On the 22nd of May 2013, the Italian Senate took a bold and first-of-its-kind step in promoting stem cell therapy. After a public and media uproar against the therapy, the ruling party temporarily allowed patients who were already undergoing this therapy at a hospital to complete their scheduled treatment and also promised a purse of 3 million euros to setup a clinical trial for a therapy it has already given approval to.
What is Stem Cell Therapy?
Stem Cell Therapy takes advantage of pluripotent (capable of giving rise to different cell types) nature of stem cells and works on the principle that introduction of such cells into damaged tissue can help in repair of the damaged area and thereby in treatment of a disease. Stem Cell Therapy is being looked up to as being the mode of treatment for several disorders such as Parkinson's disease, various cancers, cardiac failure and even genetic disorders such as Spinal Muscular Atrophy.
What is happening in Italy?
A private lab called Stamina Foundation Onlus has been promoting stem cell therapy in Italy since 2009. Its founder Prof. David Vannoni strongly believes in the potential of stem cell therapy and has been privately helping patients with extreme neurodegenerative disorders by transplanting mesenchymal stem cells in an attempt to help them recover from their debilitating diseases. While it may seem that Prof.Vannoni is carrying out a humane task, his method of treatment has not gone through the mandatory clinical trial and lacks sufficient evidence in government records that it is actually effective.
This is where the Stamina Foundation has met strong criticism from other scientists involved with stem cell research and also media and public for not having sufficient evidence to show that the method of treatment actually works. Legally, any drug that is to be administered to a patient must have an approval from the drug regulatory body of the country. However, the treatment offered by the Stamina Foundation has no approval from Agenzia Italiana del Farmaco (AIFA), Italy's drug regulator.
How could AIFA allow this to happen?
More than AIFA allowing this, the Stamina Foundation excused itself from regulatory approval by citing two major points:
- First, the therapy Stamina Foundation offers involves extraction of mesenchymal stem cells from the bone marrow of the patient, transfer to a lab setup where they are grown and then injected back into the patient, where they are expected to reach affected/ damaged areas or organs and take form of healthy local tissue. Since, the bone marrow cells are extracted from the same patient, the Stamina Foundation claims that they are not medicines from a external source but mere transplants and require no regulatory approval.
- Second, as per Italian law, an unapproved drug can be used on 'compassionate grounds' for a patient who is critically ill and has no alternative form of therapy available. Since, most of such disorders have no cure, the Stamina Foundation had a free run with its stem cell therapy and was able to sign up patients for its treatments.
Trouble began, when the AIFA visited the lab premises of the Stamina Foundation and found that the lab did not meet the basic standards of Good Manufacturing Practice (GMP) set for medicines and immediately sought a ban for their treatments. While, like the Stamina Foundation, one may argue, that these cells are not medicines, the promoter of the therapy, Prof. Vannoni himself has gone on record to say that culture of the extracted stem cells under GMP standards might reduce its efficacy. Such statements derail any faith one may have in a prospective treatment.
In addition to this, Prof. Vannoni is a mere promoter of this therapy and is nowhere involved in the research that is required for progression of such a new line of medicine. The therapy the Stamina Foundation is promoting was developed by Ukrainian scientists who are missing at this point in time and only has records in the European Patent Office, as filed by Prof. Vannoni and Mr. Molino, an unseen character during the development of this story. Claiming success with no visual clinical history or background (Prof. Vannoni is a Professor of Psychology and an expert at persuasive communication) has therefore attracted the ire of multiple stem cell researchers around the globe. Their combined angst can be seen in the commentary published by 13 authors in the EMBO Journal titled, Regulation of stem cell therapies under attack in Europe: for whom the bell tolls, which was where we at Coffee Table Science, also became aware of this issue.
While scientists from US, UK, Australia and many developed European countries were furious for having to present statistically significant data to prove the efficacy of the therapies and receive grants for further research, loop holes in the Italian framework have allowed the Stamina Foundation to have a free hand to recruit patients for its self-proclaimed therapy. Coming under pressure from celebrities and families of the patients undergoing treatment at the Stamina Foundation, the government not only reversed the ban that AIFA had imposed but also promised a full wallet to convert their operation into a clinical trial to obtain data on the efficacy of the therapy. The Senate's decision has the following local and global implications:
1. The AIFA, the chief regulator of drugs in the country, now seems powerless against the Stamina Foundation since it could not exercise control on an company that is merely 4 years old.
2. If proven successful, scientists who have dedicated decades of their life tryin
g to perfect stem cell therapy to their regulatory standards and appease their ethics and grant committees, would have lost to an apparently sub-standard yet effective method of treatment.
3. If successful, thanks to the support from the government, the loop-holed legal framework and sidelined drug regulator, AIFA, Italy has the potential of becoming the destination for stem cell therapy, ahead of US, UK and other European majors, who have spent years of time and billions in funds to take their research forward.
4. The decision could also start a trend where legislators can overpower administrators and divert the course of research, development and treatments in the country.
5. Other alternate therapies, which have been largely denounced by Western medicine, could see a rise in adoption by patients and their families, even after rejection from regulatory bodies.
Whether the Italian government has made a blunder or carried out a master stroke will only be revealed once the results of the study are released. For now, let us know what you think about this in the comments below. If you had an ailing relative or friend in need of a therapy, would you really care, if the treatment was approved or not?