CIRM Funded Trial for Parkinson’s Treats First Patient

Dr. Krystof Bankiewicz

Brain Neurotherapy Bio, Inc. (BNB) is pleased to announce the treatment of the first patient in its Parkinson’s gene therapy study.  The CIRM-funded study, led by Dr. Krystof Bankiewicz, is one of the 64 clinical trials funded by the California state agency to date.

Parkinson’s is a neurodegenerative movement disorder that affects one million people in the U.S alone and leads to shaking, stiffness, and problems with walking, balance, and coordination.  It is caused by the breakdown and death of dopaminergic neurons, special nerve cells in the brain responsible for the production of dopamine, a chemical messenger that is crucial for normal brain activity.

The patient was treated at The Ohio State University Wexner Medical Center with a gene therapy designed to promote the production of a protein called GDNF, which is best known for its ability to protect dopaminergic neurons, the kind of cell damaged by Parkinson’s. The treatment seeks to increase dopamine production in the brain, alleviating Parkinson’s symptoms and potentially slowing down the disease progress.

“We are pleased to support this multi-institution California collaboration with Ohio State to take a novel first-in-human gene therapy into a clinical trial for Parkinson’s Disease.” says Maria T. Millan, M.D., President and CEO of CIRM.  “This is the culmination of years of scientific research by the Bankiewicz team to improve upon previous attempts to translate the potential therapeutic effect of GDNF to the neurons damaged in the disease. We join the Parkinson’s community in following the outcome of this vital research opportunity.”

CIRM Board Member and patient advocate David Higgins, Ph.D. is also excited about this latest development.  For Dr. Higgins, advocating for Parkinson’s is a very personal journey since he, his grandmother, and his uncle were diagnosed with the disease.

“Our best chance for developing better treatments for Parkinson’s is to test as many logical approaches as possible. CIRM encourages out-of-the-box thinking by providing funding for novel approaches. The Parkinson’s community is a-buzz with excitement about the GDNF approach and looks to CIRM to identify, fund, and promote these kinds of programs.”

In a news release Dr. Sandra Kostyk, director of the Movement Disorders Division at Ohio State Wexner Medical Center said this approach involves infusing a gene therapy solution deep into a part of the brain affected by Parkinson’s: “This is a onetime treatment strategy that could have ongoing lifelong benefits. Though it’s hoped that this treatment will slow disease progression, we don’t expect this strategy to completely stop or cure all aspects of the disease. We’re cautiously optimistic as this research effort moves forward.” 

Other trial sites located in California that are currently recruiting patients are the University of California, Irvine (UCI) and the University of California, San Francisco (UCSF). Specifically, the Irvine trial site is using the UCI Alpha Stem Cell Clinic, one of five leading medical centers throughout California that make up the CIRM Alpha Stem Cell Clinic (ASSC) Network.  The ASSC Network specializes in the delivery of stem cell therapies by providing world-class, state of the art infrastructure to support clinical research.

For more information on the trial and enrollment eligibility, you can directly contact the study coordinators by email at the trial sites listed:

  1. The Ohio State University: OSUgenetherapyresearch@osumc.edu
  2. University of California, San Francisco: GDNF@ucsf.edu
  3. University of California, Irvine: chewbc@hs.uci.edu

The Most Important Gift of All

Photo courtesy American Hospital Association

There are many players who have a key role in helping make a stem cell therapy work. The scientists who develop the therapy, the medical team who deliver it and funders like CIRM who provide the money to make this all happen. But vital as they are, in some therapies there is another, even more important group; the people who donate life-saving organs and tissues for transplant and research.

Organ and tissue donation saves lives, increases knowledge of diseases, and allow for the development of novel medications to treat them. When individuals or their families authorize donation for transplant or medical research, they allow their loved ones to build a long-lasting legacy of hope that could not be accomplished in any other way.

Four of CIRM’s clinical trials involve organ donations – three kidney transplant programs (you can read about those here, here and here) and one targeting type 1 diabetes.

Dr. Nikole Neidlinger, the Chief Medical Officer with Donor Network West – the federally designated organ and tissue recovery organization for Northern California and Nevada – says it is important to recognize the critical contribution made in a time of grief and crisis by the families of deceased donors. 

“For many families who donate, a loved one has died, and they are in shock. Even so, they are willing to say yes to giving others a second chance at life and to help others to advance science. Without them, none of this would be possible. It’s the ultimate act of generosity and compassion.”

The latest CIRM-funded clinical trial involving donated tissue is with Dr. Peter Stock and his team at UCSF. They are working on a treatment for type 1 diabetes (T1D), where the body’s immune system destroys its own pancreatic beta cells. These cells are necessary to produce insulin, which regulates blood sugar levels in the body.

In the past people have tried transplanting beta cells, from donated pancreatic islets, into patients with type 1 diabetes to try and reverse the course of the disease. However, this requires islets from multiple donors and the shortage of organ and tissue donors makes this difficult to do.

Dr. Stock’s clinical trial at UCSF aims to address these limitations.  He is going to transplant both pancreatic islets and parathyroid glands, from the same donor, into T1 patients. It’s hoped this combination approach will increase beta cell survival, potentially boosting long-term insulin production and removing the need for multiple donors.  And because the transplant is placed in the patient’s forearm, it makes it easier to monitor the effectiveness and accessibility of the islet transplants. Of equal importance, the development of this site will facilitate the transplantation of stem cell derived beta cells, which are very close to clinical application.

“As a transplant surgeon, it is an absolute privilege to be able to witness the life-saving organ transplants made possible by the selfless generosity of the donor families. It is hard to imagine how families have the will to think about helping others at a time of their greatest grief. It is this willingness to help others that restores my faith in humanity”

Donor Network West plays a vital role in this process. In 2018 alone, the organization recovered 702 donor samples for research. Thanks to the generosity of the donors/donor families, the donor network has been able to provide parathyroid and pancreas tissue essential to make this clinical trial a success”

“One organ donor can save the lives of up to eight people and a tissue donor can heal more than 75 others,” says Dr. Neidlinger. “For families, the knowledge that they are transforming someone’s life, and possibly preventing another family from experiencing this same loss, can serve as a silver lining during their time of sorrow. .”

Organs that can be donated

Kidney (x2), Heart, Lungs (x2), Liver, Pancreas, Intestine

Tissue that can be donated

Corneas, Heart valves, Skin, Bone, Tendons, Cartilage, Veins

Currently, there are over 113,000 people in the U.S. waiting for an organ transplant, of which 84 % are in need of kidneys.  Sadly, 22 people die every day waiting for an organ transplant that does not come in time. The prospect of an effective treatment for type 1 diabetes means hope for thousands of people living with the chronic condition.