Tuesday, May 17, 2022

Re-Thinking Drug Development Roadmap In United States

March 21, 2008 by  
Filed under Health Care Policy, Virtual BioTechs


No Insurance. No Money. No Drugs. No Hope.

We Need A New RoadMap For The Future

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 10+ 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 $180,000 a year for a highly experimental drug treatment for their two children afflicted with Niemann Pick Type C. Who can afford a single experimental drug at this price?  Like millions of others, many Niemann Pick Type C children have no health insurance.  No insurance. No money.  No drugs.  No hope.

The dire situation we are faced with our drug development process is happening across disease states — this is not a unique problem to Niemann Pick Type C. The FDA is in a major crisis and we can no longer continue on this path when millions of lives are at stake. The Addi and Cassi Fund, named in honor of our five year old identical twin girls, plans to bring attention to the drug development crisis and to push for change.

We are building one of the first “Virtual Pharmaceutical” models in the United States with the help of Silicon Valley heathcare company CollabRx. Our Virtual Pharmaceutical is named "SOAR-NPC" (Support Of Accelerated Reserach for Niemann Pick Type C) and we are focused on creating a drug therapy pipeline for NPC afflicted children around the world. If global pharmaceutical companies and the FDA won’t help us save our children by rapidly bringing new drugs to market, we must find solutions ourselves in order to attack and treat a rare and fatal disease like NPC.

One of the greatest resources we have as a nation is our National Chemical Genomics Center. The robotic technology at the NCGC which is pictured above should continue to be invested in by our government — NCGCs technology can find existing drugs sitting on shelves that can be repurposed and used to treat diseases they were not originally intended for. Currently, we are funding the NGCG to test thousands of approved drugs as well as natural supplements against multiple Niemann Pick Type C cell lines in the hopes of finding immediate targets to treat afflicted children.

Every dollar donated to The Addi and Cassi Fund goes directly to cutting edge research projects to rapidly accelerating finding immediate treatments for brain diseases. 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 alternative approaches to the current drug development process.

We must seek treatments today and cures tomorrow!

A Boy With A Very Big Heart

March 18, 2008 by  
Filed under Get Involved

I was laying in bed the other afternoon after staying up all night emailing scientists the results of Addi and Cassi’s brain scans when the doorbell rang.  It was Christopher Serio with an envelope in hand.  Chris is 11 years old and is the son of our friend and former neighbor, Bob.   “Here, this is for Addi and Cassi,” he says and proceeds to hand me an envelope decorated with a red heart, a sunshine and pine trees.   “I just counted up all my savings from when I was little.  I have $653 dollars and I want to give Cassi and Addi $150 dollars of my savings for research into Niemann Pick Type C.” I open the envelope and it’s full of cash.  I almost burst into tears but I somehow hold it together and give him a great big hug.   I think about how generous this young man is.  It turns out that this is an entire year of his allowance.   Would anyone else we know give us an entire year of their savings to help with research?  I am so inspired by Chris and his generosity.   As I watch him walk down the driveway and get into his Dad’s truck, I hold out hope that Addi and Cassi will someday be competing for his attention.  A lovely young man with a very big heart.  That’s Christopher Serio.