Ride Ataxia is making a big impact toward treatments for FA by directly funding research. When every day is critical to each of us it is important to recognize milestones as they pass to keep ourselves motivated and pushing forward as fast as possible.
The new FARA newsletter will be hitting mailboxes over the next couple of weeks and there is one section of special interest to anyone who has supported Ride Ataxia either as a donor or a participant. The section is at the end of the Update on FARA's Research Grant Program and it is called Recently Awarded Named Grants. This section includes the descriptions of two research grants which put the total funded research for Ride Ataxia at a staggering $1.2 million since 2007. The first is named The Phillip Bennett Translational Research Award in honor of one of the original pioneers of FA research fundraising. This award is made possible with funds from Ride Ataxia NorCal and the Phillip Bennett Memorial Fund. The second award is called The Kyle Bryant Translational Research Award. The title and description of the two newly awarded grants is at the bottom of this post.
There are a couple of reasons to be excited about these two specific grants.
The Phillip Bennett Translational Research Award, given to Gino Cortopassi at UCDavis, is for the continuation of a project that started with funds from Ride Ataxia in 2009. Dr. Cortopassi has been hard at work and has produced some impressive results with his high throughput screening program in the last two years, and we want help advance his work towards the finish line. An additional exciting factor about Dr. Cortopassi's project is that he is looking at existing drugs that have already been approved for use in humans which carries significant potential to reduce the development time to clinical trials in FA.
Dr.Sarsero's project is a new approach to FA which aims to fix the root cause of the disease - a defective gene. Gene therapy is an area of research that FARA's scientific advisers have identified as a need because of the potential for a profoundly curative therapy. Dr. Sarsero's research is part of the plan to fill that need.
Ride Ataxia funded grants have advanced three drug candidates in our treatment pipeline and will add new candidates in the near future. For a full list of all the other Ride Ataxia funded grants and progress updates please check: http://rideataxia.org/research.php . It is clear that Ride Ataxia has made a significant, tangible impact on advancing research. Further updates to these projects will be posted as they become available.
When Ride Ataxia originated with our first cross country trip in 2007 our goal was to raise $30,000. We had no idea where the money was going to come from, we just threw the goal out there and gave it our best. We raised $40,000 during that trip and were amazed by the generosity of our friends and family. Ride Ataxia provided a platform from which to talk about FA with people who cared about us and they responded with immense generosity. Over the past few years Ride Ataxia has provided the platform for thousands of people to talk about FA from a place of pride and empowerment rather than a place of fear and helplessness.
We reach our goals because of collaboration from friends, family, cycling enthusiasts and sponsors; the most significant of which is the steadfast support of Outback Steakhouse.
As we cross the $1 million mark we think about where we have come from with our sights fixed on the finishline: treatments and a cure for FA.
Recently awarded grants Phillip Bennett Translational Research Award Principal Investigator: Dr. Gino Cortopassi, University of California, Davis
Repurposing existing approved drugs for FRDA therapy using a novel high-throughput screening assay and a library of 1640 drugs that have already been approved for use in humans, Dr. Cortopassi identified 40 drugs that protect FRDA patient cells from death. The screening assay is based on the sensitivity of FRDA fibroblasts to the thiol oxidant diamide. The goal of this project is to determine the mechanism of action of these protective drugs. Dr. Cortopassi will also examine their relative potency and their efficacy in cell and animal models in preparation for clinical testing in humans. Because these drugs have already been approved by the FDA for other purposes, this “repurposing” approach has substantial potential to reduce the lag time between laboratory testing in cell and animal models and approval of the drug as a treatment for FRDA. Thus, determining the mechanism and efficacy of these drugs is a top priority, because of the potential for rapid translation to FRDA patients.
Kyle Bryant Translational Research Award Principal Investigator: Dr. Joseph Sarsero, Murdoch Children’s Research Institute, Melbourne, Australia
Correction of FRDA iPS cells by non-viral gene therapy. FRDA is an inherited progressive disorder of the nervous system and muscles that results in the inability to coordinate voluntary muscle movements. Improper heart function is also a common and life-threatening condition of the disease. The genetic defect that causes FRDA results in reduced levels of an essential protein termed frataxin in all cells of the body. Stem cell therapy has the potential to repair or replace damaged tissues and restore organ function in individuals with FRDA. Major advances in stem cell technologies have led to the development of ‘embryoniclike cells’ from adult human tissue. These cells, known as induced-pluripotent stem (iPS) cells, have essentially the same properties as embryonic stem cells, and thus can be used to derive any mature cell type. Prior to the transplantation of nerves or heart cells derived from FRDA iPS cells, it will be necessary to restore frataxin protein to levels compatible with normal cell function. In this project Dr. Sarsero and his collaborator, Dr. Mirella Dottori, propose a means to correct the defect inherent in FRDA iPS cells by a gene therapy approach that will restore normal FRDA gene expression and does not leave any ‘genetic scars’ in the cells. The strategy addresses major safety concerns for the clinical use of iPS cells and should facilitate compliance with regulatory agency requirements for the approval of the use these cells in transplantation medicine.