No insurance. No money. No drugs. No hope.

The Push For Change: Re-Thinking Drug Development To Treat Diseases

Of 7,000+ diseases that affect humankind, treatments for only 200 of these diseases are being actively pursued by pharmaceutical and biotechnology companies. With the cost of bringing a new drug to market now exceeding $1 billion dollars and taking up to 15 years to test and develop, global pharmaceutical companies are focused on return on investment. 

Meanwhile, millions of people suffering from all types of diseases will never see new drugs reach them or their loved ones in their lifetime.   As a nation, we must unite to think about the drug development process in a different way and push for change.

Today, one family is faced with paying $225,000 a year for a highly experimental drug treatment for their two children afflicted with NPC.  Who can afford a single experimental drug at this price?  No one!  Hundreds of children around the world have no access to health insurance.  No insurance. No money.  No drugs.  No hope.

This dire situation with drug development is happening across diesase states — this is not a unique problem to Niemann Pick Type C.  The Addi and Cassi Fund, named in honor of our four year old identical twin girls, plans to bring attention to this drug development issue and to push for change for all.  

We are building one of the first “virtual pharmaceutical” models in the United States and we are focused on creating a treatment therapy pipeline for Niemann Pick Type C (NPC) afflicted children around the world.  If global pharmaceutical companies won’t invest in helping us by bringing new drugs to market, we must rethink how we attack and treat rare diseases like NPC.   The FDA is in a major crisis.   We can no longer continue on this path when millions of lives are at stake.

One of the greatest resources we have as a nation is our National Chemical Genomics Center.  The robots and technology at the NCGC should continue to be invested in by our government — this technology can find existing drugs sitting on shelves that can be repurposed and used to treat diseases they were not originally intended to treat.   Currently, we are seeking out known drugs as well as natural supplements and vitamins including DHA from fish oil and Alpha Lipoic Acid that are proving effective in early studies on neurological diseases.

Every dollar you donate to The Addi and Cassi Fund goes directly to research projects that are rapidly accelerating finding innovative therapies for NPC.   Translational research – studies that bridge the gap between the discoveries in the laboratory with the need to find ways to intervene in the disease process and treat patients who are living with illnesses - is the roadmap for the future.   With your help, we can build this roadmap and prove there are alternatives to current drug approaches.  Treat today, cure tomorrow!