We're ready for scrutiny (continued).

We are ready because scrutiny means careful and detailed examination. And the more careful and detailed the better.

As I reviewed last week, making an investment in a biotech company is complicated. How can a non-scientist investor or a scientist investor evaluate the likelihood of a novel therapy to work? Here's where AI can be useful.

Grok scrutinized our program and pitch, and concluded that we were "clever" and "data-driven." Specifically, Grok wrote that our peer-reviewed publication showing that dopamine levels inside neurons are elevated, which would serve as a logical basis for the disease, "is a valid and innovative estimation approach for inferring cytosolic dopamine levels, particularly in the absence of direct, non-invasive measurement tools. It builds on well-established neuroscience principles and has been applied in peer-reviewed research to support the hypothesis of elevated cytosolic dopamine in Parkinson's disease."

You will find announcements with great frequency claiming to discover the cause of a disease with the promise of a treatment to follow. And unfortunately, most of those times the data show something different than the headlines - the data only show a correlation between an abnormality and a disease. That does not mean that it is a treatable cause. This is why Grok concluded that "direct validation in living systems would strengthen it further."

In 2024, I published an article in the Journal of Neurology that summarized the data showing direct validation in 9 living systems that are accepted models of Parkinson's.

The basis for launching the phase 2A clinical trial of RB-190 asa dopamine-reduction therapy is compelling.

And then last week - there was more. An article was published in eLife that showed that mice with increased dopamine production in neurons also manifested cellular and behavioral changes typical of Parkinson's, including the loss of axons before loss of neuron cell bodies, calcium overload and changes in movement. This is an article from several world-leading laboratories working together, including the Gladstone Institute in San Francisco and Brain and Mind Center in Sydney, with support from the Aligning Science Across Parkinson's Research Network, Larry Hillblom Foundation and the NIH.

We're ready for scrutiny because our approach continues to be scientifically validated as more data are collected, published and considered (with an open mind).


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About Jonathan Sackner-Bernstein, MD

Dr. Sackner-Bernstein shares his pursuit of conquering Parkinson's, using expertise developed as Columbia University faculty, FDA senior official, DARPA insider and witness to the toll of PD.
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RightBrainBio, Inc. was incorporated in 2022 to develop tranformative therapies for people with Parkinson's.