Last week, we published a blog about the first FDA sanctioned cell therapy to promote repair through myelin regeneration in Transverse Myelitis, which is set to begin in 2018. We are extremely excited about this study, and are so happy that you are excited as well!
This study requires funding. The CONQUER program at UT Southwestern secured a one and a half million-dollar gift to fund the clinical portions of the phase I trial. The TMA has been working through The Pauline H. Siegel Eclipse Fund to raise money to cover non-clinical costs of the trial. If we raise all the money needed, we expect to begin enrollment in mid-2018. Every dollar you donate to The Eclipse Fund will be matched by a generous donor up to $150,000.
The Eclipse Fund was established in memory of Pauline H. Siegel. Pauline sadly passed away on Tuesday, August 15, 2017. It was Pauline’s 1994 diagnosis of TM that catalyzed the formation of the TMA. For over 20 years, she worked with her husband and TMA President, Sandy Siegel, to educate, support and inspire others in the TMA community. Despite the difficulties she faced, Pauline always managed to see and feel the blessings of her experience, and never lost hope for a better tomorrow. Pauline lived her life trying to improve the future of other people with TM and other rare neuro-immune disorders – NMOSD, ADEM, ON, and AFM. She was a powerful advocate, and touched us all with her compassion and vitality.
Pauline’s is a legacy of hope and, in her name, we will drive research forward to find a cure for rare neuro-immune disorders and enable spinal cord repair. To learn more about The Pauline H. Siegel Eclipse Fund, please watch the video below:
The fund will be used to support and accelerate the TMA’s research portfolio to fund discoveries that will directly impact the quality of life for the members of our community. The Pauline H. Siegel Eclipse Fund will drive critical research to:
- Restore Function The first FDA approved cell therapy to promote repair through myelin regeneration in Transverse Myelitis is set to begin in 2018. The Transverse Myelitis Association, Q Therapeutics, and The University of Texas Southwestern are collaboratively sponsoring this first human safety study to treat central nervous system disease.
- Identify Genes and Causation Research at Johns Hopkins University has shown a gene mutation (VPS37A) that is currently known to be present only in Transverse Myelitis patients. The Eclipse Fund will support the validation of this gene and its role in causation.
- Improve Diagnosis Biomarkers and novel imaging techniques, once identified and validated, help accurately diagnose myelitis in the acute phase. Recent studies funded by the TMA and conducted by the Rare Genomics Institute and The Johns Hopkins Transverse Myelitis Center have revealed an antibody in TM patients that needs further investigation and has the promise of unveiling more details about the biology of the disorder.
- Investigate Novel Therapies The Eclipse Fund seeks to fund the development of scientific models of disease that can be used to test existing drug therapies. The goal is to ensure that all potential therapies for restoration and repair are investigated.