials specifically focused on CCA (Table 1). Two of them target HER2, and one targets FGFR2 on CCA cells.

Mondaca et al. showed one case with a 30% partial response of the primary lesion after trastuzumab (anti-HER2 antibody)-DM1 treatment ^[31]. Additionally, Tsurutani et al. demonstrated confirmed objective responses in HER2-expressing (IHC ≥1+) non-small cell lung cancer, salivary gland cancer, endometrial cancer, and biliary tract cancer following trastuzumab-DXd treatment. Among these cases, two showed tumor shrinkage of ≥60% ^[32]. Based on its promising results, a multicenter phase II trial of trastuzumab deruxtecan for HER2-positive unresectable or recurrent biliary tract cancer (HERB trial) is ongoing in Japan ^[33].

To target FGFR2, aprutumab ixadotin (BAY 1187982) was developed as the first ADC with a novel auristatin-based payload. A phase I trial comprising 20 patients with FGFR2-positive solid tumors, including four CCAs, revealed that aprutumab ixadotin was poorly tolerated due to a high rate of proteinuria and nephropathy. The maximum tolerance dose (MTD) was found to be below the therapeutic threshold estimated preclinically; therefore, the trial was terminated early ^[34]. The authors hypothesized that severe toxicity might be attributed to the unique combination of an auristatin W derivative payload with aprutumab. As FGFR2 fusion/rearrangement is detected in 7.4−13.6% of ICCs, improvement of anti-FGFR2 antibody-based ADC is also warranted ^[35]^[36]^[37].

3. Photodynamic Therapy

Since the ancient Egyptian era, light has been used in medicine to treat skin diseases such as psoriasis and vitiligo with psoralens. At present, photodynamic therapy (PDT) is extensively used in infectiology, dermatology, gynecology, urology, and oncology ^[38]. Since the first modern medical concept of photodynamic therapy was introduced in 1900 by Raab and von Tappeiner based on the incidental effect on malaria-causing protozoa, the first-in-human PDT of tumors was performed by von Tappeiner for skin tumors in 1903, according to the literature ^[38]^[39]^[40]^[41]. Approximately 70 years later, Diamond et al. published a groundbreaking report demonstrating the anticancer effect of PDT with hematoporphyrin and white light on rat gliomas in both in vitro and in vivo settings ^[42]. Notably, the PDT effect was limited to the area where hematoporphyrin and white light coexisted, thus achieving highly targeted treatment. The mechanism of PDT is explained by the following phases: (1) entering the cell of the photosensitizer; (2) photoexcitation of the photosensitizer from the ground energy state to the exited state; (3) energy transfer from the photosensitizer to the biomolecules from its surroundings (type I reaction) or to the oxygen molecule (type II reaction); (4) production of reactive oxygen species (ROS): superoxide anion radical (O₂^•−), hydroxyl radical (OH^•) by type II, and singlet oxygen (¹O₂) by type I inside the cells; and (5) oxidative stress resulting in the destruction of cancer cells ^[43]^[44] (Figure 1B). Furthermore, recent studies indicate that immunogenic cell death via apoptosis and necroptosis can occur after PDT, leading to the release of danger/damage-associated molecular patterns that can activate an adaptive immune response ^[45]^[46].

In 1976, Kelly and Snell conducted the first human study to evaluate the effects of PDT using a hematoporphyrin derivative (HpD) in five patients with bladder cancer. Their study resulted in one successful case, demonstrating the potential of PDT in cancer treatment ^[47]. Nowadays, PDT has been approved for the treatment of various types of cancer, including head and neck, brain, lung, esophagus, breast, prostate, bladder, skin, pancreas, and bile duct cancers ^[48]. Many researchers have made efforts to improve the efficacy of PDT through the refinement of photosensitizers and light sources, as described below.

4. Photoimmunotherapy

Photoimmunotherapy (PIT) is a novel anti-cancer therapy developed in 2011 by Kobayashi H. et al. that theoretically selectively kills targeted cancer cells with no damage to normal cells ^[49]. PIT comprises a unique photosensitizer called “IR700.” This water-soluble photo dye consists of silicon phthalocyanine and hydrophilic side chains (IRDye 700DX N-hydroxysuccinimide ester). In addition, PIT incorporates a specific molecule that enables binding to a target cell, which can be an antibody or a low-molecular-weight compound. Examples include the combination of IR700 with an anti-EGFR antibody or an affibody combined with IR700 as antibody/affibody-photosensitizer conjugates (APCs) ^[50]^[51]. IR700 is different from photosensitizers used in PDT because IR700 has a more than five-fold higher extinction coefficient (2.1 × 10⁵ M⁻¹cm⁻¹ at the absorption maximum of 689 nm) ^[52] than that of conventional photosensitizers, such as the HpD (1.2 × 10³ M⁻¹cm⁻¹ at 630 nm), meta-tetrahydroxyphenylchlorin (2.2 × 10⁴ M⁻¹cm⁻¹ at 652 nm), and mono–L-aspartylchlorin e6 NPe6 (4.0 × 10⁴ M⁻¹cm⁻¹ at 654 nm) ^[53]. Although PIT also requires a light source emitting near-infrared light (NIR) with a wavelength of 689–700 nm (NIR wavelength is usually defined as 650–1700 nm) ^[49]^[54], its mechanism differs from that of PDT (Figure 1C). In PIT, the destruction of the target cell starts with a chemical change of IR700 in APCs bound to the cell due to the release of hydrophilic side chains of IR700 after NIR irradiation. This process forms water-insoluble aggregates of APCs or APC-antigen complexes on the cell surface molecules, leading to physicochemical changes within the APC-antigen complex that reduce cell membrane integrity because of damage to transmembrane target proteins. Subsequently, water flows into the cytoplasm, causing cell swelling ^[55]. In contrast, the mechanism of PDT is based on inner cell destruction by ROS (Figure 1B). In addition, PIT also results in further immunogenic cell death that is initiated by the maturation of immature dendritic cells with released tumor antigens from the treated cancer cell in the adjacent microenvironment. After cancer cell-targeted PIT, CD8+ T cells newly primed by a larger repertoire of cancer antigens proliferate in the treated tumor beds. Finally, anticancer host immunity can be strengthened after cancer-cell-targeted PIT ^[55]. Although many investigations of PIT and clinical PIT for head and neck cancer use lasers as an NIR light source as well as PDT, LEDs can also produce NIR and yield a PIT effect on CCA cells ^[56]. As described above, CCA can develop in the extrahepatic and intrahepatic bile ducts that cannot be approached/visualized by an endoscope, while a special catheter with LEDs, such as a biliary drainage tube, can be placed for PIT at the peripheral CCA ^[56]. Therefore, a dedicated PIT device for CCA would shed light on PIT for CCA.

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Subjects: Gastroenterology & Hepatology

Contributors MDPI registered users' name will be linked to their SciProfiles pages. To register with us, please refer to https://encyclopedia.pub/register :

Masaki Kuwatani

Naoya Sakamoto

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Update Date: 25 Jul 2023

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