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!

The eyes have it. Vanna Belton and Doug Oliver can see again after adult stem cell therapy.

Adult Stem Cells Restore Sight To The Blind

March 13th, 2016 – Stem cell patients are making news! PFSC brings together all the reports on Doug Oliver and Vanna Belton, both of whom have regained vision thanks to a pioneering trial led by Dr. Jeffrey Weiss.

First, THANK YOU! to all PFSC subscribers for responding to the FDA Part 15 Hearing registration drive. As hoped, the overwhelming response caused the FDA to postpone the April 13th hearing, and there will be a collaborative workshop w/ stake holders. We look forward to open dialog with the FDA. More info forthcoming as soon as we learn of it.

Now the leaders in the Senate are looking at ways to advance cellular therapy. Patients will be listened to as constituents. At the end of this post, look for information on how you can support this legislation, which is being introduced on March 15th, 2016. [Update: delayed until March 16th, so we have another day to email our Senators.]

Doug Oliver

Doug Oliver

To help readers understand what this legislation could do for the health of Americans, PFSC will run a series of posts highlighting the positive outcomes from clinical trials, and patient stories. For conditions ranging from orthopedics, to stroke and autoimmune diseases. Starting with this story on vision recovery previously thought to be impossible.

Doug Oliver’s vision has been deteriorating since he was 30 due to a form of macular degeneration called Autosomal Dominant Drusen. By his early 50’s, Doug was legally blind, and could no longer drive or see faces.

Doug’s story was covered by Nashville Fox Affiliate WZTV, in a year-long series of reports by Mikayla Lewis which tracked his recovery from legally blind to regaining his driver’s license. His vision changes are documented in his medical records from exams at Vanderbilt University Eye Institute; he has released these records to the public as part of his effort to spread awareness of this treatment.

Highlighted facts of Doug’s case:

  • May 2015, the time of his first television interview with FOX, (before treatment), his vision was 20/400 in the right eye and 20/2000 in the left.
  • Oct 2015, at the follow-up interview (after treatment in Aug 2015), his vision had improved to 20/40 and 20/40 visual acuity range, which is legal for driving.
  • Dec 2015, Doug received his driver’s license.
  • Mar 2016, his vision is 20/30 in his right eye and 20/40 in the left.
  • Mar 11th, 2016 Mikayla Lewis returns to see Doug taking his wife for a drive “Stem Cell Therapy Helps Blind Man See“.

In his latest interview, Doug addresses a common confusion. When people hear “stem cells” they think of therapies that utilize embryos. The reporter includes an explanation by former Senator Bill Frist, MD that research on use of embryonic cells is fading. Adult stem cells from one’s own tissues are proving to be the safer and more effective method.

Vanna Belton

Vanna Belton

Vanna Belton’s vision was 20/3000 in both eyes, after she lost her sight in a matter of weeks in 2009 due to a severe onset of optic neuritis, leaving her completely blind. Vanna is still considered legally blind, but following stem cell therapy in the SCOTS trial in 2014, she can now see enough to no longer need a white cane. Vanna’s case presentation has been published in Neural Regeneration Research Journal, and is currently being submitted for follow up peer-reviewed publication.

The reports on Vanna’s case span the range from positive, factual, to a little histrionic. Patients considering such an investment in experimental therapy that has not yet reached publication or approval need to have all the information available.

The Baltimore Sun report by Meredith Cohn, an experienced health journalist, falls into the balanced journalism category, exploring the pros and cons. The next two reports are from science blogs, recapping Meredith’s story. Science Alert echoes the balanced tone, while the negative terms like “renegade” indicate the Inverse blog is using sensational terms to attract viewers. Yet the author hedges her bets at the end with “Weiss’ work is important because it’s pushing the frontiers of science well beyond what we even think is possible.”

Is this press release the Principal investigator, Dr. Jeffrey Weiss, a board certified retinal surgeon and ophthalmologist, explains the Florida-based Stem Cell Ophthalmology Treatment Study (SCOTS). It is registered with the National Institutes of Health with identifier NCT01920867. It lists the conditions treated by SCOTS, as well as study contacts. The study uses the patient’s own stem cells obtained under anesthesia from the bone marrow. These cells are injected around and potentially in the eye.

In the above media reports Dr. Weiss states 278 patients have been treated, and 60 percent of his 278 patients with macular degeneration, glaucoma and other diseases have regained some sight.

Like many of the cellular therapies offered in the U.S. the SCOTS trial is a patient-funded clinical trial; in this case treatments cost $21,000, which insurance does not cover because the procedure is investigational. Doug raised the funds for the treatment from his local community and through a GoFundMe campaign. Vanna spent her wedding budget to pay for treatment and got married at the courthouse instead.

The SCOTS trial is tracking patient results and submitting early initial case reports like Doug and Vanna’s to peer-reviewed publications. This is the first step in the process leading to publishing the results on all of the patients to determine safety and efficacy. If results warrant it, the therapy could be approved. Multiple trials and many years are usually required for approval, but the new legislation being introduced to the Senate HELP Committee could speed this approval process.

MARCH 2016 CELLULAR THERAPY LEGISLATION INTRODUCED

Senators Mark Kirk and Susan Collins are poised to introduce this legislation, because they recognize the hardship placed on patients with difficult to treat diseases, who are left with no option but to raise funds for cellular therapy, and many have to travel outside the U.S. to received treatment.

Mar 16, 2016: The Kirk/Collins bill will be introduced to the Senate HELP Committee.
Apr 06, 2016: The bill is scheduled for markup (approximate date).

YOU CAN HELP PASS STEM CELL LEGISLATION

Click TAKE ACTION for step by step instructions and draft letters you can email your Senators today. Your story can make it clear why this bill will help patients, whether you are a patient, or know someone who needs stem cells. If you are in a state that has a Senator on the HELP Committee, we urge you to schedule an in person meeting during recess March 27th-April 4th.

Patient For Stem Cells will be monitoring this legislative process and reporting developments as they happen. This may finally bring together all the stake holders, the regulators, legislators, researchers, physicians and patients, so that cellular therapy finally gets the high priority that a breakthrough therapy deserves.

Please use the share buttons below spread this post far and wide so our Senators get plenty of emails!

If you have a stem cell story you’d like us to cover, or wish to report on your interaction with your Senator, please Contact PFSC.

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FDA Public Hearing On Stem Cell Access – Registration Deadline Jan 8th

Wear this T-Shirt to the FDA hearing!  Available from  Bioinformant.

Wear this T-Shirt to the FDA hearing!
Available from Bioinformant.

Update: Over 600 event registrations arrived at the FDA, causing cancellation of the hearing. Thank you everyone, your voice is being heard! For all the details, see the 3/16/2016 BioInformant post.
———————–
The FDA has announced a 1 day public hearing to obtain input on the four recently issued draft guidances relating to the regulation of stem cell therapy in the U.S. The restrictive proposed regulations will prevent countless patients from receiving this life-changing treatment. The FDA will consider information from medical professional as well as the public/patients on the finalization of these guidances on April 13, 2016.

Read examples here of how stem cell therapy has been making news for saving patients from disabling, even life threatening illness. The FDA regulations would further limit or stop access to these treatments in America.

WE NEED TO LET OUR COLLECTIVE PATIENT VOICE BE HEARD!!!

Step 1: Register for the FDA Public Meeting

April 13, 2016 8:00 a.m. – 5:00 p.m.
FDA’s White Oak Campus
10903 New Hampshire Avenue
Silver Spring, Maryland 20903
Directions
To register by Jan 8th 2015, send an email to: CBERPublicEvents@fda.hhs.gov

Please register even if you can’t attend in person. We are requesting webinar access for those who cannot travel.
Provide the following info in your email.

1. Full name
2. Titles
3. Affiliation
4. Address
5. Email
6. Phone number
7. Your comment

For Affiliation, here are some suggestions:
• Patients For Stem Cells
• Alliance for the Advancement of Cellular Therapies (AACT)
• Stem Cell Pioneers

Comments are not required, but you can include one about your concerns. Please save it for submitting to the Federal Register. Instruction on how to do this will follow.
Here are some comment examples:

As a “No Option Patient” with a disabling chronic disease, I would like the option to be treated with my own cells, especially when all other FDA approved therapies have failed me. Please consider this when rendering your decision on the minimal manipulation of stem cells.

As a patient who has received cell therapy with quality of life benefits I can attest I wouldn’t be alive today if it wasn’t for the adult stem cell therapy I have received. I should have the right to this therapy here in the U.S. without having to travel offshore.

I am a caregiver of a once vibrant, active and intelligent human being. It is heartbreaking to watch the deterioration of a loved one who could potentially benefit from cell therapy. This a travesty beyond measure.

Step 2: Inform your representative

The FDA is a non-elected appointed regulatory body. Citizens have little recourse to object to the control they are trying to take of the practice of medicine. Please inform your elected Representatives of what is happening.

To find your Representative’s contact and email info, click here.

Step 3: Become an informed advocate

Here is the FDA public notice on the meeting:
Federal Register Notice: Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue-Based Products; Public Hearing; Request for Comments

You will notice that no attempt is made to translate the technical medical jargon into plain language. That’s why we are very lucky to have the support of AACT, whose directors are some of the leading cellular medicine physicians in the world. The healing they have witnessed in the patients they have treated has motivated them to stand up the FDA.

AACT will be providing updates and further education on the FDA’s actions, as well as further instructions on how to submit comments to the FDA before and after the April 13th meeting. Citizen/Patient Membership is free, but consider a donation if you can.

To join AACT click here: AACT Membership

Here are the topics AACT will be addressing:

    1) Overview of regenerative cells and potential usefulness to medical practice, explaining the scientific data available for us of non-embryonic stem cells, their safety and ethical acceptance.
    2) Forcing US citizens to leave home as medical tourists for untested therapies.
    3) Importance of the physician’s role in the “Practice of Medicine” and the physician patient covenant.
    4) Patient’s Bill of Rights including access to all potential treatments regardless of costs.
    5) Patient’s “Right to Try” laws and the off label use of regenerative cells.
    6) Economic (a) and Academic loss (b) associated with restrictive regressive regulations
    a) Allowing for patient funded clinical studies in the US.
    b) Establishing general and disease specific patient registries designed to collaboratively advance the science and clinical protocols.
    7) Bioethical concerns associated with delaying/denying the aggressive testing and development of quality of life improving and life saving therapies.
    8) The de facto exclusions of these therapies for children in the US.

Thank you for your efforts!