Q Therapeutics Approved to Start Trial of Cell Therapy Aiming to Restore Myelin

Written by Patricia Silva, PhD | July 26, 2017

Clinical trial approved for cell therapy

A cell therapy intended to boost myelin regeneration — Q-Cells by Q Therapeutics — has received a green light from the U.S. Food and Drug Administration to proceed with a clinical trial in patients with transverse myelitis (TM), a disease that like multiple sclerosis is characterized by myelin damage.

FDA approval of the company’s Investigational New Drug (IND) application allows researchers to start a Phase 1/2 clinical trial in which nine patients will receive increasing doses of the treatment.

Q Therapeutics hopes trial results will support further development of Q-Cells to treat other diseases that lack healthy glial cells, for instance, the myelin-producing oligodendrocytes in multiple sclerosis.

Q-Cells are what researchers call glial-restricted progenitor cells. In plain language, it means that they are stem cell-like cells that are destined to become glial cells, which in the brain and spinal cord perform numerous and indispensable functions, including myelin formation.

“This is another milestone in our quest to bring effective treatments for devastating CNS [central nervous system] diseases and injuries to the clinic,” Steven Borst, CEO and chairman of Q Therapeutics, said in a press release.

In the study, “Transplanted human glial-restricted progenitors can rescue the survival of dysmyelinated mice independent of the production of mature, compact myelin,” published in the journal Experimental Neurology, Piotr Walczak and colleagues at Johns Hopkins University showed that mice without myelin survived for longer when treated with the cell therapy.

“We have long believed that Q-Cells’ unique ability to repair and support CNS nerve cells is fundamental to treating many CNS disorders. The ability of these cells to replicate once injected, migrate, differentiate into mature glial cells and repair myelin, as demonstrated by Dr. Walczak’s lab, further highlights the power of this therapeutic approach,” said Mahendra Rao, MD, PhD, Q Therapeutics’ scientific co-founder and chief strategy officer.

Interestingly, the study reported that benefits did not correlate in time with the formation of new myelin. Animals became better before new myelin was fully formed, suggesting that the treatment might have additional beneficial effects.

Other data from animal studies also support the idea that brain and spinal cord disease or injury can be treated by delivering healthy glial cells.

“Our approach uses the glial cell’s natural ability to repair and support nerve cells in the CNS. Q-Cells hold great promise not only for those people with rare diseases such as TM and ALS [amyotrophic lateral sclerosis], but for the many people worldwide who live with MS [multiple sclerosis], spinal cord injury, and stroke,” Borst said.

The company is also planning to launch a trial in ALS patients.

“The FDA’s clearance of this IND is yet another validation of our collaborative and purposeful approach to move Q-Cells into the clinic,” added James Campanelli, PhD, vice president of Research and Development at Q Therapeutics. “We are eager to move forward with this trial and optimistic that Q-Cells will prove effective in treating human CNS injury and disease.”

About the Author

Patricia Silva, PhD Patrícia holds a PhD in medical microbiology and infectious diseases from the Leiden University Medical Center, Netherlands, and completed a postdoctoral research fellowship at the Instituto de Medicina Molecular, Lisbon, Portugal. Her work in academia was mainly focused on molecular biology and the genetic traits of infectious agents such as viruses and parasites. Patrícia earned several travel awards to present her work at international scientific meetings. She is a published author of several peer-reviewed science articles.

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Laurie Mcdonald

How can I get on the trials for cell therepy ?

Carol Cornell

I also am interested in being in the trials for cell therapy. I have had TM for 4 years now.

Cindy Budrow

Would love to get on a trial. Can I get more information.

Robert Siddle

I am a Transverse Myelitis sufferer and would like to be considered for the stem cell therapy.

Kathleen Lapkowski

I have TM . Would love to be a guinea pig in this study !! How does one go about it?

Karen Smith

I want to be on the trail list

Karen Smith

Traverse myelitis

Tina Aguilar

Yes please. This would be amazing. I would love a little normal back from what I lost to Transverse Myelitis.

Ann Miriam

Will it work on long term TM patients Now have TM 27 yrs ??

Marian Rodriguez

I need a trial please for my 11 month baby

Tim ONeill

I have had TransverseMyelitis since 2009 and would like to participate in the trial.

Mary DeBoda

I'm an MS pt. I'm in CA and would love to bein this clinical trial!

Janice Wlodarek

I have transverse myeltis for 2 years now. Would like to be on the trial list.

Mercedes Castillo

Is there age requirements?

Doug Hawthorne

Have had HSCT, 18 months post. MS progression has been halted. Could I be considered a good candidate for this trial?

Candace

I have leukodystrophy. Would I be eligible for the stem cell?

HOWARD

I have MS Diagnosed 2001
Lost a lot! Please help me.

Martin M Segal

How can I be considered for the trial? I would sign up immediately.

Lori Ybarra

I also have TM and would love to be on the list to help. Please let me know what I can do to help! Would love to get my Legs and feet back! Iam 3 weeks in from my diagnosis .

Erik

Has your TM got any better?

Susan Lee

Do you think it might help Trigeminal Neuralgia

Angie Jones

My brother was diagnosed with TM aim June 2014. He is interested in becoming part of the clinical trial.

mark bolam

Anything going to happen in the UK ??

Joseph hill

I have tm and want to be part of the clincal trail how can i go by doing that

Marilyn Antonsanti

I've had TM for 38yrs. Can I participate in this trial? Please

Giuseppina Mariano

Hi, I'm 16 months in TM. I would like to be on this trial. How can I sign up?

Jordan

Have had TM since 2011, and can only move my right arm. I'd like to be a candidate.

Damir Vrbanic

How is possible to get involved in a trial.My son is A TM patient from last year and he is 6years old,He can not walk,his left leg is paralized.Thank you.

Hi Damir and others wondering about a trial,
This trial has not been launched yet, and I don't know what criteria the company will have to include patients. It might be a good idea to sign up for our newsletter, as we commonly include information about clinical trials that are recruiting participants.

LAURA VINCENT

I’m at end stages of a rapid, advanced, aggressive, progressive form of M.S. as told to me from day one 8 years ago when I was diagnosed with the rarest form of M.S. ever documented(even possibly another rare autoimmune disease masking itself as M.S.) by a worldwide M.S. specialist! I was literally running around on ta Firday, and by Saturday night I was in the hospital and totally paralyzed up to my tongue. They thought I was having a stroke in the ambulance en route to the hospital! Finally after removing myself from DNR after 7 days in the hospital/by my neurologist's request, on his hands and knees begging me to please let him help me, after 7 more days of very high doses of methyl prednisone he brought back everything except for my left leg, I never did walk again. Now 8 years later, I merely have the use of my right arm (which I believe is flaring right now). I was also part of the Tysabri trial when I was initially diagnosed & I had all extreme, adverse effects and was taken off immediately. I also had adverse effects to Interferon & Copaxone and was immediately taken off those as well! I haven’t been on any immunosuppressive therapies aside from large doses of methyl prednisone infusions ever since. Thanking you in advance for your prompt response/at your earliest convenience regarding if/when I would be eligible for this triaI? ~Sincerely, Laura Vincent ?

Hi Laura,
I am truly sorry to hear about your extremely aggressive disease course.

The trial has, however, not been launched yet. And we at MS News Today are not involved in the research, we are merely reporting on various research and drug development efforts and providing information about trials.
A soon as we get information about this study, we will cover it here. It might, therefore, be good to sign up to our newsletter, as we would include such information there.

Michal k

Can this help for genetic disease PMP22
If so, I would be happy to participate in the research

Phil Harness

When will the clinical trials start for ALS patients?

Ray Smith

I have TM and I am very interested

Timothy ARMSTRONG

My grandson contracted TM at age 1 he is 11 now, this research is very exciting for our family, were hoping for positive results in stage one testing. He might qualify for stage two participation. Even if there is only a small measurable improvement in test subjects it would still be a leap of success for those suffering from this rare disease. God speed and prayers will be with you.

Jeremy Potter

I have TM and would love to know how this study has progressed and how I can get involved.

Sheri Nielsen

I have had Transverse Myelitis since 1975. I am in the middle of patients, I can walk with a walker and a brace for foot drop. My damage is T5 to T7. I would love to be in this trial. I just turned 60.