Participants Needed for Survey about Starch Desferal (S-DFO)

This is a wonderful opportunity to express your thoughts about a new chelator and why you think it is needed and what this type of chelator could mean to the quality of your life. This survey is open to patients and parents because chelation is an issue that involves both. I have long believed that starch desferal (S-DFO) would be a chelator that would offer a solution to the non-compliance issue and provide an alternative to those who are tired of dealing with the many side effects of the current oral chelators and the nuisance and pain of nightly desferal injections. In other words, this is a chelator that most patients would seriously consider using if it was on the market. To get to market, funds need to be raised to run trials and for this, investors are needed. What you have to say about this chelator can have a strong effect on potential investors. Please think about what a one hour per week IV chelation that lasts all week would mean to your life. Think about how much easier combination chelation would be. Think about what chelation with very mild if any side effects would mean. Imagine using desferal without the subcutaneous injections, without the bumps, skin irritation and pain. This type of survey was done in our group before Exjade was marketed. The patients have a lot of influence on what can happen, so please be ambassadors for yourself and think about the prospects of long term chelation with the drug you currently use and whether you believe this is a permanent chelation solution for yourself or whether you will need a new chelator at some point. I think most will agree that a safer and easier to use chelator is greatly needed.

Biomedical Frontiers, a Minnesota based company founded by four University of Minnesota scientists, is preparing to enter Phase 2 Clinical Trials for  a new drug therapy.  This therapy has the potential to dramatically improve the quality of life for Thalassemia patients around the globe.

Biomedical Frontier's lead drug, S-DFO, is a long-acting intravenous iron chelator.  Initial clinical study results suggest that a one hour, once a week infusion of S-DFO can replace the lengthy, burdensome regimen of current iron chelation therapy which involves daily drug infusions lasting between 8-12 hours. 

Biomedical Frontier is launching a website to educate potential investors about S-DFO and the challenges patients face with current treatment programs.  The company would like to share patient stories and perspectives on its website to tell a more compelling story to the investment community.

Would you be willing to share part of your story with the Biomedical Frontiers team?  Your participation will help Biomedical Frontiers advance towards the next phase of commercializing this powerful new drug.  Your story could be shared in a quick phone call or email and possibly be used on the company website.  Confidentiality would be ensured.
 
(Because of uncertainty about FDA rules concerning direct contact with patients by drug companies, Janet has asked that her email address be removed and that people wishing to contact her, do so through me).  You can help change the world.

Give your opinions, tell them your thoughts. You may have unique perspectives that only patients and parents would have. My observations over the years tell me that S-DFO would save many lives if it can get to the market and I have no doubt that Lisa and many others would still be alive today if this was available then. Lisa took a 4 hour IV of desferal once weekly and it was nowhere near enough, even combined with her subq desferal, which she could not take as often as needed due to the skin reaction and burning. This would have solved her problem. I think most of us knew someone who would still be here today if chelation had fewer and milder side effects. This is your chance to make something positive to happen.

We are very excited to participate.  Little A is very excited about it too, I cannot tell you the state of his stomach right now.  He has so many bumps and bruises. 

Thanks for letting us know about this opportunity Andy, thanks for being an advocate for us and for always looking out for our best interest,

Sharmin

Thanks for informing us Andy,

Can we participate as Little Z is not on desferal anymore  i'd be willing to participate.

Zaini.

Thanks Andy,

That will be on my first thing to do after work.

Thank you for the info, andy..

How can i participate?my son is 2yrs 5 mths old and he has not started his desferal yet.will he be eligible?but we are eager to participate

thank you for your concern

Me too will do

manal

Please direct any questions about this survey to Janet in the email provided. (Because of uncertainty about FDA rules concerning direct contact with patients by drug companies, Janet has asked that her email address be removed and that people wishing to contact her, do so through me).

i have emailed them and waiting for what happens next..Hopefully i can participate

 

I emailed them,lets see what happens.

Zaini.

Hi Guys,

I got a reply from Biomedical Frontiers,

   Zaini,
Thank you for your willingness to help.  We so appreciate it and need your participation to help us.


Biomedical Frontiers is a small company.  We don't have the resources that large drug companies do, but we do have a powerful, long acting iron chelator that provide effective therapy in one hour, once a week.


Our challenge is raising money to get to Phase 2 Clinical Trials.  This is no small task.  However, we believe, with the help of patients, we will get there. 


I will email you a survey for your answer, and also a Release of Information Form. The Release of Information form allows us to use your comments on our website.  (if you prefer, we would use only your first name and last initial to maintain confidentiality.)


Look for my next email tomorrow.


Sincerely,


Janet Hallaway
Biomedical Frontiers

Tariq got the same reply.

Zaini.

Hello everyone,

Finally i got another reply from Biomedical Frontiers.

Hi Zaini,
I'm sorry it's taken so long for me to get back to you.  We are developing the Biomedical Frontiers website -- we hope it will be up soon.  Here's where I could use your help.  We are trying to describe what's it's like to have to go through iron chelation therapy every day.   We really need to describe the physical and the emotional challenges of what it's like to be be required to use a therapy that lasts 8-12 hours a day.  So, would you be willing to answer these questions in your own words...


1. What type of iron chelation therapy does your daughter presently use?
2. How long has she been using this treatment?
3. What are the frustrations and challenges of administering this treatment?
4. If your daughter is using or has used deferoxamine therapy, how long does it take to administer this?
5. How would your daughter's life be different if she did not have to go through the regimen of administering deferoxamine treatment.
6. How would your daughter benefit from a once a week, one hour chelation therapy that provided the same benefits as her current treatment?

Thank you, Zaini.  I so appreciate your help.  I  will keep you informed of our progress. 

Regards,
Janet Hallaway
Biomedical Frontiers
(Because of uncertainty about FDA rules concerning direct contact with patients by drug companies, Janet has asked that her email address be removed and that people wishing to contact her, do so through me...Andy)./quote]

Although Little Z is not on desferal right now,but we've been there and done that,so i know what a difference it can make,and i'd still love to have S-DFO and ferriprox,for me its the best combination.

Zaini.

I have also contacted Janet and I am awaiting her response.  I think that S-DFO and exjade would be a good combination for little A to continue on once this trial is complete.  I hope it works out soon.

Sharmin

I have received the same reply too
I encourge any one who has expierence with chelation to answer the above questions in an attempt to help them. You can pass them to me or you can email them directly. I am sure any of your answers will make a difference

manal

Hi Manal,
I'm sorry it's taken so long for me to get back to you.  We are developing the Biomedical Frontiers website -- we hope it will be up soon.  Here's where I could use your help.  We are trying to describe what's it's like to have to go through iron chelation therapy every day.   We really need to describe the physical and the emotional challenges of what it's like to be be required to use a therapy that lasts 8-12 hours a day. 

Would you know of any transfusion dependent patients who would be willing to answer these questions?
It would be very helpful to hear how patients describe their current treatment.

1. What type of iron chelation therapy do you use?
2. How long have you been using this treatment?
3. What are the frustrations and challenges of administering this treatment?
4. If you use or have used deferoxamine therapy though a pump, how long does it take to administer this?
5. How would you benefit from a once a week, one hour chelation therapy that provided the same benefits as your current treatment?

Manal, thank you for your help.  I will keep you informed of our progress (and send you the link for the website when it's done).

So who else is ready to participate,you know the questions now,just mail the answers to me or Manal and we'll pass them on to Janet Hallaway,in my last email she said they'll respect patient's privacy too,so its not necessary to use your real name if you don't want too.

Zaini.

I just replied with answers