Dr. MacDougall studies a range of health issues primarily affecting children and young adults. His work includes examining how Epidiolex, a cannabidiol-based medication, can help manage seizures and anxiety in children with difficult-to-treat epilepsy. He also investigates innovative treatments for chronic migraines and evaluates methods for restoring fertility in young women with rare genetic conditions like androgen insensitivity syndrome. Furthermore, he focuses on improving healthcare practices for children with hearing loss and enhancing support for healthcare providers through mentorship programs.
Key findings
73% of children showed improvement in both anxiety and seizure rates after 4-6 weeks on Epidiolex.
In a study of 37 patients with chronic migraines, nearly 25% reported their headache days were reduced by over half when using dual calcitonin gene-related peptide antagonists.
39.9% of children with permanent hearing loss had chronic conditions, compared to 8.1% of their peers, and they made 25% more emergency visits.
In patients with Becker muscular dystrophy, treatment with Sevasemten led to a 70% drop in creatine kinase levels, indicating reduced muscle damage.
17.1% of older patients with relapsed or refractory acute myeloid leukemia achieved complete remission with I-apamistamab, compared to none in the standard care group.
Frequently asked questions
Does Dr. MacDougall study epilepsy?
Yes, he studies the use of Epidiolex, a cannabidiol medication, to help reduce seizure frequency and anxiety in children with epilepsy.
What treatments has Dr. MacDougall researched for chronic migraines?
He has researched the effectiveness of using dual medications that block migraine-related molecules to reduce the frequency of headaches.
Is Dr. MacDougall's work relevant to children with hearing loss?
Yes, he has studied healthcare utilization patterns in children with permanent hearing loss to improve their treatment and support.
What does Dr. MacDougall do for young women facing fertility issues?
He investigates fertility preservation techniques, including tissue cryopreservation in young women with conditions like androgen insensitivity syndrome.
How can Dr. MacDougall's research help healthcare providers?
His work on mentorship for nurse practitioners is aimed at better preparing them for their roles and improving patient care.
Publications in plain English
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Metopimazine Mesylate (NG101) in Participants With Gastroparesis.
2026
The American journal of gastroenterology
Loesch J, Hamza E, Pasricha PJ, Nee J, Cline M +8 more
Plain English This study looked at the safety and effectiveness of a medication called NG101 for treating gastroparesis, a condition that causes nausea and stomach discomfort. It involved 161 participants over 12 weeks, and while NG101 did not significantly reduce nausea severity compared to a placebo, patients reported feeling better about their nausea overall during the treatment period. This research is important as it shows NG101 may be a safe option with some positive outcomes, especially for patients with idiopathic gastroparesis.
Who this helps: This helps patients suffering from gastroparesis, particularly those with idiopathic gastroparesis.
Health Service Utilization in Children With Permanent Hearing Loss: A Nested Case Control Study.
2026
Otology & neurotology : official publication of the American Otological Society, American Neurotology Society [and] European Academy of Otology and Neurotology
Olds J, Fitzpatrick EM, Nicholls SG, Rosella LC, Spruin S +5 more
Plain English This study focused on children with permanent hearing loss (PHL) and how often they use healthcare services compared to other children. Researchers found that 39.9% of children with PHL had chronic complex conditions, compared to just 8.1% of their matched peers. Additionally, these children made 25% more emergency visits, 44% more outpatient visits, and stayed in the hospital for 62% longer than the control group in their first two years of life. Understanding these patterns helps improve healthcare planning and support for affected children.
Who this helps: This helps healthcare providers and families of children with hearing loss.
Gonadal Tissue Cryopreservation for a Girl with Complete Androgen Insensitivity Syndrome.
2026
Journal of pediatric and adolescent gynecology
Szybka M, Balaji A, Gelson E, Prakash A, MacDougall J
Plain English This study focused on a 16-year-old girl with complete androgen insensitivity syndrome (CAIS), a condition where individuals have female characteristics despite having male genetic markers. After removing her testes due to cancer risk, doctors preserved part of the tissue to explore possible fertility options. The finding that there were no definite germ cells in her tissue is important because it informs future decisions about fertility preservation for patients with CAIS.
Who this helps: This benefits young women with CAIS and their families, as well as healthcare providers navigating treatment options.
Barriers and facilitators to implementing parent-led infant pain care in rural settings: A qualitative descriptive study using the Theoretical Domains Framework and COM-B Model.
2026
Canadian journal of pain = Revue canadienne de la douleur
Benoit B, Cassidy C, van Wijlen J, Campbell-Yeo M, Hendra S +5 more
Plain English This study looked at how to better involve parents in caring for their babies' pain during medical procedures in rural areas. Researchers interviewed 24 people, including healthcare providers and parents, and found 32 key issues affecting this involvement, such as access to resources and social support. Addressing these issues is important to improve pain management practices for infants, leading to better care and outcomes in rural settings.
Who this helps: This benefits parents and healthcare providers in rural communities.
Evaluating dual calcitonin gene-related peptide antagonists for chronic migraine prevention.
2026
Headache
Graves KS, To J, Paige H, Kennedy AG, Sprouse-Blum AS +2 more
Plain English This study looked at whether using two medications that block a molecule linked to migraines—one given by injection and one taken orally—can help people with chronic migraines. Among 37 patients, those who used both medications reported an average decrease of headache days, with almost a quarter seeing their headaches cut by more than half. This matters because it shows that combining these treatments can provide relief for patients who haven't had success with other options.
Who this helps: This helps patients suffering from chronic migraines who need more effective treatment solutions.
Preliminary Prospective Study of Pharmaceutical-Grade Cannabidiol for Seizure Frequency, Anxiety, and Comorbid Symptoms in Pediatric Epilepsy: Associations With Circulating Endocannabinoids and Lipid Biomarkers.
2026
Journal of child neurology
Hopkins S, Weber A, MacDougall J, Evanski J, Marusak HA +1 more
Plain English This study looked at how Epidiolex, a medication made from cannabidiol (CBD), affects seizure frequency and anxiety in children with hard-to-treat epilepsy. Out of 12 children, 73% showed improvements in both anxiety and seizure rates after taking Epidiolex for 4 to 6 weeks, with minimal side effects. These findings are significant because they suggest that Epidiolex not only helps control seizures but may also reduce anxiety in kids who struggle with both issues.
Who this helps: This helps children with epilepsy and their families, as well as the doctors treating them.
Randomized Phase III SIERRA Trial ofI-Apamistamab Before Allogeneic Hematopoietic Cell Transplantation Versus Conventional Care for Relapsed/Refractory AML.
2025
Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Plain English The study compared a new treatment called I-apamistamab to standard care for older patients with difficult-to-treat acute myeloid leukemia (AML) who needed a stem cell transplant. Researchers found that 17.1% of patients receiving I-apamistamab achieved a lasting complete remission, while none in the standard care group did. This treatment is significant because it offers hope for improving outcomes in a patient group that currently has very few effective options.
Who this helps: Older patients with relapsed or refractory AML.
Impact of Nonmedical Switches From Reference Infliximab to Biosimilars on Disease Control Within a Rheumatology Practice.
2025
The Journal of pharmacy technology : jPT : official publication of the Association of Pharmacy Technicians
Jankowska M, Dessureault K, MacDougall J, Sowers M, Merchand M +1 more
Plain English This study looked at the effects of switching patients with rheumatologic diseases from the original infliximab medication to cheaper biosimilars. Out of 48 patients who switched, 8 (about 42%) experienced a flare-up of their condition after the change, and a quarter of those had delays in receiving treatment due to insurance issues. This is significant because it highlights that many patients may lose control of their disease after switching, suggesting that careful monitoring is crucial during the transition.
Who this helps: This helps patients with rheumatologic diseases and their doctors by emphasizing the need for close follow-up after switching medications.
A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy.
2025
Muscle & nerve
Donovan J, Silverman JA, Barthel B, DuVall M, Madden M +7 more
Plain English This study tested a new drug called Sevasemten in healthy adults and those with Becker muscular dystrophy (BMD). Researchers found that, after treatment, people with BMD experienced significant decreases in muscle injury markers: creatine kinase levels dropped by 70%, fast skeletal muscle troponin I by 98%, and myoglobin by 45%. These findings show that Sevasemten might help reduce muscle damage, which is important for improving the lives of people with BMD.
Who this helps: This benefits patients with Becker muscular dystrophy.
Plain English This study looked at oral food challenges, which are tests used to diagnose food allergies. The researchers found that while these tests are expensive and risky, they are still the best way to determine whether someone can safely eat a potentially harmful food. Proper training and standardized procedures for these tests are crucial to keep patients safe during the process.
Who this helps: Patients with food allergies and their healthcare providers.
Student Integration into Hospital Clinical Pharmacy Services in an Advanced Pharmacy Practice Experience: A Process Evaluation.
2025
The Canadian journal of hospital pharmacy
Kennie-Kaulbach N, Milner N, Davies H, Greco M, MacDougall J +1 more
Plain English This study looked at how to effectively include pharmacy students in hospital clinical services during their training. It involved 71 students and 166 supervisors, with interviews showing that both groups found the students' involvement beneficial for learning and patient care. The research identified six key areas that helped students learn and contribute meaningfully to the pharmacy team, which is important for improving training programs.
Who this helps: This benefits pharmacy students and their supervisors in hospitals.
Supporting Nurse Practitioners Through Virtual and Hybrid Mentorship: Insights From Program Design and Delivery in Nova Scotia.
2025
Nursing leadership (Toronto, Ont.)
Dunlop M, Lloy B, Laprise S, Cashen N, Lane T +3 more
Plain English This study looked at how a mentorship program helps newly graduated nurse practitioners (NPs) in Nova Scotia adjust to their new roles. It found that the first six months are crucial for NPs as they start to define their professional identity, but many still face challenges with their work and support after a year. The research highlights the need for flexible mentorship that adapts to the changing needs of NPs.
Who this helps: This benefits new nurse practitioners as they transition into their careers.
Persistent myeloid cell reprogramming despite miltefosine treatment in leishmania-infected macaques.
2025
iScience
Picard M, Boutrais S, Rodrigues V, Fortier Y, Borde C +13 more
Plain English This study looked at how well miltefosine, a drug used to treat visceral leishmaniasis (a serious disease caused by parasites), works in monkeys infected with the disease. The researchers found that even after treatment, the parasites were still present in various tissues, such as the spleen and bone marrow, three months later. The study shows that miltefosine doesn’t completely clear the infection and leads to significant changes in immune cells that could keep the infection going, which is crucial for understanding how to better treat this disease.
Who this helps: This information benefits doctors and researchers working on treatments for visceral leishmaniasis.
The absorption properties of ZrOnanoparticles in the THz and sub-THz frequency ranges.
2024
RSC advances
MacDougall J, Namai A, Jia F, Yoshikiyo M, Ohkoshi SI
Plain English Researchers studied the ability of small zirconia nanoparticles to absorb terahertz (THz) and sub-terahertz waves, which are important for technologies like 5G communication. They found that the smallest nanoparticles (around 2.6 nm) had the highest absorption value of 1.11 mm, compared to larger particles that absorbed much less (0.04 and 0.18 mm). This is important because better absorption materials can help reduce noise in THz technologies, improving their performance.
Who this helps: This helps engineers and developers of wireless technologies.
Draft Genome Sequences of Three Bacterial Species from Aquatic Habitats near Washington, DC.
2023
Microbiology resource announcements
Leff RT, MacDougall J, Pavloudi C, Oliver L, Slattery K +2 more
Plain English The study explored bacteria found in freshwater ponds near monuments in Washington, DC, by analyzing their genetic material. Researchers identified three types of bacteria, two of which might be new species, but these could not be revived for further study. This research is important for understanding the diversity of bacteria in our environment and their potential roles.
Who this helps: This helps scientists and environmental researchers studying aquatic ecosystems.
Long-term heat-storage materials based on λ-TiOfor green transformation (GX).
2023
Chemical communications (Cambridge, England)
Ohkoshi SI, Yoshikiyo M, MacDougall J, Ikeda Y, Tokoro H
Plain English This research focused on a new type of heat-storage material made from lambda-trititanium-pentoxide, which can hold onto heat for longer periods than traditional materials. The study found that this ceramic can store thermal energy efficiently and release it when needed, significantly broadening its potential uses. This discovery is important because it could lead to better ways to use waste heat energy, helping reduce energy consumption in various industries.
Who this helps: This benefits industries looking for more efficient energy solutions.
Oocyte cryopreservation in mosaic Turner syndrome with polycystic ovaries.
2023
F&S reports
Sienko A, Prakash A, MacDougall J
Plain English This study looked at a woman with a specific type of Turner syndrome and polycystic ovary syndrome (PCOS) who successfully had her eggs retrieved and frozen. After going through two rounds of treatment, she ended up with 19 frozen eggs. This is important because it shows that women with Turner syndrome mosaicism can have options for preserving their fertility, even if they also have PCOS.
Who this helps: Patients with Turner syndrome and their healthcare providers.
Strategies to maximize clinical efficiency while maintaining patient safety during the COVID-19 pandemic: an interview-based study from private practice dermatologists.
2022
The Journal of dermatological treatment
Yim KM, Yim RM, Gaspard S, MacDougall J, Armstrong AW
Plain English This study looked at how COVID-19 changed the way dermatologists in cities provided care for their patients. Researchers found that during the height of the pandemic, patient visits dropped by over 65%, but by July 2020, this decline improved to about 15%. Even with fewer patients, there were more urgent skin issues and cancer concerns, highlighting the need for dermatologists to adapt their practices, such as using telehealth and adjusting operations to keep both staff and patients safe.
Who this helps: This benefits both patients and dermatologists by ensuring timely care while maintaining safety during the pandemic.
Operationalizing integrated needs-based workforce planning at Nova Scotia Health in response to the COVID-19 pandemic.
2022
Healthcare management forum
MacKenzie A, MacQuarrie C, Murphy M, Piers G, Philopoulos K +7 more
Plain English This study looked at how Nova Scotia Health organized its workforce to address challenges during the COVID-19 pandemic. They found that there were significant shortages of staff in various healthcare roles, which would impact patient care. By taking action to increase the number of qualified workers, they were able to ensure that enough personnel were available to care for COVID-19 patients during the third wave of the pandemic, which is important for managing healthcare effectively moving forward.
Who this helps: This helps patients and healthcare providers by ensuring that there are enough staff to care for those affected by COVID-19.
Codevelopment of Implementation Interventions to Support Parent-Led Care for Pain in Infants: Protocol for a Qualitative Descriptive Study.
2022
JMIR research protocols
Benoit B, Cassidy C, van Wijlen J, Correll Q, Campbell-Yeo M +4 more
Plain English This study looked at how to improve the use of simple, effective methods for easing pain in infants, like breastfeeding and skin-to-skin contact, which are often overlooked in hospitals. Researchers explored the challenges and support needed for parents to use these pain relief techniques during medical procedures and worked on designing practical solutions. Ultimately, their findings aim to enhance pain care for infants, which is crucial for improving their health and safety.
Who this helps: This helps infants and their families receive better pain management during medical care.
30 Gbit/s PAM4 transmission using an 8-GHz directly modulated multi-section laser.
2022
Optics express
Troncoso Costas M, Reza AG, Putta H, Sharma A, Jain G +6 more
Plain English This study looked at a new type of laser that can send data very quickly over fiber optic cables. The researchers found that this laser can successfully transmit data at 30 gigabits per second over distances of up to 75 kilometers without needing extra amplifiers. This is important because it could improve internet speeds and connect more people, particularly in areas where upgrading existing infrastructure is challenging.
Who this helps: This benefits internet service providers and their customers.
Oral ENT-01 Targets Enteric Neurons to Treat Constipation in Parkinson Disease : A Randomized Controlled Trial.
2022
Annals of internal medicine
Camilleri M, Subramanian T, Pagan F, Isaacson S, Gil R +32 more
Plain English This study looked at a new treatment called ENT-01 to help relieve constipation in people with Parkinson's disease (PD). Over 25 days, patients taking ENT-01 had an increase in their weekly complete bowel movements from 0.7 to 3.2, while those on a placebo only increased from 0.7 to 1.2. This is important because constipation can significantly impact the quality of life for people with PD, and ENT-01 not only improved bowel movements but also showed signs of helping with related symptoms like dementia and psychosis.
Who this helps: This helps patients with Parkinson's disease who suffer from constipation and related gastrointestinal issues.
An AUC Target Simulation for Vancomycin in Patients With Class III Obesity.
2021
Journal of pharmacy practice
Langton MM, Ahern JW, MacDougall J
Plain English This study focused on how to better determine the right dose of vancomycin, an antibiotic, for patients with severe obesity. Researchers found that using a method focused on achieving a specific concentration over time (target AUC) required less medication—about 2,783 mg compared to 3,995 mg with the traditional method of monitoring trough levels—making it a more effective option. This matters because it could lower the chances of kidney damage from too high a dose of the drug in these patients.
Who this helps: This helps patients with class III obesity who need vancomycin treatment.
Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial.
2021
Neuromuscular disorders : NMD
Finkel RS, Finanger E, Vandenborne K, Sweeney HL, Tennekoon G +13 more
Plain English This study tested a new drug called edasalonexent in young boys with Duchenne muscular dystrophy, a condition that weakens muscles over time. The research found that when the boys took 100 mg/kg of edasalonexent daily, their disease progression slowed, and their muscle health improved, with fewer signs of inflammation. These findings are important because they suggest that edasalonexent could help preserve muscle function in boys with this condition and guide future research.
Who this helps: This helps boys with Duchenne muscular dystrophy.
Toward Chemical Validation of Leishmania infantum Ribose 5-Phosphate Isomerase as a Drug Target.
2021
Antimicrobial agents and chemotherapy
Dickie EA, Ronin C, Sá M, Ciesielski F, Trouche N +8 more
Plain English This study focused on a specific enzyme called ribose 5-phosphate isomerase B (RpiB) in the parasite Leishmania infantum, which causes serious tropical diseases. Researchers tested many small chemical pieces (fragments) to see if they could effectively block this enzyme and found that some of these fragments could successfully stop the growth of the parasite. These findings are important because they identify RpiB as a potential target for new drug development, which is crucial for treating infections caused by this parasite.
Who this helps: This helps patients suffering from leishmaniasis and other infections caused by related parasites.
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.
2021
Journal of neuromuscular diseases
Finkel RS, McDonald CM, Lee Sweeney H, Finanger E, Neil Knierbein E +22 more
Plain English This study tested a new drug called edasalonexent in children aged 4 to under 8 years who have Duchenne muscular dystrophy, a condition that weakens muscles. Over a year, 131 kids took either edasalonexent or a placebo, and while the overall results didn’t show a clear improvement in muscle function measures, younger children (under 6 years old) did show some positive effects. This matters because starting treatment early may help slow down muscle decline in these young patients.
Who this helps: Younger patients with Duchenne muscular dystrophy.
DPP inhibition alters the CXCR3 axis and enhances NK and CD8+ T cell infiltration to improve anti-PD1 efficacy in murine models of pancreatic ductal adenocarcinoma.
2021
Journal for immunotherapy of cancer
Fitzgerald AA, Wang S, Agarwal V, Marcisak EF, Zuo A +9 more
Plain English This study looked at a new treatment method for pancreatic cancer, specifically how a drug called BXCL701 can improve the effectiveness of another cancer treatment known as anti-PD1 therapy. The researchers found that using BXCL701 with anti-PD1 resulted in increased immune cells, like natural killer (NK) cells and CD8+ T cells, in tumors and reduced tumor growth—up to 50% smaller tumors in mice. Importantly, this combination treatment led to lasting immune responses that even cleared some tumors after further exposure, highlighting the potential for better outcomes in patients.
Who this helps: This benefits patients with pancreatic cancer by offering a potential new treatment option.
Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial.
2020
JAMA neurology
Howard JF, Nowak RJ, Wolfe GI, Freimer ML, Vu TH +81 more
Plain English This study looked at a new treatment called zilucoplan for patients with moderate to severe generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Among 44 patients, those who received the higher 0.3 mg/kg dose of zilucoplan saw their muscle weakness scores improve by an average of 6 points over 12 weeks compared to those on a placebo, which suggests the treatment is effective. This improvement matters because it shows that zilucoplan can help patients feel better and perform daily activities more easily without the commonly experienced side effects from traditional treatments.
Who this helps: Patients with moderate to severe generalized myasthenia gravis.
Antileishmanial Drugs Modulate IL-12 Expression and Inflammasome Activation in Primary Human Cells.
2020
Journal of immunology (Baltimore, Md. : 1950)
André S, Rodrigues V, Pemberton S, Laforge M, Fortier Y +3 more
Plain English This study looked at how three antileishmanial drugs—amphotericin B (AmB), miltefosine, and pentamidine—affect human immune cells. The researchers found that all three drugs slowed down the growth of certain immune cells, but AmB also triggered the release of specific inflammatory signals that could affect immune responses. Importantly, they discovered that manipulating those signals could potentially restore the immune cell growth inhibited by AmB.
Who this helps: This research benefits patients being treated for leishmaniasis, as it provides insights into how treatments can affect their immune systems.
Normosmic idiopathic hypogonadotropic hypogonadism due to a novel GNRH1 variant in two siblings.
2020
Endocrinology, diabetes & metabolism case reports
Sagi SV, Joshi H, Whiles E, Hikmat M, Puthi VR +5 more
Plain English This study looked at two siblings who both had a rare genetic condition called normosmic idiopathic hypogonadotropic hypogonadism (IHH), which causes delayed puberty and fertility issues. They found that both siblings had a new genetic variant affecting a hormone crucial for puberty, with the boy showing symptoms by age 17 and the girl by age 16. This research is important because early diagnosis and treatment can help affected individuals develop normally and avoid long-term health and psychological problems.
Who this helps: Patients with hypogonadotropic hypogonadism and their families.
Proton Pump Inhibitor-Induced Galactorrhea in a Kidney Transplant Recipient: A Friend or Foe?
2020
Case reports in transplantation
Prikis M, MacDougall J, Narasimhadevara N
Plain English This study looked at a kidney transplant patient who developed unexpected milk production and high levels of prolactin while taking a medication called omeprazole to treat stomach issues. The researchers found that the omeprazole might not be properly removed from her body due to her kidney condition, leading to harmful effects on her kidneys and the unusual symptom of galactorrhea. This is important because it highlights the need for careful evaluation of common medications in patients with kidney problems to prevent serious side effects.
Who this helps: Patients with kidney transplants and their doctors.
A prospective multicenter observational study of cell-mediated immunity as a predictor for cytomegalovirus infection in kidney transplant recipients.
2019
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons
Kumar D, Chin-Hong P, Kayler L, Wojciechowski D, Limaye AP +7 more
Plain English This study looked at how well a blood test can predict cytomegalovirus (CMV) infections in kidney transplant patients. Researchers tested 583 patients and found that those who had a positive test result had a much lower risk of CMV infection (3.0%) compared to those with a negative result (19.5%). This is important because being able to accurately predict CMV infections can help doctors protect patients after transplant.
Who this helps: This benefits kidney transplant patients and their doctors.
Randomised clinical trial: a placebo-controlled study of subcutaneous or intradermal NEXVAX2, an investigational immunomodulatory peptide therapy for coeliac disease.
2019
Alimentary pharmacology & therapeutics
Truitt KE, Daveson AJM, Ee HC, Goel G, MacDougall J +2 more
Plain English This study looked at a new treatment called Nexvax2 for people with celiac disease, aiming to help them tolerate gluten. Researchers compared how well the treatment worked when given as an injection under the skin (subcutaneous) versus just below the skin (intradermal). They found that both methods provided similar levels of the treatment in the body, and the subcutaneous method helped reduce negative immune reactions to gluten, with mild side effects reported.
Who this helps: This benefits patients with celiac disease looking for better ways to manage their gluten intolerance.
Elevated serum interleukin-2 after gluten correlates with symptoms and is a potential diagnostic biomarker for coeliac disease.
2019
Alimentary pharmacology & therapeutics
Tye-Din JA, Daveson AJM, Ee HC, Goel G, MacDougall J +6 more
Plain English Researchers studied how gluten affects people with coeliac disease by giving them a small amount of gluten and measuring their symptoms and a protein called interleukin-2 in their blood. They found that 92% of coeliac patients showed elevated interleukin-2 levels four hours after eating gluten, which matched the worsening of symptoms like nausea and vomiting. This research highlights that tracking interleukin-2 levels can help doctors diagnose coeliac disease in patients who already follow a gluten-free diet.
Who this helps: This helps patients with coeliac disease and their doctors.
Differentiating Familial Chylomicronemia Syndrome From Multifactorial Severe Hypertriglyceridemia by Clinical Profiles.
2019
Journal of the Endocrine Society
O'Dea LSL, MacDougall J, Alexander VJ, Digenio A, Hubbard B +8 more
Plain English This study examined how to tell apart two conditions that cause high fat levels in the blood: familial chylomicronemia syndrome (FCS) and multifactorial severe hypertriglyceridemia (sHTG). Researchers looked at data from 154 patients and found that they could correctly diagnose 45 out of 49 patients with FCS and 99 out of 105 patients with sHTG, achieving an impressive accuracy of 93.5%. This is important because correctly diagnosing these conditions can improve patient care and treatment options.
Who this helps: Patients with these metabolic disorders and their healthcare providers.
A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration.
2018
Blood cells, molecules & diseases
Peterschmitt MJ, Cox GF, Ibrahim J, MacDougall J, Underhill LH +2 more
Plain English This study looked at the side effects experienced by 393 adults with Gaucher disease type 1 who took eliglustat, an oral medication. Out of these patients, 334 reported having one or more side effects, but 92% continued with the treatment, and only 3% stopped because of a side effect. Most of the side effects were mild or moderate, with none being serious enough to require stopping the drug, which is important for ensuring that patients can maintain their treatment.
Who this helps: This benefits patients with Gaucher disease type 1 and their doctors by providing clarity on the safety and tolerability of eliglustat.
Inhibitors of Trypanosoma cruzi Sir2 related protein 1 as potential drugs against Chagas disease.
2018
PLoS neglected tropical diseases
Gaspar L, Coron RP, KongThoo Lin P, Costa DM, Perez-Cabezas B +11 more
Plain English Researchers studied a protein from the parasite that causes Chagas disease to see if they could develop new drugs to treat the illness. They found that a compound called BNIP Spermidine (BNIPSpd) was the most effective at inhibiting the protein and showed potential similar to an existing drug, but it didn't work in a mouse model for the disease. This is important because Chagas disease is a major health issue in Latin America, and finding new treatments could greatly help patients.
Who this helps: Patients with Chagas disease.
Qiao R, Xia Y, Feng X, Macdougall J, Pepper J +12 more
Plain English Researchers developed a new technique to study liquids under high pressure using soft x-rays. They found that a specialized thin membrane could contain liquids under pressures greater than 60 bars while allowing x-rays to pass through. This advancement is important because it enables detailed analysis of liquid properties at high pressure, which can improve our understanding of various materials and chemical processes.
Who this helps: This benefits scientists and researchers studying advanced materials and chemical reactions under high-pressure conditions.
Utility of the T-SPOT.TB test's borderline category to increase test resolution for results around the cut-off point.
2018
Tuberculosis (Edinburgh, Scotland)
Rego K, Pereira K, MacDougall J, Cruikshank W
Plain English This study looked at how the T-SPOT.TB test, used to detect tuberculosis (TB) infections, can more accurately identify cases that fall near the test's cut-off point by including a "borderline" category. Researchers analyzed nearly 646,000 tests and found that 1.8% were borderline. Among these borderline results, 59.2% turned out to be negative, while 20% turned positive upon retesting, indicating that the borderline category can help better determine a person's TB status and prevent misclassifications.
Who this helps: This benefits patients and doctors by providing clearer test results, especially for those at risk for TB.
The crystal structure of the Leishmania infantum Silent Information Regulator 2 related protein 1: Implications to protein function and drug design.
2018
PloS one
Ronin C, Costa DM, Tavares J, Faria J, Ciesielski F +5 more
Plain English This study examined the structure of a protein called LiSir2rp1 from the Leishmania infantum parasite, which causes the disease leishmaniasis. Researchers found that this protein has a distinct shape that is similar to proteins from yeast and humans, revealing important details about how it interacts with other molecules. Understanding this protein's structure is important because it could lead to new drugs targeting it, which is crucial for the survival of the parasite and could help treat infections in patients.
Who this helps: This helps patients suffering from leishmaniasis.
Validation of standard operating procedures in a multicenter retrospective study to identify -omics biomarkers for chronic low back pain.
2017
PloS one
Dagostino C, De Gregori M, Gieger C, Manz J, Gudelj I +13 more
Plain English This study focused on developing a standardized method for collecting and analyzing biological samples to find potential new markers for chronic low back pain (CLBP). Researchers tested their procedure across five different centers and confirmed it was effective, ensuring that the samples collected were consistent and reliable. This is important because it lays the groundwork for future research aimed at creating personalized treatments for CLBP, a condition that affects millions of people.
Who this helps: Patients suffering from chronic low back pain.
The Future of Drug Development for Neglected Tropical Diseases: How the European Commission Can Continue to Make a Difference.
2017
Trends in parasitology
Pierce RJ, MacDougall J, Leurs R, Costi MP
Plain English This paper discusses how the European Commission can improve funding for developing new drugs for neglected tropical diseases (NTDs). The authors recommend that the EC focus on supporting the entire process of turning promising compounds into drugs ready for clinical trials. Making these changes could lead to more effective treatments for diseases that currently lack sufficient attention and funding.
Who this helps: Patients suffering from neglected tropical diseases.
Impact of Ethylene Glycol Toxicity on Donor Organ Viability: A Case Report.
2017
Transplantation proceedings
Mendelson NL, Fitzgerald A, Kumar A, Pineda JA, MacDougall J +4 more
Plain English This study examined the use of kidneys from deceased donors who died due to ethylene glycol (EG) poisoning to find out if they can still be safely transplanted. The researchers discussed the challenges and potential processes for using these kidneys, emphasizing that every possible organ should be considered to address the shortage of transplantable organs. This is important because there's a rising number of patients on waiting lists who could benefit from these organs, potentially saving lives as we improve the use of all available kidneys.
Who this helps: This helps patients in need of kidney transplants.
'Omics' biomarkers associated with chronic low back pain: protocol of a retrospective longitudinal study.
2016
BMJ open
Allegri M, De Gregori M, Minella CE, Klersy C, Wang W +18 more
Plain English This study is examining the biological markers related to chronic low back pain (CLBP) to better understand its causes and improve treatment. Researchers will collect data from 12,000 people, including 4,000 with CLBP and 8,000 without, focusing on genetics and other biological factors. The goal is to identify specific genetic traits and other biomarkers that could help personalize and enhance treatment for those suffering from this condition.
Who this helps: Patients with chronic low back pain.