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FDA Provides Guidance for Early Stage Trials of Gene and Cellular Therapy

Jul 15, 2015 1:00:47 PM / by Maria

Gene and cellular therapy is different from the drugs that have come before -- and so are the dangers. Guidance from the FDA is available to avoid pitfalls.

It's a brave new world over at the pharmacy. In the past, so-called "small molecule" oral drugs were just about all there was for pharmaceutical intervention. Later, injected large drugs, often protein-based, attracted unprecedented attention. Now, gene and cellular products are all the rage. Whether it is CAR T cells to attack cancer, stem cells for regenerative medicine, or genetic interventions that affect which genes are expressed, the newest generation of therapies look quite different from a bottle of pills, and they have attracted considerable interest all around the globe.

cellular therapy Apples and oranges: Genetic and cellular therapies aren't the same as more traditional drugs. Many of the challenges are different, too. Image credit:

Although gene and cellular therapy hold promise in addressing knotty medical problems, new challenges come with the new opportunities. A few patients had developed malignancies because new genes drove cancer, or new cells developed into tumors. Others have been victim of severe immune reactions to treatment. What's more, such products are rarely ingested; the route of administration may be more invasive -- and risky -- than a simple injection. Manufacturing these treatments can be a whole different ballgame, as well. Because of the limited availability and shelf life of many of these products, a snag in the manufacturing process could mean that the patient is unable to receive treatment.

What's more, the data collected during a clinical trial may require a different interpretation than data from a small molecule. If, for example, a gene or cellular therapy is detected at a certain concentration at a certain time, the implications might not be comparable to those from a traditional study. Lastly, these new therapies may remain in the body for an extended period, long after a traditional drug would have been eliminated. What does that mean for patients and their doctors?

That is why the FDA has come up with a finalized guidance document for researchers interested in pursuing gene and cellular therapy in early clinical trials, the trials that focus on safety and tolerability.[1] Among the recommendations, the FDA discusses choosing appropriate doses, selecting the right study population, and proper monitoring of patients. Product characteristics are deliberated on. The FDA even provides suggestions for meeting with regulatory authorities and submitting an Investigational New Drug application.

Like any new frontier, gene and cellular therapy has potential dangers lurking out of direct sight. Roadmaps like the one provided by the FDA will make the route to safe and effective treatments easier to find. HemaCare is a strong believer in "Quality in, quality out" and offers a number of cellular therapies that reflect our philosophy.





Topics: clinical trials, Stem Cell Therapy, Stem Cells, Basic Research


Written by Maria

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