CORAL GABLES, FL / ACCESSWIRE / March 26, 2015 / Two months ago, Vital Therapies (VTL) announced full enrollment for a pivotal phase 3 trial of its ELAD system. Now that it's fully enrolled, it's only a matter of time before results are announced. What is ELAD? It's essentially an external liver support system for patients with chronic liver disease, or CLD, of which there are over 100,000 cases each year in the US according to the CDC, and which causes over 1 million deaths each year in China due to endemic viral diseases like Hepatitis C. CLD is actually the fifth leading cause of death in the People's Republic.
Until Vital began testing ELAD, the idea of an external liver was considered impossible. The liver is responsible for an estimated 500 biological functions, so it is impossible to mimic liver function without using actual liver cells. It's not like kidney function, which mainly filters the blood and can be mimicked by dialysis to a certain extent outside the body. The problem is, actual liver cells, or hepatocells, cannot survive outside the body. They begin to die almost immediately.
However, there is one kind of liver cell that can indeed survive outside the body indefinitely: hepatoblastoma cells, or to use the common language, liver cancer. And that's what the ELAD system is based on.
Liver cancer inside the body is deadly not because the cells themselves do anything detrimental, but because they don't stop growing, spread to other parts of the body and crowd out other organs. But outside the body and no threat to the patient, liver cancer cells can perform very similar functions to normal liver cells. In a sentence, ELAD grows hepatoblastoma cells, packs them by the pound into cartridges called the ELAD system, and uses them as an external liver to patients with CLD and those awaiting transplant.
Vital is trying to be discreet about the use of liver cancer cells in its lead product, only mentioning the word "hepatoblatoma" once in its official description of the system, calling them instead "human-derived C3A liver cells." Nevertheless, the function performed by these cells is similar to normal hepatocytes, though not exactly the same. The main differences are that C3A cells do not process ammonia as effectively, and C3A cells produce a large amount of a waste protein called AFP. These compounds, however, can by removed by dialysis.
With Phase 3 results due in April and overall survival at 91 days the primary endpoint, the stock has been steadily rising 41% since the beginning of February. With results only weeks away, the market may be smelling something. This specific trial is treating patients with alcohol induced liver failure, but if ELAD works in this indication, there is little reason it wouldn't function in the same way for patients with liver failure due to other causes like hepatitis C.
Speaking of Hepatitis C and External Blood Filters, Meet the HemoPurifier
As original as using liver cancer cells to function as an external liver is, there's another company working on an external blood filter of perhaps equal originality: using insecticides to filter viruses directly out of blood.
The idea of filtering out viruses from the blood is actually not science fiction. It's not quite standard of care for viral infections yet either, but it is definitely no longer strictly relegated to the realm of childhood fantasies. Aethlon Medical's (AEMD) Hemopurifier is the device that may yet break the viral-filtration barrier. In fact, the first stage of a two-stage clinical trial process for FDA approval is well under way.
While the process of filtering a virus out of the blood may seem complicated to the point of being magical, the concept behind the Hemopurifier is actually quite simple. Filtering viruses out can't be done simply by passing blood through tiny holes, because viruses are so small that doing such would filter everything out of the blood and leave the patient with nothing but salt water. And if you try to put some of the filtered material back in, you'll just end up putting the viruses back in, too.
But say you wanted to filter tiny iron filings out of a pile of marbles. All you'd have to do is use a magnet and you're done. The Hemopurifier uses what can be called a virus magnet made out of a material called galanthusnivalis agglutinin, or GNA. GNA is a naturally occurring molecule that comes from a plant called snowdrop. GNA is in a class of molecules called lectins, which are proteins that stick to sugars. Lectins are most widely employed in the food industry because they are mild pest controlling insecticides mostly nontoxic to humans. Genes that encode lectins have been crossed into mass-produced grains in order to ward off insects, and GNA specifically has been tested with wheat as an insecticide as well.
The special quality of GNA, however, is not its insecticidal potential for the food industry, but that it specifically sticks to sugars that happen to be on the surfaces of most viruses that circulate in the blood stream. These virus sugars are called glycoproteins, which are proteins with carbohydrate (sugar) tentacles that viruses use to attach themselves to cells and inject their genetic code into them. Glycoproteins are the hooks, the grapplers of viruses that let them infect your cells, and GNA grabs on to them like glue, allowing the rest of the blood to pass through.
The Hemopurifier basically consists of a tight cylindrical packing of GNA with plasma filters. It is attached as an added filtration unit to an existing dialysis machine, so no additional hardware is required.
Does it work?
While a large scale clinical trial is yet to be done with the Hemopurifier (Aethlon is currently enrolling patients 2 and 3 of 10 in a feasibility study) there is hard evidence that the Hemopurifier pulls viruses out of the blood with little to no side effects. In a 12-patient study of HCV patients conducted in India, no adverse events were reported, and 10 patients achieved a sustained virologic response, the technical term for a cure with HCV. 7 of those 10 acheived a rapid virologic reponse, which means a cure within 30 days of treatment. But the concreteness of the results is what they found in the Hemopurifier after each treatment on average, which was 300 billion copies of HCV.
There is even evidence that the filter works for Ebola as well. In late October 2014 when the last Ebola epidemic was at its peak in West Africa, Time Magazine reported that one patient from Sierra Leone arrived in Germany and progressed to multiple organ failure. He was treated with several experimental therapies including the Hemopurifier and was eventually cured. Doctors cannot say conclusively that the Hemopurifier cured him, but they can say conclusively that immediately before the filtration, his viral load was 400,000 copies per mL, and after it was only 1,000. It was this episode that prompted Time to name the Hemopurifier one of the top 25 inventions of 2014.
Regulatory Pathway
To get to where Aethlon is now was not easy. Convincing the FDA to allow even a feasibility study took 5 years of negotiations. Aethlon is now working with DaVita (DVA) to treat 10 patients with end stage renal disease who also happen to have HCV. Since they are on dialysis anyway, it's only a matter of plugging in the Hemopurifier to the machine they are already on and monitoring the virus. 3 patients have been treated out of 10, with results scheduled around October.
After that, since the Hemopurifier is not a new biologic drug, the regulatory pathway it must traverse from there to approval only requires one more pivotal clinical trial after the completion of the feasability study. There is no phase 3 like there is for Vital's ELAD.
Applications
The potential for the Hemopurifier does not end at HCV or even Ebola. Since almost all viruses are glued to GNA naturally, the device could theoretically work on any virus as long as it circulates in the blood stream, including incurable ones like herpes, and even HIV for which it has already been tested with encouraging results including a 55% reduction in viral load. For patients that have developed a tolerance to whatever antiviral therapy they have been using, it could be the only alternative once resistance takes hold. Viruses can develop resistance to a drug, but they cannot resist being glued to GNA in a filter.
Despite being public for 16 years already, Aethlon has been very frugal, burning through only $80M since inception in 1999. Vital, on the other hand has an accumulated deficit of over $140M. Much of that has to do with with extremely expensive nature of a pivotal phase 3 trial that Vital is currently conducting. ELAD, even though an external filter, is still a biologic and requires a phase 3. The Hemopurifier does not, since it is not a biologic.
Potential Low Cost
Hepatitis C has been a huge driver of the biotech sector with annual sales breaking through the $10B annual mark for Gilead's (GILD) Sovaldi last year and Abbvie's (ABBV) Viekira Pak expected to sell $3.5B this year. But Sovaldi is literally $1,000 a pill and Viekira is only 12% less than that. Judging by the low burn rate that Aethlon has had since inception 16 years ago, the cost of the Hemopurifier will likely be much lower.
The next milestone will be the completion of the feasibility study later this year, followed by a pivotal efficacy trial. And while mega $10B blockbusters like Sovaldi only treat HCV, the hemopurifier can theoretically treat any blood borne virus. Disruptive indeed.
CONTACT: Adam Heimann
BiotechStocks.com
[email protected]
SOURCE: Biotechstocks.com