Gene Therapy and Synthetic Merlin

Article By: Lori A Davila
Date: December 07, 2011

On 11/30/11, in a Webinar organized by NF Network, Dr Scott Plotkin MD, PhD, shared information for NF2 and of the many important topics discussed included Gene Therapy when a question was asked in reguards to Synthetic Merlin. Merlin, is the tumor suppressor protein involved in NF2 (Neurofibromatosis Type II).

The Gene Therapy topic came up towards the end at about 53minutes 47seconds in, after the discussion on Avastin and the FDA. Below is paraphrased, on what was asked and his response. Watch the video to see exactly what was asked and the exact response Dr Plotkin gave.

Question

Has anyone in the the scientific community created Synthetic Merlin?

Answer
Dr Plotkin:

Synthetic Merlin has not been created, but it is not necessary because Natural Merlin can easily be found and either extracted from cells, or produce it to reintroduce normal Merlin as a form of gene therapy.

Prevention trials are being looked at to restore the normal balance of the Merlin protein to prevent tumors from forming, or replace normal Merlin and then give drugs that can cause more Merlin to be produced.

Scientists are learning about the approach of restoring the protein function or protein levels in other genetic conditions like Muscular Dystrophy or Cystic Fibrosis. In the next 10 years doctors hope to try to restore normal levels of Merlin for NF2 and try to see what the effect on cell function is.

Conclusion

Gene Therapy will eventually be able to likely stop all tumors from growing, if not be able to fight tumors we already have when Merlin is replaced. When that happens we can then consider Stem Cell treatments to address the nerve damage the tumors have caused.

However, we are still years away from gene therapy. A lot of research needs to be done for us to get to that point and we need to be looking into what we can do till then to keep the tumors in check, and minimize nerve damage. Also, stem cell therapy are still new and until more research is done in regard to stem cell therapy, should be considered dangerous at this point in time.

Massachusetts General Hospital - 11/30/11: Dr. Plotkin - Webinar Transcript