Manufacturing life

FDA approves Israel-developed stem cell ‘factory’ for research

Pluristem is moving into mass-production of its placenta-based muscle repair system

Pluristem's first bioreactor for the enrichment of mesenchymal adherent stromal cells (ASCs) from cord blood (Photo credit: Courtesy)
Pluristem's first bioreactor for the enrichment of mesenchymal adherent stromal cells (ASCs) from cord blood (Photo credit: Courtesy)

An Israeli-invented process to speed up injury repairs got a big boost last week when the FDA approved the use of mass-produced manufactured therapeutic stem cells produced by Pluristem Therapeutics for use in studies on treating injuries. The Placental Expanded (PLX) stem cell products, derived from human placenta, will be produced in a large facility in northern Israel. With the manufacturing process approved, the next step, the company hopes, is widespread FDA approval for use of the cells in treating injuries and muscle diseases.

It was actually the second big regulatory “win” for Haifa-based Pluristem in recent weeks. In February, Germany’s Paul Ehrlich Institute (PEI), which regulates medical treatment in the country, approved in January the company’s manufacturing process for production of mesenchymal-like adherent stromal cells (ASCs) derived from full term human placentas. The approval came just after the company announced positive results in a study where patients who suffered from a muscular injury were successfully treated with Pluristem cells.

Placenta is an organ in mammals which links the fetus to the mother for transfer of oxygen and nutrients and gets ejected when a baby is born. While useless to the mother and baby, placenta, which is rich in hormones and proteins, is used in number of ways commercially, such as in the manufacture of cosmetic products and shampoo.

Over the past several years, animal placenta – especially horse placenta – has been used by alternative physicians to repair sports injuries. Placenta, according to researchers, enhances cell repair, and speeds up the healing process significantly. Using human placenta – because it is more compatible with the human genome – Pluristem processes and enhances the cells, using proprietary methods. The result, said the company, is “a drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases.

“The PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an ‘off-the-shelf’ product that requires no tissue matching prior to administration,” according to Pluristem. Injected into the area of an injury, the company said, the cells work their “magic,” greatly enhancing the repair process.

So far, Pluristem’s technology is being used on a limited basis. The FDA has approved the technology in treating several conditions, including Buerger’s disease and aplastic anemia, for which there are no other treatments.

The company has also made headlines. In 2012, for example, the technology was used to extend the life of a seven year old girl in Israel suffering from bone marrow failure. Although it is not approved in Israel for widespread use, the Health Ministry gave doctors at Hadassah Hospital in Jerusalem special permission to try Pluristem’s therapy after a bone marrow transplant failed. Doctors reported that her red blood cell count – an indication that her marrow was performing properly – had increased significantly after the girl was injected with PLX cells. Unfortunately, the girl died a few months later.

The death of that patient pulled down the value of Pluristem shares, just as the original news on the treatment’s efficacy in the case had pushed share values up. The company went through a similar gyration in January. Pluristem’s shares are traded on the NASDAQ, the Tel Aviv Stock Exchange, and the Frankfurt Stock Exchange, and shares on all three fell on January 20 – over fears that the results of phase I/II clinical trial of the Pluristem cells’ effectiveness in treating muscle injuries would not be positive.

Shares on the NASDAQ were down 19% on the 20th over those fears – but were back up by 20% the next day, after the company reported favorable results in that test, with the cells “well tolerated” by patients who were given the experimental treatment for their injuries. The test results, said Pluristem chief executive Zami Aberman, was “a very important study not only for Pluristem but for the cell therapy industry in general. Based on the results, we intend to move forward with implementing our strategy towards using PLX cells in orthopedic indications and muscle trauma.”

The test involved patients who were suffering from a gluteal muscle injury resulting from hip replacement surgery, mostly caused by osteoporosis. Over 300,000 people undergo the surgery in the US along each year, and according to industry sources, the global hip replacement market is worth over $7 billion, with many of the patients requiring treatment for muscle industry. Of course, Pluristem’s therapies would be applicable in many other treatment scenarios, the company said.

Key to implementing that vision will be ramping up production of the PLX cells for widespread use, as expected regulatory approvals for more procedures comes in, said Aberman. “We believe we have the largest, scalable, most efficient, most consistent and controlled process for manufacturing cell therapies,” he said. “We have established our leadership position in the industry by focusing on our 3D commercial scale cell manufacturing processes.

“To be a successful company in the industry, we believe it is imperative to possess and control the manufacturing processes we have developed at Pluristem,” said Aberman. “We believe this FDA approval, combined with the approval given by the Paul-Ehrlich-Institute (PEI) of Germany, is an indication that these regulatory bodies see our proprietary 3D manufacturing process as a valid and sustainable commercial scale solution for potential cell therapies.”

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