RARE DISEASE
Ankylostomiasis
Ankylostomiasis
Ankylostomiasis
Synonyms: Ancylostomiasis, Hookworm infection
Synonyms: Ancylostomiasis, Hookworm infection
Synonyms: Ancylostomiasis, Hookworm infection
Drug discovery
1
drug
With orphan designation
Overview
Ankylostomiasis, caused by Necator americanus and Ancylostoma species, is a soil-transmitted helminthiasis characterized by iron-deficiency anemia, fatigue, abdominal pain, and growth impairment in children. Transmission occurs via larval skin penetration or ingestion. Diagnosis relies on stool microscopy for eggs. The WHO prioritizes mass drug administration (MDA) with albendazole or ivermectin in endemic regions [1][2][11].
Burden
Accounts for ≈4 million DALYs annually, primarily due to chronic anemia and developmental delays [4][7].
Moderate/heavy infections (>2,000 eggs/g stool) correlate with hemoglobin deficits, exacerbating malnutrition and maternal-fetal complications [4][14].
Economic losses exceed $7 billion/year from reduced productivity and healthcare costs [7][13].
Therapies
First-line: Single-dose albendazole (400 mg) or ivermectin (200 mcg/kg), effective against adult worms [1][3].
Pediatrics: Albendazole is contraindicated under 2 years; ivermectin requires weight >15 kg [1][17].
Anemia management: Iron supplementation combined with anthelmintics in endemic areas [2][12].
Categories: rare infectious diseases
Research Papers
1,107 drug discovery papers about Ankylostomiasis, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,107 drug discovery papers about Ankylostomiasis, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-08 | Unmasking the Hidden Culprit: Capsule Endoscopy Reveals Diffuse Hookworm Infestation as a Cause of Obscure Gastrointestinal Bleeding and Refractory Anemia-A Case Report.
A 70-year-old woman with transfusion-dependent anemia and ongoing gastrointestinal bleeding had inconclusive findings on imaging, upper endoscopy, and colonoscopy. Capsule endoscopy revealed the diagnosis: diffuse hookworm infestation causing active bleeding throughout the small bowel and colon. Targeted anthelmintic therapy resolved her anemia completely.
2026-06-29 | Profound Iron Deficiency Anemia Associated With Endoscopically Visualized Nematode Infestation Mimicking Gastric Malignancy: A Case Report From Trinidad and Tobago.
Severe iron deficiency anemia (IDA) secondary to parasitic infestation is uncommon in modern clinical practice but remains an important diagnostic consideration in endemic regions and vulnerable populations. We present the case of a 61-year-old male patient who presented to the emergency department following a syncopal episode and fall and was found to have profound anemia with an initial hemoglobin level of 2.5 g/dL and a serum ferritin of 1.96 ng/ml. Initial diagnostic workup with contrast-enhanced computed tomography of the abdomen and pelvis excluded acute intra-abdominal hemorrhage but demonstrated marked thickening of the gastric fundus and body, raising concern for an underlying gastric malignancy. Laboratory investigations additionally revealed raised eosinophil percentage on complete blood count analysis. Following hemodynamic stabilization and packed red blood cell transfusion, the patient underwent esophagogastroduodenoscopy. Endoscopic evaluation revealed short-segment Barrett's esophagus (Prague classification C0M2) and multiple motile nematodes attached to the duodenal mucosa, consistent with an endoscopically visualized nematode infestation involving the proximal small bowel. In the context of endemic exposure, raised eosinophil percentage, profound microcytic anemia, and low ferritin, this was considered morphologically suggestive of hookworm infestation. Further dietary and social history revealed habitual barefoot outdoor exposure and regular consumption of wild meat, supporting environmental risk factors for parasitic infection. Stool microscopy was negative, and histopathology did not identify parasitic organisms or malignancy. The patient was treated with albendazole and proton pump inhibitor therapy, with subsequent clinical improvement, recovery of hemoglobin levels following transfusion and anti-helminthic treatment, and sustained hematologic stability on follow-up. This case highlights the importance of considering parasitic infection in the differential diagnosis of severe microcytic anemia and gastric wall thickening, particularly in endemic settings. It also demonstrates how infectious gastrointestinal pathology may mimic advanced malignancy on cross-sectional imaging.
2026-06-10 | A novel hookworm vaccine candidate identified through immunomic profiling of human protective immunity.
Hookworms infect nearly half a billion people, causing approximately 2.1 million disability-adjusted life-years lost. While anthelmintic drugs are available, they do not prevent reinfection. A vaccine could provide long-term immunity, but none currently exists. We recently demonstrated that human subjects could be safely vaccinated with irradiated hookworm larvae, eliciting humoral and cellular immune responses that induce partial protection against challenge infection with non-irradiated parasites. Using immunomic approaches, we identified IgG targets in vaccinated individuals by screening a proteome microarray containing recombinant proteins sourced from the secretome of Necator americanus. We characterized the most immunoreactive N. americanus proteins and identified their orthologs in the rodent hookworm Nippostrongylus brasiliensis. These N. brasiliensis proteins were expressed recombinantly and tested for efficacy in a mouse challenge model. Our lead vaccine candidate NBR17057 - a homolog of Na_11335 provided robust protection against infection, evidenced by >90% reductions in intestinal worm burden and fecal egg counts, and elevated antigen-specific IgG levels. Vaccination also stimulated IgA+ B-cells, IgG1+/IgA+ plasma cells, and IgG1+/IgA+ germinal centre B-cells in inguinal lymph nodes, indicating gut immunity. Additionally, high levels of IgG1+/IgA+ B-cells, germinal centre B-cells, and plasma cells in the spleen suggest protection against systemic larval migration. The presence of IgG1+/IgA+ memory B-cells in both the lymph nodes and spleen indicates potential long-term immunity against reinfection. Our findings suggest that a N. brasiliensis L3 larval-stage protein NBR17057 and its homolog in N. americanus Na_11335 could serve as a promising subunit vaccine candidate targeting the infective larval stage of human hookworms.
2026-06-07 | Soil-transmitted helminth infections among migrants and travelers: a 10-year retrospective study.
Soil-transmitted helminth (STH) infections, caused by Ascaris lumbricoides, Trichuris trichiura, and hookworms, are widely distributed in tropical regions and significantly contribute to morbidity and mortality. These parasites share similar life cycles, and treatment is primarily based on benzimidazoles. This study aims to describe the clinical and epidemiological characteristics of STH cases among migrants and travelers who attended the Vall d'Hebron-Drassanes International Health Unit between 2014 and 2024, the treatments administered, and their efficacy. This retrospective descriptive study reviewed STH cases associated with migration or travel during the period 2014-2024. Diagnosis was established either through identification of eggs or larvae in stool samples or through the detection of adult Ascaris lumbricoides worms in digestive samples, always associated with a migratory process or international travel. Clinical and epidemiological data were collected and analyzed using SPSS. A total of 361 STH cases were identified, with a mean age of 32 years. Of these, 77% were migrants, 15.7% were travelers visiting friends and relatives (VFR), and 7.2% were travelers. Hookworm infection was diagnosed in 126 cases, Trichuris trichiura in 113 cases, Ascaris lumbricoides in 87 cases, and 35 patients presented with multiple STH infections. Forty-six (12.7%) were symptomatic, predominantly with gastrointestinal symptoms, and 38% presented with eosinophilia. Ninety-five percent of patients received benzimidazole-based treatment, and therapeutic failure was observed in 7.8% of cases. Cure rates were highest for A. lumbricoides (nearly 100%) and remained above 90% for T. trichiura, whereas hookworm infections showed lower rates, ranging from 83% with albendazole to 92% with mebendazole. Multiple STH infections showed, proportionally, higher rates of therapeutic failure. Most STH cases were diagnosed in asymptomatic migrants and VFRs, highlighting the importance of screening for imported diseases in migrant populations. The observed increase in therapeutic failure with commonly used regimens highlights the need to standardize treatment protocols and consider the incorporation of new drugs and combination therapies.
2026-06-04 | Transgenic hookworm secretes anti-tetrodotoxin human single chain antibody.
Biologics, protein- and peptide-based drugs derived from living organisms or cell lines have emerged as effective therapies across a broad range of indications. However, parenteral administration and the need for frequent dosing increase costs and limit compliance, creating an urgent need for innovative platforms capable of continuously, safely, and efficiently delivering sustained biologics in situ within the host. Here, we describe a bioengineered hookworm platform to manufacture and deliver biologic therapeutics in vivo. As proof of concept, we engineer the Ancylostoma ceylanicum secretome by inserting a human single-chain variable fragment antibody (s16-HuScFv) into its genome. Transgene expression does not perturb surrounding gene expression, and heritable transgenesis is confirmed. The s16-HuScFv transgene product is secreted into host circulation and partially neutralizes tetrodotoxin. Given the availability of controlled human infections, our disease-agnostic bioengineered hookworm platform offers a next-generation approach to address a suite of chronic human diseases, and with a single-dose administration, could potentially produce and deliver biologic medicines within the human host for years.
2026-07-08 | Unmasking the Hidden Culprit: Capsule Endoscopy Reveals Diffuse Hookworm Infestation as a Cause of Obscure Gastrointestinal Bleeding and Refractory Anemia-A Case Report.
A 70-year-old woman with transfusion-dependent anemia and ongoing gastrointestinal bleeding had inconclusive findings on imaging, upper endoscopy, and colonoscopy. Capsule endoscopy revealed the diagnosis: diffuse hookworm infestation causing active bleeding throughout the small bowel and colon. Targeted anthelmintic therapy resolved her anemia completely.
2026-06-29 | Profound Iron Deficiency Anemia Associated With Endoscopically Visualized Nematode Infestation Mimicking Gastric Malignancy: A Case Report From Trinidad and Tobago.
Severe iron deficiency anemia (IDA) secondary to parasitic infestation is uncommon in modern clinical practice but remains an important diagnostic consideration in endemic regions and vulnerable populations. We present the case of a 61-year-old male patient who presented to the emergency department following a syncopal episode and fall and was found to have profound anemia with an initial hemoglobin level of 2.5 g/dL and a serum ferritin of 1.96 ng/ml. Initial diagnostic workup with contrast-enhanced computed tomography of the abdomen and pelvis excluded acute intra-abdominal hemorrhage but demonstrated marked thickening of the gastric fundus and body, raising concern for an underlying gastric malignancy. Laboratory investigations additionally revealed raised eosinophil percentage on complete blood count analysis. Following hemodynamic stabilization and packed red blood cell transfusion, the patient underwent esophagogastroduodenoscopy. Endoscopic evaluation revealed short-segment Barrett's esophagus (Prague classification C0M2) and multiple motile nematodes attached to the duodenal mucosa, consistent with an endoscopically visualized nematode infestation involving the proximal small bowel. In the context of endemic exposure, raised eosinophil percentage, profound microcytic anemia, and low ferritin, this was considered morphologically suggestive of hookworm infestation. Further dietary and social history revealed habitual barefoot outdoor exposure and regular consumption of wild meat, supporting environmental risk factors for parasitic infection. Stool microscopy was negative, and histopathology did not identify parasitic organisms or malignancy. The patient was treated with albendazole and proton pump inhibitor therapy, with subsequent clinical improvement, recovery of hemoglobin levels following transfusion and anti-helminthic treatment, and sustained hematologic stability on follow-up. This case highlights the importance of considering parasitic infection in the differential diagnosis of severe microcytic anemia and gastric wall thickening, particularly in endemic settings. It also demonstrates how infectious gastrointestinal pathology may mimic advanced malignancy on cross-sectional imaging.
2026-06-10 | A novel hookworm vaccine candidate identified through immunomic profiling of human protective immunity.
Hookworms infect nearly half a billion people, causing approximately 2.1 million disability-adjusted life-years lost. While anthelmintic drugs are available, they do not prevent reinfection. A vaccine could provide long-term immunity, but none currently exists. We recently demonstrated that human subjects could be safely vaccinated with irradiated hookworm larvae, eliciting humoral and cellular immune responses that induce partial protection against challenge infection with non-irradiated parasites. Using immunomic approaches, we identified IgG targets in vaccinated individuals by screening a proteome microarray containing recombinant proteins sourced from the secretome of Necator americanus. We characterized the most immunoreactive N. americanus proteins and identified their orthologs in the rodent hookworm Nippostrongylus brasiliensis. These N. brasiliensis proteins were expressed recombinantly and tested for efficacy in a mouse challenge model. Our lead vaccine candidate NBR17057 - a homolog of Na_11335 provided robust protection against infection, evidenced by >90% reductions in intestinal worm burden and fecal egg counts, and elevated antigen-specific IgG levels. Vaccination also stimulated IgA+ B-cells, IgG1+/IgA+ plasma cells, and IgG1+/IgA+ germinal centre B-cells in inguinal lymph nodes, indicating gut immunity. Additionally, high levels of IgG1+/IgA+ B-cells, germinal centre B-cells, and plasma cells in the spleen suggest protection against systemic larval migration. The presence of IgG1+/IgA+ memory B-cells in both the lymph nodes and spleen indicates potential long-term immunity against reinfection. Our findings suggest that a N. brasiliensis L3 larval-stage protein NBR17057 and its homolog in N. americanus Na_11335 could serve as a promising subunit vaccine candidate targeting the infective larval stage of human hookworms.
2026-06-07 | Soil-transmitted helminth infections among migrants and travelers: a 10-year retrospective study.
Soil-transmitted helminth (STH) infections, caused by Ascaris lumbricoides, Trichuris trichiura, and hookworms, are widely distributed in tropical regions and significantly contribute to morbidity and mortality. These parasites share similar life cycles, and treatment is primarily based on benzimidazoles. This study aims to describe the clinical and epidemiological characteristics of STH cases among migrants and travelers who attended the Vall d'Hebron-Drassanes International Health Unit between 2014 and 2024, the treatments administered, and their efficacy. This retrospective descriptive study reviewed STH cases associated with migration or travel during the period 2014-2024. Diagnosis was established either through identification of eggs or larvae in stool samples or through the detection of adult Ascaris lumbricoides worms in digestive samples, always associated with a migratory process or international travel. Clinical and epidemiological data were collected and analyzed using SPSS. A total of 361 STH cases were identified, with a mean age of 32 years. Of these, 77% were migrants, 15.7% were travelers visiting friends and relatives (VFR), and 7.2% were travelers. Hookworm infection was diagnosed in 126 cases, Trichuris trichiura in 113 cases, Ascaris lumbricoides in 87 cases, and 35 patients presented with multiple STH infections. Forty-six (12.7%) were symptomatic, predominantly with gastrointestinal symptoms, and 38% presented with eosinophilia. Ninety-five percent of patients received benzimidazole-based treatment, and therapeutic failure was observed in 7.8% of cases. Cure rates were highest for A. lumbricoides (nearly 100%) and remained above 90% for T. trichiura, whereas hookworm infections showed lower rates, ranging from 83% with albendazole to 92% with mebendazole. Multiple STH infections showed, proportionally, higher rates of therapeutic failure. Most STH cases were diagnosed in asymptomatic migrants and VFRs, highlighting the importance of screening for imported diseases in migrant populations. The observed increase in therapeutic failure with commonly used regimens highlights the need to standardize treatment protocols and consider the incorporation of new drugs and combination therapies.
2026-06-04 | Transgenic hookworm secretes anti-tetrodotoxin human single chain antibody.
Biologics, protein- and peptide-based drugs derived from living organisms or cell lines have emerged as effective therapies across a broad range of indications. However, parenteral administration and the need for frequent dosing increase costs and limit compliance, creating an urgent need for innovative platforms capable of continuously, safely, and efficiently delivering sustained biologics in situ within the host. Here, we describe a bioengineered hookworm platform to manufacture and deliver biologic therapeutics in vivo. As proof of concept, we engineer the Ancylostoma ceylanicum secretome by inserting a human single-chain variable fragment antibody (s16-HuScFv) into its genome. Transgene expression does not perturb surrounding gene expression, and heritable transgenesis is confirmed. The s16-HuScFv transgene product is secreted into host circulation and partially neutralizes tetrodotoxin. Given the availability of controlled human infections, our disease-agnostic bioengineered hookworm platform offers a next-generation approach to address a suite of chronic human diseases, and with a single-dose administration, could potentially produce and deliver biologic medicines within the human host for years.
Access all drug discovery papers and probability of success in trials forecasts:
Access all drug discovery papers and probability of success in trials forecasts:
Drug Discovery Landscape
1 orphan drug designation for Ankylostomiasis.
1 orphan drug designation for Ankylostomiasis.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
flubendazole | small molecules | FDA | 2024-10-17 | — | Zero Point Five Therapeutics |
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