“Gene therapy is no longer the future, it’s happening now”


Recently, NF2 BioSolutions bursted into the world of NF2 and its growth is continuous. Nicole Henwood is warm, willing, persistent and also a doctor, specialist in genetics and mother of a child diagnosed with NF2. In this interview she talks about this non-profit organization, whose driving force is to achieve a cure for the disease.

How was NF2 BioSolutions born?
We were born because we were needed. There was no group dedicated solely to NF2 in the US and certainly no other group looking at gene therapy. The need was there and we came into the NF2 community at the right time.
How would you explain what gene therapy is to someone who does not know anything about it?
First I would send them to our website (www.nf2biosolutions.org) because we have a lot of information about this on the site. But in short, gene therapy is any therapy that involves genes/DNA. If the problem is caused by a missing gene, there are ways of replacing that missing gene, using something called a vector, to carry the missing gene into the cells. If there is a mutation, or mistake, there are ways to fix or edit the gene, for example with a product like CRISPR-cas9. There are many other ways to use gene therapy but these two are the most common.
Gene therapy, is it a future therapy or something that is already accessed?
Gene therapy is no longer the future, it’s happening now. In 2017 the FDA approved the very first gene therapy to replace a nonfunctional gene in a rare disease that causes blindness. Many other applications are already waiting to be reviewed by the FDA and thousands of clinical trials are taking place to treat genetic disorders.
What are the short-term objectives of NF2 BioSolutions?
Our short term objective is to raise enough money to start a preclinical program to study how gene replacement therapy will work to slow or halt disease progression in NF2. Once we get something that works in cells and animal models then we can head toward clinical trials. We are also working with a researcher who uses gene therapy to kill Schwannoma tumor cells.
How can patients and their environment help achieve that goal?
The most important thing patients can do is educate themselves and their friends and family about the power of gene therapy. We have a page dedicated to gene therapy successes in other diseases on our website, this is good place to start.
It has been proposed to change the name of the NF2. Do you agree with that?
This doesn’t concern me very much, by the time we get around to changing the name I hope we have a treatment that will make this disease so insignificant that the name won’t matter. “A rose by any other name would smell as sweet”. NF2 by another is still the same disorder, and no matter what it’s called we will never stop fighting to put an end to it.
Interview and edition: Florencia Sarratea and Lluis Martínez.
Revision: Pía Errozarena and Ana Massa.
Images courtesy of Nicole Henwood to AMANDOS.

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