We believe that those who’ve been diagnosed with terminal diseases should be able to use every existing means in their fight for survival.  In support of that belief, we target our projects to overcome the challenges that block patients’ access to existing developmental therapies.

U.S. Code of Federal Regulations, FDA code, and FDA testimony in the U.S. Supreme Court have clearly established the need and appropriateness of Compassionate Use of developmental drugs in the treatment of immediately life threatening diseases.  That means the regulatory restrictions are minimal when terminally ill patients and their doctors decide to pursue drugs that are not yet FDA approved for marketing.  It also means the criteria for evidenced efficacy are low in the case of immediately life threatening disease and where the patient has no other effective treatment options.

Nevertheless, the level of required investigatory approval can still make it difficult to pursue Expanded Access Programs (EAP), Phase 3 clinical trials, and ultimately full marketing approval.

Serious economic obstacles are routinely encountered by small drug companies that develop only one product and have no other sustaining revenue sources.  And in the case of rare disorders, a small patient population represents a diminished return on investment for specialized drugs. Furthermore, the approval process, even when working smoothly, has so many built-in delays that a drug sponsor can burn through its operational budget long before the drug is approved for human trial.  Due to these challenges, companies often find that the development programs of promising life saving drugs are unprofitable and unwise to continue.

But the nonprofit sector is uniquely able to bridge the economic obstacles in a particular drug’s development path.

We negotiate independently with 3rd party contract manufacturers, research clinics and trial administrators to structure deals at cost.   And as charitable cause we can broaden public support and partner with foundations to provide stable funding for drug production in ways that a shareholder owned company could not.

Why are we devoted to this?  Because our first concern is for the lives of existing patients, not research scientists, and not shareholders.  And those currently suffering from quick-killer disorders like ALS are being ignored in the slow march toward a cure.