Newsletter - Winter 2016
Dear Friends,
I hope this newsletter finds you well. Though it may be hard to believe, 2016 is almost at a close and, once again, I look forward to sharing some of what we, at the Usher III Initiative, have been working on over the past 12 months.
First and foremost, I would like to thank all of you who have so generously supported us over the years in our mission to find a treatment or cure for the blindness caused by Usher syndrome type 3. Your donations are what helps to fund our groundbreaking work. No other non-profit or government agency funds Usher III treatment development, and without the funding that you have so generously provided, we could never have achieved all that
we have — nor, for that matter, all that we hope to achieve in the coming years.
Since the last newsletter, I am proud to announce that we have hired physician and surgeon Dr. Mahdi Farhan to serve as our Scientific Director. Dr. Farhan comes to the Usher III Initiative with over 14 years of clinical experience, in addition to his years of experience working within pharma development at Roche, as well as extensive knowledge of global drug development practices. It is under his leadership that we hope to accelerate the process of moving from the clinic to the lab. (See Page 3 for a more in-depth interview.)
In addition, we are very excited about Dr. Yoshikazu Imanishi’s recent publication in Nature, and are encouraged by the subsequent interest that has been shown in our novel molecule for Usher III patients since that publication.
In other news, Congress recently passed a landmark act known as the 21st Century Cures Act. The bill, which among other things grants the NIH the authority to fund higher risk research, says that the “least burdensome practices must be adhered to in approving drugs” and that patient experience data must be incorporated into the FDA’s decision-making processes, will help accelerate pharmaceutical development and clinical trials for patients with all diseases.
For those who are wondering how they may be able to help, I urge you to register with the Usher Syndrome Coalition Patient Registry. The development of any treatment depends on the registry to move forward with clinical trials, as well as to generate the interest and investment necessary from pharmaceutical companies. To start the process, and for additional information regarding the registry, simply log onto the Usher Syndrome Coalition’s Patient Registry website at www.usher-registry.org. Second, if you have not already done so, I encourage you to get genotyped.
Last, but not least, if you are able to do so, please consider making a donation to the Usher III Initiative to help accelerate the first-ever treatment for
Usher III patients, either online by clicking this button or by mail (see the bottom of page 5 for additional information, including mailing address).
From all of us to all of you, we wish you and yours a wonderful holiday season and a healthy, happy and peaceful new year.
Sincerely,
Cindy Elden
Co-founder, Usher III Initiative
Did you know you can donate online? Just click here or log onto http://usheriii.org/how-you-can-help/ & click the donation link.
SPOTLIGHT ON…
DR. MAHDI FARHAN, SCIENTIFIC DIRECTOR, USHER III INITIATIVE
INTERVIEWED BY ANDREW D. DECANNIERE
Dr. Mahdi Farhan is a Physician and surgeon with over 14 years of clinical experience. Born and raised in Jordan, he pursued his passion for Medicine and was educated at the University of Sheffield Medical School, England, where he received his MB ChB. Dr. Farhan also spent time practicing surgery and gained his fellowship at the Royal Colleges of Surgeons of Edinburgh and London.
Married with two young children, Dr. Farhan lives near London in the United Kingdom, and has recently worked for Roche Pharmaceuticals as Group Clinical Director within the Therapeutic areas of Immunology, Ophthalmology and Infectious Diseases and was the Site Head for the Pharma Development Group in Roche, U.K. Dr. Farhan’s career within the Pharmaceutical industry has spanned more than 15 years and across multiple disciplines including clinical science, early/late drug development, pediatric drug development and exploring innovative pathways to develop and deliver therapies to patients in an efficient and timely fashion.
Andrew DeCanniere: How did you come to be involved with the Usher III Initiative?
Dr. Mahdi Farhan: I learned of the Usher III Initiative via my colleague and mentor, Dr. Tony Adamis, who is a professor of ophthalmology as well as a member of the Usher III Scientific Advisory Board, and I want to make a difference. After I met Cindy Elden, who has Usher III syndrome, I realized the tremendous burden that the syndrome places on her quality of life and the restrictions it puts on her ability to achieve what she is capable of achieving. A therapy must be found for her and many people like her, in order to save their vision and hearing from deteriorating with time. Developing life-changing medicines that make a difference to people’s lives has always been rewarding to me, especially where therapies do not exist.
DeCanniere: What kinds of work or research have you been doing for the Usher III Initiative? What are you working on currently?
Dr. Farhan: Extensive and excellent research has been done in the last ten years which culminated in the generation of a small molecule (BF844) that has the potential to help individuals with Usher III. We are at the point where we need to concentrate our efforts on testing BF844 in the clinic. My involvement, thus far, has been focused on understanding the biology / molecular basis for the syndrome and on establishing a development pathway to take BF844 initially into pre-clinical and then clinical studies. Thus, over the course of 2017, we hope to take BF844 into manufacturing and start testing in animals to ensure its safety, before starting the first-in-man study.
Of course, there are many challenges to overcome, and several steps to be taken, to get human trials started. To increase our chances of success, we are planning to meet with the FDA to prepare for an IND submission. This will be followed by similar meetings with other health authorities to establish a fast but meaningful development program.
Simultaneously, it is critical that we continue to work with external collaborators and identify potential partners, as well as seek additional funding, to support the continuation of clinical development. We believe that the data generated in 2017 will make BF844 a more attractive, and less risky, prospect for partnering. In addition, we are investigating the ability of BF844 to be a potential therapy for other diseases that are still not adequately treated.
The mechanism of action of BF844 (improving the function of mis-folded and mis-located proteins in the cell) lends itself to being applicable to treat other diseases associated with a decrease in protein stability due to genetic mutation. Thus, we are planning a crowd-sourcing exercise to utilize the wisdom of the global scientific and clinical communities to help us identify diseases that might respond to BF844, thus optimizing the value of BF844 to patients.
DeCanniere: And what are the implications of this work or this research for the Usher III community?
Dr. Farhan: Achieving our goals in 2017 would constitute huge progress, and we would be a step closer to finding a therapy for individuals with Usher III. The work that is involved in understanding the molecular basis of the syndrome, developing animal models that exhibit the syndrome and establishing a clear clinical development paradigm will speed up (and pave the way for) generating therapies that soon will counter the deleterious effects of the syndrome and offer many a better quality of life.
DeCanniere: What are the plans going forward with respect to research? Where do you see the Usher III Initiative a year or two from now?
Dr. Farhan: We hope that by the end of 2017, we have obtained an IND and manufactured BF844 in sufficient quality and quantity, and are well positioned to move toward clinical trials.