These results are as good as could possibly be expected. They have proven the drug candidate increases ATP, and decreases cell death from apoptosis. In addition it works across multiple mutations and it was dose dependent (the higher the dose the better the response). For this stage of the trial it is essentially a perfect result. 2 years is also pretty speedy for bringing this drug to a clinical trial.
There are many things still to do. For example they have only tried two levels of dosing 5 and 10. At 15 microlitres perhaps this drug produces the same level of ATP as the native cell. Maybe that is not required and the level of ATP at 10 microlitres despite still having a slightly higher degree of apoptosis is adequate to protect clinical sight. Perhaps we will find out that the candidate is toxic and there is no safe dose in the clinical setting or maybe it will have very low toxicity and we can deliver a large dose.
It is very rare for a drug to be a complete cure for a condition. Benefits from many oncology drugs are often measured in weeks or months but are still considered clinically effective. Small delays in going blind will be appreciated by clinicians and patients. But at this stage the possibility of a very large benefit is still open. For what the trial was aiming to achieve this is a very good result. We still have a lot more steps to go but so far so good.
At least that’s my opinion.
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