Pedal 4 Paul
Make a gift to The ML4 Foundation in honor of Paul today!
Wow! Is it May already? I guess the terrible winter snow has set my calendar into a time-warp, and I am quite late in organizing my 2026 Pedal4Paul bike ride! However, my bike is tuned up, my route is planned, and I am so ready to take to the streets of Queens, N.Y. For my 13th annual ride in support of the ML4 Foundation.
It is with great joy and gratitude to all of you, that I can report that the progress in research made over the last several years has brought us to the brink of having an actual gene therapy treatment for ML4 that is being prepared for FDA approval. Since 2019, pre-clinical research at MGH, and later with coordination by the team at UMass for development of a Natural History Study, visits by 20 patients have been seen at MGH. The collection of patient data will support a ‘New-Drug Application’ to the FDA. Now, after further collaboration with the UMass Gene Therapy Center, manufacturing of the gene therapy began in 2024, toxicity studies were performed, and we now anticipate the final application to the FDA to be submitted in 2026. This is a complicated process requiring multiple efforts by many of our research teams, and especially by the families who have travelled to Boston for the Natural History Study.
This accomplishment is something I could not have dreamed of in 1995, when I joined the Board of the ML4 Foundation. Since its founding in 1982 by a small group of families, steadfast dedication and devotion has led us to this phenomenal achievement.
Paul has remained strong and brave throughout this past year. Despite several hospitalizations for medical and surgical care, he is happy & loving, greeting each day as a new adventure with wonders that amaze him. Whether petting animals in a woodland park, listening to a jazz band, hearing a rock musical or opera in a theater, or just being with friends and family in an Italian restaurant, his smile brings delight to all those who are with him.
Thank you all for your continued support of our efforts, and your concern for Paul and all those affected by ML4.
-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
