Newsletter - Winter 2019
Dear Usher III Initiative Friends,
As we near the end of 2019, I am very excited to share with you the progress we have made over the past several months! Your support has helped us achieve so much and we want to thank you for your generosity.
Cindy Elden, Co-founder and President of the Usher III Initiative
We are now closer than ever before to reaching our goal to halt the progression of vision and hearing loss in patients with Usher III. This year, our proprietary compound, BF844, underwent a battery of pre-clinical tests to study its safety and pharmacokinetic properties, including toxicity studies, a genotoxicity study, a cardiac safety study, and a number of cellular assays investigating BF844’s biologic properties. We are thrilled to report that these studies suggest no overt toxicity.
Given our promising results to date, we are preparing to meet with the FDA in early 2020. At this meeting, we will discuss the design of the remaining safety studies as well as the design of the human clinical trials, which we expect to commence in 2021.
Excitingly, there is also early evidence that BF844 has the potential to treat a number of other diseases, including some hereditary cancers and neurological diseases. If treatment of these alternative indications is successfully demonstrated, our drug development program will be more likely to attract interest (and funding) from the pharmaceutical industry.
In addition to our scientific achievements, we invite you to check out our new website, which is up and running. We are committed to providing resources to benefit the Usher community, and our new site makes it easier for patients to register with the USHtrust and My Retina Tracker. You will also find news and resources to learn more about the disease and various ways to get involved.
Our newsletter feature this year is an interview with the youngest new member of the Usher Initiative community, Mila Durlacher. Mila, an eight year old diagnosed with Usher III, and Sarah, her mother, were both gracious enough to answer a few of our questions. I am sure you will agree with me that Mila’s spirit and resilience serve as an inspiration to us all!
We hope that you will join us by continuing to support our work as we get closer and closer to treating patients who suffer from Usher III. Our ambition is to make 2020 the most important year for the Usher III Initiative to date—a year during which we can accomplish all that is required to begin clinical trials. Now, at this critical moment, our costs are increasing exponentially. Your contributions helped us to achieve so much this year, and we hope that this holiday season you will consider donating to the Usher III Initiative again.
Thank you again for your support and for being part of our Usher III community. We at the Usher III Initiative wish you and your family a very happy holiday season and a healthy New Year!
Warmest regards,
Cindy Elden
GET INVOLVED!
In addition to donations, we need lots of other help from the Usher III community! You can help by:
REGISTERING!
If you or someone you love has Usher III make sure they are registered on BOTH patient registries. This is critical as we get closer to clinical trials.
GET GENOTYPED!
If you have only been clinically diagnosed it is very important to get confirmation of the specific genetic disease you have. For more information on genotyping, visit the FFB’s Retina Tracker.
HOST A FUNDRAISER!
Encourage your friends and family to support a cause that means so much to you and the Usher III community. We’re happy to help - please contact us for ideas and other assistance.
INTERVIEW WITH MILA
What do you like to do with your friends?
Right now I’m very into my American Girl dolls. I just got them holiday clothes and presents and they work in an ice cream truck and I have a classroom where they can go to school. I also love school. They have knotted hair so I have to brush it a lot. They have hearing aids and glasses as accessories just like me, so that’s pretty cool. It’s fun to pretend. We also make videos and be silly together. I love my friends.
What do you want to be when you grow up and why?
I want to be a lot of things. I love animals so a Veterinarian, but I’m also a great singer and actor and love Broadway shows. So that would be fun too! I’m hoping to get my own dog really soon. Maybe someday a hearing/seeing dog to keep me company and keep me safe.
I know you want to go Japan, can you tell us more about what you want to see when you go?
Sushi, sushi, and more sushi! I know there is a part of Tokyo that has tons of Hello Kitty everything, so that’s on my wish list. I know it’s really far, but I love exploring. Japan seems so different from New York City.
Where else in the world are you interested in going?
I really want to go to Rome, Italy. It seems really historical and great and old. My mom went to Venice, and I’ve read I should go there soon. And I’d definitely eat so much pasta, food is my thing! We are trying to go to Hawaii for Spring Break. I love the beach and the sound of the waves.
If you could take anyone with on your travels, who would that be?
My good friend Zoe. She is in the picture I sent from the Walk for Hearing. She is my best friend and Cheerleader! I love her so much.
What music are you listening to these days?
I love the music from Frozen (that's the play I’m in right now) I love Katy Perry, Taylor Swift, and Sean Mendes. I’m looking forward to music being able to go directly into my hearing aids someday. (Please soon Mom!!!)
INTERVIEW WITH SARAH
What are your hopes for Mila?
Our hope for Mila is that she finds her place, and what she wants to fight for! We are just trying to be there for her, teach her that she can do whatever she wants to do, and give her the tools to navigate the world with her deficits. We try not to use the word disability in our house!”
In thinking about her daughter’s future and how she copes with Usher III, Sarah explains that at the moment Mila’s attitude is relatively lighthearted, she can make jokes about some of the frustrations and talks about wanting a seeing eye dog.
But I know that down the road when she’s a teenager and when she’s in college she’s not going to want to laugh about it. We’re just trying to get her ready for when those feelings do come. She got mad the other day that she had to wear her hearing aids to a sleepover and then I see her stomp over and put them on and I asked her, "How did you come to that conclusion?" and she comically said "Well I don’t want to not be able to hear!" I’ve met some older people with Usher III, who have cochlear implants. I’m assuming that that will be something that (Mila) does down the road, and it will be scary for her, but I know that she’ll still feel like “Well, I want to hear!"
Sarah and I discussed some of the challenges facing Usher III families. One was the prohibitive cost of many of the genetic tests and therapeutic devices.
Mila’s hearing aids were not covered by insurance and Mila's hearing aids, associated costs and genetic testing totaled $9000. I can’t imagine having not been able to do that for her. That’s where the challenge is.
When discussing challenges that Mila has faced at school, Sarah told us that she works with the institution and the teachers to give her daughter the best opportunity to learn. She wants Mila to get out of school what other kids get out it.
I think that kind of advocacy is important. That’s part of what’s hard at 8 years old, (needing) people around her who can help explain "This is what she needs, give it to her." is really important. That’s what it takes!
I asked Sarah if she had any recommendations for people about how to get involved and help, both with patients and loved ones.
Without having Megan [Kuhlmey] - an awesome audiologist - we never would have met Cindy (Elden), so I think this is a big struggle. It would be really great, if once you get a diagnosis you also got a link from your doctor that allowed you to opt in to a group of other patients. There wouldn’t be any pressure, and if you’re not ready right after you’re diagnosed, or even for years after, that’s OK. The important thing is to make the option available for people who want it. I think that we’re living in a time when the more you know the better you are, and communities can form even on something as simple as Instagram. Even that would make a difference!
Our groundbreaking research and development into finding drug therapies that will slow, and if possible halt, the progression of both vision and hearing loss in individuals diagnosed with Usher III is only possible because of the contributions we receive from donors like you. Any amount of support you can provide is greatly appreciated by us and everyone affected, like Mila and her family. Thank you!
OR mail your check, payable to the Usher III Initiative, to:
Usher III Initiative
191 N. Wacker Dr., Suite 1790
Chicago, IL 60606
If you have any questions please email us at info@usheriii.org