August 2016 Update – Big News!

Intravenous Gene Therapy Update

AMAZING news! We recently received a detailed update on the progress of the Auburn/UMass GM1 intravenous (IV) gene therapy research program. Work is truly active towards the creation of a clinical trial in human children! The details of the progress towards creating an IV gene therapy clinical trial in the United States are below.

The Cure GM1 Foundation is supporting development of IV gene therapy for GM1 gangliosidosis as follows:

A. 2 treated GM1 cats are currently 32 months old and doing well (untreated GM1 cats live 8 months). The treated cats behave and function normally. Their primary abnormality is weight loss.

B. The funds are supporting a regulatory consultant, who will inspect vector production facilities as needed and who is writing the pre-IND (investigational new drug) document for submission to the FDA. This is the first step in approval of a clinical trial, and the FDA has 2 months after submission to comment on the pre-IND document. The regulatory consultant will respond to the FDA’s comments by filing a formal IND application for final approval of a clinical trial.

C. Funds are supporting the initial steps of vector production, in which a test batch is made from highly purified DNA stocks.

D. A portion of the funds will be used to perform toxicity and bio distribution studies in normal mice, a step required by the FDA to demonstrate safety of the vector that will be used to treat children.

These are the 4 main uses of the funding so far. A and B are underway. We are waiting to begin production of the test batch in C. Studies in D can not begin until the precise method of production is determined.

The road to clinical trials is arduous. We are truly fortunate that decades of research has been conducted to arrive at this point. The exact timing of the clinical trial is still to be determined and the exact parameters of the clinical trial are also still to be determined.

We still hope the trial could be in 2017. The determination of the exact date depend on the regulatory requirements and the manufacturing schedule. Hopefully, as long as all the stars align, the trial will commence by the end of 2017.

Although we are very encouraged by this wonderful news, many steps remain and significant funding is still required for manufacturing and testing.  Continued support of this program and the Cure GM1 Foundation will help guarantee that this trial becomes a reality.

Children’s lives are at stake and this treatment was needed over 130 years ago when this category of diseases was first identified.

Please help support Cure GM1 on this incredibly important mission!



A few other highlights from recent efforts to bring more attention to our tremendously worthy cause.

A Mother’s Original Song for a Cure

Click on the image to listen!


Marilee Leishman created an incredibly beautiful and moving song that truly speaks for all those affected by GM1 Gangliosidosis.  Please click on the image above to learn more about the song.

Porter’s Annual Fundraiser and Upcoming 4th Birthday

All those affected by GM1 know how truly momentous birthdays are!  Porter’s family will be holding his 2nd annual birthday fundraiser on October 1st in Alabama.

Jessica Kelly, a GM1 researcher in Auburn is also running a marathon in honor of Porter and all those who suffer from GM1.  To learn more about the run or to purchase a t-shirt, please click the image below.


More details to follow on Porter’s fundraiser!

Back to School T-Shirts for Iris

A friend and supporter created an original design to help support GM1 awareness and to contribute to research.  To learn more about this project, please click the image below.


Run the Berkeley Marathon to Raise Awareness


Some kind folks have expressed interested in running the Berkeley Half Marathon for our Sweet Iris and for a cure for GM1! The race is November 20th, 2016. To register and to learn more see below:

If you are interested let us know. If you don’t live nearby, you can participate virtually! Just skip the Berkeley Half Marathon registration, run, and fundraise.  Please let us know if you would like to join!

Thank you!

Thank you from the bottom of our hearts for your support!  The progress made so far would not be possible without all those who have contributed so generously!  We are truly grateful.  Do not hesitate to contact us with any questions or concerns.  Thank you.

August 2016 Update – Big News!
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