Parkinson's Disease as Many Diseases

During the World Parkinson Congress in Phoenix, several presentations focused on new ways to classify Parkinson’s disease subtypes, based on the perspective that Parkinson’s is not one, but many diseases with overlapping scientific and clinical characteristics.

The proposed biological classification system should integrate what we know about the phenotype of the disease (how it appears to a patient or an observer) and the genotype of the disease (how genes are expressed or are changed – called mutations). Those of you who want to understand the scientific details can start here.

Consider what we call genetic causes of Parkinson’s. Whether considering GBA1, LRRK2, PRKN/Parkin, DJ-1 or others, the genetic abnormality is not the cause of Parkinson’s. It does meaningfully increase the likelihood of developing Parkinson’s but if you have one of these, it does not mean you are destined to develop Parkinson’s. So we need more than these genetic markers to create a useful classification system.

This is different from diseases such as Cystic Fibrosis, Sickle Cell or Huntington’s that are described as having 100% penetrance, which means everyone with the gene develops the disease.

Focus first on diagnostic tools, disease understanding or effective treatments?

Right Brain Bio has been fortunate to secure funding to keep our program moving forward with a primary focus on developing our therapeutic (RB-190).

• We reformatted and manufactured the drug (RB-190).
• We’ve established relationships with Parkinson’s experts in advisor roles and as clinical investigators.
• We’ve identified biomarkers and are developing new ones to verify that RB-190 is working in people the way we expect.
• We secured the tools to assure efficient and reliable execution of the trial (electronic database and related tools).
• We’re now putting together the applications to secure permission to launch the Phase 2A clinical trial.
• And we are in discussions with several people who seem likely to make the investments we need to keep moving forward.

We receive emails daily asking for updates and specifics — especially about when and where the trial will start and how people can participate. We are currently drafting our proposal (which will address such matters) in the formal application to ask regulators for permission to conduct the trial. Then we need to wait for their response before we can share the answers to such questions, which we expect near the end of 2026.

That puts us on schedule to have clinical data in 2027. If the clinical impact of RB-190 and the effects on the biomarkers are what we expect, we’ll be a big step further towards our goal — to transform the lives of millions affected by Parkinson’s.