Recent information came to light that Ionis is still continuing pursue a drug for myotonic dystrophy. The first trial ended and was not successful. What we learned is that the team was impressed that progress was made. Rather than pursue this initial drug they may be switching to a new more highly improved drug. This makes sense as the cost to pursue a drug is high $$$$ and you want your best candidate forward.
The IDMC meeting will be in San Fransisco this year. Make plans to attend the scientific sessions or the meeting of the Myotonic Dystrophy foundation
Please save September 5th – 9th, 2017 for the IDMC-11 conference being held in San Francisco, California. If you are interested in receiving updates about IDMC-11, please sign up online at www.idmc11.org.
Welcome to the IDMC website, home of the International Myotonic Dystrophy Consortium (IDMC, or International Dystrophia Myotonica Consortia). This site is dedicated to the community of scientists, physicians and health care providers who have taken up the fight against Myotonic Dystrophy, a progressive neuromuscular disease that effects people and families around the world.
In a instant news email the Myotonic Dystrophy Foundation (MDF) released information that the Ionis Pharmaceutical Drug DMPK-2.5Rx research project has been canceled. The drug DMPK-2.5Rx did not work, and did not get the correct amount of therapeutic drugs into the cells of the patients with myotonic dystrophy. The company may still continue research on a more potent combination but the current trial is halted.
This is hard to hear news for the myotonic community. This is the second drug in development to fail. This new drug is part of a number of new generation of interest drugs in trying to find a drug to treat the disease. There are still a number of drugs in development but the Ionis one was the most advanced. Perhaps the information in this trial will be of help to the other drugs in development. For those in the late stages of the disease the length of time to find a treatment that is FDA approved in unlikely now.
There continues to be some “off label” treatments including erythromycin and some NSAIDS as well as Actinomycin-D but none have had any proven human effect.
More information below.
Ionis Pharmaceuticals Reports on
DMPKRx Phase 1/2 Clinical Trial
When my son was born I had no idea that he would be an Over 5 Million Dollar Man.But he was born as a premmie and our medical bills started even before Day 1. The major cost of the disease concentrations on respiratory symptoms. For young people with this disease a strong effort is made to stabilize and improve respiratory function. The recovery period for individuals with this disease is very lengthy.
Moms confinement before delivery $200,000
85 day NICU stay $1,400,000
Ear Tube Placements (8) 40,000
Special Education Grade 0-6 200,000
Heel Cord Surgery 25,000
Special Education grade 7-14 210,000
Caregivers state of Ca 2012-2016 450,000
Hospitalization 2010 (80 days) 875,000
Hospitalization 2015 (79 Days) 850,000
Hospitalization 2016 (89 days) 950,000
Cardiac Monitoring 40,000
Currently we are at US$5.2 Million in Hospital and Medical Care for him. The insurance has picked up all the costs incurred, but still its a high price.
In the USA the incident of congenital myotonic dystrophy is 1/100,000. That computes to about 3,500 individuals with the disease. At a five million dollar overall cost the math computes to an overall projected cost could top 18.2 billion.
If the 5 million is a outlier the cost would still be in the neighborhood of $10 billion overall cost. That’s a very high cost to society.
In a study published in December 2015 in a peer review journal researchers from Japan and Poland found that a commonly used antibiotic might assist in the treatment of Myotonic Dystrophy. This is a sort of stunning discovery as there is no treatment identified to treat the disease. Treatment now consists of reducing symptoms.
The researchers first began by screening antibiotics. In a screen of 20 antibiotics 2-3 were found to have some potential with the disease.When screening the drugs they first used mice cells and lab equipment to find the most promising compounds (drugs). . Erythromyicin was found to have the highest attraction to the RNA CUG expansion (The opposite of CTG repeats in the DNA) Erythromycin was the drug that the researchers chose to study. Click here for the screening graph Muscleblind and Various antibiotics and compounds