May 2017 Cure GM1 Update

Fundraising Update

$84K was raised in the month of April!  

This organization started out with not a single cent towards GM1 research and Cure GM1 is now closing in on having raised $2M!    Thank you to all those who have contributed and supported our efforts.  We are truly grateful!  Every donation matters.  Before Cure GM1, there was no other nonprofit entirely dedicated to this condition.    Cure GM1 funds have already been put towards advancing research and building a community to rally around those impacted by GM1 Gangliosidosis.  We must continue to fight for more progress!

Research 

Thus far, Cure GM1 has contributed funds to 3 projects thus far listed here. We will continue to build our research portfolio and to draw scientist’s attention to GM1 Gangliosidosis.    There is much to do and there is always more we could be doing to advance our cause!

This month, let’s highlight the new UC Davis project.

Currently there is no cure or effective treatment for GM1 gangliosidosis.  Supportive care is available to ease symptoms and extend lives, however, there is no therapy to reverse or delay progress of the disease.  Therefore, a focus on developing treatments where beta-galactosidase activity can be restored or replaced needs to be developed.

The use of genetically modified blood cells which express the wild-type beta-gal gene could potentially alleviate GM1 symptoms.  By transplanting blood cells which express and secrete wild-type beta-gal, neurons would be able to take up the correct enzyme.  This strategy has the potential to stop the progression of GM1 due to the renewed degradation of gangliosides by the presence of wild type beta-gal.

As a new treatment approach for GM1 gangliosidosis, blood stem cells will be genetically modified with wild-type beta-gal encoding lentiviral vectors for use in an autologous bone marrow transplant setting.  Safety and efficacy of these cells will be evaluated to correct the GM1 disease phenotype.  The safety of lentiviral vector transduced blood stem cells will be evaluated both in vitro and in vivo and the effectiveness of the treatment will be evaluated in a mouse model of GM1 disease.

Our approach has the potential to be a one-time treatment and may potentially correct the disease phenotype if enough wild type beta-gal enzyme can be delivered to the neurons to degrade the gangliosides.

Advantages of a stem cell gene therapy approach for GM1 include:

  1. Wild type beta-gal expressing immune cells are capable of secreting the therapeutic proteins.
  2. Constitutive expression of wild type beta-gal via blood cells may allow for a one-time treatment.

Friends of Cure GM1

We are creating a new email list for those who want to stay connected with the foundation and learn about small and large ways they can help. To be added to this list, send us a message on our contact page with the following in the comment box; “Please add me to the Friends of Cure GM1 Email list”. Please consider joining this email list. There are so many wonderful people out there. Together we can make a difference and truly bring hope to the GM1 community and vanquish this truly awful disease.

  • Our 2017 2nd Quarter Fundraiser (April – June) is a Cure GM1 Spring Clean Yard sale or Lemonade stand Fundraiser! This is a great and pretty easy way to help the Foundation. Please let us know if you plan to benefit children with GM1 by doing a yard sale or lemonade stand in your area and we’ll add it to our events calendar!

  • 2017 3rd Quarter Campaign (July – Sept.) will be a Cure GM1 Walk/Ride/Run – Stay tuned for information on this virtual walk/run coming soon during 3rd Quarter!

 

Check out the new Cure GM1 Shop!

 

Cure GM1 Registry

To attract bio tech companies, we need to find as many people who suffer from GM1 Gangliosidosis as possible. Furthermore, the more concrete data, the better.  Information about the patient population can impact trial design and the level of interest from bio techs.  Rest assured, GM1 is a rare disease, but we’re already in contact with far more families than expected.  Let’s keep improving the registry and get in touch with more GM1 families!  All personal identifying information is anonymous unless specific permission has been granted otherwise.

Every Donation Matters

Your donation today will help save children’s lives.

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All donations are tax exempt and we will send you a receipt for your tax records so long as the necessary contact information is provided.  There are no paid employees at Cure GM1 and the maximum amount humanly possible is being put towards saving children’s lives.

May 2017 Cure GM1 Update

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