Firstly, it is common knowledge that conventional drug discovery is a very expensive and painfully slow process and in many cases, and exiting drug can be used to treat a neglected disease or prove to be a safe and more effective alternative to current therapy. Treating a rare disease comes at a six to seven-figure cost. Such numbers not only bankrupt the health system but also the families.
In many cases, pharmaceutical companies show little or no interest to investigate the potential use of a conventional drug to treat or prevent another disease. This largely occurs when the drug has lost its exclusivity or patent or sometimes both and there is marginal financial incentive left for the stakeholders.
On the other hand increasing failure rates, poor bioavailability, poor safety, high cost and above all limited efficacy is very convincing grounds for an alternate approach to drug discovery.
Exploring established non-cancer drugs for anticancer activity provides an opportunity rapidly to advance therapeutic strategies into clinical trials.
Having Omics data at our disposal that we did not a few decades ago, with well-equipped informatics it has become common knowledge that dic=verse diseases have common molecular origins.
It is not difficult to be able to feel the pain of those unfortunate parents who can do nothing but to wait. If the thought of the pain those children undergo can not keep you awake at night, then one can only imagine what will?
Knowing that we can reduce those numbers and knowing that we can repurpose those drugs to offer a better life to these patients and yet we look away is not an act of humanity. We are all parents and will be someday. Let us all work together and bring more medical equality.