The Best Investment We'll Ever Make
My name is Dan Brendtro, and I need for your help to cure a rare childhood disease. With $75,000, we will fund a research study that is ready to begin immediately (December 2017). (With more than $75,000, we can expand the study!)
The disease we are trying to cure is called Friedreich's Ataxia, or “FA.” It is an aggressive disease with no known cure or treatment. It affects about 4,000 people nationwide, including my 16-year-old daughter, Raena.
In simplest terms, FA is a genetically-linked protein deficiency that attacks nerve and heart cells. Typically, FA patients will require a wheelchair before high school graduation, and suffer from teenage-onset heart disease. Mortality often occurs in their 20’s.
With a little luck, and by asking the right questions, we discovered a novel, new idea for treating the disease which is not currently being pursued anywhere on the planet. This idea involves the relationship between the frataxin protein (FXN) and an enzyme called peroxiredoxin-3 (Prdx3). This connection was discovered back in 2012, during a small, one-year collaboration between Sanford Research and the Mayo Clinic. The lead researcher (from the Mayo Clinic) retired shortly thereafter. That study was never published, nor were any steps taken for a follow-up study. Through a combination of limited funding and a lack of infrastructure, this data sat in a drawer for five years, unused, and largely forgotten.
With your help, we will launch the follow-up study within the next month. The money we raise will go directly to the nuts and bolts of biomedical research: test tubes, chemicals, and hiring research staff. Our partner lab has the experience and capacity to start quickly; they have provided a detailed budget, as well a detailed project summary, which you can read about below.
As an FA parent, I know that we are in a race against the clock to find a cure before it is too late. I feel it every day. This study shows genuine promise, based on fascinating new science. I am fully on-board and donating my own money.
Would you consider investing, too?
What We Need & Why We Need It Now
The normal life cycle for projects like this would take a year or two (or more) just to get off the ground. Why? It's a product of traditional funding mechanisms: someone has an idea for a study, writes a letter of intent, waits for feedback, submits a grant proposal, waits to hear back, is awarded the grant, and then, finally, gets funded (or, doesn't get the grant, and repeats the whole process again the next year).
We are eliminating all of the delay built into a typical funding cycle. By funding this project with private funds, we can launch the study the moment we get to $75,000. After launch, we expect to have the first sets of data within 4 months. In other words, by the spring of 2018, instead of submitting a grant proposal for funding, we will already know if our study is on the right path. With a progressive disease like FA, every day, week, and month counts. We are going to start something in 3 weeks that normally takes up to 2 years.
We have partnered with a trusted, qualified research facility. The study will be conducted at the Vitiello Lab of Sanford Research, a state-of-the art, 300,000 square foot facility in Sioux Falls, SD. Sanford Research has attracted top talent from around the world to perform basic, translational, and clinical research under one roof. Priority research areas include pediatric and rare diseases and cellular therapies.
We have partnered with the right scientist, who brings a unique perspective. Dr. Peter Vitiello was involved in the prior FA study, and he and his staff are ready and willing to continue where they left off. Dr. Vitiello's area of expertise is in "redox biology," which includes the study of oxidative stress upon cells. This niche was the precise reason that he was brought in to the previous study: FA is believed to be caused by oxidative stress, although the molecular mechanism is not yet understood. Dr. Vitiello spends 100% of his time on research, and his lab is funded by the National Institutes of Health to study redox biology in lung cells. This project is an opportunity for him to lend his knowledge and skills to researching a new, uncharted area in FA.
This is necessary research. In the past six weeks, Dr. Vitiello has reviewed the new literature, contacted the (now-retired) director of the prior study, and consulted with leading FA researchers. The unanimous consensus is that this new research shows definite potential, and that the very first step is conducting basic science to demonstrate a link between FA and the Prdx3 antioxidant enzyme. If successful, we expect that this research will form the basis to catapault into a long-range, multi-year project towards a cure.
This is some pretty amazing stuff. The working hypothesis is that the loss of frataxin protein in FA patients causes an accumulation of toxic chemical by-products within cell mitochondria, which then harms the nerve and heart. We believe that the Prdx3 enzyme plays a key role in neutralizing those chemical by-products...sort of like a full-time, live-in custodian that keeps our mitochondria clean. But in an FA patient, Prdx3 activity is diminished or impaired. What's remarkable is that this same enzyme process is potentially linked to dozens of other diseases, meaning that if we can unlock and unleash Prdx3 levels for my daughter, we can potentially also pave the way to cure countless other disorders, syndromes, and diseases.
The troops are ready today. In addition to instant funding, we can also begin immediately because Dr. Vitiello already has the right person on his staff to conduct the day-to-day work (she's a recent Ph.D. graduate with expertise and interests in mitochondrial biology an disease). Upon launch, Dr. Vitiello will reassign her to our study. And this means we can bypass the 3 to 4 month lag time that it would normally take to recruit, hire, and train new staff. In total, the use of private funding will save at least 1 or 2 years of time.
The price tag is achievable. In a prior fundraising attempt this year, we exceeded a $26,000 goal in two weeks, for general FA research. We fully expect to meet our goal in this campaign, because the funds are specifically earmarked for an urgent need.
How the funds will be allocated. The project budget is $75,460 for 12 months. This includes around $50,000 for staffing (two Ph.D's, one full time, one supervisory/part-time); $7,000 for lab supplies and reagents; $8,000 for research mice; $6,500 for cell cultures; and $3,500 for 3rd-party testing. More detailed descriptions are listed to the right (or below on a mobile device).
This project is fully scalable. If we are able to raise more than $75,000, we can expand the scope of the project; procure more cell lines; run more experiments; evaluate the DNA and enzyme responses in additional mice; and hire another full-time researcher to perform the work twice as fast. (For the scientists out there, here are the research questions: Question 1: Does Prdx3 regulate ISC complex formation and activity?; Question 2: Does loss of Fxn influence Prdx3 function? Question 3: Does manipulation of Prdx3 alter FA mitochondrial physiologies?)
Who We Will Help
My daughter Raena spends part of her day in a wheelchair, and her condition is getting worse, a little more each day. She is 16. She can no longer paint her own fingernails. She struggles to hold her phone steady to take a selfie. She loves acting but can't take parts that involve a lot of walking (which is basically all of them).
This disease is often fatal to patients in their 20's. As of right now, she is 3 years plus 3 weeks away from turning 20.
Raena's dream is to attend Stanford as an English and theater major. My dream is for her to accept her Stanford diploma in 2022, on her own two feet, and then to live a long and happy life as a writer and an actress.
That means we need a cure in the next five years....for Raena....for the 4,000 other patients just like her....and for the 8,000 parents out there, just like me, who dream of reaching the finish line. I have met some of these other parents....I can't even tell you how worried, desperate, and eager they are to find a cure. Let's do this. Are you in?
Risks & Challenges
There are numerous risks and challenges with projects like this. Here are three:
The study can't begin until it is fully funded. So what happens if we don't reach our funding goal? If we can get at least a third of the way toward our goal, we can demonstrate our commitment to this project to outside funding sources, and ask them to match our participation to get this launched. If all else fails, we will donate these proceeds to another 501(c)(3) researching this disease.
The research timeline is dependent upon the breeding cycle of mice. Part of this project will involve waiting for shipments of mice to arrive; waiting for them to breed; waiting for their offspring to reach maturity; and testing the mice to identify those with compatible genetic make-up. We believe we have built enough time in to the schedule to accommodate those delays.
The data may not support the hypothesis. It is possible that after only a few months of the study, we may determine that the Prdx3 enzyme plays no likely role in FA. Then what? We will shift gears and redirect the efforts farther up the chain, and try to unlock the rest of the mitochondrial pathway, using the same mice, the same 'redox' science, and the same lab.
Other Ways You Can Help
If you are not able to contribute financially at this time, please consider helping us get the word out...share this post with your friends on Facebook, or email them a link (www.TheFinishLine.org). There are some "Indiegogo share tools" up in the right hand corner, next to the "Back It" button.
My daughter and I are also planning on participating in the "Big Sur 21 Miler" in April 2018 to raise awareness for FA. We would love to have you run (or roll) along with us. Contact us for more details.
If you're sold on this idea, please click the "Back It" button above and choose a gift of any size; or click on any of the research descriptions with specific dollar amounts. (If you would prefer to pay by check, rather than credit card, please make your check payable to: "The Finish Line Fund," and mail it to Dan Brendtro, P.O. Box 2583, Sioux Falls, SD 57101
Thank you very much for your time.