Predictive medicine can change our tomorrows today. Regenerative medicine can replace artificial body parts with lab grown technologies while genetic breakthroughs can save families from generations of genetic disability. It is possible that new knowledge of human genetics and cell biology is likely to transform medical practice. Three likely scenarios could evolve:

•Genetics will lead to the classification of diseases on the basis of the underlying genetics or biochemistry, rather than by symptoms alone leading to preventive rather than crisis orientated treatments.
•Genetic information will identify people who are likely to respond to drugs, or to be harmed by them (pharmacogenetics). This is already possible with certain psychotropic drugs on an experimental level but has not trickled down into mainstream medicine.
•Genetic variation will be a new ‘susceptibility factor’, permitting monitoring and early treatment or, perhaps prevention, of an increasing proportion of common, multifactorial diseases, such as coronary heart disease, hypertension, stroke, cancer, diabetes and Alzheimer’s disease. Even stress management can be amplified with knowledge of individual genotypes

It is the genetic variation susceptibility factor which is considered to be the change maker for the advent of predictive medicine. This could lead to regenerative medicine on a cellular (somatic) level or even in vitro gene manipulation (germ line therapy) which could prevent intergenerational transfer of genetic disabilities.
Predictive medicine, when it comes, will be based on a much wider use of genetic testing, at present the gap between what the healthcare system is geared up and trained to deliver and what is scientifically viable is huge. For example there are treatments approved for traumatic brain injury that are effective but most be given within a couple of hours of trauma. This can’t happen now because emergency room personnel are not adequately trained or equipped to diagnose MTBI… As with any new technology applied to health in the context of a complex delivery system, implementation is not going to be simple.

First, of course, there needs to be demand from medical personnel and the general public. Typically wide spread change will only take place after the following criteria are established:

•Demonstration of clinical effectiveness and patient safety – through statistically valid clinical trials
•Cost-effective for general use – through economic analysis of trials and other data;
•Standardization of technology, and quality control – generally through outside regulation of suppliers and laboratories;
•Allocation of resources;
•Recruitment and education and training (or retraining) for health workers – including specialists, MDs, nurses, counselors and technicians. For instance a surgeon who makes a good living performing spinal fusions and cervical repairs will need significant convincing, retraining and motivation to become an early adopter of treatment that makes the previous way of doing business obsolete.

Predictive, regenerative medicine may be the wave of the future. History teaches us that the way to greatness is to find a way to serve many. My dream is to witness a generation of scientists and medical professionals join in unity with a foundation of integrity to build a tomorrow for the patients and public who have make their careers possible.

References:

Materials adapted from Open University Course Materials (accessed july,2009)

Adult stem cell viability is on many minds. I have had queries from UN ambassadors to children of only nine asking me about stem cells. The curiousity is international but the questions are the same. People are not sure who to trust and they need stem cells now. Many can not travel to other countries because they are too ill and others lack funding for private stem cell clinics. Some of these feel the FDA or the NHS is unreasonable in thier demands for testing while others feel patient testimonials should be outlawed. My own training is in mental health/psychology so patient to patient information is all I can offer.

I personally like patient testimonials especially when they are accompanied by forums where people discuss how and if the treatment worked and what the hurdles were in getting treatment. I have often learned more from groups of patients with an experience than from professionals with just a theory. Forums are not meant to be professional research, they are peer to peer information only. Google stem cell or regenerative medicine forums and you will get many choices.

I agree clinical trials and time are important to assess treatments but realistically it will be 15 or 20 years before long term results are on the table for clinical trials. There are some companies that have obtained FDA approval for trials, others are listed at clinicaltrials.gov It may be useful to look at this Doctor’s description of FDA off label drug use for drugs to understand how the rules were set up and to get an inkling of how this could all translate to cell biology.

My concerns are that engineering any living object is not a simple process and what looks simple from the outside in a needle in/needle out sort of process is very complex from a laboratory perspective and all the answers may not be in. Some clinics are reputable, give good patient care and patients are reporting good long-term results. Some patients are fortunate enough to get into a university sponsored trial that is tied to a major research hospital. Other clinics are still using methods that were proven ineffective many years ago. Patients are vulnerable and need protection sometimes even from themselves. I can no longer count the number of friends I have lost to questionable therapies after spending family fortunes in a quest for a cure. There are no easy answers…

Having said this and knowing from personal experience the agony of chronic unrelenting pain and the sadness of life lost because of disability I would not likely wait until the votes were in but would join in the age old clamor of patients trapped by pain “Just fix me!” The International Society for Stem Cell Research has released guidelines which are helpful to use when considering any new therapy. The PDF is available here

“The procedure is totally simple and cheap,” reports UNSW’s Dr Nick Di Girolamo (lead author of research study), “Unlike other techniques, it requires no foreign human or animal products, only the patient’s own serum, and is completely non-invasive. This preliminary trial was conducted on three people, two with extensive corneal damage resulting from multiple surgeries to remove ocular melanomas, and one with the genetic eye condition aniridia. The patient with aniridia had damage in both eyes so stem cells were taken from the conjunctiva area. Because stem cells have not yet differentiated into specific cells they could grow into the cells that were needed. Each patient’s sight improved significantly after only a couple of months

Here is how it works. Less than a millimeter of tissue is taken from the ocular surface of the patients own eye. It takes a couple of hours to prepare the eye and put the contact lens with the baby cells in place and the patient goes home. The stem cells are cultured on a post surgical contact lens which is then placed onto the damaged cornea for 10 days, during which time the cells are able to re-colonise and heal the damaged eye surface. Apparently it took some experimentation to find a lens that could be successfully used as a scaffold for the cells.

The scientists on this research project see this therapy as a simple way to restore sight for eyes damaged by scarring, chemotherapy and a range of other disorders. They suggest that all is needed is a simple lab and qualified medical personnel putting it within reach of even third world countries

Di Girolamo, Nick; Bosch, Martina; Zamora, Katherine; Coroneo, Minas T.; Wakefield, Denis; Watson, Stephanie L. A Contact Lens-Based Technique for Expansion and Transplantation of Autologous Epithelial Progenitors for Ocular Surface Reconstruction. Transplantation, 2009; 87 (10): 1571 DOI: 10.1097/TP.0b013e3181a4bbf2