Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” is hosting its seventh annual DART to the Finish Charity Walk on Saturday, September 28, 2024, at 9:00 a.m., at Greenwich Point Park in Old Greenwich, Connecticut. This fun and family-friendly, accessible two-mile walk is $30 for adults 22 years and older, $15 for participants aged 10 to 21, and free for children 10 years old or younger. Virtual walkers from anywhere in the world are welcome to join with a $30 donation to DART. To learn more and register, visit danasangels.org or dartevents.org. Registered participants also benefit from DART’s Reward Program by referring other friends and family to join and can be awarded with fun gift certificates!

“We are so excited to be hosting our seventh annual DART to the Finish Charity Walk,” said Andrea Marella, co-founder of DART. “This event brings together our community to support vital research into Niemann-Pick Type C disease, a rare and devastating disorder that has impacted two of our four children, and 200 children other children in the United States. Every dollar raised goes directly to funding groundbreaking medical research that bring us closer to better treatments and a cure.”

The funds raised at this year’s walk will support several crucial research initiatives, including the work of Dr. Antony Cougnoux in the lab of Dr. Forbes “Denny” Porter, who are with the NPC research team at the National Institutes of Health (NIH). Dr. Cougnoux’s research, which is co-funded by DART and its SOAR-NPC (Support Of Accelerated Research) members, including the Hide and Seek Foundation, focuses on expanding the understanding of NPC beyond the traditionally studied areas of the brain, constructing reference sources to measure treatment effectiveness across multiple organs, and improving the efficacy of gene therapy. Additionally, DART and SOAR-NPC are supporting the work of Dr. Marina Kolocha, a regulatory expert whose efforts are accelerating drug development and approval processes for NPC, with a focus on clinical trials and biomarker research. Dr. Kolocha has been involved in the successful approval of more than 200 drugs, including those for rare and ultra-rare diseases. She is specifically working on developing a research plan for the SOAR-NPC collaborating scientists and the first comprehensive NPC guidance, which will be submitted to the FDA, as well as highlighting the needs for the development of clinical trials and navigating the regulatory process for the approval of biomarkers. Biomarkers have proven to be a critical component, as they are measurable indicators of a condition or disease and are an important piece of the therapeutic puzzle for NPC. DART and SOAR-NPC’s work has been instrumental in moving NPC treatments closer to FDA approval and offering hope to the approximately 200 children in the U.S. affected by this debilitating disease.

In addition DART and other SOAR-NPC partners are funding Dr. Stephanie Cologna at the University of Illinois - Chicago, who has developed a test to measure a protein called Calbindin D, in spinal fluid of NPC patients. Other biomarkers for NPC are also being tested through collaboration across SOAR-NPC labs at the NIH, UPenn, Michigan and Illinois-Chicago. Some of these biomarkers are potentially beneficial to many neurological diseases and some are more specific to NPC. One of these biomarkers is Neurofilament light chain, or NfL. Importantly, the FDA recently approved a drug for amyotrophic lateral sclerosis (aka Lou Gehrig’s disease) based on NfL levels in blood from ALS individuals.

All of this work is laying the crucial groundwork for an important clinical trial for gene therapy in patients with NPC. DART has played a pivotal role in advancing NPC research and treatment options, contributing to the launch of numerous clinical drug trials and the development of the first newborn screening test for NPC and 13 other rare disorders, which has been led by Dr. Melissa Wasserstein at Montefiore Children’s Hospital in the Bronx, New York. The newborn screening - ScreenPlus - is now in eight hospitals throughout New York and has actively enrolled more than 18,000 newborns who have been screened for NPC and other disorders.

DART and SOAR-NPC funding is also supporting work across the globe with the funding of a clinical drug trial in Spain using the medication Efavirenz for treating NPC. The initial results from the first year showed progress; DART and its SOAR-NPC collaborator the Hide and Seek Foundation are providing the funding for the 2023-2024 time-period with hopes to show even further benefits.

The Marella’s daughter, Dana, the inspiration behind DART, was diagnosed with NPC at the age of eight and fought a courageous battle for 12 years before her passing in 2013. Her younger brother Andrew, also diagnosed with NPC, is currently benefiting from clinical trials supported by DART, allowing him to live a fuller life and recently celebrated his 25th birthday in June - a particular milestone for NPC patients, who prior to DART’s efforts, often only survived into their teens. Today, Andrew works at the Prospector Theater in Ridgefield, Connecticut, an inclusive workplace that provides opportunities for individuals with disabilities.

“Niemann-Pick Type C is a devastating disease that robs children of their ability to live a healthy life,” said Phil Marella, co-founder of DART. “But through the support of our community and the progress in research, we are making strides toward better treatments and ultimately a cure. The DART to the Finish Charity Walk is a key part of that effort, and we are grateful to everyone who joins us in this fight.”

To date, DART has raised more than $6.5 million to support NPC research, including its collaborative drug development program, SOAR-NPC. SOAR-NPC’s efforts are supporting life-saving clinical trials, with more on the horizon - and the work benefits more than just NPC patients.

“The work that DART and SOAR-NPC have done have implications beyond NPC,” noted Phil Marella. “NPC research may also help millions suffering from Parkinson’s disease, HIV/Aids, Ebola, heart disease, stroke, Alzheimer’s disease and other disorders that appear to be related to cholesterol. In fact, the two recent FDA approved medications for Alzheimer’s disease were created by pinpointing a genetic biomarker - one of the same that is shared by NPC patients. Now we are looking at more than 20 different biomarkers that can help dictate a better path for treatment.”

To date, DART gives special thanks to the generous sponsors of this year’s DART to the Finish charity walk including Landis Partners, Trinity Church, Corient Private Wealth, Jusko Wealth Management Group, Prospector Theater, Palmer Point Marina, Manfredi Jewels, Greenwich Shell, Splash Car Wash, and Horseneck Wine and Spirits.

To learn more about Dana’s Angels Research Trust (DART) or to register for the DART to the Finish Charity Walk, visit dartevents.org. To learn more about Dana’s Angels Research Trust (DART), visit danasangels.org, on Facebook @danasangels, Instagram @danasangelsNPC and Twitter @danasangels.