About Kristin Comella

Ms. Comella has over 15 years’ experience in corporate entities with expertise in regenerative medicine. She was recently named number 24 according to Terrapin’s list of the Top 50 Global Stem Cell Influencers. Ms. Comella has pioneered a variety of stem cell therapies including cord blood derived cells, bone marrow cells, muscle cells and adipose cells for use in many different applications. She has developed a wide range of regenerative products and techniques that have been successfully implemented into the clinic. She also led the team that gained the first ever FDA approval for clinical trials using a combined cell and gene therapy product. Ms. Comella has been a member of the Bioheart Inc. senior management team since 2004 and is currently serving as the Chief Scientific Officer and board member. Bioheart is a publically traded company focusing on the discovery, development and commercialization of autologous cell therapies for the treatment of degenerative diseases. Since joining Bioheart, she has played a major role in managing the product development, manufacturing and quality systems of cellular products. In addition, Ms. Comella is currently and actively serving on multiple boards in the stem cell arena. She was co-founder and Chief Executive Officer of Stemlogix, LLC for veterinary medicine. Ms. Comella has years of cell culturing experience including building and managing the stem cell laboratory at Tulane University's Center for Gene Therapy. Previously, she worked as a research engineer for Osiris Therapeutics developing stem cell therapies for osteoarthritis. Ms. Comella holds an M.S. in Chemical Engineering from The Ohio State University and a B.S. in Chemical Engineering from the University of South Florida.

Kristin Comella
Chief Scientific Officer
BIOHEART INC.
13794 NW 4th Street, Suite 212
Sunrise, FL 33325
Tel: (954) 835-1500
Fax: (954) 845-9976


Questions & Answers

Q: There is industry criticism from some (mostly those in academia) that claim most doctors/commercial clinics are just in it for the money and that costs for "dubious" treatments are vastly overpriced to enable a huge profit margin. They also rail on about safety and lack of published data showing efficacy. Some criticize patient funded trials. It's a never ending tirade made to look as if every doctor/company is raking in millions off of desperately ill patients. Can you enlighten us on the actual costs of doing business in this industry for a company such as yours?
A: Oftentimes, emerging technologies are met with skepticism and criticism. When stents were first introduced as an option for patients having a heart attack, many famous cardiologists criticized them because of the risk of restenosis (blockage). Yet stenting is now a common cardiac procedure. Cellular medicine has existed since the 1960s and is commonly used for cancer patients under the term Bone Marrow Transplant. These treatments were never put through double blind placebo controlled trials; nevertheless, we accept them as the standard of care. In addition, cellular medicine for degenerative diseases has been the subject of thousands of animal studies and clinical trials. Many of these studies date back to the 1980s. I think a fair question to ask a patient who has failed to benefit from traditional medicines and therapies is “how many studies would you like to see before you try to harness your body’s own healing potential”. Most patients are willing to try something experimental and, provided that companies are clear on the possible risks versus rewards, these therapies should not be withheld from the public.

Bioheart has spent over $125 million researching cellular therapies for patients. We are committed to bringing more treatments forward and all revenue that is brought into the company is put towards advancing this science. Most physicians and scientists in this field are interested in gathering data and moving the science forward. Our FDA phase 3 MARVEL Trial for congestive heart failure patients is budgeted to cost $10 million dollars for 100 patients. Trying to complete double blind placebo controlled trials is very expensive and there is limited funding for companies who are trying to complete these trials. Rather than being a scam, patient funded trials are an important tool for providing access to these therapies.

Q: Is Bioheart still partnered with ThriveMD in a study using adipose derived stem cells for MS? The ThriveMD site states that the cells will be administered intravenously and/or intrathecally to maximize the central effect. Is the trial divided up into groups using the different methods? Do you have any early results you could share that might indicate which method seems to be producing better results? What is the expected time frame for completion and publication?
A: We are working with ThriveMD as well as several other investigators on an IRB study for MS. We have just begun this study so results are not available yet. Patients will receive both an IV and intrathecal delivery. We are hopeful that the cells can cross the blood brain barrier and provide some benefit. This trial may take several years to complete and publish. In the meantime, we are also offering these experimental treatments at several centers throughout the U.S. including the clinic in Ft. Lauderdale: http://www.usstemcellclinic.com

Q: What are your thoughts on the current FDA draft guidelines that propose new restrictions on cellular therapies in the U.S. and would this affect any of the trials Bioheart is involved in?
A: The FDA has recently released a DRAFT guidance document regarding the use of human cell and tissue products from adipose tissue and has requested comments from the public on this guidance. Please note that guidance documents represent the FDA’s “current thinking on the scope” of the topic. “FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the FDA’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in FDA’s guidances means that something is suggested or recommended, but not required.”
Many of you have been safely performing adipose cellular therapies in clinic for years and we should duly notify the FDA. Hopefully they will take this in to consideration when drafting the final guidance document. The current document specifies the following:

“In regard to HCT/Ps from adipose tissue, we generally consider the exception in 21 CFR 1271.15(b) to apply only if the HCT/P from adipose tissue is for autologous use, is removed and implanted within a single operation or in a limited number of predetermined operations in order to achieve the intended effect, and does not undergo processing steps beyond rinsing, cleansing, or sizing. Limited handling such as rinsing and cleansing to remove debris would allow the HCT/P from adipose tissue to retain the structural function, while other processing steps such as cell isolation, cell expansion, or enzymatic digestion generally would not. Thus, if such other processing steps are performed that prevent the HCT/P from adipose tissue from remaining “such HCT/P,” the establishment manufacturing the HCT/P from adipose tissue would generally not be considered to meet the exception under 21 CFR 1271.15(b).”

With the current draft above, the FDA would recommend that adipose procedures be regulated as a drug through the FDA. Just to clarify, a guidance document is considered a suggestion but not a requirement. If a physician can demonstrate that they are taking the necessary safety precautions for their patients, we do not believe that the FDA would be able to enforce this recommendation. We also do not believe that the current draft guidance document will affect our ability to offer in-clinic cell therapy from fat to our patients. However, we will be providing a comment to the FDA to help them draft the guidance document and I encourage you to do the same. Please see the comment that we have provided to the FDA below.

Use the following link to comment:
http://www.regulations.gov/#!documen...14-D-1856-0001

“I oppose the draft exemption in its current form. Adipose cells are currently being using by thousands of physicians in clinic with very few safety issues reported. Provided that these HCT/Ps are prepared in clinic and immediately delivered back to the same patient, the FDA should not have jurisdiction over this procedure as it is not a drug. Physicians have been trained in sterile and aseptic techniques and have been performing surgeries on patients using autologous tissues for many years. Using a patient’s own cells or tissues in clinic is not a drug and the FDA should not regulate it.”

Q: I contacted the ThriveMD clinic and was told the patient payment for the MS trial was $8500. This seems reasonable compared to other U.S. "same day" stem cell clinics which charge up to $20,000. I've read criticism from academic stem cell researchers who criticize patient funded trials. What is your opinion on this?
A: We try to keep the cost of our patient funded treatments low. Our priority is helping patients and gathering data. We work with a network of physicians throughout the world and their fees start around $5000. Our patients understand that these are experimental treatments and we make no guarantee or claim of outcomes. Many of the patients who come to us have failed traditional therapies and are interested in trying something new.

Q: Will Bioheart be doing other trials for neurological disease? Which cellular therapy do you think will get approved first in the U.S.?
A: We are currently running several FDA trials as well as many IRB studies. In addition, we are offering experimental therapies for interested patients willing to pay out of pocket. For Bioheart, MyoCell, our product for congestive heart failure is closest to FDA approval. It is very challenging for companies to get these products through FDA approval as it costs hundreds of millions of dollars to complete clinical trials.

Q: How do you overcome the brain blood barrier with your stem cell treatments when many experts say that approximately 95% of the stem cells never make it to the brain?
A: Unfortunately very little is known about the true mechanisms of action of cellular therapy. It is possible that when cells are placed IV, many are caught in the lungs and very few may actually cross the blood brain barrier (BBB). However, several recent publications have shown evidence that the cells may be able to cross the BBB. At this point, it is more important to determine if the cellular treatments are safe and efficacious for the specified indication. The percentage of cells that are able to cross the BBB may be irrelevant if the patients are responding to treatment. Improving the percentage of cells that cross can be part of second and third generation therapies.

Q: What is the difference between anaerobic and aerobic cancers in terms of treatment? Anaerobic cells produce oxygen as exhaust, whereas most cells do not. How lethal are anaerobic cancers vs aerobic cancers?
A: My expertise is in cellular medicine. We do not currently treat patients with cancer.

Q: What drew you into working with cellular therapies? What have you found frustrating about it and what has been the most personally satisfying?
A: I think that the body’s natural healing mechanisms are fascinating. The ability to harness this natural healing potential to reverse the effects of degenerative diseases or injuries is very powerful. We have a lot to learn about regenerative medicine but we are now starting to realize the potential by bringing these therapies to clinic. With any new therapies, there are challenges to bringing them to market. In addition, it is difficult to navigate the regulatory environment because these therapies are unlike any others currently available to patients. Cellular therapies should not be regulated in the same way as drugs and devices and many regulatory bodies are trying to establish new rules and guidelines. I am not sure that a person’s own cells should be regulated in the same way as a drug that is manufactured. It is important to advance science with patient safety as the primary interest. We have treated patients whose lives have completely changed for the better because of regenerative medicine and this is why I love this field!

Q: The Alliance for Regenerative Medicine says its mission is to advance regenerative medicine by representing, supporting and engaging all stakeholders in the field, including companies, academic research institutions, patient advocacy groups, foundations, health insurers, financial institutions and other organizations. That sounds like a huge and noble effort, but when I look at the member list, it seems more like a group involved with promoting cellular therapies as patented medicines. Johnson and Johnson is a member for instance. Are they just promoting more funding by government for research or do you see them as a group that will be significant in bringing about regulatory change that could benefit large numbers of patients who want access to their own stem cells?
A:I have not had much interaction with ARM so it is difficult for me to comment specifically on their goals. Nevertheless, it is important to bring cellular medicine to public awareness. This needs the cooperation of researchers and physicians from many different institutions so that we can bring the best therapies forward to patients. In the meantime, it is in the best interest of patients to give them access to the therapies that we can perform now. Provided that patients are able to give informed consent, it is their right to try experimental therapies that utilize tissues from their own body. I have recently joined the advisory board for the Academy of Regenerative Practices (www.regenerativeacademy.com). I’m very impressed with their message and desire to educate physicians and medical practitioners about the current cellular medicine techniques being used safely in the clinic. This can help expand the therapies that are available to patients worldwide.