Cyclists’ Hot and Hilly Trek Funds HLH Research at Cincinnati Children’s

“700-Miles to Hope” Riders Help Close Gap on Fatal Immune Disorder

Friday, September 30, 2016

As bicyclists today complete the grueling “700 Miles to Hope” from Natchez, Miss. to Cincinnati Children’s, they aren’t just finishing a very long bike ride. They arrive as the research their annual trek supports starts to close the gap on an immune disorder that ends up killing close to half of the children who get it.

Riders end this year’s trip by presenting a check to the Cincinnati Children’s HLH Center of Excellence for $250,000. During the annual rides, cyclists raise funds through the Matthew and Andrew Akin Foundation, donating a total of $850,000 in the last four years. The ride and foundation were created by Kristin and Justin Akin of St. Louis, whose sons Matthew and Andrew both died from hemophagocytic lymphohistiocytosis, or HLH.

HLH throws the immune system into irreversible overdrive. Children die from uncontrolled inflammation that destroys their blood cells and immune systems. Too often misdiagnosed and detected late, HLH has few treatments with the exception of bone marrow transplant, a procedure that comes with complications and risk.

Years of research – funded in part by family efforts like this week’s ride – is making a difference. Physicians at Cincinnati Children’s have led efforts to better understand the disease, help other physicians diagnose it, and develop new therapies. They also are helping lead a clinical trial that tests a new drug (NI-0501) for the disorder.

“We are already seeing the benefits as many more children are surviving thanks to the innovative research done here and the resources pushed along by the Akin Foundation,” said Ashish Kumar, MD, PhD, a physician in the Cancer and Blood Diseases Institute at Cincinnati Children’s who is completing his 3rd ride. “I am hopeful but we are far from done. There are children who still lose their battle after suffering for a long time.”

Joining Kumar and the group of riders are five other Cincinnati Children’s colleagues and parents who have lost a child to HLH, including Justin Akin – who started out simply taking long bike rides to remember his boys – and his wife, Kristin. They can finally see the tide turning against HLH.

“Tremendous progress is being made in the fight against HLH, and many lives have been saved because of increased awareness and creation of the HLH Center of Excellence,” Akin said. “More patients are getting diagnosed correctly and our foundation is very proud that we've helped so many of them transfer their care to the HLH experts at Cincinnati Children’s. The goal of our foundation is to save one life at a time and I think we're accomplishing that goal.”

Key advances at the Cincinnati Children’s HLH Center of Excellence include:

• A clinical trial showing encouraging early clinical results for NI-0501. Developed by the Swiss pharmaceutical company Novimmune, the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for NI-0501 – which is designed to expedite the development and review of new therapies for serious or life threatening conditions. Novimmune has been a supporter of the annual bike ride and fundraising effort.
• Cincinnati Children’s physicians have lectured at major medical schools across the country on new methods for diagnosis and management of HLH, helping lead to earlier accurate diagnosis.
• Ongoing annual conferences on weekends following conclusion of the “700 Miles to Hope” ride. The well-attended conferences focus on education for families and physicians who confront HLH.
• Numerous children with HLH have been able to receive advanced treatment for their conditions at Cincinnati Children’s because of increased awareness and funds raised by efforts like the Matthew and Andrew Akin Foundation.

Additional Information

• 700 Miles to Hope video promo
• Read the story of HLH rider Sam Chamberlin, whose daughter battled the disease