Stem cells and patents: a potent mix
pharmafile | June 10, 2010 | Feature | Research and Development | Stem cells, stem cell
In recent years, there has been much debate about whether or not the use of stem cells in medical research should be prohibited on ethical or moral grounds. Similar battle lines are being drawn up in the legal world over the patentability of stem cell related innovation.
Stem cells hold out the promise of having a very wide application, and could be used in the process of medical research, and as therapies in their own right. Researchers around the world are now investigating and developing their use in regenerative and therapeutic medicine and also in drug discovery and toxicology. Their huge clinical potential lies in the fact that they can be grown to large numbers in culture and then manipulated to differentiate into specific cell types for onward application.
Researchers are now investigating the transplantation of specific cell types into patients to replace damaged or diseased cells of the same type restoring function. In the future, it may even be possible to grow organs from stem cells in a laboratory for transplantation into patients.
Broadly speaking stem cells can be separated into two categories, embryonic stem cells (ESCs) and post-embryonic stem cells (also referred to as adult or somatic stem cells (ASCs) (see below).
Introduction to patents
The development of stem cell technology is heavily regulated and takes a significant investment of time and money to create a usable product. Research and development needs to be carried out at an early stage – stem cells with the necessary characteristics will need to be derived, produced, isolated and stored in sufficient number and quality for onward clinical application without degradation (such application will also require research).
Thereafter, clinical trials and strict testing is necessary to ensure safety and efficacy. Patent protection allows companies (and their sponsors) to recoup some of these costs.
A patent is not an automatic right. An application needs to be made to the relevant patent office and will be granted if the invention is novel (not previously disclosed to the public), includes an inventive step (over what is already known) and is capable of industrial application. An applicant should ensure that it does not disclose its invention to the public prior to applying for patent protection. Otherwise, this self disclosure may prevent the applicant being able to show that the invention is novel at the application date. To this end, the applicant should ensure that strict confidentiality provisions are in place at an early stage.
Once granted a national right is created which has effect in that territory alone, for example, a UK patent will prevent third parties infringing the patent in the UK but not in any other country. Currently, there is not a European-wide patent. However, it is possible to make a centralised application, via the European Patent Office (EPO), which creates a bundle of national rights through a single application and thus reduces administrative costs.
A patent grants the owner a monopoly right preventing third parties using the patented technology. The patent owner has an unopposed period, generally lasting for a maximum of 20 years, to recoup its research costs. In return for this monopoly right the owner must disclose the technology to the public.
A patent can protect a product (a drug for example) or a process (the method of manufacturing that drug). Take a simple process: A + B → C (where A and B are starting materials and C is product). If product C meets the patentability requirements it will be possible to apply for a patent to protect it. If product C is not afforded patent protection, perhaps it is a natural product or its patent has since expired, it might still be possible to protect a novel method of manufacturing product C from materials A and B.
The rights afforded to a process patent are narrower than those under a product patent. The owner of a product patent can prevent third parties from selling, importing, using or manufacturing the protected product by any means in the country where the patent is granted.
Whereas, the owner of a process patent can prevent unauthorised use of that process in the country where the patent is granted (and subsequently the sale, importation or use of any product created using that process).
Certain matter is excluded from patentability. For example, it is not possible to patent the human body, including the various stages of its formation and development. It is therefore generally accepted in the majority of jurisdictions that totipotent cells (cells which can develop into an entire human being) are excluded from patentability. The main exception to this rule is the US where totipotent cells are capable of patent protection.
Patentability of stem cells in the UK
Cells are generally patentable and are assessed like any other invention, that is, the cells must be novel, inventive and capable of industrial application.
Cells are deemed to be novel if they have been isolated or purified from their natural environment or reproduced outside the body.
The patentability of stems cells however is a controversial subject and an area of law which is rapidly evolving. There are harmonised European prohibitions preventing patents being granted for inventions which concern the “uses of human embryos for industrial or commercial purposes” or are contrary to public morality. However, these prohibitions have been interpreted differently by Member States.
The issue of animal welfare and morality has been debated at length and it is generally accepted that an invention is not necessarily contrary to morality if, in weighing up the suffering of the animal and the potential medical benefit to man, the advantages outweigh the disadvantages.
Further, as the derivation of animal stem cells does not involve the use of ‘human’ embryos the above mentioned prohibitions do not apply and it is therefore possible to get a UK patent for animal stem cells (or a process to obtain or that uses such cells). Likewise, as the derivation of ASCs does not involve the use of human embryos, deprive the right to life, or cause suffering to life it is possible to get a UK patent over ASCs (or a process to obtain or that uses such cells).
The patentability of ESC related technology is controversial. The UK Intellectual Property Office (UK IPO) will not grant patents for processes of obtaining stem cells from human embryos (i.e. they will not grant a patent protecting a process of deriving human ESCs, as such a method would use human embryos).
However, until recently the UK IPO would grant patents for ESCs (or processes that use ESCs), the justification being that such is not a process that uses an embryo but is a product derived from an embryo (or technology that uses the derived product). But it appears that its position has changed following the EPO’s decision in the Wisconsin Alumni Research Foundation (WARF) patent application (For more on the WARF case in the US, see bottom of left page).
WARF applied to the EPO for patent protection with respect to an embryonic stem cell culture and methods for maintaining and using such cells. The EPO rejected the application (initially and at appeal) stating that as the ESC culture was derived from human embryos, which would inevitably be destroyed as part of the process, the ESC culture itself and any use of the ESC culture would be contrary to public morality and thus prohibited from patentability. It is therefore likely that if ESCs could be obtained without destroying a human embryo the ESC culture and any use thereof might be patentable via the EPO.
The WARF decision effectively prevents ESC related patent applications being made through the administratively efficient route of the EPO.
That said, the WARF decision is not binding on national IP offices and nothing prevents an applicant applying to each national IP office separately. Whether or not an ESC patents will be accepted will be a matter for each national office governed by its national laws.
Although the EPO’s decision is not automatically binding in the UK, it appears that the UK IPO has decided to adopt a similar stance. In February 2009 the UK IPO amended its briefing note on ‘inventions involving human embryonic stem cells’ to explain that it will grant patents for inventions involving human embryonic pluripotent stem cells provided that, at the filing or priority date, the invention could be obtained by means other than the destruction of human embryos.
Stem cell patents in other jurisdictions
For the reasons discussed here, the majority of jurisdictions will grant patents involving animal stem cells or ASCs. However, the patentability of human-ESC related technology differs from country to country. Roughly speaking, the United States, Canada and Australia allow ESC technology to be patented whereas France, Germany and Spain do not.
The problem is that in order to secure a patent in a specific jurisdiction, the invention needs to be disclosed to the public. Unless the relevant technology is capable of patent protection in all countries targeted for commercial exploitation, a company will have a decision to make with respect to patent strategy. For example, a biotech company might be able to apply for patent protection over ESC technology in the US but not in the UK. The granting of the US patent would prevent third parties from exploiting the invention in the US, but the disclosure required to obtain the patent would allow third parties to exploit the invention in the UK.
Bearing this in mind, biotech companies may opt not to apply for patent protection but instead impose strict confidentiality oblig-ations for as long as practicably possible (which is inevitably difficult due to the need to publish the research in scientific journals and as part of clinical trials).
EMBRYONIC STEM CELLS (ESCS)
ESCs are isolated from a blastocyst, a 4-6 day old embryo consisting of around 100 cells. ESCs are obtained from the inner cell mass of the blastocyst, which inevitably means destroying the embryo (however, new research could allow ESCs to be obtained without preventing embryo development). As a result, there has been much debate about the ethical dimension. However, ESCs have a versatile characteristic which makes them attractive to research scientists as they are able to develop into any type of cell – they are ‘pluripotent’. So for example, ESCs can be manipulated under certain conditions to develop into liver cells, or cells of the nervous system, heart, skeletal muscle, blood, etc. However, an ESC can’t develop into an entire human being (they are not totipotent).
ADULT OR SOMATIC STEM CELLS (ASCS)
ASCs derive from cells already developed past their embryonic state and are generally categorised according to their tissue of origin, i.e. neural stem cells (nervous system) and haematopoietic stem cells (blood). As ASCs derive from post embryonic cells their ability to differentiate is limited to a small number of specific cell types generally dictated by their tissue of origin (they are multipotent), for example, a ASC that is derived from liver tissue may, under the appropriate conditions, be able to differentiate into cells to replace old or damaged tissue in the liver. However, it is unlikely that the same ASC could differentiate into specialist cells to replace damaged tissue in the brain.
Rob Jacob is an associate at law firm Stephenson Harwood. Email: rob.jacob@shlegal.com Website: www.stephensonharwood.com
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