PHOTOS: Dana’s Angels Research Trust Fundraising Walk at Tod’s Point Benefits from Sunshine and a Great Turnout

The 9th annual Dana’s Angels Research Trust (DART) “Dart to the Finish” charity walk at Tod’s Point benefited from beautiful weather and a good turnout on Saturday.

The annual event raises funds to support research, education and treatments for Niemann-Pick type C, often referred to as “childhood Alzheimer’s.”

Niemann-Pick type C is a rare, progressive genetic disorder where the body cannot properly move cholesterol and other fats within cells. This trapped fat builds up in organs like the brain, liver, and spleen, causing severe physical and neurological damage over time.

Andrea and Phil Marella, who founded DART, addressed the crowd before the walk and shared a recap on the development of therapies for children with Niemann-Pick type C. Their daughter Dana, died in 2013 from complications resulting from the rare disease.

Mr. Marella said although research slowed during, by 2024 one drug combination was approved by the FDA, and a week later a second drug was approved for a total of three therapies children with Niemann-Pick type C can receive.

Mr. Marella said their son Andrew, who has Niemann-Pick type C, has been on Beren Therapeutics drug, adrabetadex, for since he was 13 for a total of 14 years.

“We expecting an FDA approval on November 17,” he said.  “The drug is an absolute miracle.”

Mr. Marella added that he hoped that in a month or so, there would be a total of four drugs approved for Niemann-Pick type C (NPC).

“That is really an incredible feat for any disease, especially an ultra rare one,” he said, adding that DART and their research partners have so many efforts underway, including undergraduate medical science research and newborn screening being tested.

“Hopefully we’ll end up on the newborn screen list so we’ll be testing all the kids right away,” he said. “Obviously early intervention is incredibly important.”

Mr. Marella introduced three people from the National Institutes of Health, including Dr. Cristin Davidson. He described the trio from the NIH as the hub of the wheel on the effort to find repurposed drugs – drugs previously approved by the FDA – that might apply to Niemann-Pick type C.

Dr. Davidson said AAV9 (Adeno-associated virus serotype) is a first generation gene therapy that has shown promise in many diseases, though she said, “neurological diseases are not necessary the best ones for this because we don’t correct a lot of cells in the central nervous system.”

“We partnered with a company named Apertura Gene Therapy. They have a new capcid they developed to cross the brain barrier and hit the cells we want to hit, she said. “Hopefully from a neurological aspect we’ll have a greater impact.”

She said the new capsid requires a one-time administration, whereas the other three – miglustat, arimoclomol and N-acetyl-L-leucine –  require repeated administration.

“We have worked with a few companies over the years for gene therapy, and Aperture is very engaged. They’re already having conversations with the FDA, and they’re very interested in moving this forward. We’re hopeful it will be a short timeline – even though two or three years sounds like a long time, we started this in 2015 and God willing, before 2030, we’ll have something in patients.”

For more information about Dana’s Angels Research Trust or make a donation to further research into Niemann-Pick type C head to the DART website.