While most drugs developed in the United States today consist of natural or synthetic compounds, living drugs are comprised of fully functional cells, selected and modified to target certain diseases. In this article we look at living drugs and their potential to treat a range of virulent diseases, including cancer.
One area in which living drugs demonstrate particular potential is in the field of immunotherapy, as a type of cancer treatment that involves stimulating a patient’s own immune system to fight disease.
The first living drug received FDA approval for use on cancer patients in 2017
A new cancer therapy approved by the FDA in 2017, CAR-T involves extracting a patient’s own white blood cells. The cells are genetically reprogrammed to recognize and destroy cancer cells before being reintroduced into the body.
CAR-T therapies are tailor-made for each patient, marking a shift from conventional cancer therapies such as chemotherapy or surgery. The “living drug” therapy effectively reprograms a patient’s own immune system to make it more effective at seeking out and destroying cancer cells.
Novartis is responsible for developing CAR-T, which costs around $475,000 per treatment. Medical practitioners have reported that the drug has an 83% success rate in curing acute lymphoblastic leukemia.
Dr. Scott Gottlieb of the FDA explained that this new approach to treating cancer is exciting, marking a new era in medical innovation and giving scientists the ability to reprogram a patient’s own body to effectively cure itself.
The therapy, marketed as Kymriah, has demonstrated effectiveness in treating acute lymphoblastic leukemia. Dr. Gottlieb points out that new technologies such as cell and gene therapies hold the potential to transform the way we treat patients today, creating an inflection point in our ability to treat—and potentially cure—a host of intractable diseases.
Acute lymphoblastic leukemia is widely regarded as one of the more treatable forms of cancer. Nevertheless, in some patients, traditional cancer therapies have proven ineffective. Physicians often use Kymriah in those cases where other treatments have not shown to be effective.
Dr. Stephan Grupp of Children’s Hospital of Philadelphia was the first physician at the facility to use Kymriah on a pediatric patient. Since receiving Kymriah, the patient, who had been near death, has now been cancer-free for over five years.
In a survey of 63 patients treated with Kymriah, 83% were found to be in complete remission within three months of treatment. Nevertheless, this new therapy does come with a health warning. It can lead to potentially fatal cytokine release syndrome due to the rapid proliferation of CAR-T cells in the body. The side effect can be controlled with other drugs.
Dr. David Maloney of the Fred Hutchinson Cancer Research Center hailed the FDA’s decision to license this new living drug as a major milestone in terms of finding a cure for cancer. He indicated that this breakthrough could potentially pave the way for the development of many new immunotherapy-based treatments targeted to treat a variety of cancers.
Can living drugs cure genetic diseases?
A pill containing millions of live bacteria could prove to be an effective tool in fighting disease. Many hereditary genetic disorders interfere with the processes involved in creating specific proteins, substances that are vital to the body’s ability to grow, develop, and function properly.
One example is phenylketonuria (PKU), a disease caused by the lack of an enzyme necessary for breaking down protein, causing toxic chemicals to accumulate in the blood and leading to permanent brain damage. In many countries, the PKU test forms part of a newborn’s “heel prick” screening, in which blood is collected and analyzed.
PKU was traditionally treated with a special low-protein diet that patients would need to adhere to for the rest of their lives. While this could prove somewhat challenging, researchers from Synlogic, a Massachusetts biotech company, may have found a solution.
In clinical trials, the team introduced therapeutic genes to the guts of PKU patients. Researchers hoped to stimulate the patients’ bodies to begin producing the missing enzyme, effectively correcting the digestive disparity caused by PKU and retraining the patients’ bodies to start breaking down protein.
Until relatively recently, scientific understanding about the role of the trillions of bacteria that reside in the human gut has been fairly limited, but recent studies have revealed millions of different genes present in bacterial genomes, outnumbering our own genes by around 150 to 1. Scientists hope to apply this newfound knowledge to the development of living drugs targeting PKU and other debilitating genetic diseases.
Are living drugs safe?
While living drugs have proven effective at curing disease, particularly acute lymphoblastic leukemia, scientists regard the practice as an experimental treatment and therefore reserve it for those cases where traditional therapies have proven ineffective.
need much more research into living drugs to gauge their safety and effectiveness.
However, the results revealed so far are extremely encouraging, with many leading scientists believing that the practice could be revolutionary, effectively reprogramming the human body to heal itself on a cellular level—from the inside out.