Stem Cell 'Teething Problems' -- Pricing to Manufacturing, the European View

From Europe this morning comes a perspective on the "three obstacles" that are blocking cell therapy from the mainstream.

It is a subject, of course, that resonates in the United States and particularly in California, where the $3 billion state stem cell agency is running out of cash and needs a boost.

The article appeared on Labiotech, which bills itself as "leading digital media covering the European biotech industry." Written by Helen Albert, the piece actually lists more than a trio of "teething problems" for the stem cell industry. They are:

• "A reliable, consistent and scalable manufacturing system" 
• "Excessively high pricing"
• Safety, regulation and a knowledgeable workforce

Albert interviewed a number of industry executives in Europe. She also mentioned Orchard Therapeutics, which has a tie to California.  It has received an $8.5 million award from the state stem cell agency, formally known as the California Institute for Regenerative Medicine.

The United States is ahead of the game on regulation, according to the folks in Europe. Albert wrote, 

"The US regulators are currently in the lead when it comes to keeping up with the research and being open to changing their processes, according to (Alan O’Connell, partner at Irish life sciences VC Seroba). Although he says Europe is not too far behind. 'Certainly, the EMA (European Medicines Agency) is responding, but from my perspective, the FDA is taking the lead, at least currently.'"

The Albert piece said, 

"With the first CAR T-cell therapies (technically a combination of gene and cell therapy) to treat blood cancers gaining US and European approval last year and global investment of over €6.7B in cell therapy in 2018, the question on everyone’s lips is ‘is cell therapy about to hit the big time?’"

Labiotech's answer? "2019 certainly appears to be the start, if not the end, of cell therapy’s move to the big time."

California's "Huge Ecosystem" for Stem Cells: The View from Europe

Some folks in Europe are worried about stem cell research, particularly about organizations like California's $3 billion stem cell agency.

The alarm was sounded just yesterday in Horizon, which calls itself  "The EU Research and Innovation Magazine."

The article in question (the second most popular on its web site at midday today) was headlined:

"Europe is in danger of being out-innovated in regenerative medicine"

Ton Rabelink, Arno Massee Fotografie

The piece consisted of an interview with Ton Rabelink, professor of internal medicine and head of Leiden University in the Netherlands. He cited the California agency as a "huge ecosystem" for developing much needed stem cell therapies.
nephrology at

Rabelink said that the European Medicines Agency is wrestling with finding the "the right mechanisms to support the field."

‘It is very important that they do this because the regulatory landscape in the US and Japan has changed over the past two or three years to accommodate recent advances. For example, Japan has an early access programme for treatments that seem promising but are not yet proven to work. If they appear safe in say, 40 patients, then doctors can start applying them. The US has created the 21st Century Cures Act that allows for clinical trials for stem cell therapies and fast-track access to market for those that appear effective."

Rabelink said,

"The risk is that if we don’t organise locally here in Europe, we’ll end up having to buy these treatments from those countries. We’ve already seen this with genetically modified cells, so-called CAR cells, to attack tumours in leukaemia. The treatment works quite well but costs about €500,000 ($582,670) per patient.

"It's very interesting to look at what happens in the rest of the world. You really need ecosystems — academia, but also legislatures, manufacturing and, of course, finance. The US has huge ecosystems like CIRM, the California Institute for Regenerative Medicine, which was founded through taxpayers’ money following a referendum, and invests about $250 million per year in this space. (The agency actually was created by a ballot initiative, which is much different than a referendum.)

The Dutch researcher continued,

"The situation in Japan is even more remarkable. The early breakthroughs were made by a Japanese scientist so they consider regenerative medicine almost as their national invention. And, of course, Japan has an ageing population so the concept is very appealing. The government set up a planned economy around regenerative medicine and adapted its regulatory framework, putting national systems in place to oversee quality and safety and organising private-public collaborations, bringing together academic institutions and big pharma. Fujifilm, which was originally a photographic company, is devoting resources to stem cell research and using its film technology to make biomembranes.’