Oct 11, 2016

How Long Does It Take to Develop a New Therapy for a Blinding Eye Disease?

This is a tough question to answer, but one thing is for certain: you are bound to get a different answer, depending on who you ask. This question—and the answers to it—are at the core of what we are trying to do here at Fighting Blindness Canada (FBC). FBC’s singular goal is to develop new treatments for blinding eye diseases. It’s why we do what we do. How long before we succeed? We went looking for answers.

We asked every single scientist who receives funding from FBC to explain how much time it will take for their research to reach a stage when it can be tested in a clinical trial (this is when an experimental treatment is tested in people). We also asked them what additional resources they would need to get their research ready to test as a treatment.

Not surprisingly, we received a full range of answers. Does this mean that some people are right, and others are wrong? Not quite. Within the FBC-research community, there is a diverse array of experiences and expertise, which informs how people answer the question. Moreover, each research project is focused on a unique problem that fits into a web of complementary research programs, which are all at different stages of development. While FBC-funded scientists are already testing their findings in clinical trials, others are in a laboratory setting working on models of disease, hoping that their findings will influence the effectiveness of emerging treatments.

Everyone agrees on one thing: funding is absolutely essential for the development of new sight-saving therapies.

According to FBC-funded scientist Dr. Catherine Tsilfidis, the amount of time it takes to generate a new treatment depends on many factors: “It depends on the type of disease and on the degree of progression of the disease. Some diseases are already being treated using gene therapy, while others are much more difficult to treat. It depends on the prevalence of the disease in the population. Diseases that are very rare are much more difficult to fund than those that are common, and funding determines everything.”

Dr. Tsilfidis emphasizes the critically important role that funding plays in the development of new therapies: “Funding accelerates research. Without research funding, nothing is possible.”

Let’s look at Dr. Tsilfidis’ gene therapy research as an example.

Dr. Tsilfidis’ research targets a symptom that is shared by many blinding eye diseases: the death of photoreceptor cells (these are the eye’s light-sensitive cells). XIAP gene therapy targets this shared final endpoint of retinal disease that results in blindness—the death of the photoreceptors. XIAP is pronounced “zie-app” and stands for X-Linked Inhibitor of Apoptosis. XIAP acts as a general therapy that can be applied to many different types of disease, including retinal ischemia, retinal detachment, retinitis pigmentosa and Leber’s Hereditary Optic Neuropathy.

Four years ago, FBC was very proud to award funding to support Dr. Tsilfidis’ XIAP research. We were excited for three main reasons:

1. Her research on XIAP gene therapy was effective at saving vision in multiple eye diseases;
   2. She had secured excellent complementary funding to support her research from the Canadian government; and
   3. Her XIAP research was so close to the clinic—indeed, she seemed just steps away. We hoped and predicted that she would be testing XIAP gene therapy in patients living with a variety of different degenerative eye diseases in the very near future.

Although Dr. Tsilfidis had money from the Canadian government to support her research, she did not have adequate funds to conduct the critical pre-clinical tests that are required before a new therapy can be tested in people. So, she applied to FBC for funding to do special pre-clinical tests, including toxicity testing, and also generate GLP (which stands for “good laboratory practice”) grade vector, which is essential before testing a new treatment in people. Funding these tests aligned with FBC’s goal of developing new treatments and we were very grateful to receive a grant from the Krembil Foundation to support this work!

Dr. Tsilfidis is now hopeful that a clinical trial with XIAP therapy will be possible in 2-3 years. To get there, she describes how she will need “help with regulatory approval and to make the appropriate contacts with industry to help to fund the trial.” Looking ahead, Dr. Tsilfidis is optimistic because “the progress made in the past few years, and with the toxicity testing made possible by the current FBC grant, we are in a good position to approach the regulatory agencies (FDA, Health Canada) to obtain approval for using XIAP therapy in patients.”

Dr. Tsilfidis emphasizes that it is really hard to create an accurate timeline. Because of this, she is worried about letting people down if everything doesn’t go exactly as planned! But we know that timelines are important, so we applaud her for making these bold yet cautious predictions about when her research will be ready for the clinic!

Thanks to funding from key FBC supporters such as the Krembil Foundation, Dr. Tsilfidis is closer to making XIAP therapy a reality.

You can support research like Dr. Tsilfidis’ today:

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** Over the next few months, we will be sharing stories about how different FFB-funded scientists answered your questions about when and how their work will impact blindness today and in the future. Stay tuned to learn more about how your donations are making a difference.