From the recently approved second-gene therapy to treat cancer to the discovery of Amish gene mutation that claimed to help prevent from acquiring Type 2 Diabetes and may have increased our longevity, there’s no doubt that gene therapy is making some noise in the health industry. For those who are confused as to what gene therapy does and how it affects our body, it is the process of altering human cells and tissues in order to procreate a situation in order to create or produce the desired effect. Since gene therapies and breakthroughs proved to be more than useful to the public nowadays, the FDA had issued a new guideline for gene therapy.

FDA Had Issued a New Guideline last Thursday For Gene Therapy Studies and It Also Aims to Crack Down Rogue Clinics

The FDA wants to speed up the review of gene therapy studies as well as its implementation and practice of medical clinics in order to discover more ways to treat illnesses like cancer and diabetes. However, along with the review selection process for approval, the FDA also warned those rogue clinics not licensed offering dangerous or untested and unapproved treatments to their patients. They warned the public that going on to these rogue clinics may actually jeopardize the patient’s health and well-being rather than healing their diseases.

“These concepts are no longer the stuff of science fiction,” said Scott Gottlieb, the commissioner of the F.D.A., “but rather, real-life science where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness.”

For those legitimate clinics conducting gene therapies experiments that have the potential to treat untreatable diseases, as well as those experiments which yield medical breakthrough can now apply to the FDA and expedited their review. The test would require clinical trials with a much faster process as amended by Congress under the 2016 21st Century Cures Act. Once approved by the said agency, the clinics can now produce more products for market availability.

FDA Also Acknowledge the Fact that with the advancement of gene therapy, questionable centers had also emerged to exploit the technology.

The FDA warned the public, as well as those rogue clinics and questionable centers offering treatments for everything. Their offers range from sore knees up to heart disease with questionable and unapproved treatment methods and facilities. The FDA also emphasized that the products these rogue clinics sell use the patient’s own fat in order to produce an enzyme to “treat” their illnesses, as they claimed. The FDA will definitely look into these treatments and ban them from getting their products approved as well as canceling their professional license. The FDA is committed to track down these rogue clinics and close their business before they can jeopardize the public’s safety. However, for those legitimate clinics, this can be very good news for them.

“This is an incredibly significant development for the gene therapy, regenerative medicine sector,” said Michael J. Werner, a partner with Holland & Knight and co-founder of the Alliance for Regenerative Medicine.

Gene Therapy Manufacturers Will Immensely Benefit With this Hastened Approval Review Process

Mr. Warner is pleased to know about the FDA’s new movement, reiterating that their latest actions would benefit the manufacturers and this would create a social pathway to the market. These gene therapies would not only help the patients on healing from the illnesses they’re suffering. But that it can also help with their long-term maintenance and therapeutic treatment. So far, the FDA had only approved two drug products under the gene therapy category, namely:

• Kymriah, from Novartis
• Yescarta, made by Kite Pharma

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The Food and Drug Administration will speed up the approval process of gene therapies

Both gene therapies involve altering the patient’s own immune cells in order to fight leukemia or lymphoma. While the third product that the agency approved aimed to correct a gene defect, it enables some patients to inherit blindness or other eye diseases. The product was made by Spark Technologies. All of these products so far were approved by the FDA in only a span of a year. With the FDA speeding up the review process, Mr. Warner is positive that we’ll be able to have access to more gene therapies that could help improve our health. The only downside to these gene therapies as of now is that they’re still expensive to avail since they’re not available in the mass market at the moment.