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Thread: S 2689: (REGROW) congress to the rescue on stem cells? (not)

  1. #1

    Default S 2689: (REGROW) congress to the rescue on stem cells? (not)

    Here's what many of us have feared. I cannot support this proposed legislation. Thanks to member Jennifer for this info.

    May 4, 2016
    Richard Jaffe, Attorney

    In my last post on stem cells, I painted a pretty bleak picture. Based on warning letters and recent FDA guidance documents, the clinical use of autologous stem cell therapeutics is essentially over. The only way patients are going to be able to receive autologous stem cell transplants for non-homologous use is either in FDA approved clinical trials or outside the United States.

    This hasn’t gone unnoticed in Congress. In March, 2016, a bill was introduced, by Senator Mark Kirk and others which purports to help. It doesn’t. It creates an illusion of a solution that changes nothing. The bill does not address the core problem which is that autologous stem cells, even if cultured, should not be regulated as drugs, because under any reasonable analysis, they are not. As I suggested in my previous article, the feds are clamping down on stem cells because of the claims made by transplanters and because of the culturing of stem cells. The FDA’s response is draconian and the Kirk bill doesn’t help in any meaningful way.

    Under the proposed bill, ostensibly a new regulatory pathway is created. But before we talk about the new path and why it’s nonsensical window dressing, let’s briefly review the traditional approval path. Under current FDA law, to get a drug approved, a drug sponsor needs to file an IND (investigational new drug application). This is a very big deal in terms of time, expense and volume of paperwork. All available information about the safety and efficacy of a drug must be submitted. Usually this information comes from animal studies. (However, if there is controlled clinical use or foreign studies, which data is submitted which can be used to bypass phase 1 and go right to phase 2 studies.)

    All available pharmacokinetics information (basically mechanism of action and interactions) must also be submitted. A protocol setting out all of the details of the dosage, entry criteria, contraindications, endpoints, and a myriad of other aspects of intended use information must be included in the application. In addition, the proposed informed consent form must be submitted for FDA review, as well as information about the study’s IRB (Institutional Review Board. Also, the sponsor must include information about the principal investigators and sub investigators who will administer the investigational drug to the patients/study subjects. Basically, the IND has to convince the FDA that it is safe to administer the drug to humans and there is some reason to believe that the drug will work as well in humans as it did in animals, (or in humans prior if there is prior controlled clinical use). In other words, that there is some reasonable expectation of efficacy. After the IND is submitted (which usually consist of tens of thousands of pages), the FDA has 30 days to review the filing. If the sponsor doesn’t hear from the FDA in that time, the trials can proceed. Oftentimes, the FDA has questions which have to be answered, which begins the back and forth between the sponsor and FDA. This process can move with the alacrity of the shifting of tectonic plates.

    Under the Kirk bill, a supposedly new and streamlined FDA drug approval path is created. It’s called “conditional approval.” The bill gives the FDA one year to come up with some kind of framework to conditionally approve a stem cell application, if the sponsor demonstrates preliminary evidence of safety and a reasonable expectation of efficacy, short of phase 3 levels of proof/. See section 351B (a) of the proposed bill.

    On its face, this test seems similar to what is required to obtain an IND, except the Kirk bill seems to assume that the treatment has already been used on humans and there is some legitimate clinical trials data supporting safety and efficacy.

    Hmmm. Odd. I thought the bill was supposed to make it easier for a person to get his/her own stem cells? But this seems to require more evidence of efficacy than might otherwise be required of a sponsor to obtain a regular old IND.

    These two Kirk bill requirements (preliminary evidence of safety and controlled clinical evidence of a reasonable expectation of efficacy) of course gives the FDA complete discretion in determining whether the proposed study meets the criteria. If the sponsor succeeds in this threshold showing, then it has five years to submit the data in an NDA (new drug application).

    However, in detailing the additional requirements for obtaining “conditional approval, Section 351B (b) (8) requires that that the sponsor submit an IND in order to treat any patients under the “conditional approval.”

    OK, so everything which has to be submitted in a regular IND has to be submitted for “conditional approval” because you can’t get conditional approval without submitting an IND.

    So in terms of both substance and paperwork, the bill seems more cumbersome than the requirements for an IND, at least an IND with a couple different protocols. So what’s the point of the conditional approval thing? I haven’t figured that out yet. Seems that the Kirk bill is a waste of time and energy, despite the fact that something like 80 organizations support it. Maybe the devil is in the details. One could always hope that the FDA will actually come-up with a more streamlined process to allow patients to receive stem cells, but I’m just not feeling it from what is in the bill.

    My underlying problem is that I don’t think a person’s cells are drugs or should be regulated/prohibited as “unapproved new drugs.” There seems to be something fundamentally odd and wrong about the federal government regulating a person’s own tissue when the tissue or part of it is reinjected. I get that the separation should be done in a sterile and effective way, (read that the cells aren’t contaminated or destroyed by the separation process) and that the centrifuges should be validated and FDA cleared. But regulating a person’s own biological material which is removed and then reinjected, well that just seems crazy intrusive, or just plain crazy to me, except in the Bizzaro regulatory world. But actually, the FDA used to agree with my view, in part at least, because up until the first warning letter in 2012, the FDA interpreted 21 USC 1271.15 as stating that separating stem cells from tissue or structure was the practice of medicine and not subject to FDA regulation as long as the product was reinjected in the same surgical procedure. The FDA only changed this reasonable position based on the perceived abuse by stem cell transplanters (at least that’s my interpretation).

    In that regard, I also get that government agencies might or should be concerned about these exaggerated claims. But the state medical boards can and do police physicians based on their web sites and information conveyed to patients. I think the state medical boards are in a better position to handle enforcement actions against unsupported claims. And of course, this is more in-line with the seeming intuitive notion that a person’s tissue or cells are not drugs just because the material is removed and reintroduced into the body. And both the FDA and the FTC have the statutory jurisdiction to go after the transplanters for false claims if they think the medical boards aren’t doing their job. But prohibiting what is essentially a surgical procedure seems like an unnecessary and complete overreaction to the problem.

    So what needs to be done? It’s not complicated. Congress should statutorily overturn the FDA stem cell guidance documents and warning letters by passing a law that autologous stem cell transplantation is the practice of medicine, even for non-homologous use, and even when the cells are cultured/expanded. Let the state medical boards police the autologous stem cell transplant physicians in terms of their claims and therapeutic use. Texas is already doing just that. Organizations or other sponsors can still do clinical trials. People will still enroll in trials the way they do now, (or did before the warning letters) because of the financial incentives to the patients in enrolling in clinical trials (the drugs are free and so are many associated costs). But at least those who decide to have their own stem cells reinjected in their bodies will have the option to do so without government interference, and in my view, that’s a good thing.

    Any brave Congress folk willing to take on the FDA?

    Richard Jaffe
    First treatment in 2007. Pioneering ever since.


  2. #2


    So, are you giving up on the bill based on that one blog post?

  3. #3


    I have sent messages to Senator Kirk and Rep. Coffman (he's from my state and is co-sponsoring it) that they need to rethink REGROW to clear up any doubts for same day procedures.

    Dr. Centeno opposes it in its current form and he is an expert on regulatory matters. He says it will restrict same day procedures for orthopedic issues, lung disease, etc. It's very complicated quite frankly, but I don't want something that makes it worse for those procedures than what we have currently. It's the usual - politicians getting into issues that they truly don't understand. If they really want to do something, change the onerous regulations that the FDA has imposed on procedures that really are the practice of medicine and get the ball rolling for procedures that fall outside of those categories. Enough thumb sucking! People are suffering.

    On the other hand -

    By Bernard Siegel, Executive Director, Regenerative Medicine Foundation

    The REGROW Act- an urgent demand for a new FDA regulatory pathway to accelerate safe cell therapies

    In the midst of one of the most partisan political seasons in memory, a significant piece of legislation is making its way through Congress with bipartisan, bicameral support. The REGROW Act — Reliable and Effective Growth for Regenerative Health Options that Improve Wellness — seeks to move the field of medicine dramatically forward by creating a new regulatory pathway with conditional or temporary approvals for safe, regenerative cell therapies. Its broad support is a telling indication of its importance and its promise.

    It is hard to overstate the urgency of this opportunity. Regenerative cell therapy is one of the most exciting and promising areas of medicine today. Cell therapy is bringing enormous advances to the areas of cardiology, neurology, oncology, ophthalmology, and orthopedics. New studies show that cell therapy holds great promise for patients with Alzheimer’s disease, Parkinson’s disease, heart disease, diabetes and cancer.
    Why does cell therapy need a new regulatory pathway?

    Because it is a unique form of treatment that does not appropriately belong in either of the FDA’s two approval pathways: the practice of medicine or the development of biologics and drugs. The FDA currently regulates cell therapy as a drug. Requiring all therapeutic cell products to go through the lengthy and expensive Biologics Licensing Application (BLA) process and initial Phase 3 trials is too high a regulatory bar, especially since some forms of cell therapy, such as bone marrow transplants, have been in use for decades and have been proven safe.

    Even more to the point, after 15 years of requiring cell therapy developers to pursue approval through the BLA process, the FDA has not yet issued a single approval. This bottleneck has real-world consequences for patients every day who cannot receive new treatments that could alleviate suffering or even cure their conditions.

    This is not just some ivory tower academic debate about the power and reputation of the FDA. This is not about the bottom line for a few companies and their minions that have decided that they have vested financial interest in maintaining the status quo. Here’s the reality: Some patients die waiting.

    I’ve been working as an advocate for cell therapies for 14 years, and I have never seen the convergence of technologies and progress that we see today in areas such as imaging, nanotechnology, gene editing and cell reprogramming. We have largely moved beyond the societal debate about embryonic versus adult, or tissue-specific, stem cells. Today we understand that all of these cells hold promise in different ways as valuable tools in the clinical toolkit.

    We are at an important juncture and the time to move forward is now. The U.S. is lagging behind other nations that have already developed accelerated approval pathways for cell therapy. The FDA is arguably the world’s most important regulatory agency, and should seize the opportunity to guide the appropriate development of this emerging field. The innovation and cross-disciplinary work being done in the lab should be a model for our regulatory and legal systems.

    I am not advocating that we compromise safety. Far from it. We must have appropriate standards and boundaries. And that is why we need an innovative approval pathway designed specifically for cell therapy. We must be smart about preparing for the era of precision medicine and the sweeping changes regenerative cell therapy will bring.

    Recently I’ve been studying and writing about important patient advocacy movements. In the mid-20th century, medical philanthropist and health activist Mary Lasker, who said she was “opposed to heart attacks and cancer and strokes the way [she was] opposed to sin,” fostered important medical research and revolutionized the American Cancer Society. Polio vaccines were a giant clinical trial, and parents in the 1960s offered up their children as research subjects to help face down a health emergency. The AIDS/HIV epidemic was eventually addressed and the field advanced after public protests gained national attention. Breast cancer research was underfunded until public advocacy campaigns brought about change.

    I believe that regenerative medicine represents the next consumer movement, and the REGROW Act gives us a foundation to build on. Public awareness and coalition-building will help move the needle on this, and advance the field of medicine, just as it has in the past.
    Recently I spoke with a mother whose precious son died at age 14 from graft-versus-host disease after nearly two years of grueling treatment for cancer. His last hope was prochymal cell therapy, but it was not approved for use, and the FDA did not grant a compassionate use exception. In Japan, patients are receiving this therapy, and the survival rate for graft-versus-host disease has leapt up. There is no easy way to explain this discrepancy to that grieving mother.

    The human cost of further delay is too great to justify. We cannot be a society that might cure a mouse of diabetes but spend 10 years and billions of dollars bringing that benefit to humans. We owe it to ourselves to break down barriers and move the field forward.

    Frederick Douglass said, “Power cedes nothing without a demand.” The REGROW legislation, backed by scientists, physicians, patients, and — incredibly — legislators on both sides of the aisle, is our demand.
    First treatment in 2007. Pioneering ever since.


  4. #4


    I realize the legislation is a double-edged sword that affects same day procedures adversely and of course I hate that, but for those of use whose interests are more aligned with getting expedited access to cultured cells, the positives would seem to outweight the negatives...assuming this bill would actually fulfill its alleged purpose to speed up the approval process. We would all like to our cells completely deregulated, but unfortunately I'm pretty sure that that's unrealistic in the kind of country we have today.
    Last edited by gh253; 05-10-2016 at 03:53 AM.

  5. #5


    Quote Originally Posted by gh253 View Post
    assuming this bill would actually fulfill its alleged purpose to speed up the approval process. We would all like to our cells completely deregulated, but unfortunately I'm pretty sure that that's unrealistic in the kind of country we have today.
    I agree that those that are looking for a way to get clinically relevant, legal treatment in the U.S. using expanded (cultured) cells, it could be a quicker route to see that happen if we assume, as you point out, the bill will actually do that. I think it will so for those who patients I would like to see it happen, however, why not put some pressure on the legislators to make sure they don't take away what we already have? It's the usual hurry to get a bill introduced without really knowing the negative impact it might have. If politicians want to control our existence, they need to get it right from the beginning.

    I don't think it will pass at this point, but I could be wrong. There are also the embryonic advocates clamoring that the bill is restrictive in that it only includes adult stem cells.
    First treatment in 2007. Pioneering ever since.


  6. #6


    Quote Originally Posted by gh253 View Post
    I'm pretty sure that that's unrealistic in the kind of country we have today.
    It's time to take back our country from a big, bloated government. Why in the world are people content to let the FDA control our lives? While I am happy for this lady, what about those that aren't as fortunate to be able to try an experimental treatment? Why is the FDA picking and choosing? Why does the FDA continue operating under an antiquated system?
    A Mouse that Roared: Lori Alf's Fight Against Cancer and the Miracle Cure that Saved Her
    MAY 6, 2016

    Carl H. June, M.D., who spends his days in a sterile research laboratory, had never met the woman who crossed the room and hugged him at a conference in New York City last fall. But she had a good reason. Just one year earlier June had saved her from a blood cancer that might have killed her within weeks.

    Lori Alf, now 50, was the first volunteer for a clinical trial using immunotherapy—which boosts the body’s natural defenses—to fight a blood cancer called multiple myeloma. The highly experimental trial was run by June at the University of Pennsylvania. To him, Alf had been only a code ending in 01, for the first anonymous patient. But with her hug, Alf personified the millions of cancer patients June and his colleagues hope to save by transforming a patient’s own immune system into a cancer killer.

    During his presentation later that day at the Multiple Myeloma Research Foundation’s Leadership Circle Summit, June choked up as he showed Alf’s before and after slides. In the former, round liquid myeloma tumors clog 95 percent of her bone marrow, crowding out all but a whisper of healthy cells—barely enough to survive. The second slide is completely clear. Alf’s cancerous blood tumors had vanished within a month or two after her treatment.

    How? Researchers withdrew some of her immune system’s white blood cells, transformed them in the lab into hunters retrained to track down and kill her cancer cells, then let them loose in her body.

    And it worked: Alf is cancer-free. That’s nothing short of a medical miracle.

    Findings by June and his researchers—as well as new studies—suggest that immunotherapy could fight more than two dozen types of cancer. Before too long, for example, blood cancer patients could walk into outpatient clinics and get one-time, 10-minute intravenous drips of their own commercially reprogrammed immune cells and become cancer-free within weeks.

    As for Alf: “I was supposed to die. But here I am,” she says. “I feel as if I was placed into one of the lifeboats from the Titanic—and I can’t help looking back and wishing that everyone on that ship could join me.”

    A Mean Cancer

    Before Alf got very lucky in the clinical trial, she was profoundly unlucky. Back in 2009, the smart, speed-talking mother of three, who runs an Olympic-class ice rink in West Palm Beach, Fla., thought she had a worsening case of bronchitis. Her family doctor delivered the bad news: Alf had multiple myeloma, a treatable but incurable blood cancer that affects fewer than 90,000 Americans—and rarely someone so young. She was 43.

    “I walked out to my car,” she says, “slumped down onto the hood and stayed there paralyzed, crying.”

    But within a week, she and her husband, Chris, took control of what became a seven-year ordeal to keep her particularly aggressive cancer in check. While many myeloma patients find therapies that allow them to enjoy the semblance of normal health for years, Alf lurched from one disappointing treatment to another, including a debilitating stem cell transplant using her own blood. Few therapies helped her for more than months at a time. “I had a really mean cancer,” she says.

    Her lead myeloma specialist, the renowned Kenneth C. Anderson, M.D., of Boston’s Dana-Farber Cancer Institute, agrees: “Lori had one of the worst myelomas I have ever seen.”

    By 2014, Alf was down to 80 pounds, with a grim prognosis.

    In desperation, she and her doctors were considering her second stem cell transplant, this time with cells from her sister, who is not a perfect genetic match. The doctors told her that the complications triggered if the transplanted cells attacked her body rather than helped her, known as graft-versus-host disease, could end her life.

    One Last Chance

    There was one other option. Scouring the internet, Alf and her husband discovered something so new even her own doctors hadn’t heard about it. University of Pennsylvania researchers were organizing clinical trials to reprogram the immune systems of seriously ill myeloma patients like her. Instead of using chemotherapy drugs to attack the cancer, immunotherapy assaults cancer cells from within with a patient’s own re-engineered immune system.

    Alf had long believed immunotherapy—which avoids graft-versus-host complications by using the patient’s own cells—held the key to curing her cancer. She was determined to get into the trial funded by drug manufacturer Novartis. She told her then-16-year-old daughter Caterina, a competitive figure skater, “If they reject me, you go into that doctor’s office and cry your eyes out. Make a scene until they change their minds.”

    But Alf was exactly the type of patient the researchers wanted—seriously ill with the abnormality they were targeting, yet hopefully strong enough to survive the rigors of the trial itself.

    The stakes were high: The immunotherapy hadn’t even been tested on animals. University researchers were moving fast to treat the seriously ill myeloma volunteers under expedited “breakthrough” status from the U.S. Food and Drug Administration and did not stop to experiment, even on mice.

    Alf was “the mouse model.”

    In a process similar to kidney dialysis, she was hooked up to a machine that collected a type of white blood cells called T cells. “Then the lab made my T cells very angry,” she explains. Actually, the lab transformed the T cells with a protein called chimeric antigen receptor (CAR) into what they hoped would be an army of hunters that would recognize her enemy myeloma cells and kill them.

    Twelve days later, the doctors dripped the red, garlicky-smelling CAR T cells into her veins. The next day, she felt sicker than ever. But that was about what the doctors had hoped: In their earlier trials with leukemia patients, when the immune system’s army overwhelmed the enemy, the warfare commonly triggered high fevers, nausea, muscle pain and sometimes serious neurological symptoms.

    Fortunately, Alf was spared the worst of that and was well enough to be discharged a week later to a nearby hotel. Still, she continued to feel “jittery, itchy, twitchy and miserably nauseous.”

    But her blood tumors decreased—then disappeared. “I’m sure we killed my mother cancer cells,” she says of the stem cells where cancer originates. More than one of her oncologists agree. Nothing else explains her remarkable recovery.

    “I cry from happiness every time I see her,” says Alf’s Florida oncologist Robert J. Green, M.D. “When she went to Pennsylvania, I didn’t think I’d see her alive again. Lori is proof that these new immunotherapies do magical things.”


    Nearly two years later, Alf’s blood is totally normal, and she’s finding joy in the smallest moments of life, “like the smell of my dog, Versace,” she says.

    Husband Chris marvels at the resilience of the woman he fell in love with 27 years ago. And their three teenagers cherish a bedtime ritual they started in childhood, Caterina says: “We all go in and kiss her goodnight.”

    Whenever possible, Alf shares her story at conferences and events in hopes of raising more research money for the treatment that saved her life.

    The mighty mouse roars on.

    The Health Care Detective™ Frank Lalli is producing a book for Simon & Schuster this fall on how to get affordable health care.

  7. #7


    Quote Originally Posted by Bobcat View Post
    It's time to take back our country from a big, bloated government.
    There are 130 million voters and maybe a few thousand who want their freedom. Hate to say it but this society is so corrupt and irrational that I can't the government getting anything other than worse in our lifetimes. We may even be teetering on the edge of an outright dictatorship.

  8. #8


    This is why there is concern that the bill is going to benefit Pharma, not doctors who are doing same day procedures which should be considered the practice of medicine.

    SA biotech company backing fed stem cell bill to cut red tape from regulatory pathway
    May 10, 2016,
    W. Scott Bailey
    Reporter/Project Coordinator
    San Antonio Business Journal

    San Antonio-based biotech firm StemBioSys Inc. has put its full support behind a proposed federal law that supporters contend will break down unnecessary barriers to medical innovation and accelerate advancements in regenerative medicine.

    One of the challenges in stem cell research, industry leaders contend, is that the Food and Drug Administration has yet to create a regulatory pathway for regenerative medicine that recognizes its uniqueness, impeding the delivery of potentially life-saving products. That hurdle has given companies in other countries, where the safety and efficacy of such treatments have already been acknowledged, a leg up on American entities like StemBioSys.

    Senate Bill 2689, also known as the REGROW Act, was jointly introduced by U.S. Senators Mark Kirk (R-Illinois), Joe Manchin (D-West Virginia) and Susan Collins (R-Maine). Backers said if approved, it could substantially promote more stem cell innovation, requiring the FDA to work with industry stakeholders to develop standards for stem cell research that could lead to the safe delivery of more regenerative medicine products.

    “Stem cells are a big frontier in medicine,” said Davis, one of the industry leaders who is working to drum up support for the bill.

    Davis recently participated on a national panel convened in Washington at the Bipartisan Policy Center to address the issue. He told me there has been a willingness from Texas political leaders on both sides of the aisle to weigh the merits of the proposed legislation.

    The bill would create more flexibility for companies like StemBioSys engaged in stem cell research and development.

    “For StemBioSys, the legislation would mean that the therapies we are beginning to look at could get to market much more quickly and efficiently than in today’s process,” said StemBioSys CEO Bob Hutchens.

    Davis said SB 2689 would be a game changer for regenerative medicine and for StemBioSys.

    “This would give us an opportunity to push forward our technology,” he said. “We are hopeful both sides can work together on this.”

    W. Scott Bailey covers health care, tourism, sports business, economic development; he also plans and edits some special reports.
    First treatment in 2007. Pioneering ever since.


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