♦ Only 4 Adverse Events Reported Against 570 Stem Cell Clinics

A Freedom Of Information Act (FOIA) request to the FDA has revealed only 4 reports of adverse events against a list of 570 U.S. stem cell clinics.

This low number of FOIA reports demonstrate that:

  • There is clearly not an established pattern of harm.
  • There is no significant trend of harm, just fear of harm.
  • Fear of harm is not a basis for crafting health policy. Proof of harm is as essential as proof of safety.

Pervasive hype, both overly positive and overly negative hurts us all. Dr. Feel Good and Chicken Little have no place at the table of social or health policy of such gravity to our lives and the lives of those we love.

Here is the response to the request, followed by links to the 4 reports.

Cc: “ORAHQ ACRA ESS FOI-DISCLOSURE STAFF”
Sent: Monday, August 22, 2016 2:45:07 PM
Subject: FOI Request 2016-6466

8/22/2016

Dear Requestor,
The attached records are being provided by the Office of Regulatory Affairs (ORA)Information Disclosure in response to your request #2016-6466 dated 08/08/16 for records from the Food and Drug Administration pursuant to the Freedom of Information Act regarding:

ADVERSE EVENTS RE CELLULAR THERAPIES

Your request is granted in part.

After a thorough review of the responsive records, we have determined that portions of the documents are exempt from disclosure under FOIA exemptions (b)(4) and (b)(6) of the FOIA 5 U.S.C. § 552, as amended and delineated below:

Exemption (b)(4) permits the withholding of “trade secrets” (TS) and “commercial confidential information” (CCI). Disclosure of this information would impair the government’s ability to obtain necessary information in the future and cause substantial harm to the competitive position of the person from whom the information was obtained. Under the balancing test of this exemption, we are withholding all proprietary information identified as TS and CCI.

Exemption (b)(6) permits the withholding of information which, if released, would constitute a clearly unwarranted invasion of personal privacy. In this case, it was determined that there is no countervailing public interest qualifying under the standard set forth, under exemption (b)(6), to release the personal identifying information of certain third parties.
ORA considers your request closed. If you have any questions about this response, you may contact Anna Postell at 301-796-6488.

Sincerely,
Anna Postell
Program Analyst

These are the adverse event reports:
APPLIED_Consumer Complaint 127882_Redacted.pdf
APPLIED_Consumer Complaint 131004_Redacted.pdf
APPLIED_Consumer Complaint 141021_Redacted.pdf
APPLIED_Consumer Complaint 144501_Redacted.pdf

Note: The request was granted in part, This doesn’t mean there are more than 4 records and the FDA is withholding them. It means the privileged types of information (b)(4) and (b)(6) are not provided, which you can see have been redacted in all of the 4 records.



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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!