A Report: NPC Drug Trials
Untreated NPC Cat – Falls Over
Treated With VTS-270 – Cat Appears Normal
The data provides a strong foundation for confidence.
For the Phase I trial and the Rush iIND patients, most symptoms of NPC progression seemed to slow, stabilize or even improve after receiving VTS-270 treatment.
VTS-270 treatment is sometimes difficult, but brings results.
Although patients must go to the clinical site every two weeks, Vtesse's travel agent will coordinate travel bookings and reimburse travel expenses.
While it may cause some anxiety, many clinicians are experienced in giving drugs intrathecally (ie. via a lumbar puncture), and a number of NPC patients have already undergone the procedure and found it to be only a minor difficulty.
An adults-only safety trial to repurpose Vorinostat for NPC.
Through the use of high-throughput screening, researchers at the NIH discovered that Vorinostat, an HDAC inhibitor used to treat cancer, has potential to treat NPC.
How the Trials Are Scored
- Data from humans
- Data from large animals (e.g. cats)
- Data from small animals (e.g. mice)
- In vitro data (e.g. cell cultures)
A Detailed Explanation
- Benefits Score = a number between 0 and 1; if the expected benefit is both prolonged life and improved quality of life, it's a 1.0; if the expected benefit is only one or the other, 0.75; if the expected benefit is uncertain, 0.5.
- Data Weight = 2 for in vitro models, 2 for small animal models, 2 for large animal models, and 4 for humans.
- Data Quality = a number between 0 and 1; the more published papers and the more independent verification of the drug, the closer the score is to 1.0.
- Data Weight x Data Quality = multiply the data quality by the data weight for one type of data (e.g. in vitro).
- Sum of (DW x DQ) = the sum of Data Weight x Data Quality for all types of data (ie. in vitro, small animal model, large animal model, & humans).
- FDA Breakthrough Bonus = a number between 0 and 1; if the drug was granted FDA Breakthrough Therapy status, then a bonus of 1.0 is scored.
- Other Disease Bonus = a number between 0 and 0.5; if the drug was tested for another disease and was safe, then a bonus of 0.5 is scored.
- Difficulty Penalty = a number between 0 and 1; if the drug is easy to use, then 0 points are removed; if the drug is difficult to use, then a penalty of 1.0 is assessed.
- Control Group Penalty = a number between 0 and 1; if the drug has no control group, then a factor of 1.0 is used as a multiplier; if the trial has a 2:1 ratio active:control, then a factor of 0.83 is used as a multiplier (a penalty of 1/6th).