Ride Ataxia Exceeds $1 Million In Funded Research

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.

1. 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.
2. 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.

Ride Ataxia Orlando

Building on the momentum we established with our ride in Tampa, FL last year, the Orlando location will provide a well supported, challenging ride for all riders.  The Orlando ride will feature 16, 25, and 50 mile routes allowing participation from all ability levels.  All routes will feature strong SAG support, fully stocked rest stops, and what has been called "The best post ride food ever" provided by Outback Steakhouse and Carrabba's Italian Grill.  All routes will start and end at Waterfront Park in Clermont, FL.

• The 16 mile route will take place on the South Lake Trail heading east with a rest stop and turn around point at mile 8.  This out-and-back route will give riders the option to ride a shorter length if they choose. View the 16 mile route here: http://rideataxia.org/orlando/images/16map.png.
• The 25 mile route will travel around Lake Minneola, out to Groveland to our first rest stop at Lake David Park and back.  This route will include varied traffic conditions and is recommended for intermediate and advanced riders.  View the 25 mile route here: http://rideataxia.org/orlando/images/25map.png.
• The 50 mile route will travel around lake Minneola, out to Groveland, up through Mascotte, around through Ferndale and back to Clermont.  This route will also include varied traffic conditions and is recommended for advanced riders only.  View the 50 mile route here: http://rideataxia.org/orlando/images/50map.png.

The Orlando location will provide access to one of the country's premier cycling atmospheres and will allow us to partner with strong teams from Outback Steakhouse, Carrabba's Italian Grill, and Catrike.  The ride will also receive support from Coca-Cola and Brown and Caldwell.

We look forward to seeing you on the road or trail for Ride Ataxia Orlando on November 6, 2011.

For all the details and to register please visit rideataxia.org/orlando.

Registration is open for Ride Ataxia Philadelphia 2011

The 3rd Annual Ride Ataxia Philadelphia will offer 5, 10, 25, and 50-mile route options starting and ending at Limerick Community Park in Limerick, PA on October 9, 2011. The ride will once again feature well marked courses and cue sheets, fully stocked rest stops, and strong SAG/mechanical support provided by Bike Sport, Tri County Bicycles, and Spinners Mobile Bike Repair. Additionally the ride includes what has been called "the best post-ride food ever!" with a delicious meal provided by our presenting sponsor Outback Steakhouse plus Carrabba's Italian Grill and Bonefish Grill. You will not leave hungry!

Last year Ride Ataxia Philadelphia welcomed 400 riders and raised $150,000 for Friedreich's ataxia (FA) research!

Please follow these simple instructions to guide you through the registration and fundraising process for Ride Ataxia Philadelphia.

1.  Register for the ride

• Go to rideataxia.org/philly
• Click the button at the upper left that says "Register to Ride". This will take you to the Ride Ataxia Philadelphia Active.com registration page
• At the Active.com registration page, click on the green button to the upper right that says "Register Now"
• Pick your preferred ride length (If you are registering as a team of 10 to receive the discounted rate, you must choose a team Captain to create the team.  After the team has been created each individual team member can register and find the team under the name created by the captain, max is 10 per team for registration), read and agree to the waivers, fill in your date of birth and click "Agree and Continue"
• Fill out all your information and answer the questions.  Scroll down and click "continue"
• Fill in your payment information and click "Pay Now".  Note:  Your registration fee goes to FARA; the "Processing Fee" goes to active.com. 

2.  Now that you have registered to ride, you need to create your personal fundraising page...There are two ways to do this. 

       a.  After you register to ride (step 1 above) you will receive two confirmation emails, one is a receipt for your payment and one has instructions on how to set up your fundraising page.  Follow the instructions in the email.

       b.  Follow the instructions below:

• Go to rideataxia.org/philly
• Click the button at the upper left that says "Donate Now".  This will take you to the event fundraising site.
• Click on the link in the uper left that says "register"
• If you were a participant last year, choose "I already have an account", enter your email and password and then click next (please skip the next bullet).  If this is your first Ride Ataxia event choose "I need to create an account" (this is completely separate from the active.com registration in step #1) then click "next"
• Fill in your information and click "next"
• Set your fundraising goal, AIM HIGH! Enter your display name and click "next"
• If you would like to create a team choose "Create a team".  If your friends have already created a team that you want to join. click "search for an existing team". Note:  Everyone on a team will all have an individual fundraising page with an individual total and those individual totals will be combined for the team total on the team page.  All team and individual totals will be combined on the event page. Note: there is no limit to the number of people you can have on a fundraising team.  If you registered 2 teams of 10 in step 1 above they can all be on the same team for fundraising. If you do not want to join a team choose "Do not join a team".  Click "next".
• confirm your info and click "next".
• you will receive a confirmation email which will take you to your page where you can upload a picture and write a personal message to your potential donors.  Here is an example: https://sna.etapestry.com/fundraiser/FriedreichsAtaxiaResearchA/raphiladelphia/individual.do?participationRef=968.0.418554997
• copy the link for your page, paste it in an email or on facebook, twitter...tell everyone about your efforts to help cure FA!