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FARA Ambassador Program

FARA Ambassadors are a united team of patient volunteers living with FA who are committed to supporting FARA in the search for treatments and a cure.

The Ambassadors are a service team within the FARA organization. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community. Together we seek to know more about the research and pharmaceutical pipelines being developed through FARA in order to be better prepared to represent the FA community when opportunities arise to educate the medical community and potential donors. When meeting with scientific groups, pharma partners, and the FDA, our purpose is to promote awareness of the patient perspective of living with FA. We believe our dedicated support is key to continued success toward our ultimate goal of treatments and a cure.

FARA Ambassador Program Mission Statement:

The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

The Ambassador Blog

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This blog is a vehicle for the voice of the FARA Ambassador Program and features posts from Program participants and friends from the FA community on a wide range of topics.

The blog features posts about FARA events, articles about living with FA, spotlights on individuals throughout the FA community, weekly interviews to “Meet the Community,” and more. We hope that you will be inspired, uplifted and encouraged through the FARA Ambassador Blog!


Patient Perspective: Sean's Experience Participating in a Clinical Research Study

PatientPerspectiveSeanBaum1Everyone has a part they can play, a role that is essential to our mutual fight against Friedreich’s Ataxia and other rare conditions. For me, I sometimes wish I was a billionaire philanthropist who could fund entire facilities and programs devoted to finding cures. However, it’s safer just to keep my billions to myself. Someone else can be the philanthropist. Actually, someone else will have to do all the philanthropy because my bank account isn’t eligible. Like I said, “sometimes I wish…”

After I was diagnosed in 2007 and every year since then, I ask myself, “what can I do, what part can I play?” A few years ago I got connected with UCLA and enrolled in the ongoing natural history study efforts. They monitor the disease and its progression overall within my body. This study isn’t designed necessarily to identify a cure. However, I believe it is absolutely crucial to the understanding of the disease which directly contributes to cure-discovering efforts and research. Although the data collected from me may or may not help me specifically, I know that it will help someone down the road and I’m happy to be able to invest myself in that manner.

I can do more though, and I want to do more. Several months back I learned about a new clinical trial and I was immediately interested. Sure, there is always some apprehension when talking about a brand new drug that hasn’t been tested in humans yet, but I haven’t felt that the risks were that high compared to the risk of having FA. You are probably aware of some of the symptoms and ultimate prognosis when it comes to living with FA; how much worse can it get? For me, I consider the role I can play and the effect this may have on the bigger picture as a way of managing and contributing to the risk of living with the disease. If I can’t give billions, what can I give? 

Drug Access and Supplements

The below article is Part Four of a series that began in the Winter 2015/2016 edition of the FARA Advocate.

"Drug Development Series: Drug Access and Supplements"

By Jane Larkindale, PhD

Patients with FA are, understandably, impatient for new treatments. Many say that they are willing to accept some degree of risk in taking a new treatment, if it has the potential to be beneficial for them. So, how can a patient get a potential treatment, when there is nothing on the market that has been approved for FA? Firstly, we need to bear in mind that if a treatment has not been approved for FA, it has not been fully tested in patients – there is a real risk that it will be ineffective, or worse still it could harm FA patients. However, if the patient is willing to risk that, there are several ways to try.

Supplements vs. Drugs: The most common unapproved treatment taken by FA patients is probably idebenone, which is available as a dietary supplement, but not currently as a drug. Idebenone was tested in clinical trials for FA, but failed to result in a statistically significant change in the outcome measure, and no further trials are underway. Patients have, however, continued to take forms of the drug that are available as a supplement. Dietary Supplements are governed by the FDA, just as drugs are, but the barriers to sales are much lower. The seller of a supplement does not have to prove to the FDA that it has an effect in any specific disease (and cannot claim that it does). In contrast, a drug developer has to prove through extensive tests that their drug is both safe and effective in specific diseases. The FDA stringently governs how a drug is manufactured, and exactly what is in the tablet – this is not the case with a supplement. Thus, supplements may be available on the market which a patient might think helps his or her disease, and he or she would be able to access it. However, there is no stringent proof of what is in that supplement, or that it has an effect. Further, if a drug form of that supplement is approved by the FDA (as Santhera hopes idebenone will be for other diseases), the owner of that drug can ask the FDA to step in to prevent sales of the supplement form, to protect patients with the disease indicated from taking a form of the drug that is either dangerous or ineffective. In the idebenone example, FA patients would then no longer be able to buy the supplement that they have previously been taking, once a drug form was on the market for another indication. 

Ambassador Program Applications 2016

APFARA Ambassadors are FA patient volunteers who bridge the gap between research, patient care and awareness of Friedreich’s Ataxia. Ambassadors are positive and supportive, peer representatives for the FA community dedicating their time and effort to raise awareness and funds for FARA.

The FARA Ambassador Program (AP) meets on the first Thursday of each month via WebEx. A large contributing factor to the effectiveness and success of this program is being present and active. Planning committees and project resposibilities are determined at the meetings along with sharing of research updates and FARA news.

The AP focuses on several different projects throughout the year. Some examples include:

- Contributing posts to the Ambassador Program blog (curefa.org/ambassadors)
- Writing thank you cards to donors and organizers for FARA events
- Monthly FA Hangouts
- Speaking at fundraisers, community events, pharmaceutical/research organizations
- Fundraising
- Awareness: Rare Disease Day, FA Awareness Day, International Ataxia Awareness Day
- Communicating with newly diagnosed families
- Helping others in the FA community with their fundraising projects 

Patient Perspective: Alex's Experience Participating in a Clinical Research Study

PatientPerspectiveAlex1I’ll admit, I was a bit nervous starting my first clinical trial. A 5 hour drive and overnight stay gave a clear distinction that I wasn’t in my comfort zone. How difficult would the testing be? How stressful would the next few months be during the trial? When would I be able to leave and get lunch (I was very hungry!)? I settled my nerves (FA medical pun intended) and reminded myself why I was doing this…

I won’t claim to be selfless and heroic, saying I was doing this for the FA community. To be honest, I was hoping to personally benefit from the treatment. I had done extensive research on my own, consulted with my physician, and decided the mechanism of this drug was most promising at my stage of progression. Of course, I want the drug to be successful and move forward to a treatment for everyone. But my main motivation to get involved now was to see if my health might improve (trial is still underway… can’t share any details of how I’m feeling…). 

Patient Perspective: Jamie & Jean's Experience Participating in the Natural History Study

PatientPerspectiveJamiePlourdeI have been participating in the Natural History Study for as long as I can remember; for me, it just made sense to participate since I was going to see Dr. Lynch in Philadelphia anyway for my yearly visit. It seemed like a no brainer, especially since there's no extra traveling or work required.

This study is completely painless: to be honest I didn't think they were doing any research at first, I thought it was just part of my check up. You do have to answer some questions about your health and give cheek swabs. Dr. Lynch is such a fun doctor, he makes it seem like you aren't at a check up at all. I can honestly say that his research is enjoyable. I usually spend most of the visit laughing, and when I'm leaving I feel optimistic. I don't know how many people can say that their research is "fun"? :) 

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