GREENWICH, Conn., September 30, 2024 – 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 proud to announce that its 7th annual DART to the Finish Charity Walk, held on September 28, 2024, at Greenwich Point Park in Greenwich, Connecticut, along with virtual walkers throughout the world from California to South Korea, incredibly raised more than $160,000 so far to support Niemann-Pick Type C (NPC) disease research and treatments. This exciting fundraising milestone comes just after the U.S. Food and Drug Administration (FDA) approved the two first-ever therapies for NPC: Miplyffa combined with Miglustat, and a second therapy, Aqneursa. These approvals represent significant advances in the treatment of this rare, neurodegenerative disease, which has devastated the lives of many children and young adults and is so rare that it impacts only an estimated 900 children and young adults worldwide.
NPC, which many note has the properties of “childhood Alzheimer’s,” is a genetic cholesterol storage disorder that causes progressive neurological deterioration, affecting movement, speech, cognition and mobility. Until now, there were no FDA approved treatments, despite clinical trials of therapies being conducted for almost 20 years, of which DART has been a major funder.
“These new therapies mark a turning point in the battle against NPC, offering hope for a brighter future for those affected by the disease,” noted Phil Marella, co-founder of DART. “We’ve worked long and hard to find a treatment or multiple treatments that would help slow the progression of this devastating disease that robs children of their childhood and their lives. We are proud to note that the success of basic research funded by DART and its NPC community partners, plus all of the awareness that has come from the cyclodextrin clinical trial which the NIH has been conducting with a drug called Adrabetadex, has created interest in NPC as a disease for biotechs to pursue. We are hopeful that Adrabetadex will go before the FDA in 2025 and hopefully be approved as well.”
Phil Marella noted that the recent FDA approved combination of Miplyffa and Miglustat offer a combined approach to treating NPC. While Miglustat—originally approved for Gaucher’s disease—has been used off-label by NPC patients to slow disease progression, the addition of Miplyffa brings a complementary mechanism that enhances cellular function with the goal of the combination significantly delaying neurological decline and improving quality of life for patients, as demonstrated by clinical trials.
Aqneursa, the second newly approved drug, is designed to address the accumulation of harmful lipids in the brain and other organs, a hallmark of NPC. Clinical trials have shown that it effectively slows disease progression and improves neurological function, particularly mitigating the severe effects of cerebellar ataxia, a condition that impacts balance and coordination.
The funds raised from this year’s DART to the Finish Charity Walk will support additional critical research initiatives, including those led by Dr. Antony Cougnoux at the National Institutes of Health, and Dr. Marina Kolocha who is helping coordinate other global collaborations under DART’s SOAR-NPC initiative. This research focuses on developing new treatments, understanding the disease mechanisms, and moving closer to a cure for NPC. In addition, DART has been instrumental in helping fund 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.
“We want to thank everyone who joined us to walk to help us get closer to a cure for NPC,” noted Andrea Marella, co-founder of DART and mother to Andrew, who is now 25 years old and battling NPC. Two of the Marella’s four children were diagnosed with NPC. Sadly, the Marella’s lost their daughter Dana in 2013, just shy of her 20th birthday, after battling the disease since she was a child. Andrew is doing better, thanks to the advances in treatments that have been funded by fundraisers held by Dana’s Angels Research Trust throughout the past 20 years. “This walk, and the incredible $160,000 it has raised so far, is a testament to the unwavering support of our community,” noted Andrea Marella. “Every dollar raised brings us one step closer to life-saving treatments for NPC patients.”
Since its founding, DART has raised more than $6.5 million to fund research into NPC, and this year’s walk further advances those efforts. DART’s impact extends beyond NPC, as the research it supports has implications for other neurological diseases such as Parkinson’s, Alzheimer’s, ALS and heart disease. The organization remains committed to funding research and clinical trials that offer hope to NPC patients and their families.
The DART to the Finish Charity Walk was made possible thanks to the generosity of sponsors including Landis Partners, Trinity Church, Corient Private Wealth, Jusko Wealth Management Group, The Charles A. Mastronardi Foundation, The Jones Family, The Bai Family, Prospector Theater, Palmer Point Marina, Manfredi Jewels, Costco Wholesale, Greenwich Shell, Gault Family Companies, Chilly Bear, Threads & Treads Greenwich, Greenwich Emergency Medical Services, Inc., Horseneck Wine, Banc Raiders and Splash Car Wash.
To learn more about Dana’s Angels Research Trust (DART), visit danasangels.org, on Facebook @danasangels, Instagram @danasangelsNPC and Twitter @danasangels.
