DC Dispatch: Taxpayers Supporting Cancer Treatment Won’t See Price Relief
Kite Pharma is preparing to launch its CAR-T immunotherapy cancer treatment, which it developed with the help of the U.S. government. CAR-T was initially developed by a team of researchers at the National Cancer Institute, and Kite pays millions a year to the government to support its research. As a result, Dr. Arie Belldegrun, Co-founder of Kite, says stock is worth $170 million—and Kite investors are in the middle of a gold rush. While some may say the government wouldn’t be able to develop this possibly life-saving drug alone, critics believe this method forces taxpayers to pay for drugs in development, as well as when they buy it.
This single treatment therapy would cost patients $200,000, and while the U.S. government has the power to force price-concessions from their private-sector partners, they’ve stated that they will not do so with Kite. The U.S. government doesn’t usually exercise this power because it would drive away much needed funding for speedy drug development. But the counter argument says that private drugmakers rely too heavily on government and academic labs to pull out of partnerships.
The National Institutes of Health currently has about 400 cooperative research agreements with companies such as Kite, and licenses hundreds of patented inventions for private-sector development. While Kite and other companies will generate millions in revenue, the government sees very little profit. Dr. Belldegrun and others believe this is the way things should work, as new and much needed drug discoveries would never be made available otherwise.
Medical Devices: Building Kidneys for Medical Testing
Binghamton University researchers Gretchen Mahler and Courtney Sakolish, PhD, developed a reusable, multi-layered, microfluidic model kidney that has porous growth substrate, physiological fluid flow, and passive filtration of the capillaries where waste would be filtered from blood in a human kidney. The artificial kidney is intended to be a more effective replacement for animal pre-clinical drug testing.
This model is the first to simulate glomerular filtration and is proving to more accurately replicate human kidney responses than traditional testing methods. “We found that the more complex, dynamic culturing conditions (like those used in this project) are necessary to accurately predict renal drug toxicity in human systems,” says Sakolish. “When we compared physiological renal function and drug toxicity in traditional static culturing against our new model, we found significant differences in the ways that cells behaved. In our platform, cells looked and acted like those that you would find in the body, showing more sensitive responses.”
TeleMed Text: Blood Bacteria Now Identified in Hours
Engineers from University of California San Diego recently developed a desktop diagnostic tool that detects a wide range of harmful bacteria in just hours, rather than days. It detects small traces of bacteria in a massive amount of blood cells, and then melts the bacterial DNA into about 20,000 extremely small (20 picoliters) reactions. The DNA comes apart as it melts and is imaged and analyzed by a machine that has proven 99% accuracy in identifying DNA from bacteria—specifically Listeria monocytogenes, a food-borne bacterium that causes about 260 deaths per year in the United States, and Streptococcus pneumoniae, which causes everything from sinus infections, to pneumonia, to meningitis. The error rate for traditional methods can be up to 22.6%.
“Analyzing this many reactions at the same time at this small a scale had never been attempted before,” explains Stephanie Fraley, a professor of bioengineering at UC San Diego and the paper’s lead author. “Most molecular tests look at DNA on a much larger scale and look for just one type of bacteria at a time. We analyze all the bacteria in a single sample.”
Discoveries/Innovations: Septic Shock Treatment is in the Works
Septic shock, a condition during which the body damages its own tissues and organs by overreacting to infection, can be fatal when in the septic shock phase. In this phase, for which there is no known treatment, blood pressure drops drastically which can cause organs to fail. Yasuhiro Ogawa, Yoko Irukayama-Tomobe, and Masashi Yanagisawa of the International Institute for Integrative Sleep Medicine, have found that the neuropeptide, orexin, can improve a mouse’s chances of living through septic shock when it is subcutaneously injected.
While it was known that orexin can alter heart rate and body temperature, it wasn’t used clinically because the delivery of orexin is hampered by the blood-brain barrier (BBB). The research team discovered orexin could increase blood rate and temperature, fighting off septic shock, because the BBB becomes leaky enough to allow it to reach the brain. The next step is to test the method on primates and see exactly how orexin regulates the immune system.
FDA Update
Drug Approvals
The FDA approved Celgene’s Revlimid (lenalidomide) for use as maintenance therapy for patients with multiple myeloma following stem cell transplant. Revlimid demonstrated a 15-month and 18-month progression-free survival advantage and an overall survival of 111 and 106 months versus 84 and 88 months for patients receiving placebo treatment. Patients receive the oral drug in 10 mg doses once a day for 28-day cycles.
The FDA has approved Valeant Pharmaceuticals’ Siliq (brodalumab) to treat adults with moderate-to-severe plaque psoriasis. The injectable is for patients who have failed to respond to systemic therapy or phototherapy.
Accelerated Approval
Opdivo, a Bristol-Myers Squibb drug for previously treated locally advanced or metastatic urothelial carcinoma, is now approved by the FDA for subcutaneous injection. The drug received accelerated approval due to a bladder cancer objective response rate of 19.6%.
Updated Label
Eli Lilly’s Trulicity received an updated label for use in combination with basal insulin for adults with type 2 diabetes. The .75 mg or 1.5 mg doses can help adults control their blood sugar levels more easily with the once-weekly injectable in a pen design.
Medical Device Approval
The FDA has permitted marketing of Seeker System kit—an NIH-funded project—that can detect four rare lysosomal storage disorders in newborns by checking blood 24-48 hours after birth. Seeker System measures the activity level of proteins required for healthy lysosomal storage to immediately treat the rare disorders to avoid organ damage, neurological disability, or death.
