Scary Stuff

October 30, 2014

by Mark Dunning

It is Halloween week so it is a good time to write about something very scary. We are at a critical point in the search for Usher treatments. There are a number of promising developments in the lab that could lead to treatments. These are getting close to clinical trial but they may never reach the clinic for one very avoidable reason:

We are not in touch with enough people with Usher syndrome.

I can’t state this strongly enough. Clinical trials will be suspended, abandoned, or simply not begun at all if we cannot identify enough candidates. You need these trials and these trials will desperately need you.

In a recent post I mentioned the example of the drug Solaris. This was developed for an orphan disease that affects about 4,000 Americans. That’s about the equivalent of the number of people with Usher 1d in the US. I used the example to illustrate the cost of a clinical trial. But buried in that example is a more frightening number: 208. That’s the number of people with the disease that were involved in the trial (11 in phase I, 11 in phase II, and 186 in phase III) and it represents 5% of the total number of people with the disease in the United States. 

The pool of people needed to identify candidates for clinical trial is even larger than that. That 208 number is the final number of participants in the final trial. But remember, that is just one trial. Only 1 in 12 clinical trials result in a treatment making it to the clinic. In many cases participation in one clinical trial precludes participation in a second trial for a period of years. So the people in phase III can’t be the same from phase I. In that recent post, I estimated that to get a treatment in to clinic would take 12 phase I trials, 3 phase II trials, and 1 phase III trial. Using the number of participants in each phase from that Solaris example, that means we would actually need 351 people to participate in the trial. That’s almost 9% of the total population for that particular disease.

Hold on, there’s more. According to a Research America poll, 72% of people polled would be willing to participate in a clinical trial if asked. So to get 351 people to participate, we would need to ask 487 people. Now we’re up to 12% of the total population with that disease in the US. When you take in to account the restrictions on age, amount of vision, language, and accessibility to a trial site it becomes even harder to identify people for a trial. We might need to ask 1,000 people or more to participate to find 350 willing, appropriate candidates.

Why should these numbers scare you? Globally we, the Usher research community, are in touch with around 3,000 people with Usher. That’s for every type of Usher syndrome. The Usher Syndrome Registry is one of the largest single sources of individuals with Usher and it contains 650 people. Again that is every type of Usher syndrome. To be blunt, globally the Usher syndrome community could not quickly produce a pool of 487 people of any single Usher type for clinical trials. This is in spite of the fact that Usher impacts 400,000 worldwide.

A graph

This is tragic. In my last post I discussed four groups that have the means to provide the $50 million a year we need to fund Usher syndrome research. But governmental organizations will not fund grants that cannot produce enough candidates for clinical trials. Large, global, non-governmental organizations will not fund projects that can’t be delivered to the entire population. Pharmaceutical companies will only fund clinical trials if they see a reachable market for treatments. Right now we as a community can’t meet any of those requirements.

Identifying and maintaining contact with every person in the world with Usher syndrome is one of the goals of the Usher Syndrome Coalition. So while it is correct to write that we do not directly fund research, the truth is the efforts of the Usher Syndrome Coalition are critical to developing treatments. I would argue that at this moment in time, our effort to identify everyone in the world with Usher syndrome is THE most critical thing. 

So join the Usher Syndrome Registry today. Tell everyone you know with Usher to join the Usher syndrome registry today. Talk to every institution you know that is in contact with people with Usher and get them to let their constituents know about this urgent need today. 

Joining the registry doesn’t mean you have to be part of a clinical trial, it doesn’t mean you will be selected even if you are a willing candidate, but it will ensure that you are informed of every clinical trial. More importantly it will create a sufficient pool of potential candidates that researchers can contact through the Coalition. I have written it before. Without you, there will be no treatments. Now, more than ever, that is true.

And, since we are in the midst of our annual appeal, realize that without the Usher Syndrome Coalition there will be no registry, no coordinated outreach, no support, and far less collaboration between researchers and families. Please give what you can to support these efforts. As always, we need you.

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