A double-blind approach to restoring vision

After a much regretted hiatus, I was driven to write this blog post by the frankly extraordinary experiences I’ve had in recent weeks regarding ‘alternative’ therapies for disorders that cause blindness. Apologies for the long post that follows - if you follow it all the way to the end I promise it'll be worth it...

Firstly, a disclaimer: I am a molecular biologist, working on gene therapy for retinal disorders. You may be familiar with our work via the recent media interest surrounding the publication of results from our Phase I/II clinical trial for gene therapy for a form of early-onset sever retinal degeneration called LCA (I don't think the full article is behind a paywall so you should be able to access it, any probs please le me know!). Indeed I'm writing this from the USA where our research group has just presented the findings to a major conference.

I say this not to gloat about how fantastic our work is (although of course it is...), but rather to make a point. That is, there are many academic research groups working on several well-researched treatment options for the several forms of retinal disease that lead to blindness - indeed a group in the USA published the results from their trial at the same time as us. These potential therapies are based on research into how the various diseases are inherited, how mutated genes can be replaced or modified, and how such potential therapies can be translated from cells to animals to people. This is, after all, how science works - progress through peer-reviewed publication and controlled studies that lead to careful use in clinical trials.

Of course patients with retinal degeneration don't really care about how a treatment for their condition is developed, as long as it restores vision. But that makes it all the more important to ensure that developments such as the trials I just described are handled well by the media, that people understand how we've arrived at the stage of restoring vision to people losing their sight, how we may proceed to widen this type of therapy to other conditions. So, this is where the fun begins.

Last week I went to Madrid, to speak at a meeting organised by the Spanish Retina Foundation. They had one day of talks for a clinical and scientific audience, and one day of talks by the same speakers, to patients, relatives and carers of those with retinal degeneration (it was truly a novel experience to attend a conference in Spain without knowing a word of Spanish, although the United Nations-style live translation of lectures was amazing). I spoke on both days, describing our group's work on gene and cell therapy for blindness, describing the ins and outs of the work leading up to the trial. The patient audience was really engaged in the talk, and took a great deal of interest in our work - of course they did, they're hoping that such treatments may one day help them or their children see.

I shared the stage with a Spanish scientist who told the audience about other therapies for retinal disease, describing amongst other things some work published last year where specific anti-oxidant compounds were given to mice that suffered retinal degeneration, and there was some protective effect. This is one study, in mice, and although it's quite robust, needs repeating and refining before we tell LCA patients to rush out to Holland and Barrett to stock up on vitamin pills. But at least one member of the audience seemed to understand it as "oral anti-oxidants improve sight," because at the end of my talk he asked the following question:

As you mentioned, alternative therapies such as vitamins may improve sight. What role do you think Ayurvedic Medicine may have to play in retinal degeneration?

?!

I took a deep breath before answering - here I was representing my lab, in front of hundreds of people, and the last thing I wanted to do was lose it. My answer consisted of phrases like "we believe that therapies for such severe diseases need to be properly researched" and "should there be any evidence for efficacy of a treatment, then and only then can in be recommended" and "as the whole point of this conference was to inform you of the scientific process involved in gathering evidence for a therapy based on genetic research, I would advise that any therapies without a basis in evidence are not pursued." How else to deal with such an enquiry? Of course I don't want to be dismissive of the situation my audience was in - any therapy, any treatment, anything at all that could work would be so useful, but the use of "alternative therapies" to treat disorders in which cells in the retina die is, err, somewhat misguided. I emphasise here that I hold nothing against the chap that asked the question - being blind is unimaginable for those of use fortunate enough to read these words, and if someone tells you that X, Y or Z might work, you'd trust them. But how did we get to the stage where such "alternative" approaches are seen as being just as valid as the scientific approaches...?!

Turns out that he wasn't alone in wanting to apply woo-woo for blindness. As someone with a PhD in gene therapy for retinal degeneration, websites like this one peddling a compound called Tostitin make me cry. Classic woo tactics are used - testimonials asserting that the stuff works, guarantees of safety and efficacy, and of course a link to a site where you can pay for this compound by credit card.

The amazing 100% guaranteed treatment for Retinitis Pigmentosa that is bound to leave you amazed!

No matter how long you have suffered from Retinitis Pigmentosa we assure you that with use of Tostitin you will regain your condition faster than any other solution currently available.

Tostitin is an established treatment and produces time tested results.

Tostitin's composition is 100% safe and all its ingredients are purely natural and free from any side effects.

Not only is this dangerous, in that patients surfing t'internet could well believe that there are homoeopathic remedies for their conditions, but it totally undermines the entire research effort into remedies and treatments that are legitimate, safe, and could actually work.

Together with the Ayurvedic question from Madrid, this last week has been a strange one - on the one had, members of the research team I'm part of have been heaped with praise for their diligent, well-controlled, scientifically sound, superbly presented and ultimately successful clinical trial of gene therapy in the eye. And on the other, we have a public willing to ignore all this and jump into the unknown using so-called alternative therapies.

Sometimes I just despair...