The Niche

Investors ♥ stem cells

Enthusiasm was more apparent than scepticism at the 4th Annual Stem Cell Summit, organized by investor, analyst and stem cell fan Robin Young, head of RRY Publications. This conference brought together a motley collection of companies, both well-known and obscure, and each was given ten minutes to tout themselves to potential investors and partners. Business models included cell banking, services, therapies, medical devices and tools; even the smallest companies were often relying on multiple business models.

Several attendees were also planning on going to the Stem Cell Partnering Series, a separate conference put on in cooperation with the International Society for Stem Cell Research, which is going on at the University of California, San Diego on 26 and 27 February. I unfortunately won’t be able to attend that one, but you can get more information here.

In New York on Tuesday, Young himself acknowledged the dizzying diversity of companies represented, speculating whether the term ‘stem cell’ had become an inadequate term for business models applying to many things. Companies at the conference were funded by their own sales revenues, private investors, government grants, venture capital and big pharma partners. Several of the more established companies were recently discussed in an upbeat article in Crain’s Cleveland Business.

Perhaps my favourite talk came from Maryland-based Osiris, which has recently reported a string of successes including closing a $1.4 billion deal with Genzyme, completing enrolment of a Phase III trial and receiving encouraging results from a small but placebo-controlled clinical trial in heart attack patients. From his stance on top of the world, CEO Randy Mills could tell everyone just how hard the business was. Companies have to talk roses, especially when business looks bleak, and Mills was ready to take an opportunity to say that getting Osiris to its current point took 17 years and half-a-billion dollars.

Even the smallest stem cell companies tend to have several strings to each bow. They pursue cell banking, tools, reagents and therapies simultaneously. That makes classification difficult. It’s a little easier if one asks whether a company is selling cells, services or devices. And if the answer is cells, the next question is whether the cells are to be used as therapies or to test therapies.

Techniques to manufacture, characterize and scale-up cells were a common theme. Robert Preti, the scientific head of Progenitor Cell Therapy, among others, outlined the hazards his clients regularly fail to anticipate: product characterization, product characterization, product characterization and, oh yeah, reimbursement and delivery too.

One surprise was the number of product launches and clinical trials, sometimes even controlled clinical trials, that companies actually assigned dates to.

Some highlights, in no particular order:

Histogen is topically applying secretions of embryonic fibroblasts for hair regrowth. They make four sections on the top of each subject’s head and then compare the results of different treatments. One nice thing about skin and hair; you can have patients serve as their own control over time.

California Stem Cells, represented by scientific advisory board member and University of California, Irvine professor Hans Keirstead, plans to have an IND filed for spinal muscular atrophy by the end of the year. Babies die of this disease before they are a year old, according to Keirstead. He didn’t need to say explicitly that desperate families make for high patient enrolment or that certain death and lack of alternative treatments ease regulatory barriers.

Stemina is analyzing the metabolites secreted by embryonic stem cells and other types of cells under a variety of conditions. They’ve detected differences between neural stem cells, neural cancer stem cells and other brain tumour cells. Next steps: tying this to pathways and creating sellable products.

MaxCyte says its product can stuff suspensions of stem cells full of whatever molecule you want: RNA, protein, small molecules and its machine processes over a hundred million cells a minute with 90% viability.

Cytori, though it didn’t say so, is focusing on its Cellution machine more than cells partly because of a patent dispute. The idea is to let surgeons sort a patient’s cells by the bedside and then infuse the cells into the patient. It’s enrolling patients in a trial for breast reconstruction and has a trial underway for radiation-induced injury.

Arteriocyte and Aastrom are already in clinical trials. Gamida, Athersys, Aastrom and others promise techniques to expand cell populations. Gamida is pursuing expansion of a holy grail: cord blood. Stemcyte, the big umbilical cord banking company, is looking into trials in spinal cord injury and stroke.

Stematix wants to work with governments to send medical tourists to get procedures in regulated countries outside the US, focusing on Mexico, Singapore and Greece.

Spanish company Cellerix is using mesenchymal stem cells collected and expanded from a patient’s fat to treat complex perianal fistula, a condition in which the contents of the large intestine exit the body through an opening besides the anus. The company is evaluating its product in several clinical trials and has orphan drug designation in Europe. Roche and Novartis have signed on as investors, and its last funding round was oversubscribed, says CEO Eduardo Bravo. Even so, he noted that his and the other companies won’t have an easy ride to financial stability.

“You will know the results,” he joked, “because either we will be here [at next year’s summit] or we won’t be here.”

There was even one unscheduled company claiming to have found an abundant yet secret source of embryonic-like stem cells. The cells don’t have any of the expected cell surface markers, but they do make Oct4, said representatives of Parcell Laboratories, which, according to its website, has licensed ELA cells from Brigham and Women’s Hospital. Details on the cells’ behaviour are too vague to report just now.

At this summit last year, PrimeGen Biotech captured headlines and imaginations by announcing it had reprogrammed cells to pluripotency without using viruses and would soon be publishing results in a prestigious journal (see my blog entry and Nature story). Since then, the company does not appear to have published that paper (I searched PubMed and its website), and PrimeGen wasn’t back at the summit this year.

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