Updated: May 16, 2018 05:47 PM
A cure for cancer will hopefully be discovered one day, and cures for other diseases too. But medical breakthroughs don't happen very often. That's why this story is so significant. At the center of it all, is a little boy named Logan who happens to live right here in the Northland. A genetic missing link has been found in him and people are now asking, does Logan hold the key to a cure?
It all started about a year ago when Mia and Adam Forrestal brought their then five-year old son to a doctor. He was flare footed, which means he walked with his toes sticking out quite a bit. The Doctor also suggested a routine blood test. The next day, on February 15th, 2017, Logan's dad, answered the phone. "They called and told me they had the lab work back and said we have a medical indicator that your son has muscular dystrophy".
A painful diagnosis for any parent to hear, and painful news for a doctor to deliver. Duluth pediatric neurologist, Dr. Richard Kanoff sees the debilitating affects of this disease every day. "The moment you diagnose this problem and say muscular dystrophy, those words generally convey a life expectancy somewhere between the teens and early 20s".
Dr. Kanoff says the progression of the disease is heart-breaking to witness. "These patients go from walking ok, to not ok, to can't walk, to being in wheelchairs, to barely being able to move their hands, and usually succumb to either respiratory or heart problems to their teens or 20s".
Even more heart-breaking for parents. Logan's mom, Mia told us, "For a parent to know that they are going to outlive their child, is very hard, the hardest thing that I've ever had to come to grips with".
Muscular dystrophy is a very complicated disease. Imagine a healthy dystrophin gene looking something like a long chain of lego blocks with everything lining nes up as it should. In dushen muscular dystrophy patients like logan however, a segment is missing. In his case, what's called the 51st exon. And that gap stops muscle development and eventually the muscle cells in the body fall apart. But a brand new drug incorporating exon-skipping therapy fills the gap in the gene to help the gene function better. And this is where Logan's story takes a remarkable turn.
"So logan our boy, came back with a very special mutation", Dr. Kanoff says with a smile. Logan's special mutation or missing link, makes him an extremely rare and fortunate candidate for a drug that was recently given the government okay. "There is a brand new drug that was FDA approved last year that is specifically designed for his type of genetic mutation", Dr. Kanoff told us.
That new drug is called, Exondys 51, and in January Logan began getting it in weekly intervenis infusions. Dr. Kanoff says filling the dystrophin gene gap could be truly revolutionary. "Although we don't make normal distrophin, we seem to make something that works better, and this is life-saving, a huge breakthrough!"
Fewer than a half dozen patients in Minnesota have this specific type of gene mutation, and Dr. Kanoff says for Logan, it seems to really be working. "All of us that see him agree that he is actually functioning better now than when he was first diagnosed. The way he walks, his endurance, what he can do is all better!"
Logan was a little shy while we were visiting the family, but he's a real trooper. Now, six years old he stayed occupied with things like cartoons, caterpillars and butterflies.
Logan will have to get the weekly drug for the rest of his life. No one is making any predictions, but Dr. Kanoff says this could not only be life changing for Logan, it could be life extending and even lead to an eventual cure for this dreaded disease. Mom and dad agree, it's important to see this through.
"It's a huge deal!", Mia Forrestal says. "It's so amazing and I feel just hope! Just pure hope that we have something that might you know, in his life time, we might see a cure for this!"
As Dr. Kanoff thinks back over the many years of raising money for research for muscular dystrophy, he says, "I've watched the Jerry Lewis telethons, I stop on the corner for the firemen who have been out there for how many decades, filling the boot. This is what it was for! It's reaching fruition of what all that was for!"
Dr. Kanoff is not alone. For many of us, the term muscular dystrophy brings memories of the Jerry Lewis telethons. They were broadcast nationally for more than 40 years. Or we may also think of our local firefighters, out there at intersections for their fill the boot campaigns. Dr. Kanoff says decades of research is all leading to this. "How many years have we given our dollars and our phone calls and raised money? This is it! This is for Logan!"
And there is more good news around the corner. There are already new, next generation drugs in the pipeline that Dr. Kanoff says are designed to take this discovery even closer toward a possible cure. The hope here is that Logan will not only do better with this drug now, but that he'll also qualify for those next new drugs down the line. We wish Dr. Kanoff, Logan and the Forrestals the very best.
Updated: May 16, 2018 05:47 PM
Created: May 16, 2018 05:00 PM
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