From St Paul Pioneer Press Sept 7, 2018
Written by Ruben Rosario
Leo the lion-hearted’s success
Mayo may study parents’ non-traditional treatment for toddler’s incurable disease
Never underestimate the steps loving and refuse-to-take-no-foran- answer parents will take to find a cure or life-saving treatment for their sick child.
The parents behind the "Lorenzo’s Oil" story, which was made into a popular 1992 film, bucked doctors, studies and pessimism and uncovered a medication that arguably helped extend the life of their terminally ill son by decades.
Now meet, in a sense, their Minnesota counterparts— the St. Martins of West St. Paul. The parents, Denis and Anne, both 36, are saltof- the-earth folks. He’s an electrical foreman with the International Brotherhood of Electrical Workers, St. Paul Local 110. Until the lifethreatening medical drama involving their youngest child, Leo Victor, she w orked as an operations account manager for the Boys and Girls Club of the Twin Cities.
Long story short: The St. Martins, with the invaluable help of a close family friend, came up with a dietary formula and regimen they believe has helped improve the toddler’s battle against infantile Pompe, a serious, lifelong and incurable disease. Their effort now serves as the catalyst and inspiration for a proposed clinical trial spearheaded by Mayo Clinic physicians.
“In my career, nobody (parent) has ever come to me like that for a first visit with a research plan and a hypothesis”, Dr. Jonathan Johnson, the toddler’s cardiologist, informed me this week.
“Impressive,” Johnson added during the chat, “would be the word to use for the amount of research they had done and the amount of detail they had looked into and wanting to work with the medical community … to figure out away to advance therapy for these children.”
Let’s go back first to the bittersweet events of 2016. Leo, the couple’s second child, was born in May. A month later, the couple’s garage went up in flames during the middle of the night.
The family escaped the blaze and “made it to the driveway just in time to watch the fire jump from the garage to the house,” the couple explain on their Pompe Warrior Foundation online site.
It was during their stay in temporary housing that the couple noticed that Leo could not turn his head to the left and was sweating often through his pajamas. He later spit out his food and it appeared his muscles became weaker.
Leo’s heart, it turned out, had grown to the size of his! chest cavity, and his heart muscle was thick. What followed was a string of doctor visits that forced both parents to leave work with the blessings and support of their respective employers. A now defunct Gofundme page was started by family and friends to help defray fire and other expenses.
Leo was eventually diagnosed with infantile Pompe Disease. The disorder prevents the body from breaking down glycogen. Current life expectancy is two years or less.
Leo, now 2 years and 4 months and counting, underwent weekly enzyme-replacement infusion therapy, a ninehour- long treatment which tries to break down glycogen in the body. But its effectiveness is rated at 1 to 5 percent, according to Denis St. Martin.
Leo still produced bloodsu gar levels too high to stop the storage of glycogen. The St. Martins thought limiting their child’s intake of sugar and carbohydrates wouldhelp. But doctors “kept telling us that there’s no published medical research that dietary treatments would have any effect,” Anne said Thursday.
Enter Donald Bennett. The U.S. Army veteran was a close buddy of Anne’s youngest brother, Sgt. Frank Joseph Michael Vanek, who was killed in action in Iraq in 2007.
Both were members of Charlie Company 2nd Battalion 325th Airborne Infantry Regiment, 82nd Airborne Division.
Bennett, injured eight years ago during a parachuting mishap while training, learned about nutritional ketosis as part of a comprehensive recovery treatment put together by a former Minnesota Vikings strength coach then working for the De! partment of Defense.
Bennett, now a personal health consultant, went to work for Leo and the family of his deceased Army buddy.
While the St. Martins were at the hospital or working on rebuilding the damaged home, Bennett pored through piles of research papers on Pompe Disease, Lysosomal storage diseases and ketogenic diets from all over the world.
“I had an amazing recovery,” Bennett explained Thursday. “When you see a kid with a genetic disorder like this, why wouldn’t you apply that level of treatment I received? It seemed like common sense from my perspective.”
In February of 2017, the parents placed Leo on a diet designed by Bennett. It involves a 760-calorie-a-day regimen that roughly breaks down to 65 percent fat, 30 percent protein and about 5 percent carbohydrates. Call it Leo’s Oil. Its goal is to significantly limit glycogen intake by replacing it with an alternative body fuel rich in ketones. It seems to be working.
Leo’s blood sugar levels plummeted below 100 — the level at which the body begins storing glycogen. His health drastically improved. They shared their results with doctors, who still had doubts the diet was the main reason for the improvement.
Anne turned into what could be aptly described as a mother force of nature and tracked down physicians with the best knowledge and experience on the disease. She came upon Dr. Marc Patterson, Mayo’s chief of neurology for pediatrics and adolescents. He read their detaile d regimen and results and called to meet within a week.
"He saw the same logic we did,"Denis St. Martin said. Patterson is now a key figure in efforts to design and fund the trial study. Bennett has been asked to write up the protocol for the study.
A year ago, Johnson, Leo’s cardiologist, compared Leo’s echocardiogram images taken before the dietary supplement with one done at the Mayo Clinic. It’s like looking at the hearts of two different persons, the doctor said.
The heart chamber wall thickness is clearly visible in the first image, which appears in a video posted on the foundation’s website. The thickness has virtually disappeared in the second one, as shown in the video.
“Leo’s heart is now essentially normal,” said Johnson, who supports the proposed study.
Although the boy still has to undergo the weekly ERT treatments" we don’t see any problems at all,"he added. “We use the (echo images) now to teach fellows what Pompe looks like and what can happen with treatment. It’s such an incredible change in a really unbelievable case.”
The St. Martins and Bennett established the foundation as a way to raise more awareness about Leo’s story and success so far. They will hold an event Saturday at Luv ice cream in North St. Paul from noon to 4 p.m. The business advocates the elimination of sugar in the American diet.
The event will feature games for kids, a jazz band and Maria Emmerich, a New York Times bestselling ketogenic cookbook author.
“We know! we are doing the right thing for him, and we can continue just doing that,” Denis told me." But that’s not our goal. Our goal is to take this and apply to everybody else, and help with other diseases along the way."
You go, little Leo and company.