Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

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

13.03.2018

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.


Chemotherapeutic vandetanib bound to its main target Protein Tyrosine Kinase 6 (PTK6) in purple, which is involved in many cancers including gastrointestinal tumors and ovarian cancers. By modeling vandetanib and PTK6 complex, researchers at LSU found that the KRAS protein to also contain a similar drug-binding site and therefore to be a good match for the same drug. The computer-generated model of KRAS in gold with vandetanib depicts the predicted interaction.

Credit: Misagh Naderi, LSU

"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.

###

This work was supported by the National Institute of General Medical Sciences of the National Institutes of Health [R35GM119524].

Media Contact

Alison Satake
asatake@lsu.edu
225-578-3870

 @LSUResearchNews

http://www.lsu.edu 

Alison Satake | EurekAlert!

More articles from Life Sciences:

nachricht Quality control in immune communication: Chaperones detect immature signaling molecules in the immune system
20.09.2019 | Technische Universität München

nachricht Moderately Common Plants Show Highest Relative Losses
20.09.2019 | Universität Rostock

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: 'Nanochains' could increase battery capacity, cut charging time

How long the battery of your phone or computer lasts depends on how many lithium ions can be stored in the battery's negative electrode material. If the battery runs out of these ions, it can't generate an electrical current to run a device and ultimately fails.

Materials with a higher lithium ion storage capacity are either too heavy or the wrong shape to replace graphite, the electrode material currently used in...

Im Focus: Stevens team closes in on 'holy grail' of room temperature quantum computing chips

Photons interact on chip-based system with unprecedented efficiency

To process information, photons must interact. However, these tiny packets of light want nothing to do with each other, each passing by without altering the...

Im Focus: Happy hour for time-resolved crystallography

Researchers from the Department of Atomically Resolved Dynamics of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg, the University of Hamburg and the European Molecular Biology Laboratory (EMBL) outstation in the city have developed a new method to watch biomolecules at work. This method dramatically simplifies starting enzymatic reactions by mixing a cocktail of small amounts of liquids with protein crystals. Determination of the protein structures at different times after mixing can be assembled into a time-lapse sequence that shows the molecular foundations of biology.

The functions of biomolecules are determined by their motions and structural changes. Yet it is a formidable challenge to understand these dynamic motions.

Im Focus: Modular OLED light strips

At the International Symposium on Automotive Lighting 2019 (ISAL) in Darmstadt from September 23 to 25, 2019, the Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP, a provider of research and development services in the field of organic electronics, will present OLED light strips of any length with additional functionalities for the first time at booth no. 37.

Almost everyone is familiar with light strips for interior design. LED strips are available by the metre in DIY stores around the corner and are just as often...

Im Focus: Tomorrow´s coolants of choice

Scientists assess the potential of magnetic-cooling materials

Later during this century, around 2060, a paradigm shift in global energy consumption is expected: we will spend more energy for cooling than for heating....

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

Optical Technologies: International Symposium „Future Optics“ in Hannover

19.09.2019 | Event News

Society 5.0: putting humans at the heart of digitalisation

10.09.2019 | Event News

Interspeech 2019 conference: Alexa and Siri in Graz

04.09.2019 | Event News

 
Latest News

Quality control in immune communication: Chaperones detect immature signaling molecules in the immune system

20.09.2019 | Life Sciences

Moderately Common Plants Show Highest Relative Losses

20.09.2019 | Life Sciences

The Fluid Fingerprint of Hurricanes

20.09.2019 | Life Sciences

VideoLinks
Science & Research
Overview of more VideoLinks >>>