The series of clinical trials for a potential drug discovery and therapy can cost hundreds of millions of dollars and years of investigations. The human costs are even greater: patients with progressive terminal illnesses that may have just one shot at an unproven but promising treatment.
Clinical trials on drug discovery typically require patients to commit to a year or more of treatment, during which they are not allowed to pursue other experimental options. A long clinical trial without the backing of extensive and expensive animal data keeps patients out of tests for therapies that may have a better chance of success with an actual human.
Drug discovery has many laws that need review. “The problem is definitely not isolated to ALS. … It’s pandemic across drug discovery,” said Steven Perrin, chief scientific officer of the ALS Therapy Development Institute. “A patient in a disease like ALS, or a very aggressive oncology indication like pancreatic cancer, possibly has one shot on goal to find a treatment that might slow down their disease. … It’s our responsibility as scientists and doctors and clinicians to push the best opportunities forward for patients.”
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