September 2017 Cure GM1 Update

So many supporters this month!

Join wonderful and amazing supporters who are running and walking for GM1 children this month.  Isn’t this photo amazing?!  So many supportive people have come out this month to participate in our Run/Walk event!

Research Update

Our friends at the Cure Tay-Sachs Foundation recently shared news regarding a potential treatment being developed that could apply to both Tay-Sachs and GM1!   Please click the links below to read the Facebook posts or you can check out more information on the Cure Tay-Sachs Foundation web site. 

Relevant CTSF Facebook Posts

https://www.facebook.com/CTSF1/posts/1450120811735042

https://www.facebook.com/CTSF1/posts/1454729707940819

In addition, below is the IntraBio statement regarding the potential treatment.  Furthermore, the IntraBio team is seeing GM1 patients in Europe as part of an existing clinical trial for cerebellar ataxia in Munich.    For more information, please contact us.

IntraBio Clinical Programs

IntraBio Inc is pleased to announce significant progress in the treatment of rare neurodegenerative diseases with IB1000, a well-tolerated, modified amino acid that is administered orally. Most notably, IB1000 is showing promising effects in pre-clinical and Phase 1 observational clinical studies in patients with late-onset Tay-Sachs (TS) disease. These findings are also supported by results from in vitro cellular studies and studies in a Sandhoff animal model. In addition, patients with Niemann-Pick Type C (NPC) disease, and various subtypes of Cerebellar Ataxias (CA) have been treated with IB1000 and observed symptomatic benefit. A multi-national, randomized, controlled Phase 2/3 clinical trial of IB1000 in 108 CA patients was recently completed, with the final results expected in 2018.

IntraBio is now planning multi-national randomized, controlled pivotal clinical trials to evaluate the safety and efficacy of IB1000 as a therapeutic intervention in patients with TS, NPC and specific CA diseases. IntraBio hopes to commence enrollment in one or more of these pivotal trials in the EU in early 2018 to be followed by recruitment in North America. In these trials, the safety and efficacy of IB1000 will be determined for each indication using validated clinical and quality of life assessment scales. IntraBio will provide full details of clinical site locations and the clinical trial design including inclusion criteria as soon as they are available.

About IntraBio

IntraBio is a global leader developing novel therapies and treatments for “orphan” and neurodegenerative diseases, including lysosomal storage disorders, which have high unmet medical needs. IntraBio has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for two drug compounds, including IB1000 for the treatment of Niemann-Pick Types A, B & C diseases.

Mallory Factor

Chairman of IntraBio Inc.

 

In her own words, Professor Heather Edwards (Auburn University) shared a statement below regarding the Auburn UMass Intravenous AAV GM1 gene therapy program.

“Intravenous gene therapy for GM1 gangliosidosis is moving forward by a collaborative effort between Auburn University, University of Massachusetts and the National Institutes of Health. Studies in GM1 animals are complete, pre-IND meetings with the FDA have provided guidance for approval of human clinical trials, and AAV manufacturing for animal GLP toxicity studies is ongoing.  In addition to toxicity studies, steps remaining before the clinical trial can begin include clinical grade AAV production for human patients and completion of regulatory documents (such as the IND application to FDA).  At present, funding for the remaining activities is needed to move to the clinic as quickly as possible. Also, funding is needed for patient monitoring during the clinical trial, which will be performed by an experienced contract agency.”

Both the Dorphan and UC Davis continue to progress as well!  We will share more updates on those programs in the future.

The Cure GM1 Run/Walk is still going strong!

So far, this event has been amazingly successful and it’s been incredibly heartwarming to see the outpouring of support through t-shirt purchases, running, walking, and social media posts.  There is nothing like combining a great cause with staying healthy.

There is still time to participate and you can still help us reach our goal!  Less than $2200 to go until we reach our goal!

 

4th Quarter and Holiday Fundraising is coming!

The holidays are an excellent time to fundraise.  We’ll be rolling out our 4th quarter campaigns shortly.  Please let us know if you would like to help and we will close out this amazing year with continued progress towards a treatment for GM1!

 

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.

September 2017 Cure GM1 Update

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