Living medicines from the pharmaceutical forge
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- As a matter of necessity, the production of stem cell medicines takes place in clean rooms. Source: Apceth
12.12.2011 -
Stem cells and gene therapy – in recent years these two fields have been noticeable above all because of a series of problems and setbacks. Despite major research efforts, no approved therapies have emerged to date. Nevertheless, Apceth GmbH in Munich is bold enough to combine both approaches – and is still confident of success. The company is funded as part of the top cluster initiative ‘m4 - a new dimension in drug development’, which is based in the BioTech Region Munich. The cluster was also a winner of the Cluster of Excellence Competition in 2010.
Visitors will not have to wait long to be shown the pride of place of the company: the ‘official production license under §13 of the Medicines Act.’ This one simple document from the Federal Government of Upper Bavaria gives the company iconic status – testament the fact that Apceth is moving into completely uncharted territory with a new form of cell-based gene therapy. This was confirmed by Ralf Huss, until recently the Global Head Therapeutic Cell Initiative at Roche, and since 1 October Chief Scientist at Apceth: “I know of no comparable company anywhere in the world.” Neither pharmaceutical companies nor venture capital investors were willing to support the early but ambitious research carried out at Apceth. “Many have gotten their fingers burned as a result of new therapeutic approaches. The capital expenditure and risk appear simply too long-term,” says Huss, who has remained a partner at Apceth also during his time at Roche. Back in the day, Huss met Thomas Strüngmann through a mutual acquaintance, and was able to present his idea. And with success. The company has since received a two-digit million sum through Santo Holding GmbH, the holding arm of Strüngmannschen Family Offices.
Stem cells: “Living medicines”
Apceth focuses above all on mesenchymal stem cells (MSCs) taken from bone marrow. “Living drugs,” as managing director Guenther calls them. The most advanced aspect of their work is the enrichment of autologous stem cells taken directly from the bone marrow of patients before being enriched and readministered. These are aimed towards the treatment of arterial occlusive disease. One dose, administered systemically by infusion, consists of approximately one to two million cells per kilogram of body weight. Deep-frozen, a batch of drugs can keep for around three months – one day maybe even forever. “We require more data in order to prove this,” says Felix Herrmann, responsible for preclinical development at Apceth. A clinical phase I trial with 30 subjects is currently underway. In addition to somatic stem cell therapy, the company also exploits the tendency of existing stem cell lines to accumulate in tissues with low oxygen concentration, such as in a tumor. The trick here is to genetically alter the cells in such a way that they are able to metabolise the drug Ganciclovir from an inactive precursor into the actual medicine. “A sustainable and locally applied therapy would be a real step forward in terms of efficacy and side effects,” says Günther. Phase I clinical trials are set to begin in 2012. Laboratory tests have demonstrated that the genetically altered cells are genetically stable. It is not known whether and in which tissues they differentiate following administration. Like almost everything in Apceth, this is entirely new territory.
The third pillar: contract manufacturing
In keeping with the Strüngmann tradition, the company has a third pillar to fall back on. Even the generic drug company Hexal, founded by the brothers and eventually sold to Novartis for more than 5 billion euros, also produces for third parties. “On one hand to get a feel for the market, on the other to achieve a genuine benchmark for internal processes with respect to price/performance,” says Helmut Jeggle, who manages the life sciences side of the Family Office, and is also Chief Financial Officer at Apceth. And thus, the biotech company also produces cell therapeutics on behalf of pharmaceutical and biotech clients. In addition to their financial contribution, Apceth is also able to make use of the Hexal founders’ know-how. The clean rooms were earlier taken up by a blister packaging plant operated by a subsidiary of Santo. Expanding the headquarters at the Max-Lebsche-Platz opposite the Großhadern clinic was not possible, and so Apceth rededicated the rooms for more exciting projects after the blistering – the common packaging for most pill-based medicines – began to slow down following a change in legislation. With the additional site, Apceth was able to get around a bottleneck in the time-consuming production of stem cell therapeutics: production is highly time consuming. For example, a clean room is occupied for a whole month for just a single batch.
And the regulatory requirements are as complex as the preparation procedures. Established approval paths? Concrete examples of success? None of this is yet available. The same applies for archetypes of the Apceth therapies. Everything is new. “From the medium used to the quality assurance, all the way to cryopreservation – we’ve done everything ourselves,” says head of production Christoph Peter. And in this way the company continues to learn, as does the local government in Upper Bavaria. Other certificates certainly look set to follow.
