One patient. Two decades of science. A Nobel Prize.
One of the most honest things said about this year’s Nobel Prize in Medicine came from a laureate himself. When the call came from Stockholm, Georg Nagel admitted he thought it was too early for a Nobel Prize. As far as he knew, only one patient had actually benefited from optogenetics.
He was being modest, but he was also right. And that is exactly why this prize matters to anyone building in healthcare.
This of course resonates with us. At HITLAB, we run small sample size studies all the time so that startups can demonstrate their clinical and experiential efficacy quickly.
On Monday, the Nobel Assembly at Karolinska Institutet honored Karl Deisseroth, Peter Hegemann and Georg Nagel for their discoveries concerning light-gated ion channels and optogenetics.
The story starts somewhere no investor would have looked: Chlamydomonas, a single-celled green alga that swims toward light. Hegemann and Nagel uncovered the proteins, channelrhodopsins, that let it sense that light. Deisseroth’s team then put those proteins into mammalian neurons, and suddenly scientists could switch specific brain cells on and off with a flash of light. The key papers came in quick succession: channelrhodopsins described in 2002 and 2003, light-controlled neurons in 2005.
Neuroscience changed overnight. Researchers stopped asking only “what lights up when?” and started asking “what happens when I flip this switch?” Correlation became causation.
Nobody wrote a business case for an alga’s eyespot. It would never have survived a three-year ROI review.
1. Our timelines are fiction. Pitch decks promise transformation in 18 months. Optogenetics took roughly sixteen years to travel from the first light-controlled neurons in 2005 to the first published evidence of benefit in a person in 2021. Count from the basic research on how this alga senses light, and the arc stretches back to the 1980s. The ideas that end up changing medicine often look slow, strange, or useless at the start. An ecosystem that funds only the obviously useful is quietly optimizing for incrementalism.
2. A tool is not a treatment, and evidence is the bridge. That one patient, a man b in France who had been diagnosed with retinitis pigmentosa 40 years earlier and was left with only light perception, received a gene therapy delivering a light-sensitive protein (ChrimsonR) plus engineered light-projecting goggles, then months of training. He could perceive, locate, and count objects. He could not recognize faces. The researchers even recorded his brain activity with EEG to confirm his visual cortex was responding. It was partial, carefully measured, and honestly reported, which is exactly what real progress looks like.
In digital health, we too often skip that honesty. We confuse a working prototype with a validated intervention, and a compelling demo with a clinical outcome. Pear Therapeutics is the cautionary tale: it earned FDA authorization for prescription digital therapeutics, then filed for bankruptcy in 2023 after struggling to get insurers to pay for them. Clearance is not the same as evidence that convinces payers. At HITLAB, much of our work lives in precisely that gap: study design, meaningful endpoints, regulatory strategy. It is where most innovations die. Rarely for lack of brilliance; usually for lack of evidence.
Five years on, others are building on that result. In September, the FDA accepted Nanoscope Therapeutics’ application for MCO-010, an optogenetic gene therapy for retinitis pigmentosa, bringing this class of therapy closer to a regulatory decision. The tool is slowly becoming a treatment.
3. The future is convergence, and our systems are not built for it. The first optogenetic therapy was a gene therapy, a medical device and a light-delivery engineering problem all at once. Biology is becoming programmable. Yet our regulatory pathways, reimbursement codes and even our org charts still sort the world neatly into drugs, devices and digital. There are early signs of movement, such as the FDA’s combination-product pathway and the Medicare billing codes for digital mental health treatment devices added in 2025. But these are patches on a system built for one category at a time. The innovators who win the next decade will be fluent across all three. So must the people who evaluate them.
Here is the question we can’t stop asking: what curious, unfundable, “why does that alga swim toward light?” question are we ignoring right now because it doesn’t fit a quarterly roadmap?
And when that idea finally arrives, will we have the evidence infrastructure to carry it from one patient to millions?
Part of the answer is in that single patient. The 2021 study behind him reported on just one person, yet it was published in Nature Medicine (Sahel et al., 2021) and changed what an entire field believed was possible. It worked because it was designed well: objective tasks instead of impressions, baseline measurements taken before treatment, control tests with the goggles switched off, and results reported honestly, limits and all. Small evidence becomes significant evidence when it is rigorous, transparent and aimed at the right question.
What it can show: feasibility, usability, early safety signals, a first estimate of effect size, and which endpoints matter for the next study.
What it can’t show: that a product works across a broad population, or that it deserves to be paid for.
What makes it credible: endpoints set in advance, a baseline before the intervention, objective measures, a control condition, and limits reported as clearly as results.
That is the thinking behind the small-sample studies we run at HITLAB, where we help startups demonstrate clinical and experiential value quickly. A well-designed early study won’t replace a pivotal trial, but it can de-risk a product, sharpen the endpoints for the next study, build credibility with investors and regulators, and earn a startup the right to keep going. Every breakthrough’s evidence base starts with patient number one.
Nagel’s humility is the right posture for all of us. Celebrate the discovery. Then get to work on patient number two.
What’s the smallest study you’ve seen make the biggest difference? We’d love to hear it.
Associate Director of Research, HITLAB
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