Nav: Home

Researchers computationally find the needle in a haystack to treat rare diseases

March 13, 2018

One in 10 people in America is fighting a rare disease, or a disorder that affects fewer than 200,000 Americans. Although there are more than 7,000 rare diseases that collectively affect more than 350 million people worldwide, it is not profitable for the pharmaceutical industry to develop new therapies to treat the small number of people suffering from each rare condition. Researchers at the LSU Computational Systems Biology group have developed a sophisticated and systematic way to identify existing drugs that can be repositioned to treat a rare disease or condition. They have fine-tuned a computer-assisted drug repositioning process that can save time and money in helping these patients receive effective treatment.

"Rare diseases sometimes affect such a small population that discovering treatments would not be financially feasible unless through humanitarian and governmental incentives. These conditions that are sometimes left untreated are labeled 'orphan diseases.' We developed a way to computationally find matches between rare disease protein structures and functions and existing drug interactions that can help treat patients with some of these orphan diseases," said Misagh Naderi, one of the paper's lead authors and a doctoral candidate in the LSU Department of Biological Sciences.

This research will be published this week in the npj Systems Biology and Applications journal, published by the Nature Publishing Group in partnership the Systems Biology Institute.

"In the past, most repurposed drugs were discovered serendipitously. For example, the drug amantadine was first introduced to treat respiratory infections. However, a few years later, a patient with Parkinson's disease experienced a dramatic improvement of her disease symptoms while taking the drug to treat the flu. This observation sparked additional research. Now, amantadine is approved by the Food Drug Administration as both an antiviral and an antiparkinsonian drug. But, we can not only rely on chance to find a treatment for an orphan disease," said Dr. Michal Brylinski, the head of the Computational Systems Biology group at LSU.

To systematize drug repurposing, Naderi, co-author Rajiv Gandhi Govindaraj and colleagues combined eMatchSite, a software developed by the same group with virtual screening to match FDA approved drugs and proteins that are involved in rare diseases. LSU super computers allows them to test millions of possibilities that will cost billions of dollars to test in the lab.
-end-
This work was supported by the National Institute of General Medical Sciences of the National Institutes of Health [R35GM119524].

Louisiana State University

Related Disease Articles:

Findings support role of vascular disease in development of Alzheimer's disease
Among adults who entered a study more than 25 years ago, an increasing number of midlife vascular risk factors, such as obesity, high blood pressure, diabetes, high cholesterol and smoking, were associated with elevated levels of brain amyloid (protein fragments linked to Alzheimer's disease) later in life, according to a study published by JAMA.
Dietary factors associated with substantial proportion of deaths from heart disease, stroke, and disease
Nearly half of all deaths due to heart disease, stroke, and type 2 diabetes in the US in 2012 were associated with suboptimal consumption of certain dietary factors, according to a study appearing in the March 7 issue of JAMA.
Study links changes in oral microbiome with metabolic disease/risk for dental disease
A team of scientists from The Forsyth Institute and the Dasman Diabetes Institute in Kuwait have found that metabolic diseases, which are characterized by high blood pressure, high blood sugar, and obesity -- leads to changes in oral bacteria and puts people with the disease at a greater risk for poor oral health.
Fatty liver disease contributes to cardiovascular disease and vice versa
For the first time, researchers have shown that a bi-directional relationship exists between fatty liver disease and cardiovascular disease.
Seroprevalence and disease burden of chagas disease in south Texas
A paper published in PLOS Neglected Diseases led by researchers at the National School of Tropical Medicine at Baylor College of Medicine suggests that the disease burden in southern Texas is much higher than previously thought.
Maternal chronic disease linked to higher rates of congenital heart disease in babies
Pregnant women with congenital heart defects or type 2 diabetes have a higher risk of giving birth to babies with severe congenital heart disease and should be monitored closely in the prenatal period, according to a study published in CMAJ.
Citrus fruits could help prevent obesity-related heart disease, liver disease, diabetes
Oranges and other citrus fruits are good for you -- they contain plenty of vitamins and substances, such as antioxidants, that can help keep you healthy.
Gallstone disease may increase heart disease risk
A history of gallstone disease was linked to a 23 percent increased risk of developing coronary heart disease.
New disease gene will lead to better screening for pediatric heart disease
Cardiomyopathy, or a deterioration of the ability of the heart muscle to contract, generally leads to progressive heart failure.
Early weight loss in Parkinson's disease patients may signify more serious form of disease
A study led by a Massachusetts General Hospital investigator finds evidence of an association between weight loss in patients with early Parkinson's disease and more rapid disease progression.

Related Disease Reading:

How to Make Disease Disappear
by Rangan Chatterjee (Author)

A much-needed program to prevent and reverse disease, and discover a path to sustainable, long-term health from an acclaimed international doctor and star of the BBC program Doctor in the House.

How to Make Disease Disappear is Dr. Rangan Chatterjee’s revolutionary, yet simple guide to better health—a much-needed, accessible plan that will help you take back control of your health and your life.

A physician dedicated to finding the root cause of ill health rather than simply suppressing symptoms with drugs, Dr. Chatterjee passionately advocates and follows... View Details


Disease: The Story of Disease and Mankind's Continuing Struggle Against It
by Mary Dobson (Author)

Wear and bending to dust jacket. Pages are free of writing and markings. View Details


Diseases of the Human Body
by Carol D. Tamparo PhD CMA-A (AAMA) (Author)

Gain insight on diseases and treatment options for better patient care!


 


Explore human diseases and disorders…from a patient’s perspective!


Join the generations of students who have relied on this popular text to introduce them to common human diseases and disorders. Its concise, well-organized approach makes learning about the conditions nearly painless! Quick-reading and easy to follow, the book can... View Details


Prevent and Reverse Heart Disease: The Revolutionary, Scientifically Proven, Nutrition-Based Cure
by Caldwell B. Esselstyn Jr. (Author)

The New York Times bestselling guide to the lifesaving diet that can both prevent and help reverse the effects of heart disease

Based on the groundbreaking results of his twenty-year nutritional study, Prevent and Reverse Heart Disease by Dr. Caldwell Esselstyn  illustrates that a plant-based, oil-free diet can not only prevent the progression of heart disease but can also reverse its effects.  Dr. Esselstyn is an internationally known surgeon, researcher and former clinician at the Cleveland Clinic and a featured expert in the acclaimed documentary Forks... View Details


The Prevent and Reverse Heart Disease Cookbook: Over 125 Delicious, Life-Changing, Plant-Based Recipes
by Ann Crile Esselstyn (Author), Jane Esselstyn (Author)

The long-awaited cookbook companion to the revolutionary New York Times bestseller Prevent and Reverse Heart Disease.

“I hope you'll treat yourself to one of these recipes and just open that door. I guarantee you won't close it!"
—Samuel L. Jackson

 
Hundreds of thousands of readers have been inspired to turn their lives around by Dr. Caldwell B. Esselstyn’s Jr.’s bestseller, Prevent and Reverse Heart Disease. The plant-based nutrition plan Dr. Esselstyn advocates based on his twenty-year nutritional study—the most... View Details


The Ultimate Women's Guide To Beating Disease 2018 (...and Living a Happy, Active Life)
by Bottom Line Books (Publisher)

Learn how women live with and through one of life's greatest physical test, CANCER. Real, impactful, life changing! View Details


Dr. Dean Ornish's Program for Reversing Heart Disease: The Only System Scientifically Proven to Reverse Heart Disease Without Drugs or Surgery
by Dean Ornish (Author)

The Ornish Diet has been named the “#1 best diet for heart disease” by U.S. News & World Report for seven consecutive years!

Dr. Dean Ornish is the first clinician to offer documented proof that heart disease can be halted, or even reversed, simply by changing your lifestyle. Based on his internationally acclaimed scientific study, which has now been ongoing for years, Dr. Ornish's program has yielded amazing results. Participants reduced or discontinued medications; they learned how to lower high blood pressure; their chest pain diminished or disappeared; they felt... View Details


The Disease Delusion: Conquering the Causes of Chronic Illness for a Healthier, Longer, and Happier Life
by Jeffrey S. Bland (Author), Mark Hyman (Foreword)

For decades, Dr. Jeffrey Bland has been on the cutting edge of Functional Medicine, which seeks to pinpoint and prevent the cause of illness, rather than treat its symptoms. Managing chronic diseases accounts for three quarters of our total healthcare costs, because we’re masking these illnesses with pills and temporary treatments, rather than addressing their underlying causes, he argues. Worse, only treating symptoms leads us down the path of further illness.

In The Disease Delusion, Dr. Bland explains what Functional Medicine is and what it can do for you. While... View Details


Robbins & Cotran Pathologic Basis of Disease, 9e (Robbins Pathology)
by Vinay Kumar MBBS MD FRCPath (Author), Abul K. Abbas MBBS (Author), Jon C. Aster MD PhD (Author)

Dependable, current, and complete, Robbins and Cotran Pathologic Basis of Disease, 9th Edition is the perennially best-selling text that you’ll use long after your medical student days are behind you. A world-class author team headed by Drs. Vinay Kumar, Abul Abbas, and Jon Aster, delivers the latest, most essential pathology knowledge in a readable, interesting manner, ensuring optimal understanding of the latest basic science and clinical content. High-quality photographs and full-color illustrations highlight new information in molecular biology, disease... View Details


The Disease To Please: Curing the People-Pleasing Syndrome
by Harriet B. Braiker (Author)

What's wrong with being a "people pleaser?" Plenty!

"A fascinating book... If you struggle with where, when, and how to draw the line between your own desires and the demands of others, buy this book!"­­Kay Redfield Jamison, bestselling author of An Unquiet Mind and Night Falls Fast

People pleasers are not just nice people who go overboard trying to make everyone happy. Those who suffer from the Disease to Please are people who say "Yes" when they really want to say "No." For them, the uncontrollable need for the elusive approval of others is an... View Details

Best Science Podcasts 2018

We have hand picked the best science podcasts for 2018. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

The Person You Become
Over the course of our lives, we shed parts of our old selves, embrace new ones, and redefine who we are. This hour, TED speakers explore ideas about the experiences that shape the person we become. Guests include aerobatics pilot and public speaker Janine Shepherd, writers Roxane Gay and Taiye Selasi, activist Jackson Bird, and fashion executive Kaustav Dey.
Now Playing: Science for the People

#478 She Has Her Mother's Laugh
What does heredity really mean? Carl Zimmer would argue it's more than your genes along. In "She Has Her Mother’s Laugh: The Power, Perversions, and Potential of Heredity", Zimmer covers the history of genetics and what kinship and heredity really mean when we're discovering how to alter our own DNA, and, potentially, the DNA of our children.