Gleevec Cancer Drug Reported To Work In Alzheimer’s disease also working in Niemann Pick Type C mouse

A New York Times story is reporting that Paul Greengard (and his dog Alpha), who won a Nobel Prize for his work on signaling in brain cells, has found out that the cancer drug Gleevec, is a new potential drug target for Alzheimer’s disease and block gamma secretase activating protein.  Rudolph Tanzi and Paul Aisen [...]

IND Application Filed With FDA To Bring Cyclodextrin Into The Brain To Treat Fatal Childhood Cholesterol Disease

After months and months of work and input by doctors and researchers, Dr. Caroline Hastings at Children’s Hospital Research Center Oakland filed our second Investigational New Drug application (IND) with the FDA on July 14, 2010.
The 200+ page IND filing details our request to deliver hydroxy-propel-beta-cyclodextrin (HPBCD or CYCLO) directly into Addi and Cassi’s central [...]

Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain

The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One!
We are now working on making our own miracle  – a new compassionate use investigational new drug (IND) protocol and application that we plan to [...]

Instructions On How Cyclodextrin Solution Is Made For Addi and Cassi’s Intravenous Cyclodextrin Therapy

A number of people from around the world who have children diagnosed with the fatal cholesterol disease called Niemann Pick Type C  have asked me how our we prepare our cyclodextrin solution for Addi and Cassi’s bi-weekly IV infusions of the compound.
I asked our pharmacist at Renown Hospital in Reno, Nevada, to provide me with [...]