|
PCUT is a non-profit, clinical-stage drug development company.
PCUT identifies high-potential drug programs that have stalled due to economic obstacles and builds operating partnerships to restore manufacture, clinical trial, and expanded access (compassionate use) programs en route to FDA approval.
The company works with existing, proven-safe drugs that have potential effectiveness in treating rare life-threatening diseases. These products may be, in some cases, already approved for other indications.
PCUT is a 501c3 non-profit company with the social mission of delivering potentially life-extending treatment options to dying patients who have no other effective treatment available to them.
PCUT brings drugs to clinical trial by structuring drug specific LLC's for product development and commercialization. The social/commercial partnerships can include operating companies as well as venture philanthropy and third party capital.
The company's pilot program is a safe growth-factor complex that was FDA approved for children's primary IGF-1 deficiency in 2005 and is now being re-purposed for the treatment of ALS (Lou Gehrig's disease).
A future cure is not the same as an effective treatment today.
Our focus on today’s sufferers is important, because providing people right now with potentiially life-extending treatment is a distinctly different mission than pursuing a future cure for the disorder.
We all share the interest in eradicating life threatening disorders for future generations. But those patients who have been given a life expectancy of 2-5 years need something right now that slows the progression of their degenerative symptoms and gives them a chance to live long enough to benefit from future availability of better therapies. It is an emergency situation.
Today’s discovery-phase research takes eight to twelve years to move through translational and clinical development before becoming available to the public. Newly funded research rarely, if ever, can save the lives of ANY current sufferers of an imminently fatal disorder. When we focus solely on new research for an absolute cure, while neglecting existing, potentially effective treatment that could be put to use right now, we are throwing lives away.
We make therapies available by using the science that already exists.
PCUT sides with patients who are alive right now and who have chosen to fight for their survival. We recognize that some in the health care and science communities discourage this view, but it is essential to stand up for the patients and their families who choose to do everything possible to beat their condition.
In the case of ALS (Lou Gehrig’s disease), a specific growth factor therapy has shown promise in arresting motor-neuron degeneration, and in some cases reversing the progression of the disorder. Additionally, the product has clearly demonstrated a clean safety profile, having been clinically tested in over 160 people, and anecdotally observed in over 80 ALS patients. This science already exists because in 2006 the therapy was FDA approved for a different disorder. Unfortunately, due to economic conditions, the manufacturer was forced to abandon the program before it could be approved for ALS.
This is one example of a potentially effective therapy that could be made available within months to dying patients who have no other effective treatment option, and who otherwise face near term death. The patients cannot wait years for a cure. But we can help them by using the science that already exists.
|