New CureTalks Series - Small BioTech, The Engine Of Change And Hope For Orphan Diseases eg #Myeloma #AML #TNBC #PediatricCancer

CLICK ON THE YOU TUBE VIDEO BELOW TO VIEW THE FIRST IN THIS SERIES.

CureTalks has been so kind as to initiate a new series which will highlight the value of the contribution of small biotech to the development of most drugs for orphan diseases.  Myeloma is one of those orphan diseases, and iMids, Proteasome inhibitors, CD38 monoclonal antibodies, and these drugs in combination, are the current standard of care for myeloma treatment.  You can see the small biotech origin of some of these drugs if you CLICK HERE. The average new drug for an orphan disease is just not a big enough money maker to interest any of the large pharmaceutical companies.  As a result the FDA had developed a new program in 1983 called the Orphan Drug ACT to provide incentives, economic help, and FDA assistance to navigate the FDA approval process.  All intended to promote Orphan Drug development. This benchmark program has provided the 7000 orphan diseases with many new drugs, and since the origin of this program these companies have taken the number of orphan drugs from a total of 38 to now well over 500.  A significant improvement,  however this means the vast majority of orphan diseases still have NO APPROVED drugs.

When we say orphan disease we may think this is not a very large number of patients, however 1 in 5 cancers is an orphan cancer, and the have much lower life expectancy  than non orphan diseases.  For myeloma, we have been blessed to have several drugs available, however all major breakthroughs have come from small pharma.  The most likely to be approved are those with dual FDA designations, like orphan drug, another called fast track, breakthrough, etc.  Most all major developments have come from small pharma, and currently we have 8 myeloma drugs with dual FDA designations, but only 2 are backed by Big Pharma, the remaining 6 are Small Pharma.  Big Pharma needs no help getting their products to market, however Small Pharma brings their products to market on a shoe string budget.  They are the ones that put it all on the line, do the heavy lifting, and when they get close to success are often purchased by Big Pharma who then cash in.  I just want the opportunity to highlight these small biotechs which have provided so much to the Orphan Disease community, and hope that we can continue to keep them in the game so they can continue their treatment leadership. 

I am currently in contact with the management of Cellectar and Sellas Life Sciences to provide them with a forum to discuss the technology they bring to market, and the challenges of being small bio tech and bringing life saving drugs to market. We will have our first broadcast in October with Cellectar, which has a targeted universal drug conjugate with a potential to be used for many cancers. Both of these companies have several orphan diseases which have dual FDA designations, and therefore have a high probability of success. Cellectar has 11 dual designations with 4 for pediatric cancers, and Sellas with 3 dual designation with one in Phase 3.  I have myeloma so I have a vested interest to promote the 6 small bio tech stocks with dual designation for myeloma but limited resources  However this forum will be available to all small bio tech with FDA dual designations.   The FDA has an uncanny way of identifying the best drugs for Orphan Diseases, but unfortunately these companies struggle to fund the outstanding life saving treatments.  I hope CureTalks can help to improve the lack of understanding of the importance of small biotech.

Good Luck and God Bless your cancer journey. For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1