Finding cures for children's genetic diseases


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  • Dr. Scott Cohen
    Dr. Scott Cohen

Meet Dr. Scott Cohen

Dr. Scott Cohen works quietly, but his research speaks loudly: He determined the molecular composition of human telomerase, a factor important in 85% of all cancers. With the help of increased funding he’s now taking his research to the next level.

Q: Why do you study telomerase?
A: Most cancer cells rely on telomerase to keep growing, making it a prime target for anti-cancer treatments. There are no small-molecule inhibitors against telomerase in clinical trials, but there is a clear need for these drugs.

Q: What are you working on?
A: I’m taking a two-pronged approach to developing telomerase inhibitors. The most important goal is to determine a high-resolution structure of the telomerase enzyme. Structure tells us “how” and “why”. For example, the structure of DNA told us how genetic information is stored and passed down. Understanding the structure of telomerase will reveal how it works and how to rationally design drugs to block its function. I’m also screening small molecules for their ability to block telomerase. This screening process can reveal candidates to be developed into drugs, a process that will be aided by understanding telomerase structure.

Q: How has funding, including that from Ernest & Piroska Major Foundation, helped?
A: The Foundation has contributed $420,000 over 4 years, keeping this project afloat. Now, they have committed a further $240,000 for the next 3 years. This research is long term and high risk, but they understand its great potential. They have funded the structure work and now the small-molecule screening too, for which I am very grateful.

Q: What’s next?
A: Just a few weeks ago we reached a major milestone: we were able to observe individual telomerase molecules using a technique called Cryo-EM. So, after nine years, I’m much more confident now than just a few weeks ago of eventually solving telomerase’s structure.

Funding is what keeps Scott's research going 
each and every day. With continued support he is able to reach new milestones in the important work he does. Help us create a future without childhood diseases like cancer by donating today - and remember that every dollar counts!