Drugs and Products to Watch in 2018: Part 3

Gene therapy: Regina Au guides us through key developments in 2018

Regina Au

You may remember reading Regina's previous articles about drugs and products to watch in 2018. Part 1 delved into oncology and part 2 looked at gene editing and heart failure devices.

In part 3 Regina discusses gene therapy and shines some light on future technologies. 

Gene Therapy

Gene Therapy has been found to be effective for diseases that are a one gene mutation. The challenge is to find a delivery system, such as an associated adeno-associated virus (AAV) vector that will penetrate the cell and deliver the gene completely.

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Scientists at the University of Pittsburgh School of Medicine made significant progress in their efforts to use gene therapy to reverse diabetes. They demonstrated a gene therapy approach that transforms alpha cells in the pancreas into fully functioning beta cells. Using an AAV vector, they delivered two proteins, Pdx1 and MafA instead of a gene, into the pancreas. These proteins “reprogrammed” alpha cells into insulin-producing cells and the mice maintained normal blood glucose levels for about four months.

One potential advantage of transforming alpha cells into beta cells is that alpha cells are distinct enough that the immune system is unlikely to mistake them for beta cells and attack them. The researchers compared gene expression patterns between normal beta cells and the alpha cells that they transformed into insulin producers and determined they had achieved “nearly complete cellular reprogramming,” according to George Gittes lead author the study.

However, Gittes and his colleagues will need to further their research in determining how to obtain this same response in humans. They are also concerned that this gene therapy may be only temporary and the mice would eventually return to a diabetic state.

Closing thoughts

There are a lot of technologies and products being developed today to restore the quality of life for patients in addition to reducing morbidity and mortality for a number of diseases allowing patients to living longer. In the past, those diagnosed with cancer had a mortality rate of a 100% within months, but scientists have and are developing drugs that can keep a patient cancer-free for years.

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When I was younger watching futuristic shows such as Star Trek depicting hand-held medical scanner that diagnosed what was wrong with you, I always thought that it was only in one's imagination and that we would never get there. But today, in medicine, we have MRI and CT scanners, 3D images, 3D printing from these images and the technologies that I've discussed above and previously such as regenerative medicine and CAR-T therapy. We live in an incredible time to be so fortunate to see all these innovations come to life.

The one technology that would be interesting to see in the future if I had a crystal ball is cryogenics. Will scientists one day be able to freeze a human being and bring them back to life fully functional and live a normal life? We are on the path as scientists are already freezing human eggs but those are cells and not a human being.

For more pharma insights check out the video below!