FDA Approval of 2 “Gene Patch 2.0” Drugs Offers Hope to Those at Risk for Hereditary Illnesses Including Hemophilia & Cancer, According to Author Stephen Shrewsbury, M.D.

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The FDA approved two gene patches that successfully override defective genes to create vital missing muscle proteins for those with two lethal muscle diseases. The successful use of this drug "technology," which works like a software patch, has positive implications for those with hereditary illnesses such as heart disease, cancer, diabetes and hemophilia.

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New gene patch technology may herald medical revolution.

"“These medicines act the same way software patches work.They override faulty pieces of genetic code. If given early they may actually prevent a disease before symptoms appear.”

The first shot was fired in what eventually may be a medical revolution with the recent FDA approval of Sarepta’s EXONDYS 51, a gene patch, (also known as a therapeutic splice switching oligomer). It successfully overrides the defective gene to create a vital missing muscle protein for those with Duchenne Muscular Dystrophy (DMD). That has now been followed by the pre-Christmas Eve approval of Ionis/Biogen’s SPINRAZA for another lethal neuromuscular disease, Spinal Muscular Atrophy (SMA).

“These medicines act the same way software patches work,” says Dr. Stephen B. Shrewsbury, author of Defy Your DNA: How the New Gene Patch Personalized Medicines Will Help You Overcome Your Greatest Health Challenges (10 Finger Press/2013). “They override faulty pieces of genetic code. If given early they may actually prevent a disease before symptoms appear.”

At its worst, SMA leads to weakness within weeks of birth, then paralysis and death before age 2. There are other types that are milder and may even not appear until adulthood. Overall SMA affects 1 in 10,000 newborns. SPINRAZA works by restoring levels of the missing SMN protein.

DMD is a lethal disease that causes muscle weakness in boys. It usually leads to the inability to walk by age 12, requires breathing support in the late teens and ends in death from heart failure in the mid 20s. DMD affects roughly 18,000 boys in the United States with over 500 boys born each year with the faulty gene.

FDA approval of both EXONDYS 51 and SPINRAZA signal that this next generation gene patch technology has passed a major test. Whereas the first gene patches worked by blocking a pathway (like a traditional drug, albeit a genetic message rather than chemical one), these drugs trick the cell to replace missing proteins.

This new drug type has the potential to stop everything from inheritable forms of heart disease to cancer. They allow the body to manufacture proteins necessary for certain vital tasks that aren’t happening due to the faulty message coming from a mutant gene.

There has already been significant progress—including clinical trials at a variety of stages—on gene patches against diabetes, liver disease, multiple sclerosis, and for lipid disorders that lead to premature heart disease, as well as a number of other cardiac and rare but lethal disorders. In the next five years, many of these therapies could gain FDA approval.

“It is really encouraging to see the progress made over the last few years by gene patch development companies,” says Shrewsbury. “With the recent advent of inexpensive genetic testing from organizations like DDC and 23andMe, in the future, some of these formerly inevitable hereditary diseases will be diagnosed by a genetic test before they cause trouble – and potentially, that trouble can be then be prevented.”

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Dr. Stephen B. Shrewsbury is a physician and has been Chief Medical Officer to several biotech firms. He is the author of Defy Your DNA: How the New Gene Patch Personalized Medicines Will Help You Overcome Your Greatest Health Challenges published by 10 Finger Press. He recently helped organize the annual Rare Diseases and Orphan Drugs conference in Chicago.

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For more information about gene patches or this and other story angles, or to interview Dr. Shrewsbury, please contact Mahesh Grossman at (831) 428-2846 or AuthorsTeam(at)gmail.com

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