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Take action to protect your rights to use your own stem cells

♦ FDA wants to Stop Stem Cell Therapy AND Breast Reconstruction -Make Comments by Sept. 27th 2016

fdahearingUPDATE: Congratulations everyone! We submitted 6,952 comments! Click HERE to read the comments.

Sept. 24th, 2016 – Cellular therapy is a disruptive medical innovation which is revealing a surprising truth: our own bodies are the best source of medicine ever discovered. This is challenging many of the conventional therapies, such as drugs and surgery. The FDA’s regulatory approach starting in 1993 has been inhibitory to the wider adoption of this therapy. The current proposals, which seek to classify certain stem cell procedures as a prescription drug, which will only insure that more patients are forced to pursue medical tourism.

If you you think you or a loved one might ever need to use their own stem cells, for example to avoid a knee replacement, then please take a few moments to make comments to the FDA by Sept. 27th. Instructions provided below.

The FDA has presented 4 draft guidances, with the goal of shutting down stem cell clinics in the U.S. Readers of the PFSC blog helped start the citizen feedback process earlier this year when over 600 registrations for the FDA Part 15 hearing overwhelmed the agency. The hearing had to be postponed until Sept 12-13th 2016 and expanded to 2 days, which only allowed 5 minutes each for the 100 people who registered to speak. These presentations from scientists, cellular therapy providers and patients were mostly in opposition to the guidances. The legal and scientific details are available below in links to the testimony and articles.

FDA PROPOSED BAN ON BREAST RECONSTRUCTION TECHNIQUE
The FDA was roundly criticized for proposing that not only your stem cells, but also your fat, should be classified as a drug, requiring a decade of trials. Rick Jaffe, a lawyer with 3 decades of healthcare legal experience, raises the alarm in this passionate piece.

THE SCARIEST ATTACK ON PERSONAL FREEDOM YOU’VE NEVER HEARD OF/ THE BIG TAKEWAY: If the FDA gets its way, the most popular post mastectomy breast reconstruction procedure will become illegal, and so will many other life enhancing procedures – Richard Jaffe, Esq. The huffington Post, 09/18/2016

HOW TO MAKE COMMENTS TO THE FDA BY SEPTEMBER 27, 2016

Click HERE to submit your comments o FDA.

Tell your own story if you have had stem cell therapy, or think you want it in the future.
In your own words (or FDA will reject it) make the following points, if you support continued access to your cells:

1. Withdraw the four HCT/P guidance documents, and work with the experts on solutions
2. Stop trying to regulate a person’s use of their own body parts
3. Continue access to responsible stem cell therapy that uses the patients’ own tissue

A great deal of media attention has arisen, so you may want to save your comment, and also post it as a comment on the articles we’ve included below.

Click HERE to read comments submitted by others.

UPDATE: An amazing 6,952 comments were submitted by the deadline of Sept. 27th. FDA has never received more than 20 comments on previous guidances.

Click HERE to review the speaker agenda for the two day hearing.
Click HERE to view Day 1 of the hearing.

1:48:00 Mary Ann Chirba (Boston College)
1:53:00 and 2:58:40 Arnold Caplan (Case Western Reserve)
1:59:10 and 2:53:00 Keith March (Indiana University)

These three presenters are leaders in the field. Caplan was the first to discover the healing abilities of adult stem cells. In FDA Draft Stem Cell Guidance Documents Exposed as Improper Rulemaking, Bad Science and Heartless Public Policy Richard Jaffe comments “…these presenters and especially March and Caplan showed that the FDA’s view was biologically unsound.”

Alexey Bersenev (Yale University) posted an objective scientific review of the hearing, also noting the import of March and Caplan’s input,
Historic FDA public hearing on regulation of cell therapy in US

Click HERE to view Day 2 of the hearing.
Click HERE for a patient presentation provided by SammyJo Wilkinson.

In the last presentation at the hearing, SammyJo explains how her own stem cells put long-term multiple sclerosis into remission and recovery since 2014. She also echoed the proposals for solutions presented, such as the conditional approval plan and patient registry proposed by Arnold Caplan, Janet Marchbroda (BPC) Day 1 4:16:00, and Leslie Miller (AACT) Day 1 3:23:58.

Editorial – Improving the regulatory framework for cell therapy does not equate to deregulation
Caplan A. I., Ricordi C., CellR4 2016; 4 (4): e2109

Many moving presentations were made by patients on Day 2, about lives restored by cellular therapy from illnesses that had no effective treatment options. In our next post we will explore how the media has covered the FDA regulatory story, and how the patient experience is finally being included.

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♦ Will Real World Patient Evidence Have An Impact On Stem Cell Legislation?

Patients For Stem Cells (PFSC) encourages you to listen to this important webcast regarding the medical innovation bills that are currently before Congress, including the REGROW legislation for cellular therapy. This legislation will determine whether patients have access to life saving cellular therapies in the U.S., or must continue traveling to offshore clinics.

Real World Evidence for Safe and Effective Cures: Medical Innovation for All Americans
The webcast was hosted by the Bipartisan Policy Center (BPC) on June 23, 2016 9:00.

Click here to listen to Webcast, and scroll down the page to access the videos.

We previously covered the impact that Doug Oliver made with his Senator, Lamar Alexander (R-TN). Oliver told his story of recovery from blindness thanks to his own stem cells to Alexander, who is Chairman of the Senate Health, Education, Labor and Pensions Committee. The HELP Committee will be voting in the near future on stem cell legislation like the REGROW bill, which outlines an accelerated approval pathway to cut time and cost of the approval pathway in half. Both Alexander and Oliver will be on the BPC panel.

While PFSC has supported this effort to accelerate approval of cellular therapy, we would like to add two points to the discussion, under the declaration:

Our Cells Are Not Drugs!

1. The REGROW bill reaffirms the FDA’s erroneous jurisdiction over the use of a patient’s own stem cells.

The classification of one’s own stem cells as a drug, makes this therapy subject to the extended drug approval pathway that currently costs $1 billion dollars and takes 10 years. While the bill offers to cut this in half, it is based on the wrong premise that our own stem cells should be considered a drug. This type of therapy is more akin to banking your own blood for a later transfusion. PFSC documented this erroneous regulatory position taken by the FDA in 2012 in The History of FDA Control of Your Body.

Recently, this was further explored by Richard Jaffe, a health care litigator who has worked on many of the most significant, high profile heath care cases of the past twenty plus years, and helped organize and testified at several Congressional Hearings on various issues related to access to experimental medicine.

In an essay titled S 2689: Congress to the rescue on stem cells? (NOT) Richard explains why the REGROW bill would allow the FDA to prohibit some same day autologous stem cell procedures.

“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.”

An example is the orthopedic treatments currently offered at over 800 clinics across the U.S. This includes procedures where your own stem cells are injected into an injured arthritic hip or knee, at a cost usually under $5,000. Instead of a knee or hip replacement with costs $50,000 to $100,000 and has more risk, pain and poorer outcomes. Putting this on the drug approval pathway would delay access and multiply the cost of a procedure that has already successfully helped thousands of patients avoid disability and stop pain.

eyesTherapies already in use and accumulating safety and effectiveness data should not have to go back to the starting line. This point is clearly underscored by this Fox News article which points out the current move by the FDA further restrict cellular therapy would make cataract treatments illegal!
Cell therapy reversed blindness for 47,000 patients in 2015. So why is it against the law?

2. Just because stem cells are expanded, that doesn’t turn them into a drug.

WalkerswIn January 2016 MD Monthly Magazine published an opinion piece, by one of the founding members of PFSC, SammyJo Wilkinson. She recounted the life-saving cellular therapy she had in 2012 from Houston-based biotechnology company, Celltex Therapeutics. Celltex took her adipose-derived stem cells and cultured them in their state-of-the-art, FDA-regulated laboratory, to reach a therapeutic dose in the hundreds of millions of mesenchymal stem cells, or MSCs. Her first round of therapy was administered in Houston. However, shortly after, the FDA made a ruling that expanded (cultured) adult stem cells constituted a biological drug. Celltex was forced to move therapy to Cancun, Mexico, where SammyJo continued therapy after a year delay.

While Celltex continues to operate a best-in-class cGMP laboratory in Houston, the company exports cells to Mexico, in accordance with the Federal Commission for Protection against Sanitary Risk (COFEPRIS), which is considered the regulatory equivalent of the FDA in Mexico. Cells are used by licensed physicians at Hospital Galenia for therapeutic applications.

Adult stem cell therapy spared SammyJo from 20 years of disabling multiple sclerosis, and impending nursing home care. By 2014 her disease was in remission. Today she is still living at home with her husband. She is pain-free, and four years into recovery. Recently, she reached a milestone with the ability to stand up from her wheelchair and walk 100 feet with a walker.

In her article she quoted one of her long time stem cell heroes, Dr. Andrew von Eschenbach, former FDA director, who is also speaking at the June 23rd BPC Panel discussion. He has gone on record stating:

“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them. Take regenerative medicine… after proof of concept and safety testing, a product could be approved for marketing with every eligible patient entered in a registry so the company and the FDA can establish efficacy through post-market studies.”

The FDA ruling in 2012 claiming that expanded cells are “more than minimally manipulated” was not supported by scientific evidence, and the FDA ignored the fact that the expanded cells that saved SammyJo’s life were identical to the original cells taken from her body.

PFSC is happy to see Real World Patient Evidence given credit, and is happy to add the many success stories we have documented. This is what motivates our grassroots advocacy effort to make this therapy available to all Americans. The current legislative effort needs to take our perspective on FDA over regulation into account. We herewith submit this article as patient testimony to the Congressional hearings on regenerative medicine therapy.

If you agree with the opinions in this article. please express this to your legislators by sending an email in your own words from the one click email for at CellTherapyNow.org

SammyJo Wilkinson
Member, Patients For Stem Cells

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♦ Patients Support REGROW Stem Cell Legislation

Senator Mark Kirk

Senator Mark Kirk says patients should not have to go out of country for stem cell therapy.

In March we reported on two patients who had regained vision following stem cell therapy in the U.S. We also asked readers to contact their Congressional Reps in support of legislation to accelerate regenerative medicine, aptly named the REGROW Act, submitted to the Senate HELP Committee by Sen. Mark Kirk (R-ILL). Thanks in part to patient activism, the bill made it through the April 6th Senate markup session. Doug Oliver, one of the patients in the vision story, made a real impact with his patient advocacy. He is a constituent of Senator Lamar Alexander (R-TN). They met and discussed stem cells just before the Senate session, and the Senator recounted Doug’s stem cell treatment success!
Senator Lamar Alexander

Senator Lamar Alexander recounts a constituents stem cell success.

 
An interview with Doug Oliver was just published by USC Center for Health Journalism, ‘Using Stem Cells to Reverse Vision Loss‘.

StefanieStefanie Cowley is another patient advocating for REGROW. Within days after receiving stem cell therapy, client Stefanie’s pain level due to Multiple Sclerosis was reduced. Watch this news story to learn more about her journey to improve her quality of life.

The REGROW legislation of 2016 (S. 2689 / H.R. 4762) is bipartisan, bicameral legislation supporting faster adoption of adult, non-embryonic cellular therapy. Senator Kirk is joined by the following legislators as co-sponsors: Joe Manchin (D-WV), Susan Collins (R-ME), Representatives Mike Coffman (R-CO), Mark Takai (D-HI) and Morgan Griffith (R-VA).

03/24/2016 Fighting diseases through regenerative medicine by Rep. Michael Coffman
04/05/2016 REGROW: US Senator Mark Kirk champions hope for millions

How You Can Help Pass the REGROW Legislation

It is important to keep our voices as patients involved in the decision making process. After all, we are the primary stakeholders. Our health and well being is at stake. You can also request an in-person meeting with your Congressional reps, just like Doug Oliver had with Sen. Alexander.

CellTherapyNow.org is a website set up by supporters of REGROW. There is a webform to send email to your all your Representatives with one click, urging them to support REGROW. Also review the list of supporting organizations.

Become An Informed Advocate

Advancing a New Policy Framework for Regenerative Cell Therapy
The Bipartisan Policy Commission held a public panel discussion on 4/12/2016 with cellular therapy experts and patients, on the REGROW legislation.
Scroll down the page to watch the videos, including Arnold Caplan, PhD, whom we can all thank for his monumental discovery of the Mesenchymal Stem Cell. He has also been a leading proponent of an accelerated approval pathway.

These are some of the questions that are being addressed as the legislation takes shape.

1. The practice of medicine is exempted from 351 or 361 by 21 CFR 1271.15(b) (same surgical procedure exemption)
Does the REGROW legislation protect the POM pathway as it exists today, i.e. can I still go to an orthopedist and get my arthritic knees or torn tendons treated with a) same-day bone-marrow aspirate b) same-day SVF from adipose tissue or c) platelet rich plasma (PRP).
or, will these procedures be subject to the same new conditional approval pathway, requiring the physician to process my cells in a cGMP lab, file an IND, complete phase 1-2 FDA trials, enter conditional approval for 5 years, then file for a BLA drug license with the FDA, all of which will cost millions of dollars, and increase the cost of same day stem cells.

The same question applies to clinics treating COPD and other lung conditions with same day stem cells from bone marrow and adipose tissue.

2. Some same-day clinics offer treatment for serious neruo-degenerative conditions like ALS, MS and Parkinson’s, but all the University trials for these conditions are using higher dose expanded mesenchymal stem cells. Will same-day treatments for these more serious conditions have to follow the conditional approval pathway, similar to high-dose trials?

3. This question is for Joshua M. Hare, MD University of Miami
The best way to understand the impact of REGROW legislation is to look at specific trials. University of Miami has several cardiac trials in ph 1/2. If a trial completes ph 2 and is eligible for conditional approval, what would that mean to patients? Would we be able to pay to receive the therapy, and what might the cost be?

4. This question is for Professor Arnold Caplan
Sen. Kirk has mentioned that this bill would stop the flow of medical tourists out of the U.S. but there is one specific therapy that is available in the U.S. at a cost of $125,000, the HSCT trial at Northwestern University, for autoimmune disease like MS and CIPD. Insurance is even covering it for some patients, but those who do not qualify, or can’t afford it, are seeking the same treatment in Russia for $45,000 or Mexico for $55,000. How will the REGROW legislation help these patients so they don’t have to leave the country?

5. How will the REGROW legislation affect the 4 draft guidances the FDA was going to hold a Part 15 public hearing for on April 15th, 2016, that has been postponed until end of year.

Who is the opposition?

The opposition so far comes from Paul Knoepfler, Associate Professor at UC Davis.
04/10/2016 Alliance for Regenerative Medicine (ARM) Opposes REGROW Act, Danger to Patients Cited

Paul is an academic researcher investigating Induced Pluripotent Stem Cells (iPSC), a promising technique of manipulating skin cells into any type of cell required for repair. But this is still years away from clinical trials. Paul also blogs about patients trying to gain access to cellular therapy. In this post he is joined in concerns about this legislation by The Alliance for Regenerative Medicine (ARM), a lobbying organization for cellular companies primarily focused on cell products manufactured from donor cells, which truly fit under the category of cells=drugs, and require the traditional drug testing pathway to ensure safety.

One has to consider if their concerns are more about patient safety, or the threat to their academic and commercial interests, which could be threatened by patients gaining faster access to their own stem cells.

However, there is a place for off-the-shelf allogeneic (from a donor) cellular therapy, for conditions that prohibit using one’s own adult (autologous) stem cells. Such as graft-vs-host-disease, where the immune system is in such crisis that healthy donor cells are required. Such a GVHD product from Mesoblast has already been approved under Japan’s Regenerative Medicine Act, including insurance coverage during conditional approval. Another case is acute conditions like heart attack or stroke, where the treatment must be administered immediately, and autologous extraction would cause too much stress for a critical condition patient.

There are plenty of sick patients so there’s plenty of market share for all types of cellular therapies. Personalized medicine using one’s own autologous stem cells is the farthest along in human trials for both safety and effectiveness. Until other approaches catch up, this method should be the logical focus to save lives and alleviate suffering.

We will keep you up to date on the progress of REGROW legislation as it unfolds!