Why Britain must invest in gene therapy in the NHS

A few weeks ago I became a parent and it was a challenging time for me. However, my experience gives me a great deal of optimism about the potential of DNA therapies to save…

Why Britain must invest in gene therapy in the NHS

A few weeks ago I became a parent and it was a challenging time for me. However, my experience gives me a great deal of optimism about the potential of DNA therapies to save many lives.

At 53, my cancer prognosis was dire. Most people would not hold their breath in such a situation, but I had faith that I could do what I had done in my career and turn this situation into something better. I chose to try novel therapies to enable stem cells to turn my tumour into a type of graft that could be transplanted back into my body.

I got that all right, and because of this I now live with a rare type of leukaemia in the lower part of my lower spine.

I am one of the lucky ones. I have been fine for three years now. The cells that sent my disease into remission were not actually found on the small intestine or the muscle wall. I am grateful for the stem cells that turned my tumour into a stem cell drive. It gave me a chance at living in another life for a time and I am thankful to that therapy.

Citizens for Genomics, the BMT Foundation, the BMS (Biopharmaceuticals for Clinical Research) and its leader Corinna Hahn-Wickert knew how important it was to target the leukaemia stem cells. They provided the core and then provided the fuel to build and run the chemical reactions necessary to turn the stem cells into a healthy white blood cell. I am grateful for this therapy.

I will never forget the sequence of events that led to the discovery of this cancer drug. I immediately embarked on a path to get stem cells called DCA (a DNA short-cut) licensed as a cancer drug. DCA has been in clinical trials for nearly 20 years and it has shown both safety and efficacy. DCA has been shown to work for a broad range of cancers including melanoma, breast cancer, esophageal cancer, lung cancer, prostate cancer, colorectal cancer, ovarian cancer, head and neck cancers, small cell lung cancer, renal cell carcinoma, and serous lysosarcoma.

But DCA is far from the only gene therapy therapy available. There are two others: the miRview, which is known to work for advanced blood cancers and skin cancers; and pegvaliase, which is known to work for rare disorders of inherited organs. The CF people, who are all under 30 and have rare inherited mutations, have little to no hope of finding a safe gene therapy in this country.

Because we have low survival rates here, many patients have decided to go abroad for their clinical trials. The truth is, our clinical trials programme for gene therapy in the UK is not ready yet. The clinical research which started 10 years ago in 2005 still hasn’t finished.

I have been thinking about the process of getting my DCA treated and I would like to know how anyone can access gene therapy and this treatment on the NHS.

Well, until it is ready, we have to wait for other pathways to develop. All of the clinical research will continue to support understanding of these diseases. But when there is a cure, it will be up to us, the parents of sick children, to show the taxpayers of this country that they have made the right decision to fund their stem cell treatments.

Every day we wait, our children’s disease will be our responsibility and our responsibility alone. If there is any way we can support parents who want to provide their children with treatment we need to do it, so their children can make it home from the ward one more time.

If the government will not fund gene therapy here in the UK, we have to figure out what to do next to help our children survive the battle with this disease. If we don’t, who will? And how can they, as those taxpayers, stand up and tell us they gave us the money to do our jobs as a care worker?

One of the biggest influences of my own search for gene therapy was about COS-tech, the multi-billion pound vaccines R&D company in California. But now their focus has changed from CAR-T treatments, to gene therapy. This means that the time is ripe for England to support, fund and develop our own gene therapy company.

Genetics technology is changing all the time, and we have to keep up with it.

If we are unable to develop a gene therapy for kids who don’t live here, who will provide this treatment when they arrive? What hope will the parents have

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