FDA & Clinical Trial Process

The FDA & Clinical Trial Process For Regenerative Medicine (Stem Cell- Based) Therapies

(updated 02-22-2021)

This page is dedicated to providing information about the FDA and regenerative medicine therapies specifically, the research project launched by Summit for Stem Cell Foundation: autologous dopamine neuron replacement therapy for Parkinson’s disease.


Summit for Stem Cell Foundation was founded to support, educate, and promote the autologous dopamine neuron replacement therapy for Parkinson’s disease research project guided by principal investigator, Dr. Jeanne Loring and by, director of research and development, Dr. Andres Bratt-Leal. The organization was successful and to hasten the research project’s path from bench to bedside, transferred the brilliant body of science to a private company, Aspen Neuroscience, Inc. Summit has a good relationship with “Aspen” and continues to monitor the cell-based therapy’s progress.

Regenerative medicine is a new field. The research project working to develop an autologous dopamine neuron replacement therapy for Parkinson’s disease is the first of its kind globally. The scientific team has been working with FDA consultants and the FDA throughout the research project’s development and have established a good working relationship. There have been no FDA precedents or guidelines to follow. Studies leading to the clinical trial process and the clinical trial process are subject to the discretion of the FDA for patient safety.


The FDA has created a “Framework for the Regulation of Regenerative Medicine Products” (05/21/2019) addressing how the agency plans to support and expedite the development of regenerative medicine products. The guidance documents underscore the agency’s commitment to help bring new and innovative treatment options to patients with unmet medical needs.

Regulatory Considerations for Human Cell, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use

Same Surgical Procedure Exception: Questions and Answers Regarding the Scope of the Exception

The FDA also published two final guidances intended to aid in the effort to bring innovative, safe, and effective products to patients as efficiently as possible:

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions

Evaluation of Devices Used with Regenerative Medicine Advanced Therapies


The typical timeline for a pharmaceutical drug to be publicly accessible:

  • 12+ years for a pharmaceutical drug to receive FDA approval to begin clinical trials.
  • Average cost of US$350 million.

Once FDA approval is received, the “investigative new drug” then begins three phases of clinical trials:

  • Phase I – Establishing a drug’s safety and profile (approximately 1 year).
  • Phase II – Assess a drug’s effectiveness (approximately 2 years).
  • Phase III – Determining a drug’s effectiveness and identify adverse reactions (about 3 years).

Once Phases I,II, & III are completed

  • A 100,000+ page application is submitted
  • 2.5+ years additional to processing

Only then is final FDA approval given for physicians to prescribe.


Is the FDA doing anything to speed up the process and keep up with fast-paced scientific discoveries? In a word – yes.

The FDA is working to meet the unmet needs of people with diseases having no cure. Parkinson’s disease is a disease with unmet medical needs. Although the induced pluripotent stem cell-based therapy has not received its classification as of this date, the FDA has developed new policies to address the pace at which scientific discoveries in regenerative medicine are being made today.

Aspen Neuroscience is currently working with a newer FDA division called: “Center for Biologics Evaluation and Research (CBER)”.

CBER is working with the Regenerative Medicine field to develop new policies to “expedite” Regenerative Medical therapies availability. The following policies and programs have been created in an effort to meet patient’s needs and the challenging pace of new scientific discoveries:

Fast Track Designation: to facilitate the development and expedite review of drugs and biologics intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

The 21st Century Cures Act: modifies the FDA Approval process. The development and bring new innovations and advances to patients who need them faster and more efficiently.

RMAT designation: Regenerative Medicine Advanced Therapy (RMAT): a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:

  1. The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products meeting FDA criteria.
  2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  3. Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

Right To Try: This law is a way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.

The Right to Try Act permits/allows eligible patients to have access to eligible investigational drugs. An eligible patient is a patient who has:

  • Been diagnosed with a life threatening disease or condition
  • Exhausted approved treatment options
  • Unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
  • And has provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician

An eligible investigational drug is an investigational drug:

  • For which a Phase 1 clinical trial has been completed
  • That has not been approved or licensed by the FDA for any use
  • For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA.
  • Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA.

​If you are interested in Right to Try, you should discuss this pathway with your licensed physician.

For more information on Right to Try: View More

Compassionate Use or Expanded Access: Extended Use, sometimes call “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

  • Expanded access may be appropriate when all the following apply:
  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
  • Patient enrollment in a clinical trial is not possible.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

If you are interested in Expanded Access or Compassionate Use, you should discuss this pathway with your licensed physician.

For more information on Compassionate Use: View More