SOHO 2023 | Unmet needs in amyloidosis: early diagnosis, treating relapsed disease & progression criteria

So unmet needs, early diagnosis is definitely a major unmet need because the longer a patient goes, the more likely they’re going to have potentially irreversible damage to their organs and a higher risk of kind of early death. So early diagnosis, which people are addressing with education, strategies like artificial intelligence, NLP, different methodologies. So that’s a big unmet need. In terms of the treatment, there’s a lot, we still don’t know best therapies at the time of relapse, best combinations. There are all of these immunotherapy strategies in the myeloma space and they haven’t yet been really vetted and tested in the amyloid space. So that’s a huge unmet need. Drugs like venetoclax with patients with translocation 11;14 is a really amazing advance, though so far we don’t really have true clinical trial data, a lot of experience and case series. And then I would say the final unmet need is I think we still need to optimize our progression criteria in that space. Because in order to really get drugs approved, we need to have reliable endpoints that can easily be adjudicated and that have meaning so that regulatory agencies will approve the drugs.