Pedal 4 Paul
Make a gift to The ML4 Foundation in honor of Paul today!
Hello everyone! It’s that time of year again! When spring rolls around, I get my bike tuned up, grab my helmet, and set out on practice rides leading up to my annual Pedal4Paul ML4 Foundation fundraising event. I’m planning to ride on June 22, 2025, on a 14.3-mile ride through the lovely neighborhoods of Queens County, New York.
2025 marks my 12th year of Pedal4Paul. In those past years, the ML4 Foundation has seen amazing growth, achievements, and scientific accomplishments that once were only hoped for. Today, we stand at the brink of having an actual gene therapy! The past few years have been marked by considerable research progress, with more exciting developments ahead. We just concluded out three year prospective natural history study which took place at Massachusetts General Hospital and involved studying a cohort of 15 MLIV individuals every year over three year to learn about the disease and disease progression over time. During this time we also conducted a retrospective natural history, which involved collecting medical records from patients throughout the community to provide a deep understanding of the disease across the age spectrum. In addition, during this time, we have been developing a gene therapy for MLIV, the first treatment for the disease. Having shown excellent proof of concept, in animals, we submitted our first documents to the FDA in preparation for later clinical trials. Having received positive feedback from the FDA, we are now in the process of manufacturing this gene therapy and planning for a small clinical trial of this drug when manufacturing is complete. In addition, we have continued a rich research relationship with Dr. Diego Medina at TIGEM in Italy, a major center for rare disease research. Dr. Medina recently published important research documenting kidney pathology in individual with MLIV and is now undertaking research to examine possible pathology in liver and thyroid tissues, as well. These accomplishments are all due to your consistent, generous, and unwavering support over these many years.
Paul is now 32 years old, as loving, fun, and beautiful as ever! Diagnosed with ML4 in November, 1994 when only 2 years old, our family’s journey has been gratifying with the knowledge that all of today’s scientific results may not have been accomplished without Paul’s contributions, nor the contributions from the dozens of families whose children participated in the earliest stages of research. Their donations of blood and skin samples, exams under anesthesia, annual visits for evaluation to the NINDS at the NIH, and collection and documentation of all their clinical data, laid the groundwork for the discovery of the ML4 gene in 2001 and the description of the pathology and natural history of ML4. My family has been so fortunate to have partnered with and befriended so many of these families. Therefore, it is our great honor to say ‘Thank You’ to our friends Lily, Lauren, Jonathan, Scott, Brad, Lara, and Arielle, just to name a few of the many children and young adults who were there at the beginning of the ML4 research. You are not forgotten, so this year, as I ride my route, I will not only Pedal 4 Paul, but I will Pedal 4 All!
-Paula, Allan, and Paul Kutner
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ABOUT THE ML4 FOUNDATION
MLIV is a genetic disease caused by the mutation of a single gene that results in the loss of a protein (MCLN1). This incredibly important protein is required by the body for normal functioning: without MCLN1, children and adults with MLIV suffer from progressive and debilitating loss of gross and small motor function, go blind, develop problems chewing and swallowing, rarely have the ability to speak, cannot walk, and over time, lose many abilities that they work hard to acquire when they are young.
The funds raised by the ML4 Foundation are directed to a research program at Massachusetts General Hospital in Boston. We have been working for several years with a team there to create a gene therapy to treat MLIV. We are also funding a large-scale natural history study of MLIV, bringing MLIV patients from around the world to the MGH clinic to be seen by specialists in this disease. YOUR DONATIONS made this work possible and allow it to continue at a speedy pace!
If you prefer to donate by check, you may mail it to the following address, and note that it is given in honor of Paul Kutner.
Mucolipidosis IV Foundation
1440 Spring Street NW
Atlanta, Georgia 30309
The ML4 Foundation is a U.S. 501 (c)(3) nonprofit charitable organization.
Tax ID 13-3633501
