MD Stem Cells

SCOTS and Questions about Stem Cells

The Stem Cell Ophthalmology Treatment Study- SCOTS

MD Stem Cells announces new stem cell study for retinal and optic nerve eye diseases- largest stem cell eye treatment study registered with NIH – to determine effectiveness of stem cells in ophthalmic disease.  

       For many patients with serious eye disease, going blind is a constant fear.  The loss of vision experienced from retinal or optic nerve problems may be progressive with devastating impact on a person’s ability to live a full life.  Treatment with Bone Marrow Derived Stem Cells may hold the key to reversing blindness and restoring vision.  But the concern has been whether such stem cell treatments have enough published reports and studies to show effectiveness in a convincing way.  MD Stem Cells hopes to help provide that evidence.

       MD Stem Cells has been named Collaborator for the new Stem Cell Ophthalmology Treatment Study, abbreviated as SCOTS.   Dr. Steven Levy, President of MD Stem Cells, will function as Study Director for the clinical trial expected to continue through August 2017.  The Retinal Associates of South Florida is the study Sponsor and Dr. Jeffrey Weiss, retinal surgeon and physician, is the Principle Investigator and provider of the ophthalmic stem cell treatments.   SCOTS is being conducted in Florida.

      “We are extremely pleased to have helped design and implement this broadly encompassing retinal and optic nerve disease study” Dr. Levy exclaimed.  “We believe ours is the largest, most comprehensive registered ophthalmology stem cell study to date.  We have taken great care in powering the study and expect to obtain statistically meaningful results.  SCOTS is being conducted under an Institutional Review Board whose evaluation was rigorous.  We have satisfied every requirement for their approval.”

  
     Conditions potentially eligible for the new study include retinal diseases such as age-related macular degeneration (AMD), myopic macular degeneration, hereditary retinopathies such as Retinitis Pigmentosa and Stargardts,  as well as selected inflammatory, vascular and traumatic conditions.  Optic nerve diseases considered eligible include glaucoma, ischemic optic neuropathy, optic atrophy, optic neuritis, LHON / Leber Hereditary Optic Neuropathy, NMO / Neuromyelitis Optica or Devic's Syndrome and some trauma.  The study is focused on the ocular tissue that has sustained damage and its potential for improvement rather than a specific disease entity.     

       SCOTS is registered with the National Institutes of Health and listed on their website www.clinicaltrials.gov with identifier NCT01920867. Patients interested in whether they may participate and healthcare providers may reach Dr. Levy at info@mdstemcells.com or 203-423-9494 Eastern Time USA. 

       “We hope that the treatment will be shown to improve vision in the vast majority of individuals who are enrolled in SCOTS.  However patients considering treatment should understand that this is clinical research and individual responses cannot be predicted “stated Dr. Levy.  He added, “Our previous anecdotal experience with eye disease treated with stem cells has been positive.   With SCOTS we hope to provide strong evidence of the effectiveness of these treatments to the medical community. “

       Disclaimer: The Stem Cell Ophthalmology Treatment Study or SCOTS is an open label, non-randomized efficacy study and no guarantees of specific improvements or visual results are being made.  Any medical procedure carries risks as well as potential benefits.  The SCOTS study has different treatment arms and our principle investigator assigns patients to minimize risk and maximal potential benefit.  Depending on the arm chosen the risk of potential complications has been calculated to be from approximately 0.0008% to 5%.



 Stem Cells & The Available Medical Literature.

        We encourage patients to search the available pre-clinical and clinical literature.  Preclinical typically refers to research in animal models or sometimes in the 'test tube' .  Clinical means in medical patients.   Some of the best sites include- PubMed part of the US National Library of Medicine and the National Institutes of Health and which has over 21 million medical citations-  PLoS   the Public Library of Science which is leading a transformation in research communications -  ClinicalTrial  a searchable US database which provides patients, family members and the public with information about current ongoing clinical research studies  - EU Clinical Trials Register  a searchable database for clinical trials in Europe.
      
It is important to realize many physicians do not favor the use of adult stem cell therapy, believing there is not sufficient evidence based medicine.  In part this is because much of the preclinical and clinical research is not presented in the medical journals they are used to reading.  Because autologous or adult stem cell treatments depend on taking stem cells from each individual patient, it is not possible to create an actual 'product' out of those particular adult stem cells that can be marketed and sold.  Doing strong clinical trials with controls and large numbers of patients is expensive, time consuming and difficult.  Typical pharmaceutical drugs take many hundreds of millions of US dollars to complete the approval process.  Such investment from either the private or government sectors is unlikely any time soon.  Therefore patients will continue to rely on anecdotal evidence, preclinical studies and papers reporting on small numbers of patients to make their decision about obtaining treatment. 

       Disclaimer: 
Information presented in this website is for educational purposes only and is not a guarantee of results from stem cell treatments.  Patient results may vary from those presented in any of the links.


MD Stem Cells
412 Main Street, Suite I
Ridgefield, CT 06877  USA

  Tel: 203-423-9494
Fax: 203-905-6800
info@mdstemcells.com

The role of patient funded clinical research in advancing medical care.

 

Jeffrey N. Weiss, M.D.

Retina Associates of South Florida

Margate, Florida, 33063

RetinaAssocSF@aol.com

 

Steven Levy M.D.

MD Stem Cells

Ridgefield, CT 06897

stevenlevy@mdstemcells.com

 

 

A clinical trial, also called an interventional trial, is a type of research where participants receive a specific intervention that is dictated by a study protocol.  The intervention may be a surgical procedure, drug, device, or change to the participants behavior, such as a diet.  Clinical trials may compare a new medical or surgical approach to no intervention.  There are various sources that provide funding for clinical trials.

 

The National Institutes of Health (NIH)

 

                       The NIH is the largest biomedical funding source in the U.S.  There has been a 20% inflation adjusted decline in the NIH budget over the last decade.  Approximately 15 – 20 % of grant submissions receive funding and due to government budget sequestration, investigators receive only 90% of the approved funds.   A 2007 U.S. government study found that university faculty members spend approximately 40% of their research time navigating bureaucracy. 

 

                      A research grant is awarded to an individual at a university, but not to the same individual when he enters private practice, effectively removing an experienced researcher from government funding.  NIH grants are typically for a 3 year period and end abruptly if not renewed.  In addition, the researcher is unable to shift the funds to another project if the first project is not successful. 

                     

                      Best has demonstrated that disease advocacy has political outcomes, including direct benefits, distributive changes and systemic effects.  She has shown that advocacy groups secure gains for their members (direct benefits), that the mobilized groups receive resources at the expense of the elite political influence (distributive changes) and that such groups can change the system of political decision making (systemic effects).

                     

                      Pharmaceutical Companies

 

                      Pharmaceutical Companies are commercial enterprises organized to make money.  Approximately 75% of clinical trials in medicine are company sponsored.  Such funding may introduce bias, as the study design or interpretation is more likely to favor the drug under consideration with the ultimate goal, the attainment of FDA approval.  This is understandable as the time to develop a successful drug is 10 to 15 years and the cost to achieve FDA approval has successively increased over the decades to approximately 1.2 billion dollars at the present time.

 

                      An orphan disease is defined as any disease affecting up to 200,000 individuals in the US.  There are incentives offered to pharmaceutical companies through the Orphan Drug Act of 1983 to address the rare disease market.  However orphan diseases with larger numbers of patients or those diseases for which there may be less price resistance regarding any successful treatment may draw development efforts in an unfair way.

                       

                      Non-Profit Organizations

 

                      Like the NIH, non-profits use a panel of expert who decide the worthiness of a particular grant and determine the awarding of funding. As such, they suffer from the same problem as the NIH. 

 

                      Thomas Kuhn has pointed out that breakthrough insights frequently stem from the intersection of disciplines, not from within the discipline.  Grant reviewers are generally within the same discipline and can’t recognize the new paradigm that may have come from the work of scientists in other fields.  They remain committed to the old paradigm that has shaped their beliefs, which prompted Max Planck to say, “New scientific truth does not triumph by convincing its opponents and making them see the light, but rather because its opponents eventually die, and a new generation grows up that is familiar with it.” Also like the NIH, nonprofit organizations award grants to institutions, not to individual researchers in private practice.

                     

                      Private Funded Research

 

                      This includes a broad pool of donors with a wide range of passions that may speed progress by investing in bold ideas, gambles, and risky projects. Decisions may be made quickly, not encumbered by large bureaucracies. A sense of urgency can be respected.

 

                      Critics argue that gifts privatize research and steer resources towards areas of personal interest.  Supporters argue that it is the personal choice of the funder whether money is spent on personal goods or personal research.  Personally funded projects have a vested interest in solving the chosen problem and unlike pharmaceutically funded research, are not subject to the profit motive or market forces. 

                     

                      Patient Funded Foundations

 

                      It is not uncommon for many patients with rare diseases or their families to set up support groups, particularly with the ready availability of social networking.  In some cases the only research foundations focused on particularly rare diseases have been initiated by patients and families affected by those diseases.   There are over 7000 rare genetic diseases impacting 8-10% of the US population or about 25 million patients.   When such a disease afflicts a high profile or financially successful individual, or their family member, that particular disease can receive disproportionate attention and support. 

                     

                      Patient Funded Research

 

                      In this model, the patient pays for participating in the clinical research.  In terms of the research, this is similar to the Private Funded model discussed above.   Less common conditions and risky proposals may be studied, unencumbered by bureaucracy and coupled with the ability to make quick decisions.

                     

                      Critics argue that desperate patients can be exploited and should not pay for clinical research.  Clearly, people with resources decide how to use their resources.  Not allowing patients to pay for research would restrict their ability to use their own resources as they see fit. 

 

                      Unlike other types of funding, patient funded research is subject to market demands.  If a study is too expensive, there will be less participants, unlike the situation where the funding is provided for the patient to participate in the research.  The self funding patient will be more inclined to demand information thus providing a more thought out informed consent than in a study where free care is provided, as they want to receive their “moneys worth.”  One can argue that free medical care may be considered an exploitative inducement to participate in research. 

 

                      Patient funded studies are generally for conditions not being investigated by NIH or pharmaceutical studies and tend towards novel approaches for less prevalent medical problems.

 

                      Patient funded research has been criticized for, by its nature, it eliminates the participation of individuals who cannot afford to participate. One can make the same criticism for the other funding sources as unless the study pays the patient for participation, including travel costs, individuals may be unable to take the required time off from work, nor have the financial and personal support structure in place to engage in a study.  Since the purpose of a research study is to determine whether the clinical intervention is of benefit, and such  interventions  may carry some medical risk, one could argue that aligning financial cost with participation creates a higher level of patient engagement, risk benefit assessment and therefore informed consent.  In addition, the ultimate benefits of basic and clinical research accrue to all individuals with a particular disease irrespective of financial wherewithal.

 

                      Does profit taint the research?  One could argue that all human activity creates bias, otherwise there would be no activity.  Unless the research is self-funded, researcher are always paid by some funding source, otherwise they could not perform the research.  Frequently, both NIH and pharmaceutical funding is proportional to the number of subjects the researcher enrolls in the study, which some may consider to be unethical.  In this respect, the patient funded study differs little from other types of studies.  As pointed out previously, it is in the researcher’s interest to keep the patient cost at a reasonable level in order to encourage patient entry. 

                     

                       This paper is a discussion of actual medical studies and does not speak to the situation in which a private physician requires a patient to pay for treatment with an off-label remedy for which no protocol has been written, there is no Institutional Review Board (IRB) approval, and no data is being collected. 

 

                      The patient funded study, that has a detailed protocol, has undergone IRB review, is listed with ClinicalTrials.gov (NIH), and is publishing results, whether positive or negative, is an important form of research allowing the testing of ideas that would otherwise languish due to a lack of funding.

 

                      All human endeavors require work and effort and therefore have financial costs.  There is a range of how removed the patients are from those costs depending on the research payment methodology. 

                      

                       In the case of publically funded clinical research, the patient may have low awareness of the cost of the clinical research as the funds are taken from the general tax base of whose allocation individuals have minimal awareness. In publically funded research the cost of an individual’s participation is spread out over millions of people and therefore the percentage of cost that is the responsibility of an individual patient is low.  This low awareness of source and percentage cost of participation removes these particular financial considerations from the informed consent process.

                      

                       On the other end of the spectrum patient funded research causes high awareness of the cost of that individual’s participation and causes a high percentage of the cost to be the responsibility of the participating patient.  This high awareness enters the informed consent process as an additional consideration as patients weigh the potential personal benefit of participation. This causes a pause in this process, even for the wealthiest individuals.    

 

                       Pharmaceutical clinical research would fall in-between with the actual costs being deferred to a later time, but still assumed by the individual patient needing the successfully proven care depending on the health insurance methodology. 

                      

                       In considering the different methods of funding of clinical research, the overall benefit of properly done clinical research, from whichever funding source, should be kept in mind.   All clinical research and patient care creates costs which ultimately accrue to individuals in the society.  It is only a matter of how directly those costs are felt by individuals.

 

                      Suggested Reading

 

  1. ClinicalTrials.gov. Learn about clinical studies. Accessed December 11, 2014.
  2. U.S. Investment in Health Research – Research America. Accessed December 8, 2014.
  3. Parke DW. Current Perspective, Innovation: Risky Business. EyeNet. December 2014:12
  4. Lind SE. Fee-for-service research. N Engl J Med 1986;314:312-315.
  5. Lind SE. Dilemmas in paying for clinical research:  The view from the IRBs.  IRB: A Review of Human Subjects Research March/April 1987;9:1-5.
  6. Lind SE. Financial issues and incentives related to clinical research and innovative therapies.  In Vanderpool HY, ed. The Ethics of Research Involving Human Subjects: Facing the 21st Century. Frederick, MD: University Publishing Group, 1996:185-202.
  7. Best RK. Disease politics and medical research funding: Three ways advocacy shapes policy. American Sociological Review. 2012:77(5); 780-803.
  8. Kuhn T. The Structure of Scientific Revolutions. Chicago: University of Chicago Press, 1962.
  9. Myers ER, Alciati, MH, Ahiport, KN, Sung, NS. Similarities and differences in philanthropic and federal support for member states: A analysis of funding by nonprofits in 2006-2008. Academic Medicine. November 2012;12(1)1574-1581.

                      10. Evan S, Block JB. Perspective-Ethical issues regarding fee-for-                        service-funded research within a complementary medicine context.              Journal Alternative and Complementary Med. 7:6,2001;697-702.

                      11. Rose Bowl opponents team up to raise awareness of a rare blood                  disorder.  PBS NewsHour Episode December 31, 2014 

                              http://video.pbs.org/video/2365395300/

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