Successes
More than 100,000 patients are already receiving the critical benefits of repurposed treatments that were created in part by funding from PFC donors.
Using $300,000 in donor contributions, and matching funds from Mayo Clinic, PFC was able to support one of the first research projects to look at the mechanism of action of the drug Thalidomide - which was being explored as a treatment for the blood cancer multiple myeloma. Additional research from other groups added significant data on the mechanism of action of thalidomide and its effectiveness in myeloma with improvement in length and quality of life for myeloma patients. Thalidomide was eventually approved for use in myeloma patients by the FDA, and the drug company Celgene created a new Thalidomide-like drug that is stronger but has fewer side effects. Thalidomide and similar drugs are now being explored in dozens of other diseases!
PFC funded researchers at Children’s Hospital of Philadelphia created, in less than two years, a $10 per day repurposed treatment for children suffering from Autoimmune Lymphoproliferative Syndrome (ALPS), a rare, life-shortening disease that affects the immune system. The day this research was published, physicians from around the world began using this drug for their patients. Prior to that there was no effective treatment for these children and they typically died before age 21. After three weeks on this repurposed treatment, all symptoms of the disease disappear and have not returned for any of the patients!
Click here to read Dr. Teachey's published article
Late stage lung cancer is a disease that is difficult to treat. A new class of drugs currently on the market helps a small percentage of patients for a short period of time. A PFC funded research team at Massachusetts General Hospital discovered that when these drugs are used in conjunction with a safe, inexpensive anti-malarial drug a higher percentage of patients responded to the “new” treatment for a longer period of time.
