The Long Road for Developing Novel Treatments for People with Chromosome 18 Conditions - Part 2
Last month’s article described the general research steps conducted by the Chromosome 18 Clinical Research Center. Here, in Part 2, we identify the exciting steps on the horizon that will bring us to treatments for the currently untreatable aspects of the chromosome 18 conditions.
5. Screen thousands of drugs or potential drugs
Drug screening and development is where the previous two steps come together with the steps which we have not yet begun. Once the specific abnormality is defined for the cells in a dish, they can be assessed for their ability to behave normally after the addition of different drugs.
There are thousands of drugs that have been developed by drug companies and have even passed the first step of being approved for human use; However, they did not significantly improve the condition the drugs were developed for and were never put on the market. Conversely, there are also compounds derived from medicinal plants that can be tested. All in all, there are thousands of potential compounds that can be tested. To accomplish this drug screening effort, there are specialized facilities to do this testing using robotics to evaluate the massive number of compounds, each at different strengths. If the only identified compound is one with a minor positive effect, medicinal chemists can make minor alterations to the chemical structure to improve the compounds effectiveness. But again, success at this step is only improving or normalizing the function of cells in a dish.
6. Test candidate drugs in the mouse
Once a potential treatment compound is identified, it can be tested using the mouse with the chromosome 18 single gene defect developed for that specific aspect of the chromosome 18 condition. The mouse can be evaluated for all the aspects described above to determine if the candidate compound is actually effective in a whole animal, and at what dose. Again, if the positive effect is minor or incomplete, the compounds could be slightly chemically modified and re-evaluated.
The timeline for these drug identification steps is highly unpredictable. A successful compound that has already been approved for human use could be identified and moved to clinical trials within several years, given sufficient funding. The odds of this might be slightly better than winning the lottery. But eventually success will come – it is a matter of not giving up. Additionally, at these later steps a pharmaceutical company may become interested and help to fund these next steps because although the chromosome 18 conditions are ultra-rare, the aspects that we seek to treat are not. Therefore, a treatment we develop may have applicability to a much broader population.
7. Clinical trials
Once all these steps have been successfully completed, the next step is to move to human clinical trials. When the clinical trial steps have successfully advanced to evaluate the effect in people with a chromosome 18 condition, the study population will likely be young adults. The complicated potential and pitfalls of clinical trials is an entirely separate article with many more steps, an unpredictable timeline, and points of potential failure. If you are wondering just what success might look like, see the article entitled, “What does treatment look like?” In the August 2022 newsletter (https://myemail.constantcontact.com/What-Might-Treatments-Look-Like--New-Article-from-Dr--Cody-.html?soid=1104240642387&aid=fIDYH0YYHeg).
It may be a long path, but it is feasible, and we each have a part to play.
