Friday, July 5, 2019
Sernova touts first-in-human results from insulin-secreting cell implant for diabetes
In the first patient to receive its pancreatic cell implant for type 1 diabetes, Sernova’s insulin-secreting Cell Pouch showed no adverse events related to the therapy, and was able to help maintain blood sugar levels over at least two weeks.
In interim results from the phase 1/2 study, the transplant of purified islets demonstrated the production of insulin as well as stimulated C-peptide, a biomarker of the pancreas’ effectiveness in responding to rising glucose levels after eating.
Additionally, the regenerative Cell Pouch was found to integrate well with the blood and lymph vessels of the patient—who saw a weight loss of about 14 pounds (6.35 kg), or about 12% of total body weight.
“The first of two doses of islets transplanted into the Cell Pouch is showing safety and early indicators of efficacy. Importantly, demonstration of glucose-stimulated C-peptide and insulin present in the bloodstream is definitive proof of islet survival and function in the Cell Pouch,” said principal investigator Piotr Witkowski, director of the pancreatic and islet transplant program at the University of Chicago.
Using a continuous glucose monitoring system, the patient saw an 87.5% reduction in hypoglycemic events compared to baseline over a two-week period, according to Sernova. In addition, stimulated blood levels of C-peptide and insulin were observed 90 days after the transplant, in a mixed meal tolerance test.
“These results are an important first step towards a paradigm shift in the treatment of this debilitating disease. Our team is looking forward to reporting longer-term results in enrolled patients as the trial progresses,” Witkowski said. The data were presented at the World Congress of the International Pancreas and Islet Transplantation Association in Lyon, France.
Newly enrolled patients with severe hypoglycemia unawareness—where sharp drops in blood sugar fail to trigger the release of epinephrine and common symptoms—will be implanted with cell pouches and stabilized on immunosuppression following the development of vascular tissue and a dose of purified islets.
After six months of monitoring and an assessment of the transplant’s success, patients may receive a second dose of islet cells and will be followed for another year.
Saliva-based monitor Glucose Biosensor Systems re-files for a $23 million US IPO
Glucose Biosensor Systems (Greater China) Holdings, which is developing a saliva-based glucose monitoring system for diabetes in China, filed on Friday with the SEC to raise up to $23 million in an initial public offering.
Glucose Biosensor Systems originally filed to list on the Nasdaq in October 2018 under Regulation A+ in a $9 million to $25 million best-efforts offering with Cuttone & Co serving as placement agent; it withdrew its Reg A+ filing in March 2019.
The New York, NY-based company was founded in 2016 and booked $0.7 million in revenue for the 12 months ended March 31, 2019. It plans to list on the NYSE American under the symbol GBSG. Glucose Biosensor Systems filed confidentially on May 13, 2019. Aegis Capital Corp. is the sole bookrunner on the deal. No pricing terms were disclosed.
Karyopharm up as analysts boost targets on FDA approval
Karyopharm Therapeutics (NASDAQ:KPTI) is up 3% on its return to regular-session trading after it spiked 36% in Wednesday’s shortened session amid an FDA approval for its selinexor, for treatment of penta-refractory multiple myeloma.
Shares today hit their highest point since mid-November, while analysts responded by raising price targets.
Baird boosted its target to $25 from $15 — implying 173% upside — and modeled $150M peak annual sales for the later-line approval. It says accelerated approval de-risks the market in second-line relapsed multiple myeloma, the more meaningful market.
Meanwhile RBC raised its target to $22 from $16, pointing to the lack of the most serious caution on the label: “The label includes no boxed warning, a win, and while it describes in detail the many AEs (adverse events) associated with Xpovio and the need for monitoring, it provides clear dose- adjustment schemes and guidelines for AE management.”
Drug stocks slip after Trump talks up `favored nations’ plan
Pharmaceutical stocks dip after President Trump says his administration is working on a system that would allow the U.S. to buy drugs based on the lowest prices paid by other countries.
“We’re working on right now a favored-nations clause,” he told reporters at the White House.
Common cold virus strain may revolutionize bladder cancer treatment
A strain of the common cold virus has been found to potentially target, infect and destroy cancer cells in patients with bladder cancer, a new study in the medical journal Clinical Cancer Research reports. No trace of the cancer was found in one patient following treatment with the virus.
Researchers from the University of Surrey and Royal Surrey County Hospital investigated the safety and tolerability of exposure to the oncolytic (‘cancer-killing’) virus coxsackievirus (CVA21), a naturally occurring strain of the common cold, in fifteen patients with non-muscle invasive bladder cancer(NMIBC). NMIBC is found in the tissue of the inner surface of the bladder and is the tenth most common cancer in the UK with approximately 10,000 people each year diagnosed with the illness.
Current treatments for this cancer are problematic. Transurethral resection, an invasive procedure that removes all visible lesions, has a high tumour recurrence rate ranging from 50 per cent to 70 per cent as well as a high tumour progression rate between 10 per cent and 20 per cent over a period of two to five years. Another common course of treatment, immunotherapy with Bacille Calmette-Guerin, a live bacterium used to treat bladder cancer, has been found to have serious side effects in one third of NMIBC patients while one third do not respond to the treatment at all.
During this pioneering study fifteen NMIBC patients, one week prior to pre scheduled surgery to remove their tumours, received CVA21 via a catheter in the bladder. Examination of tissue samples post-surgery discovered that the virus was highly selective, targeting only cancerous cells in the organ and leaving all other cells intact. The virus was found to have infected cancerous cells and replicated itself causing the cells to rupture and die. Urine samples taken from patients on alternate days detected ‘shedding’ from the virus indicating that once virally infected cancer cells had died, the newly replicated virus continued to attack more cancerous cells in the organ.
Typically tumours in the bladder do not have immune cells, preventing a patient’s own immune system from eliminating the cancer as it grows. Evidence suggests treatment with CVA21 inflames the tumour causing immune cells to rush into the cancer environment, targeting and killing the cancer cells. These tumours devoid of immune cells are known as ‘cold’ areas immunologically; however, treatment with the virus causes inflammation and immune cell stimulation to create ‘immunological ‘heat’. ‘Hot’ tumours in this way are more likely to be rejected by the immune system.
Following treatment with the virus cell death was identified in the majority of the patients’ tumours. In one patient no trace of the cancer was found during surgery.
Hardev Pandha, Principal Investigator of the study and Professor of Medical Oncology at the University of Surrey, said: “Non-muscle invasive bladder cancer is a highly prevalent illness that requires an intrusive and often lengthy treatment plan. Current treatment is ineffective and toxic in a proportion of patients and there is an urgent need for new therapies.
“Coxsackievirus could help revolutionise treatment for this type of cancer. Reduction of tumour burden and increased cancer cell death was observed in all patients and removed all trace of the disease in one patient following just one week of treatment, showing its potential effectiveness. Notably, no significant side effects were observed in any patient.”
Dr. Nicola Annels, Research Fellow at the University of Surrey, said: “Traditionally viruses have been associated with illness however in the right situation they can improve our overall health and wellbeing by destroying cancerous cells. Oncolytic viruses such as the coxsackievirus could transform the way we treat cancer and could signal a move away from more established treatments such as chemotherapy.”
Journal information: Clinical Cancer Research
Successes and challenges of digital medical devices
Eric Topol, a cardiologist and digital medical researcher, and also director of the Scripps Research Translational Institute, has published a Focus piece in the journal Science Translational Medicine discussing the short history of digital medical devices, covering both success stories and current challenges.
The type of digital medical devices Topol addresses are the kinds people can use without assistance from medical personnel. They include devices that can take blood pressure measurements or count steps. He suggests the starting point for such devices was the introduction of the smartphone in 2007, a mere 12 years ago. He notes that over that short time span, engineers have created ever more powerful devices, which he finds both encouraging and disconcerting.
Allowing people to monitor aspects of their own health in real time in the real world is, of course, a good thing. But he notes that many such devices are not properly vetted through clinical trials. He also notes that one of the most exciting areas of current research surrounds the development of imaging applications—devices that can attach to a smartphone, for example, that can be used to perform ultrasound examinations.
Topol notes that biosensors have proliferated in recent years, partly due to work by companies that design them and partly due to the willingness of the USFDA to readily approve such devices. He also notes that the FDA’s Digital Health Action Plan plays a role, ensuring that all Americans have access to the best digital health care products available. Part of that effort has been the establishment of site-less digital clinical trials in which participants test a device in the real world rather than in a controlled environment. He reports that such testing has not always met with great success—in one trial, for example, a group of participants tested a smartphone app that showed an improvement in self-reported adherence to prescribed hypertension medications. But it did not lead to lowered blood pressure.
Topol points out that as digital medical devices evolve, it is likely that the time will come when they will need to consider more than just one physical feature—they will have to take a more full-bodied approach. And that will most certainly involve the development of machine-learning applications, which entail ethical issues. He notes also that as such devices improve, researchers must deal with the massive amounts of data they will generate, and how to keep it all private.
More information: Eric J. Topol. A decade of digital medicine innovation, Science Translational Medicine (2019). DOI: 10.1126/scitranslmed.aaw7610
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