Follistatin Gene Therapy

No, there has been no influence of gender on any safety or data collected to date.

Yes, we would have to evaluate you. We appreciate your efforts.

Interesting question, Not likely McD’s or KFC, but the concept is out there and in fact there are transgenic cows that are myostatin depleted, There are also other companies exploring this concept.

No, not at this time, We are very interested in screening additional patients.

Follistatin is a protein that blocks another protein called myostatin that is a negative regulator of muscle growth. Simply put, Follistatin causes muscles to get larger and stronger. It also has anti-inflammatory effects, which we believe is advantageous for IBM.

The AAV Follistatin is currently only being performed by us.

Please contact us on our website, at The Center for Gene Therapy, at Nationwide Children’s and likely you would be evaluated here by our team.

We expect to have a reasonable idea of the results in the next 6 months. However, we are pleased to state that the therapy has been safe and well tolerated to date.

Please contact us as you may need to be re-checked and we are also adding new patients to the study list. There are a multitude of enrollment criteria set by FDA.

If FDA and Medicare approve it, it appears that it would be covered by the Affordable Care Act. These are being figured out on Capital Hill! and beyond Dr. Mendell and Dr. Kaspar’s political knowledge.

Likely in future studies, if it works in the leg muscles, we can certainly discuss with the FDA.

Yes on the higher dose, we’re moving as quickly as the regulatory bodies will allow us, Again, our goal is to run a safe and best chance for success study, And in new studies, (higher doses) will be recruiting new subjects.

This is likely a good question for the FDA, given that PM and DM have currently other treatment options.

Milo Biotech is supporting the advancement of this therapy if it is successful, making a path towards getting this fully approved if we are successful in the human studies. Currently the company is private, and to our knowledge is not planning on an IPO, but may likely raise funds in the near future to advance the product.

Yes, Dr. Mendell has designed the trial to treat the most affected muscle group (quads) for the first trials. Injected muscles in our pre-clinical studies showed the greatest benefits.

Hi Mike, We actually don’t know the answer to this, but is something that we will have to explore in future trials. We do know in mouse studies that we can treat at later stages and see some benefits, but again the greatest benefits are when treated early. We will test this exact question in patients and we can hope that there will be benefit.

We have a number of measurements:strength testing, 6 minute walk test, stair climbing, getting up from chair, muscle biopsies. A great deal of data is being collected!

Safety is the primary outcome for this trial, but we are also collecting a great deal of functional measurements. We do think early treatment would have greater effects on muscles.

Our animal studies demonstrated that muscle that were injected received the greatest effect, but other muscles demonstrated strength and size improvements. These are the same things we are currently testing in our clinical trial.

This is a difficult question and really is dictated by safety and the results of the first study. We are thinking of all scenarios, but it’s too early to have this fully mapped out at this time. Something we’re working on though.

Safety is the primary goal of this first trial, We are collecting many data points on leg function (walking, climbing stairs, force measurements, as well as biopsy evaluation). With trials, we are collecting as much data as possible and can only share definitive results at the end of the first trial. We can state that the injections have been safe and well tolerated to date.

Hello Gary, First age has nothing to do with eligibility and if interested, please contact us for screening for the clinical trial. We are working with the FDA and oversight committees that are checking for safety in the testing, and if this work is positive, will promise to move the therapy forward to more patients and trials as mandated by the FDA to move this to an approved drug.

No, we are still screening patients. We would be very happy to be able to screen anyone interested.

Yes, we continue to screen patients for enrollment. The Children’s Hospital Gene Therapy website has contact information.

The first human trial is currently only in the US, and only at one site, Nationwide Children’s Hospital. We accept patients outside the country, but for a trial like this, would have to stay for at least 6 months. In the future, if successful, we of course would open to many sites.

This is a very good questions, and the truth is we don’t know. What we do know is that follistatin appears to suppress the inflammation found in muscle tissue and we are encouraged by that.

We have to get this approved by FDA as the first hurdle. The clinical trial does not cost the patient or Medicare any money, We have to fund this via grants/foundations. If a drug is approved and there are no other therapies available, Medicare would likely cover the costs, but again, it has to be an approved drug.

Thank you very much, we are excited about our research and moving this into the first human clinical trials for IBM patients. We appreciate your willingness to help us. We are working very hard on all of the regulatory process, receiving orphan drug designation status, and moving as fast as we can. What is limiting is funding for running the actual clinical trial.

Age is not a factor. Patients at this time must be ambulatory, and also not have high levels of pre-existing immunity to AAV. If this trial is effective, we would request FDA to go back and test the therapy in non-ambulatory patients.

Our ultimate goal is to target many muscle groups, but this is the first stage of trials at this time. We are pushing more systemic muscle studies currently, but those new studies are in earlier stages.

The patients are still under study and we are collecting additional information. The important point is that the injection has been well tolerated and there may be signs of benefit, but too early to put a timepoint for efficacy.

Through clinical trials there does not appear to be any benefit of IVIG treatment for IBM patients.

Right now we’re in the middle of the trial, and so far safe and well tolerated. We will have to take the data we collect safety and efficacy data to the Food and Drug Administration and move this forward as quickly and safely as possible. As we don’t have the trial Phase I completed it is hard to set a date or timing, but we will move as quickly and safely as possible to bring this forward if it is deemed safe and effective.

To date, we have not seen any side effects or adverse events related to the gene therapy or follistatin expression. This means the trial has been safe and well tolerated to date.